A Study of Rilzabrutinib in Adult Patients With Immune Th... | NCT03395210 | Trialant
NCT03395210
Sponsor
Principia Biopharma, a Sanofi Company
Status
Completed
Last Update Posted
Feb 23, 2026Actual
Enrollment
86Actual
Phase
Phase 2
Conditions
Immune Thrombocytopenia
Interventions
Rilzabrutinib
Countries
United States
Australia
Bulgaria
Canada
Czechia
Netherlands
Norway
United Kingdom
Protocol Section
Identification Module
NCT ID
NCT03395210
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
DFI17124
Secondary IDs
ID
Type
Description
Link
PRN1008-010
Other Identifier
Principia Biopharma
U1111-1260-4044
Registry Identifier
ICTRP
2023-509397-39
Registry Identifier
CTIS
Brief Title
A Study of Rilzabrutinib in Adult Patients With Immune Thrombocytopenia (ITP)
Official Title
An Adaptive, Open-Label, Dose-Finding, Phase 1/2 Study Investigating the Safety, Pharmacokinetics, and Clinical Activity of PRN1008, an Oral BTK Inhibitor, in Patients With Relapsed Immune Thrombocytopenia
Acronym
Not provided
Organization
SanofiINDUSTRY
Status Module
Record Verification Date
Feb 2026
Overall Recruitment Status or Expanded Access Status
Completed
Last Known Status
Not provided
Delayed Posting
Not provided
Why Stopped
Not provided
Expanded Access Info
No
Start Date
Mar 22, 2018Actual
Primary Completion Date
Jan 31, 2023Actual
Completion Date
Dec 11, 2025Actual
First Submitted Date
Dec 22, 2017
First Submission Date that Met QC Criteria
Jan 3, 2018
First Posted Date
Jan 10, 2018Actual
Results Waived
Not provided
Results First Submitted Date
Sep 11, 2025
Results First Submitted that Met QC Criteria
Oct 30, 2025
Results First Posted Date
Nov 14, 2025Actual
Certification/Extension (aka Delayed Results) First Submitted Date
Jan 30, 2024
Certification/Extension First Submitted that Passed QC Review
Not provided
Certification/Extension First Posted Date
Nov 14, 2025Actual
Last Update Submitted Date
Feb 3, 2026
Last Update Posted Date
Feb 23, 2026Actual
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
Principia Biopharma, a Sanofi CompanyINDUSTRY
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
Yes
Is FDA Regulated Drug
Yes
Is FDA Regulated Device
No
Is Unapproved Device
Not provided
Pediatric Postmarket Surveillance of a Device Product
Not provided
Product Exported from US
Not provided
FDAAA801 Violation
Not provided
Description Module
Brief Summary
This was a 2 part (Part A and B) adaptive, open-label, dose-finding study of PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options, with a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. The dose-finding portion of the study was completed. Part B treatment dose was 400 mg twice daily.
Detailed Description
This was a 2 part (Part A and B) adaptive, open-label, dose-finding study of PRN1008 in approximately 60 patients in Part A and approximately 25 patients in Part B.
Part A enrolled patients with ITP who were refractory or relapsed with no available and approved therapeutic options. Eligible patients had a platelet count <30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. The active treatment period was 24 weeks and the post-treatment follow-up period is 4 weeks. In the dose-finding part of the study, each patient enrolled in the study was allowed to up-titrate their dose after 28 days of PRN1008 therapy, if they did not experience a platelet response or a dose-limiting toxicity (DLT) at the last dose level. Patients who responded to PRN1008 per protocol may enter a long term-extension.
Part B of the study included approximately 25 patients with ITP who had relapsed or had an insufficient response to prior therapies. Eligible patients had a platelet count <30,000/µL on two occasions no less than 7 days apart, within 15 days before treatment began and a platelet count of ≤35,000/µL on Study Day 1 (SD1). The study consisted of a 28-day screening period, 24-week active treatment period, and a long-term extension. After the last dose of PRN1008 there was a 4-week safety follow-up period.
Conditions Module
Conditions
Immune Thrombocytopenia
Keywords
ITP
Design Module
Study Type
Interventional
Number of References to an Expanded Access Study
Not provided
Expanded Access Types
Not provided
Patient Registry
Not provided
Target Follow-Up Duration
Not provided
Phases
Phase 2
Interventional Study Design
Allocation
Biospecimen
No data available
No data is available for this block.
Enrollment
86Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
Rilzabrutinib (PRN1008) Daily
Experimental
Part A approximately 60 patients: Up to 24 weeks open-label treatment with PRN1008 400mg BID; safety and dose evaluation. Patients who respond to PRN1008 per protocol may enter a long-term extension.
Part B approximately 25 patients: Up to 24 weeks open-label treatment with PRN1008 400mg BID; safety and dose evaluation. Patients who respond to PRN1008 per protocol may enter a long-term extension
Drug: Rilzabrutinib
Interventions
Name
Type
Description
Arm Group Labels
Other Names
Rilzabrutinib
Drug
BTK inhibitor
Rilzabrutinib (PRN1008) Daily
PRN1008
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
Part A: Percentage of Participants Who Achieved 2 or More Consecutive Platelet Counts by Starting Dose Level and Overall
The percentage of participants who achieved 2 or more consecutive platelet counts, separated by at least 5 days, of >=50,000/ microliter (μL) and an increase of platelet count of >=20,000/μL from baseline, by starting dose level and overall, without use of rescue medication in the 4 weeks prior to the latest elevated platelet count. 95% confidence interval (CI) was based on the Clopper-Pearson method. The average of the 2 screening results and the Cycle 1 Day 1 result were used as the baseline value.
Up to 24 Weeks
Part B: Percentage of Participants Who Achieved Platelet Counts >=50,000/μL
The percentage of participants who achieved platelet counts >=50,000/μL on at least 8 out of the last 12 weeks of the 24-week treatment period without the use of rescue medication after 10 weeks of active treatment. 95% CI was based on the Clopper-Pearson exact method.
Up to 24 Weeks
Part A: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment Related Treatment-Emergent Adverse Events
Adverse event (AE): any untoward medical occurrence in participant or clinical study participant, temporally associated with the use of rilzabrutinib, whether or not considered related to rilzabrutinib. TEAEs: AEs that developed or worsened or became serious on or after the first dose administration of rilzabrutinib (Day 1). Any TEAEs are considered treatment-related TEAEs as per Investigator's evaluation of participant's circumstances surrounding the event, and an evaluation of any potential alternative causes to determine whether an TEAE can be considered as related to the rilzabrutinib.
From first dose of rilzabrutinib (Day 1) up to last dose + 1 (up to 294 days)
Part B: Number of Participants With Treatment-Emergent Adverse Events and Treatment Related Treatment-Emergent Adverse Events
AE any untoward medical occurrence in participant or clinical study participant, temporally associated with the use of rilzabrutinib, whether or not considered related to rilzabrutinib. TEAEs: AEs that developed or worsened or became serious during the treatment-emergent period, defined as any time after the first dose administration of rilzabrutinib (Day 1). Any TEAEs are considered treatment-related TEAEs as per Investigator's evaluation of participant's circumstances surrounding the event, and an evaluation of any potential alternative causes to determine whether an TEAE can be considered as related to rilzabrutinib.
Secondary Outcomes
Measure
Description
Time Frame
Part A: Percentage of Weeks With Platelet Counts >=50,000/μL by Starting Dose Level and Overall
The percentage of weeks in which participants achieved platelet counts >=50,000/μL in the treatment period are summarized here.
Up to 24 Weeks
Part A: Percentage of Participants With 4 Out of the Final 8 Platelet Counts >=50,000/μL by Starting Dose Level and Overall
Other Outcomes
Not provided
Eligibility Module
Eligibility Criteria
Inclusion Criteria:
Male or female patients, aged 18 to 80 years old
Immune-related ITP (both primary and secondary)
Exclusion Criteria:
Pregnant or lactating women
Current drug or alcohol abuse
History of solid organ transplant
Positive screening for HIV, hepatitis B, or hepatitis C
Accepts Healthy Volunteers
No
Sex
All
Sex/Gender Based
Not provided
Sex/Gender Description
Not provided
Minimum Age
18 Years
Maximum Age
80 Years
Standard Ages
AdultOlder Adult
Study Population
Not provided
Sampling Method
Not provided
Contacts/Locations Module
Central Contacts
Not provided
Overall Officials
Name
Affiliation
Role
Olga Bandman, MD
Principia Biopharma
Study Director
Locations
Facility
Status
City
State
ZIP
Country
Contacts
Bleeding and Clotting Disorders Institute- Site Number : 1087
Cooper N, Jansen AJG, Bird R, Mayer J, Sholzberg M, Tarantino MD, Garg M, Ypma PF, McDonald V, Percy C, Kostal M, Goncalves I, Bogdanov LH, Gernsheimer TB, Diab R, Yao M, Daak A, Kuter DJ. Efficacy and Safety Results With Rilzabrutinib, an Oral Bruton Tyrosine Kinase Inhibitor, in Patients With Immune Thrombocytopenia: Phase 2 Part B Study. Am J Hematol. 2025 Mar;100(3):439-449. doi: 10.1002/ajh.27539. Epub 2025 Jan 22.
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
Types
Not provided
Time Frame
Not provided
Access Criteria
Not provided
URL
Not provided
Results Section
Participant Flow Module
Pre-assignment Details
A total of 60 participants in Part A and 26 participants in Part B were enrolled in the study. The results are presented up to primary completion date 31 January 2023.
Recruitment Details
The study was conducted at 31 active centers in 8 countries. As prespecified in statistical analysis plan (SAP), Part A results were presented by either of the of the treatment group labels: Starting dose level (each participant was assigned to only one of the groups), dose level (dose received during the defined period. As most patients received more than one dose, a given patient could be assigned to multiple dose levels) and overall.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
Part A: Starting Dose 200 mg QD
Participants received rilzabrutinib 200 milligrams (mg) orally once daily (QD) as starting dose from Day 1 to 4 weeks. Participants were allowed to up-titrate the dose levels unless withdrawn, had a platelet response to current dose level, or the next dose level had been determined to be ineligible for further enrollment every 4 weeks up to the maximum allowed 400 mg twice daily (BID) (800 mg/day) over 24 weeks of treatment period.
Periods
Title
Milestones
Reasons Not Completed
Overall Study
Type
Comment
Milestone Data
STARTED
Baseline Characteristics Module
Baseline Analysis Population Description
Outcome Measures Module
Outcome Measures
Adverse Events Module
Frequency Threshold
5
More Info Module
Limitations and Caveats
Not provided
Annotation Section
No data available
No data is available for this block.
Document Section
Large Document Module
Document Has No Statistical Analysis Plan (SAP)
Not provided
Uploaded Document Information
Type
Includes Protocol
Includes SAP
Includes ICF
Document Label
Document Date
Document Uploaded Date
Document File Name
Prot
Yes
No
No
Study Protocol
May 15, 2024
Sep 11, 2025
Derived Section
Miscellaneous Info Module
Version Holder
Jul 10, 2026
Removed Countries
Not provided
Submission Tracking
Estimated Results First Submitted Date
Not provided
Condition Browse Module
MeSH Terms
Intervention Browse Module
No data available
No data is available for this block.
