Activity, Safety and Pharmacokinetics in Pediatric Subjec... | NCT03774082 | Trialant
NCT03774082
Sponsor
Novartis Pharmaceuticals
Status
Completed
Last Update Posted
Dec 11, 2025Actual
Enrollment
46Actual
Phase
Phase 2
Conditions
Graft vs Host Disease
Interventions
INC424
Countries
Brazil
Canada
Czechia
India
Italy
Japan
Russia
Slovakia
South Korea
Spain
Switzerland
Taiwan
Thailand
Turkey (Türkiye)
Protocol Section
Identification Module
NCT ID
NCT03774082
Obsolete or Duplicate NCT IDs
Not provided
Organization Study
CINC424G12201
Secondary IDs
ID
Type
Description
Link
2018-003296-35
EudraCT Number
Brief Title
Activity, Safety and Pharmacokinetics in Pediatric Subjects With Moderate and Severe Chronic Graft vs. Host Disease After Allogeneic Stem Cell Transplant
Official Title
A Phase II Open-label, Single-arm, Multi-center Study of Ruxolitinib Added to Corticosteroids in Pediatric Subjects With Moderate and Severe Chronic Graft vs. Host Disease After Allogeneic Stem Cell Transplantation
Acronym
REACH 5
Organization
NovartisINDUSTRY
Status Module
Record Verification Date
Nov 2025
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
May 20, 2020Actual
Primary Completion Date
Feb 25, 2022Actual
Completion Date
Aug 26, 2024Actual
First Submitted Date
Dec 11, 2018
First Submission Date that Met QC Criteria
Dec 11, 2018
First Posted Date
Dec 12, 2018Actual
Results Waived
Not provided
Results First Submitted Date
Feb 25, 2025
Results First Submitted that Met QC Criteria
Mar 28, 2025
Results First Posted Date
Apr 17, 2025Actual
Certification/Extension (aka Delayed Results) First Submitted Date
Not provided
Certification/Extension First Submitted that Passed QC Review
Not provided
Certification/Extension First Posted Date
Not provided
Last Update Submitted Date
Nov 24, 2025
Last Update Posted Date
Dec 11, 2025Actual
Sponsor/Collaborators Module
Responsible Party, by Official Title
Sponsor
Lead Sponsor
Novartis PharmaceuticalsINDUSTRY
Collaborators
Not provided
Oversight Module
Has Data Monitoring Committee (DMC)
Yes
Is FDA Regulated Drug
No
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 open-label, single-arm, Phase II multi-center study enrolled 46 participants and investigated the activity, pharmacokinetics and safety of ruxolitinib added to the subject's immunosuppressive regimen among infants, children, and adolescents aged ≥28 days to <18 years old with either moderate to severe treatment-naive cGvHD or SR-cGvHD.
Although 46 participants were enrolled,1 participant (enrolled in the ≥6y to <12y age group) received study treatment beyond protocol requirements and was excluded from analyses.
Detailed Description
Subjects were grouped according to their age as follows:
Group 1 included subjects ≥12y to <18y
Group 2 included subjects ≥6y to <12y
Group 3 included subjects ≥2y to <6y and
Group 4 included subjects ≥28days to <2y. Enrollment initiation into the youngest age group, Group 4, was subject to the availability of data in this age group from another study, as well as a review of available PK, safety, and activity data generated from Groups 1 to 3 in the current study. At least 5 evaluable participants per group were needed for the primary analysis in Groups 1, 2 and 3. No minimum number of evaluable participants were needed in Group 4. Enrollment was completed prior to the availability of the data and so no subjects were enrolled in Group 4.
After a screening period of Day -28 to Day -1: eligible subjects started study treatment on Cycle
1 Day 1 and were treated for up to a maximum of 3 years (39 cycles/156 weeks) or until early discontinuation. Subjects who discontinued study treatment for any reason earlier than 39 cycles were followed every 6 months until 3 years from their first dose of study treatment was reached.
Conditions Module
Conditions
Graft vs Host Disease
Keywords
GvHD
INC424
pediatric
Chronic Graft versus Host Disease
moderate and severe chronic graft vs. host disease
allogeneic stem cell transplant
ruxolitinib
corticosteroids
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
46Actual
Arms/Interventions Module
Arm Groups
Label
Type
Description
Intervention Names
INC424 (ruxolitinib)
Experimental
All pediatric participants received ruxolitinib twice a day (BID) in either tablet or oral solution (liquid), depending on the group they were in.
Drug: INC424
Interventions
Name
Type
Description
Arm Group Labels
Other Names
INC424
Drug
Ruxolitinib was taken orally based on age groups as follows:
Group 1 (>=12y to <18y): 10mg bid as tablet Group 2 (>=6y to <12y): 5mg bid as tablet or liquid Group 3 (>=2y to <6y): 4mg/m2 bid as liquid
Outcomes Module
Primary Outcomes
Measure
Description
Time Frame
Overall Response Rate (ORR) at Cycle 7 Day 1
ORR is defined as the percentage of participants demonstrating a complete response (CR) or partial response (PR) without the requirement of additional systemic therapies for an earlier progression, mixed response or non-response. The response is assessed per National