N/A
Intervention Model
Sequential Assignment
Intervention Model Description
Not provided
Primary Purpose
Treatment
Observational Model
Not provided
Time Perspective
Not provided
Masking Info
Masking
None (Open Label)
Masking Description
Not provided
Who Masked
Not provided
From first dose of rilzabrutinib (Day 1) up to last dose + 1 (approximately 170 days)
The percentage of participants who had at least 4 out of the final 8 platelet counts >=50,000/μL are summarized here. The final 8 scheduled platelet counts are the measurements performed in the last 8 weeks of rilzabrutinib (depending on treatment duration, not necessarily from Week 19 to Week 24) in the treatment period. 95% CI was based on the Clopper-Pearson method.
Up to 24 Weeks
Part A: Change From Baseline to the Average of Post Day 1 Platelet Counts by Dose Level and Overall
Average of post Day 1 platelet count is equivalent to average of (average of each participant's post-Day 1 platelet counts), included platelet counts up to 1 day after the date of last dose of rilzabrutinib and excluded platelet counts on or after date of rescue, if applicable. The average of the 2 screening results and the Cycle 1 Day 1 result measured on different date were used as the baseline value.
Baseline and up to 24 Weeks
Part A: Number of Weeks With Platelet Counts >=50,000/μL by Starting Dose Level and Overall
The number of weeks in which participant achieved platelet counts >=50,000/μL in the treatment period are summarized here. The number of weeks is based on the number of scheduled weekly assessments, by study day.
Up to 24 Weeks
Part A: Number of Weeks With Platelet Counts >=30,000/μL by Starting Dose Level and Overall
The number of weeks in which participant achieved platelet counts >=30,000/μL in the treatment period are summarized here. The number of weeks is based on the number of scheduled weekly assessments, by study day.
Up to 24 Weeks
Part A: Time to First Platelet Count >=50,000/μL Across All Dose Levels
Time to first platelet count >=50,000/μL during the treatment period in days was calculated as: (date of first occurrence of platelet count >=50,000/μL - date of first rilzabrutinib dosing) +1.
Up to 24 Weeks
Part B: Number of Weeks With Platelet Counts >= 50,000/μL or >= 30,000/μL and Doubling the Baseline
The number of weeks in which participant achieved platelet counts with thresholds as: >=50,000/μL or >=30,000/μL and doubling the baseline in the absence of rescue therapy (platelet counts will be censored for 4 weeks after the use of rescue medication, if given) are summarized here. Baseline is defined as the average of 3 platelet counts: 2 qualified screening platelet counts collected in electronic case report form (eCRF) and Week 1 (study day 1) platelet count.
Up to 24 Weeks
Part B: Percentage of Participants Who Achieved 2 or More Consecutive Platelet Counts
Percentage of participants who achieved 2 or more consecutive platelet counts, separated by at least 5 days, of >=50,000/μL and an increase of platelet count of >=20,000/μL from baseline without use of rescue medication in the 4 weeks prior to the latest elevated platelet count are summarized here. Baseline is defined as the average of 3 platelet counts: 2 qualified screening platelet counts collected in eCRF and Week 1 (study day 1) platelet count. 95% CI was based on the Clopper-Pearson exact method.
Up to 24 Weeks
Part B: Number of Weeks With Platelet Counts >=30,000/μL and Doubling the Baseline
The number of weeks in which participant achieved platelet counts >=30,000/μL and doubling the baseline in the absence of rescue therapy (platelet counts will be censored for 4 weeks after the use of rescue medication, if given) are summarized here. Baseline is defined as the average of 3 platelet counts: 2 qualified screening platelet counts collected in eCRF and Week 1 (study day 1) platelet count.
Up to 24 Weeks
Part B: Percentage of Participants Who Received Rescue Medication
Rescue medication is defined as any therapy used to rescue a participant (1 of intravenous immunoglobulin [IVIG], high-dose steroids, platelet infusion or anti-D immunoglobulin infusion). Percentage of participants who received rescue medication are summarized here. 95% CI was based on the Clopper-Pearson exact method.
Up to 24 Weeks
Part B: Change From Baseline in Idiopathic Thrombocytopenic Purpura Bleeding Scale (IBLS)
The IBLS is a bleeding assessment score. IBLS comprises of 11 sites for female and 10 sites for male, and each site is scored from 0 (none) to 2 (marked bleeding). The total overall score ranges from 0-22, with higher scores indicating higher presence of marked bleeding. For each participant, an IBLS score at each visit was calculated by taking the average across 11 items (10 for male and postmenopausal women) at 9 anatomical sites (8 for male and postmenopausal women). For each participant, a mean IBLS score was also calculated by taking the average across all post-baseline visits during the 24-week treatment period. IBLS average value ranges from 0 to 2. The smaller the IBLS average value is, the healthier the participants are. For change from baseline, negative value indicates an improvement. The baseline value is defined as the last available value before the first dose rilzabrutinib.
Baseline and up to 24 weeks
Part A: Percentage of Participants Who Received Rescue Medication by Dose Levels and Overall
Rescue medication is defined as any therapy used to rescue a participant (1 of IVIG, high-dose steroids, platelet infusion or anti-D immunoglobulin infusion). The percentage of participants who received rescue medication for each dose level and overall are summarized here. 95% CI was based on the Clopper-Pearson method.
Up to 24 Weeks
Part A: Percentage of Participants With Grade 2 or Higher Bleeding Event by Dose Level and Overall
The percentage of participants with intensity grade 2 or higher bleeding event are summarized for each dose level and overall. The TEAEs with standardized medical dictionary for regulatory activities (MedDRA) query (SMQ) hemorrhages were medically determined for analysis of bleeding events. 95% CI was based on the Clopper-Pearson method.
Up to 24 Weeks
Part A: Number of Participants With Idiopathic Thrombocytopenic Purpura/Immune Thrombocytopenia (ITP) Bleeding Assessment Tool (ITP-BAT) Scale by Dose Level
The ITP-BAT scale comprises of 11 grades from 0 (none) to 2 (marked bleeding), with higher scores indicating higher presence of marked bleeding, assessed at 9 anatomical sites (skin, oral, epistaxis, gastrointestinal [GI], urinary, gynecological [GYN], pulmonary, intracranial hemorrhage [HEM], subconjunctival HEM) by history over the previous week (Hx). In addition, 2 sites (skin and oral), were also assessed by physical examination (PE). The 'worst ever' bleeding experienced at each site was graded using the same system. Here, 0 indicates none; 1 indicates 1-5 bruises and/or scattered petechiae and 2 indicates >5 bruises with size >2 centimeter (cm) and/or diffuse petechiae. Each participant summed up the transformed scores across all 11 sites per visit assessment. The total overall score ranges from 0-22 with the higher score indicating worst outcome.
Up to 24 Weeks
Part A: Maximum Observed Plasma Concentration (Cmax) of Rilzabrutinib
Plasma samples were collected at specified timepoints to determine Cmax of rilzabrutinib.
Day 1 of Cycles 1, 2, 3, and 5 (each cycle 28 days)
Part A: Time of Observed Maximum Plasma Concentration (Tmax) of Rilzabrutinib
Plasma samples were collected at specified timepoints to determine tmax of rilzabrutinib.
Day 1 of Cycles 1, 2, 3, and 5 (each cycle 28 days)
Part A: Area Under the Plasma Concentration-Time Curve From Time Zero to Last Quantifiable Concentration (AUClast) of Rilzabrutinib
Plasma samples were collected at specified timepoints to determine AUClast of rilzabrutinib.
Day 1 of Cycles 1, 2, 3, and 5 (each cycle 28 days)
Part A: Area Under the Plasma Concentration-Time Curve From Time Zero to Infinity (AUCinf) of Rilzabrutinib
Plasma samples were collected at specified timepoints to determine AUCinf of rilzabrutinib.
Day 1 of Cycles 1, 2, and 3 (each cycle 28 days)
Part A: Elimination Half-Life (t1/2) of Rilzabrutinib
Plasma samples were collected at specified timepoints to determine t1/2 of rilzabrutinib.
Day 1 of Cycles 1, 2, and 3 (each cycle 28 days)
Part A: Apparent Volume of Distribution of the Drug After Oral Administration (Vz/F) of Rilzabrutinib
Plasma samples were collected at specified timepoints to determine Vz/F of rilzabrutinib.
Day 1 of Cycles 1, 2, and 3 (each cycle 28 days)
Part A: Apparent Total Clearance of the Drug From Plasma After Oral Administration (CL/F) of Rilzabrutinib
Plasma samples were collected at specified timepoints to determine CL/F of rilzabrutinib.
Day 1 of Cycles 1, 2, and 3 (each cycle 28 days)
Part B: Plasma Concentration of Rilzabrutinib
Plasma samples were collected at specified timepoints for evaluation of rilzabrutinib pharmacokinetic (PK) concentrations.
Pre-dose and 2 hours post-dose on Days 1, 29, and 57
RCCA MC LLC- Site Number : 1091
Bethesda
Maryland
20817-1915
United States
Massachusetts General Hospital Cancer Center- Site Number : 1092
Boston
Massachusetts
02114-2603
United States
Beth Israel Deaconess Medical Center- Site Number : 1099
Boston
Massachusetts
02215
United States
Mid Michigan Medical Center- Site Number : 1086
Midland
Michigan
48670
United States
New York Presbyterian Hospital/Weill Cornell Medical Center- Site Number : 1097
New York
New York
10021
United States
Pitt County Memorial Hospital- Site Number : 1095
Greenville
North Carolina
27834
United States
Seattle Cancer Care Alliance Site Number : 1098
Seattle
Washington
98109-4405
United States
Investigational Site Number : 105
Canberra
Australian Capital Territory
2605
Australia
Investigational Site Number : 104
Sydney
New South Wales
2139
Australia
Investigational Site Number : 102
Woolloongabba
Queensland
4102
Australia
Investigational Site Number : 101
Clayton
Victoria
3168
Australia
Investigational Site Number : 106
Parkville
Victoria
3050
Australia
Investigational Site Number : 103
Perth
Western Australia
6005
Australia
Investigational Site Number : 213
Pleven
5800
Bulgaria
Investigational Site Number : 214
Sofia
1431
Bulgaria
Investigational Site Number : 211
Varna
9010
Bulgaria
Investigational Site Number : 1161
Toronto
Ontario
M5B 1W8
Canada
Investigational Site Number : 1162
Montreal
Quebec
H3A 1A1
Canada
Investigational Site Number : 431
Brno
62500
Czechia
Investigational Site Number : 433
Hradec Králové
50005
Czechia
Investigational Site Number : 434
Ostrava - Poruba
70852
Czechia
Investigational Site Number : 432
Prague
12808
Czechia
Investigational Site Number : 727
Rotterdam
3015 GD
Netherlands
Investigational Site Number : 728
The Hague
2545 CH
Netherlands
Investigational Site Number : 542
Bergen
N-5021
Norway
Investigational Site Number : 541
Grålum
1714
Norway
Investigational Site Number : 981
Leicester
Leicestershire
LE1 5WW
United Kingdom
Investigational Site Number : 983
London
London, City of
E1 2ES
United Kingdom
Investigational Site Number : 980
London
London, City of
W12 0HS
United Kingdom
Investigational Site Number : 984
Birmingham
B15 2GW
United Kingdom
Derived
Kuter DJ, Mayer J, Efraim M, Bogdanov LH, Baker R, Kaplan Z, Garg M, Trneny M, Choi PY, Jansen AJG, McDonald V, Bird R, Gumulec J, Kostal M, Gernsheimer T, Ghanima W, Daak A, Cooper N. Long-term treatment with rilzabrutinib in patients with immune thrombocytopenia. Blood Adv. 2024 Apr 9;8(7):1715-1724. doi: 10.1182/bloodadvances.2023012044.
Kuter DJ, Efraim M, Mayer J, Trneny M, McDonald V, Bird R, Regenbogen T, Garg M, Kaplan Z, Tzvetkov N, Choi PY, Jansen AJG, Kostal M, Baker R, Gumulec J, Lee EJ, Cunningham I, Goncalves I, Warner M, Boccia R, Gernsheimer T, Ghanima W, Bandman O, Burns R, Neale A, Thomas D, Arora P, Zheng B, Cooper N. Rilzabrutinib, an Oral BTK Inhibitor, in Immune Thrombocytopenia. N Engl J Med. 2022 Apr 14;386(15):1421-1431. doi: 10.1056/NEJMoa2110297.
Del Pozo Martin Y. 2021 ASH annual meeting. Lancet Haematol. 2022 Feb;9(2):e92-e93. doi: 10.1016/S2352-3026(21)00384-7. Epub 2021 Dec 16. No abstract available.
FG001
Part A: Starting Dose 400 mg QD
Participants received rilzabrutinib 400 mg orally QD as starting dose from Day 1 to 4 weeks. Participants were allowed to up-titrate the dose levels unless withdrawn, had a platelet response to current dose level, or the next dose level had been determined to be ineligible for further enrollment every 4 weeks up to the maximum allowed 400 mg BID (800 mg/day) over 24 weeks of treatment period.
FG002
Part A: Starting Dose 300 mg BID
Participants received rilzabrutinib 300 mg orally BID as starting dose from Day 1 to 4 weeks. Participants were allowed to up-titrate the dose levels unless withdrawn, had a platelet response to current dose level, or the next dose level had been determined to be ineligible for further enrollment every 4 weeks up to the maximum allowed 400 mg BID (800 mg/day) over 24 weeks of treatment period.
FG003
Part A: Starting Dose 400 mg BID
Participants received rilzabrutinib 400 mg orally BID as starting dose from Day 1 to 24 weeks, unless withdrawn or had a platelet response to current dose level.
FG004
Part B: Rilzabrutinib 400 mg BID
Participants received rilzabrutinib 400 mg orally BID from Day 1 up to 24 weeks of treatment period.
FG0009 subjects
FG0011 subjects
FG0025 subjects
FG00345 subjects
FG00426 subjects
COMPLETED
FG0004 subjects
FG0010 subjects
FG0020 subjects
FG00315 subjects
FG0044 subjects
NOT COMPLETED
FG0005 subjects
FG0011 subjects
FG0025 subjects
FG00330 subjects
FG00422 subjects
Type
Comment
Reasons
Other
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG0041 subjects
Participant Uncooperative or Noncompliant
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
FG004
Adverse Event
FG0001 subjects
FG0010 subjects
FG0022 subjects
FG0034 subjects
FG004
Need of Rescue Medication
FG0001 subjects
FG0010 subjects
FG0021 subjects
FG0034 subjects
FG004
Lack of Response
FG0000 subjects
FG0010 subjects
FG0020 subjects
FG0035 subjects
FG004
Participant was Erroneously Enrolled in the Study
FG0001 subjects
FG0010 subjects
FG0020 subjects
FG0030 subjects
Participant Decision
FG0001 subjects
FG0011 subjects
FG0020 subjects
FG0034 subjects
FG004
Ongoing at the Time of Primary Completion Date
FG0001 subjects
FG0010 subjects
FG0022 subjects
FG00313 subjects
Part A: Intent-to-treat population (ITT) consisted of all participants who had enrolled into the study.
Part B: The enrolled population consisted of all participants from screened population (who signed informed consent) who had been allocated to rilzabrutinib regardless of whether rilzabrutinib was received or not.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
Part A: Starting Dose 200 mg QD
Participants received rilzabrutinib 200 mg orally QD as starting dose from Day 1 to 4 weeks. Participants were allowed to up-titrate the dose levels unless withdrawn, had a platelet response to current dose level, or the next dose level had been determined to be ineligible for further enrollment every 4 weeks up to the maximum allowed 400 mg BID (800 mg/day) over 24 weeks of treatment period.
BG001
Part A: Starting Dose 400 mg QD
Participants received rilzabrutinib 400 mg orally QD as starting dose from Day 1 to 4 weeks. Participants were allowed to up-titrate the dose levels unless withdrawn, had a platelet response to current dose level, or the next dose level had been determined to be ineligible for further enrollment every 4 weeks up to the maximum allowed 400 mg BID (800 mg/day) over 24 weeks of treatment period.
BG002
Part A: Starting Dose 300 mg BID
Participants received rilzabrutinib 300 mg orally BID as starting dose from Day 1 to 4 weeks. Participants were allowed to up-titrate the dose levels unless withdrawn, had a platelet response to current dose level, or the next dose level had been determined to be ineligible for further enrollment every 4 weeks up to the maximum allowed 400 mg BID (800 mg/day) over 24 weeks of treatment period.
BG003
Part A: Starting Dose 400 mg BID
Participants received rilzabrutinib 400 mg orally BID as starting dose from Day 1 to 24 weeks, unless withdrawn or had a platelet response to current dose level.
BG004
Part B: Rilzabrutinib 400 mg BID
Participants received rilzabrutinib 400 mg orally BID from Day 1 up to 24 weeks of treatment period.
BG005
Total
Total of all reporting groups
Denominators
Units
Counts
Participants
BG0009
BG0011
BG0025
BG00345
BG00426
BG00586
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Customized
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
<65 years
BG0008
BG0011
BG0025
BG003
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Female
BG0004
BG0010
BG002
Race/Ethnicity, Customized
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
White
BG0009
BG0011
BG002
Type
Title
Description
Population Description
Reporting Status
Anticipated Posting Date
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Time Frame
Units Analyzed
Denominator Units Selected
Arm/Group Information
Denominators
Classes
Analyses
Primary
Part A: Percentage of Participants Who Achieved 2 or More Consecutive Platelet Counts by Starting Dose Level and Overall
The percentage of participants who achieved 2 or more consecutive platelet counts, separated by at least 5 days, of >=50,000/ microliter (μL) and an increase of platelet count of >=20,000/μL from baseline, by starting dose level and overall, without use of rescue medication in the 4 weeks prior to the latest elevated platelet count. 95% confidence interval (CI) was based on the Clopper-Pearson method. The average of the 2 screening results and the Cycle 1 Day 1 result were used as the baseline value.
ITT population consisted of all participants who had enrolled into the study. It was prespecified (SAP), data was analyzed by overall and starting dose level.
Posted
Number
95% Confidence Interval
percentage of participants
Up to 24 Weeks
ID
Title
Description
OG000
Part A: Starting Dose 200 mg QD
Participants received rilzabrutinib 200 mg orally QD as starting dose from Day 1 to 4 weeks. Participants were allowed to up-titrate the dose levels unless withdrawn, had a platelet response to current dose level, or the next dose level had been determined to be ineligible for further enrollment every 4 weeks up to the maximum allowed 400 mg BID (800 mg/day) over 24 weeks of treatment period.
OG001
Part A: Starting Dose 400 mg QD
Participants received rilzabrutinib 400 mg orally QD as starting dose from Day 1 to 4 weeks. Participants were allowed to up-titrate the dose levels unless withdrawn, had a platelet response to current dose level, or the next dose level had been determined to be ineligible for further enrollment every 4 weeks up to the maximum allowed 400 mg BID (800 mg/day) over 24 weeks of treatment period.
OG002
Part A: Starting Dose 300 mg BID
Participants received rilzabrutinib 300 mg orally BID as starting dose from Day 1 to 4 weeks. Participants were allowed to up-titrate the dose levels unless withdrawn, had a platelet response to current dose level, or the next dose level had been determined to be ineligible for further enrollment every 4 weeks up to the maximum allowed 400 mg BID (800 mg/day) over 24 weeks of treatment period.
OG003
Part A: Starting Dose 400 mg BID
Participants received rilzabrutinib 400 mg orally BID as starting dose from Day 1 to 24 weeks, unless withdrawn or had a platelet response to current dose level.
OG004
Part A: Overall
All participants who received rilzabrutinib (200 mg QD or 400 mg QD or 300 mg BID or 400 mg BID) at all dose levels over 24 weeks of treatment period, unless withdrawn or had a platelet response to dose level.
Units
Counts
Participants
OG0009
OG0011
OG0025
OG003
Title
Denominators
Categories
Title
Measurements
OG00044.4(13.70 to 78.80)
OG0010.0(0.00 to 97.50)
OG00240.0(5.27 to 85.34)
OG003
Primary
Part B: Percentage of Participants Who Achieved Platelet Counts >=50,000/μL
The percentage of participants who achieved platelet counts >=50,000/μL on at least 8 out of the last 12 weeks of the 24-week treatment period without the use of rescue medication after 10 weeks of active treatment. 95% CI was based on the Clopper-Pearson exact method.
ITT population consisted of all enrolled participants.
Posted
Number
95% Confidence Interval
percentage of participants
Up to 24 Weeks
ID
Title
Description
OG000
Part B: Rilzabrutinib 400 mg BID
Participants received rilzabrutinib 400 mg orally BID from Day 1 up to 24 weeks of treatment period.
Units
Counts
Participants
OG000
Primary
Part A: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment Related Treatment-Emergent Adverse Events
Adverse event (AE): any untoward medical occurrence in participant or clinical study participant, temporally associated with the use of rilzabrutinib, whether or not considered related to rilzabrutinib. TEAEs: AEs that developed or worsened or became serious on or after the first dose administration of rilzabrutinib (Day 1). Any TEAEs are considered treatment-related TEAEs as per Investigator's evaluation of participant's circumstances surrounding the event, and an evaluation of any potential alternative causes to determine whether an TEAE can be considered as related to the rilzabrutinib.
Safety population consisted of all participants who received at least 1 dose of rilzabrutinib. It was prespecified (SAP), data was analyzed by overall and dose level and participants could be classified into multiple dose levels.
Posted
Count of Participants
Participants
From first dose of rilzabrutinib (Day 1) up to last dose + 1 (up to 294 days)
ID
Title
Description
OG000
Part A: Dose Level 200 mg QD
All participants who received rilzabrutinib 200 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG001
Part A: Dose Level 400 mg QD
All participants who received rilzabrutinib 400 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
Primary
Part B: Number of Participants With Treatment-Emergent Adverse Events and Treatment Related Treatment-Emergent Adverse Events
AE any untoward medical occurrence in participant or clinical study participant, temporally associated with the use of rilzabrutinib, whether or not considered related to rilzabrutinib. TEAEs: AEs that developed or worsened or became serious during the treatment-emergent period, defined as any time after the first dose administration of rilzabrutinib (Day 1). Any TEAEs are considered treatment-related TEAEs as per Investigator's evaluation of participant's circumstances surrounding the event, and an evaluation of any potential alternative causes to determine whether an TEAE can be considered as related to rilzabrutinib.
Safety population consisted of all participants who had taken rilzabrutinib, regardless of the amount administered.
Posted
Count of Participants
Participants
From first dose of rilzabrutinib (Day 1) up to last dose + 1 (approximately 170 days)
ID
Title
Description
OG000
Part B: Rilzabrutinib 400 mg BID
Participants received rilzabrutinib 400 mg orally BID from Day 1 up to 24 weeks of treatment period.
Units
Counts
Participants
Secondary
Part A: Percentage of Weeks With Platelet Counts >=50,000/μL by Starting Dose Level and Overall
The percentage of weeks in which participants achieved platelet counts >=50,000/μL in the treatment period are summarized here.
ITT population consisted of all participants who had enrolled into the study. Only participants with data collected at specified timepoints are reported. It was prespecified (SAP), data was analyzed by overall and starting dose level.
Posted
Mean
Standard Deviation
percentage of weeks
Up to 24 Weeks
ID
Title
Description
OG000
Part A: Starting Dose 200 mg QD
Participants received rilzabrutinib 200 mg orally QD as starting dose from Day 1 to 4 weeks. Participants were allowed to up-titrate the dose levels unless withdrawn, had a platelet response to current dose level, or the next dose level had been determined to be ineligible for further enrollment every 4 weeks up to the maximum allowed 400 mg BID (800 mg/day) over 24 weeks of treatment period.
OG001
Part A: Starting Dose 400 mg QD
Participants received rilzabrutinib 400 mg orally QD as starting dose from Day 1 to 4 weeks. Participants were allowed to up-titrate the dose levels unless withdrawn, had a platelet response to current dose level, or the next dose level had been determined to be ineligible for further enrollment every 4 weeks up to the maximum allowed 400 mg BID (800 mg/day) over 24 weeks of treatment period.
Secondary
Part A: Percentage of Participants With 4 Out of the Final 8 Platelet Counts >=50,000/μL by Starting Dose Level and Overall
The percentage of participants who had at least 4 out of the final 8 platelet counts >=50,000/μL are summarized here. The final 8 scheduled platelet counts are the measurements performed in the last 8 weeks of rilzabrutinib (depending on treatment duration, not necessarily from Week 19 to Week 24) in the treatment period. 95% CI was based on the Clopper-Pearson method.
ITT population consisted of all participants who had enrolled into the study. It was prespecified (SAP), data was analyzed by overall and starting dose level.
Posted
Number
95% Confidence Interval
percentage of participants
Up to 24 Weeks
ID
Title
Description
OG000
Part A: Starting Dose 200 mg QD
Participants received rilzabrutinib 200 mg orally QD as starting dose from Day 1 to 4 weeks. Participants were allowed to up-titrate the dose levels unless withdrawn, had a platelet response to current dose level, or the next dose level had been determined to be ineligible for further enrollment every 4 weeks up to the maximum allowed 400 mg BID (800 mg/day) over 24 weeks of treatment period.
OG001
Part A: Starting Dose 400 mg QD
Participants received rilzabrutinib 400 mg orally QD as starting dose from Day 1 to 4 weeks. Participants were allowed to up-titrate the dose levels unless withdrawn, had a platelet response to current dose level, or the next dose level had been determined to be ineligible for further enrollment every 4 weeks up to the maximum allowed 400 mg BID (800 mg/day) over 24 weeks of treatment period.
Secondary
Part A: Change From Baseline to the Average of Post Day 1 Platelet Counts by Dose Level and Overall
Average of post Day 1 platelet count is equivalent to average of (average of each participant's post-Day 1 platelet counts), included platelet counts up to 1 day after the date of last dose of rilzabrutinib and excluded platelet counts on or after date of rescue, if applicable. The average of the 2 screening results and the Cycle 1 Day 1 result measured on different date were used as the baseline value.
ITT population consisted of all participants who had enrolled into the study. It was prespecified (SAP), data was analyzed by overall and dose level and participants can be classified into multiple dose levels. Only participants with data who had more than 4 weeks of rilzabrutinib within a dose level are reported in each dose level. Participants with more than 4 weeks of study drug across all dose levels are included in overall.
Posted
Mean
Standard Deviation
platelets x10^9/Liter (L)
Baseline and up to 24 Weeks
ID
Title
Description
OG000
Part A: Dose Level 200 mg QD
All participants who received rilzabrutinib 200 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG001
Part A: Dose Level 400 mg QD
All participants who received rilzabrutinib 400 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
Secondary
Part A: Number of Weeks With Platelet Counts >=50,000/μL by Starting Dose Level and Overall
The number of weeks in which participant achieved platelet counts >=50,000/μL in the treatment period are summarized here. The number of weeks is based on the number of scheduled weekly assessments, by study day.
ITT population consisted of all participants who had enrolled into the study. Only participants with data collected at specified timepoints are reported. It was prespecified (SAP), data was analyzed by overall and starting dose level.
Posted
Mean
Standard Deviation
weeks
Up to 24 Weeks
ID
Title
Description
OG000
Part A: Starting Dose 200 mg QD
Participants received rilzabrutinib 200 mg orally QD as starting dose from Day 1 to 4 weeks. Participants were allowed to up-titrate the dose levels unless withdrawn, had a platelet response to current dose level, or the next dose level had been determined to be ineligible for further enrollment every 4 weeks up to the maximum allowed 400 mg BID (800 mg/day) over 24 weeks of treatment period.
OG001
Part A: Starting Dose 400 mg QD
Participants received rilzabrutinib 400 mg orally QD as starting dose from Day 1 to 4 weeks. Participants were allowed to up-titrate the dose levels unless withdrawn, had a platelet response to current dose level, or the next dose level had been determined to be ineligible for further enrollment every 4 weeks up to the maximum allowed 400 mg BID (800 mg/day) over 24 weeks of treatment period.
Secondary
Part A: Number of Weeks With Platelet Counts >=30,000/μL by Starting Dose Level and Overall
The number of weeks in which participant achieved platelet counts >=30,000/μL in the treatment period are summarized here. The number of weeks is based on the number of scheduled weekly assessments, by study day.
ITT population consisted of all participants who had enrolled into the study. Only participants with data collected at specified timepoints are reported. It was prespecified (SAP), data was analyzed by overall and starting dose level.
Posted
Mean
Standard Deviation
weeks
Up to 24 Weeks
ID
Title
Description
OG000
Part A: Starting Dose 200 mg QD
Participants received rilzabrutinib 200 mg orally QD as starting dose from Day 1 to 4 weeks. Participants were allowed to up-titrate the dose levels unless withdrawn, had a platelet response to current dose level, or the next dose level had been determined to be ineligible for further enrollment every 4 weeks up to the maximum allowed 400 mg BID (800 mg/day) over 24 weeks of treatment period.
OG001
Part A: Starting Dose 400 mg QD
Participants received rilzabrutinib 400 mg orally QD as starting dose from Day 1 to 4 weeks. Participants were allowed to up-titrate the dose levels unless withdrawn, had a platelet response to current dose level, or the next dose level had been determined to be ineligible for further enrollment every 4 weeks up to the maximum allowed 400 mg BID (800 mg/day) over 24 weeks of treatment period.
Secondary
Part A: Time to First Platelet Count >=50,000/μL Across All Dose Levels
Time to first platelet count >=50,000/μL during the treatment period in days was calculated as: (date of first occurrence of platelet count >=50,000/μL - date of first rilzabrutinib dosing) +1.
ITT population consisted of all participants who had enrolled into the study. It was prespecified (protocol and SAP) that data will be analyzed for overall across all dose levels, so combined data reported here. Only participants with data collected at specified timepoints are reported.
Posted
Mean
Standard Deviation
days
Up to 24 Weeks
ID
Title
Description
OG000
Part A: Overall Participants
All participants who received rilzabrutinib (200 mg QD or 400 mg QD or 300 mg BID or 400 mg BID) at all dose levels over 24 weeks of treatment period, unless withdrawn or had a platelet response to dose level.
Units
Counts
Participants
OG000
Secondary
Part B: Number of Weeks With Platelet Counts >= 50,000/μL or >= 30,000/μL and Doubling the Baseline
The number of weeks in which participant achieved platelet counts with thresholds as: >=50,000/μL or >=30,000/μL and doubling the baseline in the absence of rescue therapy (platelet counts will be censored for 4 weeks after the use of rescue medication, if given) are summarized here. Baseline is defined as the average of 3 platelet counts: 2 qualified screening platelet counts collected in electronic case report form (eCRF) and Week 1 (study day 1) platelet count.
ITT population consisted of all enrolled participants.
Posted
Mean
Standard Deviation
weeks
Up to 24 Weeks
ID
Title
Description
OG000
Part B: Rilzabrutinib 400 mg BID
Participants received rilzabrutinib 400 mg orally BID from Day 1 up to 24 weeks of treatment period.
Units
Counts
Participants
OG000
Secondary
Part B: Percentage of Participants Who Achieved 2 or More Consecutive Platelet Counts
Percentage of participants who achieved 2 or more consecutive platelet counts, separated by at least 5 days, of >=50,000/μL and an increase of platelet count of >=20,000/μL from baseline without use of rescue medication in the 4 weeks prior to the latest elevated platelet count are summarized here. Baseline is defined as the average of 3 platelet counts: 2 qualified screening platelet counts collected in eCRF and Week 1 (study day 1) platelet count. 95% CI was based on the Clopper-Pearson exact method.
ITT population consisted of all enrolled participants.
Posted
Number
95% Confidence Interval
percentage of participants
Up to 24 Weeks
ID
Title
Description
OG000
Part B: Rilzabrutinib 400 mg BID
Participants received rilzabrutinib 400 mg orally BID from Day 1 up to 24 weeks of treatment period.
Units
Counts
Participants
OG000
Secondary
Part B: Number of Weeks With Platelet Counts >=30,000/μL and Doubling the Baseline
The number of weeks in which participant achieved platelet counts >=30,000/μL and doubling the baseline in the absence of rescue therapy (platelet counts will be censored for 4 weeks after the use of rescue medication, if given) are summarized here. Baseline is defined as the average of 3 platelet counts: 2 qualified screening platelet counts collected in eCRF and Week 1 (study day 1) platelet count.
ITT population consisted of all enrolled participants.
Posted
Mean
Standard Deviation
weeks
Up to 24 Weeks
ID
Title
Description
OG000
Part B: Rilzabrutinib 400 mg BID
Participants received rilzabrutinib 400 mg orally BID from Day 1 up to 24 weeks of treatment period.
Units
Counts
Participants
OG000
Secondary
Part B: Percentage of Participants Who Received Rescue Medication
Rescue medication is defined as any therapy used to rescue a participant (1 of intravenous immunoglobulin [IVIG], high-dose steroids, platelet infusion or anti-D immunoglobulin infusion). Percentage of participants who received rescue medication are summarized here. 95% CI was based on the Clopper-Pearson exact method.
ITT population consisted of all enrolled participants.
Posted
Number
95% Confidence Interval
percentage of participants
Up to 24 Weeks
ID
Title
Description
OG000
Part B: Rilzabrutinib 400 mg BID
Participants received rilzabrutinib 400 mg orally BID from Day 1 up to 24 weeks of treatment period.
Units
Counts
Participants
OG000
Secondary
Part B: Change From Baseline in Idiopathic Thrombocytopenic Purpura Bleeding Scale (IBLS)
The IBLS is a bleeding assessment score. IBLS comprises of 11 sites for female and 10 sites for male, and each site is scored from 0 (none) to 2 (marked bleeding). The total overall score ranges from 0-22, with higher scores indicating higher presence of marked bleeding. For each participant, an IBLS score at each visit was calculated by taking the average across 11 items (10 for male and postmenopausal women) at 9 anatomical sites (8 for male and postmenopausal women). For each participant, a mean IBLS score was also calculated by taking the average across all post-baseline visits during the 24-week treatment period. IBLS average value ranges from 0 to 2. The smaller the IBLS average value is, the healthier the participants are. For change from baseline, negative value indicates an improvement. The baseline value is defined as the last available value before the first dose rilzabrutinib.
ITT population consisted of all enrolled participants. Only participants with data collected at specified timepoints are reported.
Posted
Mean
Standard Deviation
score on a scale
Baseline and up to 24 weeks
ID
Title
Description
OG000
Part B: Rilzabrutinib 400 mg BID
Participants received rilzabrutinib 400 mg orally BID from Day 1 up to 24 weeks of treatment period.
Units
Counts
Secondary
Part A: Percentage of Participants Who Received Rescue Medication by Dose Levels and Overall
Rescue medication is defined as any therapy used to rescue a participant (1 of IVIG, high-dose steroids, platelet infusion or anti-D immunoglobulin infusion). The percentage of participants who received rescue medication for each dose level and overall are summarized here. 95% CI was based on the Clopper-Pearson method.
Safety population consisted of all participants who received at least 1 dose of rilzabrutinib. Participants are classified into different dose levels according to the dose received at the start of rescue medication. A given participant can be classified into multiple dose levels. It was prespecified (SAP), data was analyzed by overall and dose level.
Posted
Number
95% Confidence Interval
percentage of participants
Up to 24 Weeks
ID
Title
Description
OG000
Part A: Dose Level 200 mg QD
All participants who received rilzabrutinib 200 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG001
Part A: Dose Level 400 mg QD
All participants who received rilzabrutinib 400 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG002
Part A: Dose Level 300 mg BID
Secondary
Part A: Percentage of Participants With Grade 2 or Higher Bleeding Event by Dose Level and Overall
The percentage of participants with intensity grade 2 or higher bleeding event are summarized for each dose level and overall. The TEAEs with standardized medical dictionary for regulatory activities (MedDRA) query (SMQ) hemorrhages were medically determined for analysis of bleeding events. 95% CI was based on the Clopper-Pearson method.
Safety population consisted of all participants who received at least 1 dose of rilzabrutinib. Participants are classified into different dose levels according to the dose received at the start of the bleeding event. A given participant can be classified into multiple dose levels. It was prespecified (SAP), data was analyzed by overall and dose level.
Posted
Number
95% Confidence Interval
percentage of participants
Up to 24 Weeks
ID
Title
Description
OG000
Part A: Dose Level 200 mg QD
All participants who received rilzabrutinib 200 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG001
Part A: Dose Level 400 mg QD
All participants who received rilzabrutinib 400 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG002
Secondary
Part A: Number of Participants With Idiopathic Thrombocytopenic Purpura/Immune Thrombocytopenia (ITP) Bleeding Assessment Tool (ITP-BAT) Scale by Dose Level
The ITP-BAT scale comprises of 11 grades from 0 (none) to 2 (marked bleeding), with higher scores indicating higher presence of marked bleeding, assessed at 9 anatomical sites (skin, oral, epistaxis, gastrointestinal [GI], urinary, gynecological [GYN], pulmonary, intracranial hemorrhage [HEM], subconjunctival HEM) by history over the previous week (Hx). In addition, 2 sites (skin and oral), were also assessed by physical examination (PE). The 'worst ever' bleeding experienced at each site was graded using the same system. Here, 0 indicates none; 1 indicates 1-5 bruises and/or scattered petechiae and 2 indicates >5 bruises with size >2 centimeter (cm) and/or diffuse petechiae. Each participant summed up the transformed scores across all 11 sites per visit assessment. The total overall score ranges from 0-22 with the higher score indicating worst outcome.
Safety population consisted of all participants who received at least 1 dose of rilzabrutinib. It was prespecified (SAP), data was analyzed by dose level and participants can be classified into multiple dose levels.
Posted
Count of Participants
Participants
Up to 24 Weeks
ID
Title
Description
OG000
Part A: Dose Level 200 mg QD
All participants who received rilzabrutinib 200 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG001
Part A: Dose Level 400 mg QD
Secondary
Part A: Maximum Observed Plasma Concentration (Cmax) of Rilzabrutinib
Plasma samples were collected at specified timepoints to determine Cmax of rilzabrutinib.
ITT population consisted of all participants who had enrolled into the study. It was prespecified (SAP), data was analyzed based on frequent sampling on Day 1 of a new, higher dosing level and reported by dose. Only participants who received rilzabrutinib with data collected at specified timepoints are reported.
Posted
Mean
Standard Deviation
nanogram per milliliter (ng/mL)
Day 1 of Cycles 1, 2, 3, and 5 (each cycle 28 days)
ID
Title
Description
OG000
Part A: Dose Level 200 mg QD
All participants who received rilzabrutinib 200 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG001
Part A: Dose Level 400 mg QD
All participants who received rilzabrutinib 400 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG002
Part A: Dose Level 300 mg BID
All participants who received rilzabrutinib 300 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
Secondary
Part A: Time of Observed Maximum Plasma Concentration (Tmax) of Rilzabrutinib
Plasma samples were collected at specified timepoints to determine tmax of rilzabrutinib.
ITT population consisted of all participants who had enrolled into the study. It was prespecified (SAP), data was analyzed based on frequent sampling on Day 1 of a new, higher dosing level and reported by dose. Only participants who received rilzabrutinib with data collected at specified timepoints are reported.
Posted
Median
Full Range
hours (h)
Day 1 of Cycles 1, 2, 3, and 5 (each cycle 28 days)
ID
Title
Description
OG000
Part A: Dose Level 200 mg QD
All participants who received rilzabrutinib 200 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG001
Part A: Dose Level 400 mg QD
All participants who received rilzabrutinib 400 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG002
Part A: Dose Level 300 mg BID
All participants who received rilzabrutinib 300 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
Secondary
Part A: Area Under the Plasma Concentration-Time Curve From Time Zero to Last Quantifiable Concentration (AUClast) of Rilzabrutinib
Plasma samples were collected at specified timepoints to determine AUClast of rilzabrutinib.
ITT population consisted of all participants who had enrolled into the study. It was prespecified (SAP), data was analyzed based on frequent sampling on Day 1 of a new, higher dosing level and reported by dose. Only participants who received rilzabrutinib with data collected at specified timepoints are reported.
Posted
Mean
Standard Deviation
h*ng/mL
Day 1 of Cycles 1, 2, 3, and 5 (each cycle 28 days)
ID
Title
Description
OG000
Part A: Dose Level 200 mg QD
All participants who received rilzabrutinib 200 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG001
Part A: Dose Level 400 mg QD
All participants who received rilzabrutinib 400 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG002
Part A: Dose Level 300 mg BID
All participants who received rilzabrutinib 300 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
Secondary
Part A: Area Under the Plasma Concentration-Time Curve From Time Zero to Infinity (AUCinf) of Rilzabrutinib
Plasma samples were collected at specified timepoints to determine AUCinf of rilzabrutinib.
ITT population consisted of all participants who had enrolled into the study. It was prespecified (SAP), data was analyzed based on frequent sampling on Day 1 of a new, higher dosing level and reported by dose. Only participants who received rilzabrutinib with data collected at specified timepoints are reported.
Posted
Mean
Standard Deviation
h*ng/mL
Day 1 of Cycles 1, 2, and 3 (each cycle 28 days)
ID
Title
Description
OG000
Part A: Dose Level 200 mg QD
All participants who received rilzabrutinib 200 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG001
Part A: Dose Level 400 mg QD
All participants who received rilzabrutinib 400 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG002
Part A: Dose Level 300 mg BID
All participants who received rilzabrutinib 300 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
Secondary
Part A: Elimination Half-Life (t1/2) of Rilzabrutinib
Plasma samples were collected at specified timepoints to determine t1/2 of rilzabrutinib.
ITT population consisted of all participants who had enrolled into the study. It was prespecified (SAP), data was analyzed based on frequent sampling on Day 1 of a new, higher dosing level and reported by dose. Only participants who received rilzabrutinib with data collected at specified timepoints are reported.
Posted
Median
Full Range
hours
Day 1 of Cycles 1, 2, and 3 (each cycle 28 days)
ID
Title
Description
OG000
Part A: Dose Level 200 mg QD
All participants who received rilzabrutinib 200 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG001
Part A: Dose Level 400 mg QD
All participants who received rilzabrutinib 400 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG002
Part A: Dose Level 300 mg BID
All participants who received rilzabrutinib 300 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
Secondary
Part A: Apparent Volume of Distribution of the Drug After Oral Administration (Vz/F) of Rilzabrutinib
Plasma samples were collected at specified timepoints to determine Vz/F of rilzabrutinib.
ITT population consisted of all participants who had enrolled into the study. It was prespecified (SAP), data was analyzed based on frequent sampling on Day 1 of a new, higher dosing level and reported by dose. Only participants who received rilzabrutinib with data collected at specified timepoints are reported.
Posted
Mean
Standard Deviation
Liters
Day 1 of Cycles 1, 2, and 3 (each cycle 28 days)
ID
Title
Description
OG000
Part A: Dose Level 200 mg QD
All participants who received rilzabrutinib 200 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG001
Part A: Dose Level 400 mg QD
All participants who received rilzabrutinib 400 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG002
Part A: Dose Level 300 mg BID
All participants who received rilzabrutinib 300 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
Secondary
Part A: Apparent Total Clearance of the Drug From Plasma After Oral Administration (CL/F) of Rilzabrutinib
Plasma samples were collected at specified timepoints to determine CL/F of rilzabrutinib.
ITT population consisted of all participants who had enrolled into the study. It was prespecified (SAP), data was analyzed based on frequent sampling on Day 1 of a new, higher dosing level and reported by dose. Only participants who received rilzabrutinib with data collected at specified timepoints are reported.
Posted
Mean
Standard Deviation
liters/hour
Day 1 of Cycles 1, 2, and 3 (each cycle 28 days)
ID
Title
Description
OG000
Part A: Dose Level 200 mg QD
All participants who received rilzabrutinib 200 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG001
Part A: Dose Level 400 mg QD
All participants who received rilzabrutinib 400 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG002
Part A: Dose Level 300 mg BID
All participants who received rilzabrutinib 300 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
Secondary
Part B: Plasma Concentration of Rilzabrutinib
Plasma samples were collected at specified timepoints for evaluation of rilzabrutinib pharmacokinetic (PK) concentrations.
PK population consisted of all enrolled participants from the safety population who had at least 1 post-baseline PK result.
Posted
Mean
Standard Deviation
ng/mL
Pre-dose and 2 hours post-dose on Days 1, 29, and 57
ID
Title
Description
OG000
Part B: Rilzabrutinib 400 mg BID
Participants received rilzabrutinib 400 mg orally BID from Day 1 up to 24 weeks of treatment period.
Units
Counts
Participants
OG000
Time Frame
Adverse events data was collected from first dose of rilzabrutinib (Day 1) up to last dose + 1, approximately up to 294 days (Part A) and up to 170 days (Part B). All-cause mortality (death) was assessed from the signing of the ICF up to primary completion date (31 January 2023) approximately 253 weeks.
Description
Analysis was performed on safety population. As pre-specified (SAP), AEs were presented by dose level and overall . A participants could be classified into multiple dose levels.
All-Cause Mortality Comment
Not provided
Arm/Groups
ID
Title
Description
Deaths (Affected)
Deaths (At Risk)
Serious Events (Affected)
Serious Events (At Risk)
Other Events (Affected)
Other Events (At Risk)
EG000
Part A: Dose Level 200 mg QD
All participants who received rilzabrutinib 200 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
0
9
1
9
5
9
EG001
Part A: Dose Level 400 mg QD
All participants who received rilzabrutinib 400 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
0
8
2
8
6
8
EG002
Part A: Dose Level 300 mg BID
All participants who received rilzabrutinib 300 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
0
12
3
12
9
12
EG003
Part A: Dose Level 400 mg BID
All participants who received rilzabrutinib 400 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
1
52
9
52
43
52
EG004
Part A: Overall
All participants who received rilzabrutinib (200 mg QD or 400 mg QD or 300 mg BID or 400 mg BID) at all dose levels over 24 weeks of treatment period, unless withdrawn or had a platelet response to dose level.
1
60
11
60
48
60
EG005
Part B: 400 mg BID
Participants received rilzabrutinib 400 mg orally BID from Day 1 up to 24 weeks of treatment period.
0
26
3
26
21
26
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Covid-19 Pneumonia
Infections and infestations
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0020 events0 affected12 at risk
EG0030 events0 affected52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
Pneumonia
Infections and infestations
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Subcutaneous Abscess
Infections and infestations
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Evans Syndrome
Blood and lymphatic system disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Immune Thrombocytopenia
Blood and lymphatic system disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Thrombocytopenia
Blood and lymphatic system disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Syncope
Nervous system disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Transient Ischaemic Attack
Nervous system disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Iridocyclitis
Eye disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0011 events1 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Pulmonary Embolism
Respiratory, thoracic and mediastinal disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Gastrointestinal Haemorrhage
Gastrointestinal disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Rectal Haemorrhage
Gastrointestinal disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Contusion
Injury, poisoning and procedural complications
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Post Procedural Haemorrhage
Injury, poisoning and procedural complications
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Traumatic Haematoma
Injury, poisoning and procedural complications
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Covid-19
Infections and infestations
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG0031 events1 affected52 at risk
EG0041 events1 affected60 at risk
EG0056 events6 affected26 at risk
Nasopharyngitis
Infections and infestations
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Upper Respiratory Tract Infection
Infections and infestations
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Anaemia
Blood and lymphatic system disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Haemorrhagic Diathesis
Blood and lymphatic system disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Abnormal Dreams
Psychiatric disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Anxiety
Psychiatric disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Headache
Nervous system disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Conjunctival Haemorrhage
Eye disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Iridocyclitis
Eye disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0011 events1 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Ear Pruritus
Ear and labyrinth disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Palpitations
Cardiac disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Haematoma
Vascular disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Hypertension
Vascular disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Cough
Respiratory, thoracic and mediastinal disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Epistaxis
Respiratory, thoracic and mediastinal disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Nasal Congestion
Respiratory, thoracic and mediastinal disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Oropharyngeal Pain
Respiratory, thoracic and mediastinal disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Abdominal Discomfort
Gastrointestinal disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Abdominal Distension
Gastrointestinal disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0022 events2 affected12 at risk
EG003
Abdominal Pain
Gastrointestinal disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Abdominal Pain Upper
Gastrointestinal disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Aphthous Ulcer
Gastrointestinal disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Diarrhoea
Gastrointestinal disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0002 events2 affected9 at risk
EG0012 events2 affected8 at risk
EG0023 events3 affected12 at risk
EG003
Dyspepsia
Gastrointestinal disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0022 events2 affected12 at risk
EG003
Gastrooesophageal Reflux Disease
Gastrointestinal disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Gingival Bleeding
Gastrointestinal disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Inguinal Hernia
Gastrointestinal disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Loose Tooth
Gastrointestinal disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Nausea
Gastrointestinal disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0002 events2 affected9 at risk
EG0012 events2 affected8 at risk
EG0024 events4 affected12 at risk
EG003
Rectal Haemorrhage
Gastrointestinal disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Toothache
Gastrointestinal disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Vomiting
Gastrointestinal disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Alopecia
Skin and subcutaneous tissue disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Petechiae
Skin and subcutaneous tissue disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Pruritus
Skin and subcutaneous tissue disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Purpura
Skin and subcutaneous tissue disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Rash
Skin and subcutaneous tissue disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Scab
Skin and subcutaneous tissue disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Arthralgia
Musculoskeletal and connective tissue disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Back Pain
Musculoskeletal and connective tissue disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Bone Pain
Musculoskeletal and connective tissue disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Joint Swelling
Musculoskeletal and connective tissue disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Myalgia
Musculoskeletal and connective tissue disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Osteonecrosis
Musculoskeletal and connective tissue disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Micturition Urgency
Renal and urinary disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Fatigue
General disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0002 events2 affected9 at risk
EG0012 events2 affected8 at risk
EG0022 events2 affected12 at risk
EG003
Influenza Like Illness
General disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Malaise
General disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Peripheral Swelling
General disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Vessel Puncture Site Bruise
General disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0002 events1 affected9 at risk
EG0012 events1 affected8 at risk
EG0022 events1 affected12 at risk
EG003
Vessel Puncture Site Discharge
General disorders
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Alanine Aminotransferase Increased
Investigations
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Aspartate Aminotransferase Increased
Investigations
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0000 events0 affected9 at risk
EG0010 events0 affected8 at risk
EG0020 events0 affected12 at risk
EG003
Arthropod Bite
Injury, poisoning and procedural complications
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0022 events2 affected12 at risk
EG003
Contusion
Injury, poisoning and procedural complications
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0007 events3 affected9 at risk
EG0017 events3 affected8 at risk
EG0027 events3 affected12 at risk
EG003
Humerus Fracture
Injury, poisoning and procedural complications
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Limb Injury
Injury, poisoning and procedural complications
MedDRA24;MedDRA 25.1
Systematic Assessment
EG0001 events1 affected9 at risk
EG0011 events1 affected8 at risk
EG0021 events1 affected12 at risk
EG003
Certain Agreements
Are all PI(s) employees of the sponsor?
No
Restriction Type
OTHER
Results Disclosure Restriction on PI(s)?
Yes
Other Details
The Sponsor supports publication of clinical trial results but may request that investigators temporarily delay or alter publications in order to protect proprietary information. The Sponsor may also require that the results of multicenter studies be published only in their entirety and not as individual site data.
All participants who received rilzabrutinib 300 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG003
Part A: Dose Level 400 mg BID
All participants who received rilzabrutinib 400 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG004
Part A: Overall
All participants who received rilzabrutinib (200 mg QD or 400 mg QD or 300 mg BID or 400 mg BID) at all dose levels over 24 weeks of treatment period, unless withdrawn or had a platelet response to dose level.
Units
Counts
Participants
OG0009
OG0018
OG00212
OG00352
OG00460
Title
Denominators
Categories
TEAEs
Title
Measurements
OG0003
OG0013
OG0025
OG00344
OG00448
Treatment related TEAEs
Title
Measurements
OG0002
OG0012
OG0022
OG003
OG000
26
Title
Denominators
Categories
TEAEs
Title
Measurements
OG00022
Treatment related TEAEs
Title
Measurements
OG00016
OG002
Part A: Starting Dose 300 mg BID
Participants received rilzabrutinib 300 mg orally BID as starting dose from Day 1 to 4 weeks. Participants were allowed to up-titrate the dose levels unless withdrawn, had a platelet response to current dose level, or the next dose level had been determined to be ineligible for further enrollment every 4 weeks up to the maximum allowed 400 mg BID (800 mg/day) over 24 weeks of treatment period.
OG003
Part A: Starting Dose 400 mg BID
Participants received rilzabrutinib 400 mg orally BID as starting dose from Day 1 to 24 weeks, unless withdrawn or had a platelet response to current dose level.
OG004
Part A: Overall
All participants who received rilzabrutinib (200 mg QD or 400 mg QD or 300 mg BID or 400 mg BID) at all dose levels over 24 weeks of treatment period, unless withdrawn or had a platelet response to dose level.
Units
Counts
Participants
OG0008
OG0011
OG0025
OG00345
OG00459
Title
Denominators
Categories
Title
Measurements
OG00027.92± 29.42
OG0010.00± NAStandard deviation (SD) cannot be calculated for a single participant.
OG00239.58± 41.30
OG00328.14± 36.41
OG00428.60± 35.39
OG002
Part A: Starting Dose 300 mg BID
Participants received rilzabrutinib 300 mg orally BID as starting dose from Day 1 to 4 weeks. Participants were allowed to up-titrate the dose levels unless withdrawn, had a platelet response to current dose level, or the next dose level had been determined to be ineligible for further enrollment every 4 weeks up to the maximum allowed 400 mg BID (800 mg/day) over 24 weeks of treatment period.
OG003
Part A: Starting Dose 400 mg BID
Participants received rilzabrutinib 400 mg orally BID as starting dose from Day 1 to 24 weeks, unless withdrawn or had a platelet response to current dose level.
OG004
Part A: Overall
All participants who received rilzabrutinib (200 mg QD or 400 mg QD or 300 mg BID or 400 mg BID) at all dose levels over 24 weeks of treatment period, unless withdrawn or had a platelet response to dose level.
Units
Counts
Participants
OG0009
OG0011
OG0025
OG00345
OG00460
Title
Denominators
Categories
Title
Measurements
OG00011.1(0.28 to 48.25)
OG0010.0(0.00 to 97.50)
OG00240.0(5.27 to 85.34)
OG00331.1(18.17 to 46.65)
OG00428.3(17.45 to 41.44)
OG002
Part A: Dose Level 300 mg BID
All participants who received rilzabrutinib 300 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG003
Part A: Dose Level 400 mg BID
All participants who received rilzabrutinib 400 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG004
Part A: Overall
All participants who received rilzabrutinib (200 mg QD or 400 mg QD or 300 mg BID or 400 mg BID) at all dose levels over 24 weeks of treatment period, unless withdrawn or had a platelet response to dose level.
Units
Counts
Participants
OG0003
OG0011
OG0026
OG00345
OG00454
Title
Denominators
Categories
Title
Measurements
OG0006.92± 22.52
OG00168.10± NASD cannot be calculated for a single participant.
OG00237.05± 29.10
OG00329.92± 41.70
OG00428.61± 39.57
OG002
Part A: Starting Dose 300 mg BID
Participants received rilzabrutinib 300 mg orally BID as starting dose from Day 1 to 4 weeks. Participants were allowed to up-titrate the dose levels unless withdrawn, had a platelet response to current dose level, or the next dose level had been determined to be ineligible for further enrollment every 4 weeks up to the maximum allowed 400 mg BID (800 mg/day) over 24 weeks of treatment period.
OG003
Part A: Starting Dose 400 mg BID
Participants received rilzabrutinib 400 mg orally BID as starting dose from Day 1 to 24 weeks, unless withdrawn or had a platelet response to current dose level.
OG004
Part A: Overall
All participants who received rilzabrutinib (200 mg QD or 400 mg QD or 300 mg BID or 400 mg BID) at all dose levels over 24 weeks of treatment period, unless withdrawn or had a platelet response to dose level.
Units
Counts
Participants
OG0008
OG0011
OG0025
OG00345
OG00459
Title
Denominators
Categories
Title
Measurements
OG0005.9± 7.8
OG0010.0± NASD cannot be calculated for a single participant.
OG00210.2± 12.0
OG0035.7± 8.1
OG0046.0± 8.3
OG002
Part A: Starting Dose 300 mg BID
Participants received rilzabrutinib 300 mg orally BID as starting dose from Day 1 to 4 weeks. Participants were allowed to up-titrate the dose levels unless withdrawn, had a platelet response to current dose level, or the next dose level had been determined to be ineligible for further enrollment every 4 weeks up to the maximum allowed 400 mg BID (800 mg/day) over 24 weeks of treatment period.
OG003
Part A: Starting Dose 400 mg BID
Participants received rilzabrutinib 400 mg orally BID as starting dose from Day 1 to 24 weeks, unless withdrawn or had a platelet response to current dose level.
OG004
Part A: Overall
All participants who received rilzabrutinib (200 mg QD or 400 mg QD or 300 mg BID or 400 mg BID) at all dose levels over 24 weeks of treatment period, unless withdrawn or had a platelet response to dose level.
Units
Counts
Participants
OG0008
OG0011
OG0025
OG00345
OG00459
Title
Denominators
Categories
Title
Measurements
OG0008.4± 11.2
OG0010.0± NASD cannot be calculated for a single participant.
OG00214.0± 14.1
OG0038.8± 9.2
OG0049.0± 9.8
30
Title
Denominators
Categories
Title
Measurements
OG00027.4± 31.86
26
Title
Denominators
Categories
Title
Measurements
OG0009.3± 10.1
26
Title
Denominators
Categories
Title
Measurements
OG00042.3(23.35 to 63.08)
26
Title
Denominators
Categories
Title
Measurements
OG0009.3± 10.1
26
Title
Denominators
Categories
Title
Measurements
OG00011.5(2.45 to 30.15)
Participants
OG00024
Title
Denominators
Categories
Title
Measurements
OG000-0.07± 0.13
All participants who received rilzabrutinib 300 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG003
Part A: Dose Level 400 mg BID
All participants who received rilzabrutinib 400 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG004
Part A: Overall
All participants who received rilzabrutinib (200 mg QD or 400 mg QD or 300 mg BID or 400 mg BID) at all dose levels over 24 weeks of treatment period, unless withdrawn or had a platelet response to dose level.
Units
Counts
Participants
OG0009
OG0018
OG00212
OG00352
OG00460
Title
Denominators
Categories
Title
Measurements
OG00011.1(0.28 to 48.25)
OG0010.0(0.00 to 36.94)
OG0028.3(0.21 to 38.48)
OG0039.6(3.20 to 21.03)
OG00411.7(4.82 to 22.57)
Part A: Dose Level 300 mg BID
All participants who received rilzabrutinib 300 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG003
Part A: Dose Level 400 mg BID
All participants who received rilzabrutinib 400 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG004
Part A: Overall
All participants who received rilzabrutinib (200 mg QD or 400 mg QD or 300 mg BID or 400 mg BID) at all dose levels over 24 weeks of treatment period, unless withdrawn or had a platelet response to dose level.
Units
Counts
Participants
OG0009
OG0018
OG00212
OG00352
OG00460
Title
Denominators
Categories
Title
Measurements
OG0000.0(0.00 to 33.63)
OG0010.0(0.00 to 36.94)
OG0028.3(0.21 to 38.48)
OG00317.3(8.23 to 30.33)
OG00416.7(8.29 to 28.52)
All participants who received rilzabrutinib 400 mg orally QD over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG002
Part A: Dose Level 300 mg BID
All participants who received rilzabrutinib 300 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
OG003
Part A: Dose Level 400 mg BID
All participants who received rilzabrutinib 400 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
Units
Counts
Participants
OG0009
OG0018
OG00212
OG00352
Title
Denominators
Categories
Skin (PE): Score 0
Title
Measurements
OG0007
OG0018
OG0026
OG00332
Skin (PE): Score 1
Title
Measurements
OG0000
OG0010
OG0023
OG003
Skin (PE): Score 2
Title
Measurements
OG0000
OG0010
OG0020
OG003
Skin (PE): Scoring not done/missing
Title
Measurements
OG0002
OG0010
OG0023
OG003
Oral (PE): Score 0
Title
Measurements
OG0007
OG0018
OG0029
OG003
Oral (PE): Score 1
Title
Measurements
OG0000
OG0010
OG0020
OG003
Oral (PE): Score 2
Title
Measurements
OG0000
OG0010
OG0020
OG003
Oral (PE): Scoring not done/missing
Title
Measurements
OG0002
OG0010
OG0023
OG003
Skin (Hx): Score 0
Title
Measurements
OG0007
OG0018
OG0025
OG003
Skin (Hx): Score 1
Title
Measurements
OG0000
OG0010
OG0024
OG003
Skin (Hx): Score 2
Title
Measurements
OG0000
OG0010
OG0020
OG003
Skin (Hx): Scoring not done/missing
Title
Measurements
OG0002
OG0010
OG0023
OG003
Oral (Hx): Score 0
Title
Measurements
OG0007
OG0018
OG0029
OG003
Oral (Hx): Score 1
Title
Measurements
OG0000
OG0010
OG0020
OG003
Oral (Hx): Score 2
Title
Measurements
OG0000
OG0010
OG0020
OG003
Oral (Hx): Scoring not done/missing
Title
Measurements
OG0002
OG0010
OG0023
OG003
Epistaxis: Score 0
Title
Measurements
OG0007
OG0018
OG0029
OG003
Epistaxis: Score 1
Title
Measurements
OG0000
OG0010
OG0020
OG003
Epistaxis: Score 2
Title
Measurements
OG0000
OG0010
OG0020
OG003
Epistaxis: Scoring not done/missing
Title
Measurements
OG0002
OG0010
OG0023
OG003
GI: Score 0
Title
Measurements
OG0007
OG0018
OG00210
OG003
GI: Score 1
Title
Measurements
OG0000
OG0010
OG0020
OG003
GI: Score 2
Title
Measurements
OG0000
OG0010
OG0020
OG003
GI: Scoring not done/missing
Title
Measurements
OG0002
OG0010
OG0022
OG003
Urinary: Score 0
Title
Measurements
OG0007
OG0018
OG0029
OG003
Urinary: Score 1
Title
Measurements
OG0000
OG0010
OG0020
OG003
Urinary: Score 2
Title
Measurements
OG0000
OG0010
OG0020
OG003
Urinary: Scoring not done/missing
Title
Measurements
OG0002
OG0010
OG0023
OG003
GYN: Score 0
Title
Measurements
OG0001
OG0011
OG0024
OG003
GYN: Score 1
Title
Measurements
OG0000
OG0010
OG0020
OG003
GYN: Score 2
Title
Measurements
OG0000
OG0010
OG0020
OG003
GYN: Scoring not done/missing
Title
Measurements
OG0008
OG0017
OG0028
OG003
Pulmonary: Score 0
Title
Measurements
OG0006
OG0018
OG0028
OG003
Pulmonary: Score 1
Title
Measurements
OG0000
OG0010
OG0020
OG003
Pulmonary: Score 2
Title
Measurements
OG0000
OG0010
OG0020
OG003
Pulmonary: Scoring not done/missing
Title
Measurements
OG0003
OG0010
OG0024
OG003
Intracranial HEM: Score 0
Title
Measurements
OG0007
OG0018
OG0028
OG003
Intracranial HEM: Score 1
Title
Measurements
OG0000
OG0010
OG0020
OG003
Intracranial HEM: Score 2
Title
Measurements
OG0000
OG0010
OG0020
OG003
Intracranial HEM: Scoring not done/missing
Title
Measurements
OG0002
OG0010
OG0024
OG003
Subconjunctival HEM: Score 0
Title
Measurements
OG0007
OG0018
OG0028
OG003
Subconjunctival HEM: Score 1
Title
Measurements
OG0000
OG0010
OG0020
OG003
Subconjunctival HEM: Score 2
Title
Measurements
OG0000
OG0010
OG0020
OG003
Subconjunctival HEM: Scoring not done/missing
Title
Measurements
OG0002
OG0010
OG0024
OG003
OG003
Part A: Dose Level 400 mg BID
All participants who received rilzabrutinib 400 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
Units
Counts
Participants
OG0009
OG0017
OG0029
OG00343
Title
Denominators
Categories
Cycle 1 Day 1
ParticipantsOG0009
ParticipantsOG0010
ParticipantsOG0025
ParticipantsOG00343
Title
Measurements
OG000154± 117
OG002287± 149
OG003273± 203
Cycle 2 Day 1
ParticipantsOG0000
ParticipantsOG0017
ParticipantsOG0020
ParticipantsOG0033
Cycle 3 Day 1
ParticipantsOG0000
ParticipantsOG0010
ParticipantsOG0029
ParticipantsOG0030
Cycle 5 Day 1
ParticipantsOG0000
ParticipantsOG0010
ParticipantsOG0020
ParticipantsOG0032
OG003
Part A: Dose Level 400 mg BID
All participants who received rilzabrutinib 400 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
Units
Counts
Participants
OG0009
OG0017
OG0029
OG00343
Title
Denominators
Categories
Cycle 1 Day 1
ParticipantsOG0009
ParticipantsOG0010
ParticipantsOG0025
ParticipantsOG00343
Title
Measurements
OG0001.43(0.500 to 2.00)
OG0021.50(1.00 to 1.53)
OG0031.50(0.500 to 4.00)
Cycle 2 Day 1
ParticipantsOG0000
ParticipantsOG0017
ParticipantsOG0020
ParticipantsOG0033
Cycle 3 Day 1
ParticipantsOG0000
ParticipantsOG0010
ParticipantsOG0029
ParticipantsOG0030
Cycle 5 Day 1
ParticipantsOG0000
ParticipantsOG0010
ParticipantsOG0020
ParticipantsOG0032
OG003
Part A: Dose Level 400 mg BID
All participants who received rilzabrutinib 400 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
Units
Counts
Participants
OG0009
OG0017
OG0029
OG00343
Title
Denominators
Categories
Cycle 1 Day 1
ParticipantsOG0009
ParticipantsOG0010
ParticipantsOG0025
ParticipantsOG00343
Title
Measurements
OG000320± 217
OG002616± 334
OG003642± 435
Cycle 2 Day 1
ParticipantsOG0000
ParticipantsOG0017
ParticipantsOG0020
ParticipantsOG0033
Cycle 3 Day 1
ParticipantsOG0000
ParticipantsOG0010
ParticipantsOG0029
ParticipantsOG0030
Cycle 5 Day 1
ParticipantsOG0000
ParticipantsOG0010
ParticipantsOG0020
ParticipantsOG0032
OG003
Part A: Dose Level 400 mg BID
All participants who received rilzabrutinib 400 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
Units
Counts
Participants
OG0009
OG0015
OG0027
OG00335
Title
Denominators
Categories
Cycle 1 Day 1
ParticipantsOG0009
ParticipantsOG0010
ParticipantsOG0025
ParticipantsOG00335
Title
Measurements
OG000331± 224
OG002653± 351
OG003703± 455
Cycle 2 Day 1
ParticipantsOG0000
ParticipantsOG0015
ParticipantsOG0020
ParticipantsOG0031
Cycle 3 Day 1
ParticipantsOG0000
ParticipantsOG0010
ParticipantsOG0027
ParticipantsOG0030
OG003
Part A: Dose Level 400 mg BID
All participants who received rilzabrutinib 400 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
Units
Counts
Participants
OG0009
OG0016
OG0027
OG00337
Title
Denominators
Categories
Cycle 1 Day 1
ParticipantsOG0009
ParticipantsOG0010
ParticipantsOG0025
ParticipantsOG00337
Title
Measurements
OG0001.36(1.09 to 1.50)
OG0021.30(1.15 to 1.95)
OG0031.32(0.780 to 3.24)
Cycle 2 Day 1
ParticipantsOG0000
ParticipantsOG0016
ParticipantsOG0020
ParticipantsOG0031
Cycle 3 Day 1
ParticipantsOG0000
ParticipantsOG0010
ParticipantsOG0027
ParticipantsOG0030
OG003
Part A: Dose Level 400 mg BID
All participants who received rilzabrutinib 400 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
Units
Counts
Participants
OG0009
OG0015
OG0027
OG00335
Title
Denominators
Categories
Cycle 1 Day 1
ParticipantsOG0009
ParticipantsOG0010
ParticipantsOG0025
ParticipantsOG00335
Title
Measurements
OG0001780± 1180
OG0021180± 611
OG0031720± 1460
Cycle 2 Day 1
ParticipantsOG0000
ParticipantsOG0015
ParticipantsOG0020
ParticipantsOG0031
Cycle 3 Day 1
ParticipantsOG0000
ParticipantsOG0010
ParticipantsOG0027
ParticipantsOG0030
OG003
Part A: Dose Level 400 mg BID
All participants who received rilzabrutinib 400 mg orally BID over 24 weeks of treatment period, unless withdrawn or had a platelet response to current dose level.
Units
Counts
Participants
OG0009
OG0015
OG0027
OG00335
Title
Denominators
Categories
Cycle 1 Day 1
ParticipantsOG0009
ParticipantsOG0010
ParticipantsOG0025
ParticipantsOG00335
Title
Measurements
OG000911± 569
OG002592± 320
OG003854± 551
Cycle 2 Day 1
ParticipantsOG0000
ParticipantsOG0015
ParticipantsOG0020
ParticipantsOG0031
Cycle 3 Day 1
ParticipantsOG0000
ParticipantsOG0010
ParticipantsOG0027
ParticipantsOG0030
25
Title
Denominators
Categories
Day 1: pre-dose
ParticipantsOG00025
Title
Measurements
OG000NA± NAMean and SD were not calculated as the values were below the lower limit of quantification (LLOQ=0.100 ng/mL).
Day 1: 2 hours post-dose
ParticipantsOG00025
Title
Measurements
OG000154.09± 131.79
Day 29: pre-dose
ParticipantsOG00022
Title
Measurements
OG0005.07± 8.61
Day 29: 2 hours post-dose
ParticipantsOG00023
Title
Measurements
OG000228.67± 173.68
Day 57: pre-dose
ParticipantsOG00022
Title
Measurements
OG00026.42± 90.43
Day 57: 2 hours post-dose
ParticipantsOG00022
Title
Measurements
OG000331.81± 151.07
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
0 events
0 affected
52 at risk
EG0040 events0 affected60 at risk
EG0051 events1 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
2 events
2 affected
52 at risk
EG0042 events2 affected60 at risk
EG0051 events1 affected26 at risk
0 events
0 affected
52 at risk
EG0040 events0 affected60 at risk
EG0051 events1 affected26 at risk
0 events
0 affected
52 at risk
EG0040 events0 affected60 at risk
EG0051 events1 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
0 events
0 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
0 events
0 affected
52 at risk
EG0040 events0 affected60 at risk
EG0051 events1 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
3 events
3 affected
52 at risk
EG0043 events3 affected60 at risk
EG0050 events0 affected26 at risk
6 events
5 affected
52 at risk
EG0046 events5 affected60 at risk
EG0055 events4 affected26 at risk
6 events
4 affected
52 at risk
EG0046 events4 affected60 at risk
EG0053 events2 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
9 events
7 affected
52 at risk
EG0049 events7 affected60 at risk
EG00513 events9 affected26 at risk
6 events
5 affected
52 at risk
EG0046 events5 affected60 at risk
EG0050 events0 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
0 events
0 affected
52 at risk
EG0040 events0 affected60 at risk
EG0052 events2 affected26 at risk
0 events
0 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
0 events
0 affected
52 at risk
EG0041 events1 affected60 at risk
EG0051 events1 affected26 at risk
3 events
3 affected
52 at risk
EG0043 events3 affected60 at risk
EG0050 events0 affected26 at risk
4 events
3 affected
52 at risk
EG0044 events3 affected60 at risk
EG0055 events4 affected26 at risk
2 events
2 affected
52 at risk
EG0042 events2 affected60 at risk
EG0050 events0 affected26 at risk
0 events
0 affected
52 at risk
EG0040 events0 affected60 at risk
EG0052 events2 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0052 events2 affected26 at risk
6 events
6 affected
52 at risk
EG0046 events6 affected60 at risk
EG0050 events0 affected26 at risk
3 events
3 affected
52 at risk
EG0043 events3 affected60 at risk
EG0051 events1 affected26 at risk
3 events
3 affected
52 at risk
EG0043 events3 affected60 at risk
EG0056 events5 affected26 at risk
0 events
0 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
30 events
22 affected
52 at risk
EG00431 events23 affected60 at risk
EG00516 events12 affected26 at risk
4 events
3 affected
52 at risk
EG0045 events4 affected60 at risk
EG0056 events5 affected26 at risk
0 events
0 affected
52 at risk
EG0040 events0 affected60 at risk
EG0054 events4 affected26 at risk
5 events
3 affected
52 at risk
EG0045 events3 affected60 at risk
EG0051 events1 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
0 events
0 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
25 events
19 affected
52 at risk
EG00427 events21 affected60 at risk
EG00511 events7 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
5 events
5 affected
52 at risk
EG0045 events5 affected60 at risk
EG0052 events1 affected26 at risk
3 events
3 affected
52 at risk
EG0043 events3 affected60 at risk
EG0051 events1 affected26 at risk
4 events
4 affected
52 at risk
EG0044 events4 affected60 at risk
EG0050 events0 affected26 at risk
3 events
3 affected
52 at risk
EG0043 events3 affected60 at risk
EG0051 events1 affected26 at risk
3 events
2 affected
52 at risk
EG0043 events2 affected60 at risk
EG0050 events0 affected26 at risk
5 events
4 affected
52 at risk
EG0045 events4 affected60 at risk
EG0051 events1 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
9 events
6 affected
52 at risk
EG0049 events6 affected60 at risk
EG0052 events2 affected26 at risk
11 events
9 affected
52 at risk
EG00412 events10 affected60 at risk
EG0051 events1 affected26 at risk
0 events
0 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
0 events
0 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0052 events2 affected26 at risk
0 events
0 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
14 events
12 affected
52 at risk
EG00415 events13 affected60 at risk
EG0053 events3 affected26 at risk
0 events
0 affected
52 at risk
EG0040 events0 affected60 at risk
EG0052 events2 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0052 events2 affected26 at risk
3 events
3 affected
52 at risk
EG0043 events3 affected60 at risk
EG0050 events0 affected26 at risk
2 events
1 affected
52 at risk
EG0042 events1 affected60 at risk
EG0050 events0 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0052 events2 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0052 events2 affected26 at risk
3 events
2 affected
52 at risk
EG0044 events3 affected60 at risk
EG0050 events0 affected26 at risk
18 events
10 affected
52 at risk
EG00420 events12 affected60 at risk
EG00510 events5 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
1 events
1 affected
52 at risk
EG0041 events1 affected60 at risk
EG0050 events0 affected26 at risk
29
OG00431
12
6
2
42
4
4
2
34
11
5
2
43
2
5
2
49
0
1
2
49
1
0
2
48
2
0
2
22
1
1
28
49
0
0
3
49
0
0
3
50
0
0
2
Title
Measurements
OG001451± 408
OG003396± 257
Title
Measurements
OG002319± 266
Title
Measurements
OG003447± 67.2
Title
Measurements
OG0012.00(1.00 to 3.03)
OG0031.05(0.500 to 2.00)
Title
Measurements
OG0021.50(0.500 to 3.00)
Title
Measurements
OG0031.25(1.00 to 1.50)
Title
Measurements
OG001986± 646
OG0031150± 1030
Title
Measurements
OG002788± 610
Title
Measurements
OG003872± 412
Title
Measurements
OG001787± 446
OG003827± NASD cannot be calculated for a single participant.
Title
Measurements
OG002818± 671
Title
Measurements
OG0011.52(1.17 to 3.89)
OG0031.34(1.34 to 1.34)
Title
Measurements
OG0021.60(1.18 to 1.88)
Title
Measurements
OG0012090± 2640
OG003934± NASD cannot be calculated for a single participant.
Title
Measurements
OG0021650± 1510
Title
Measurements
OG001743± 593
OG003484± NASD cannot be calculated for a single participant.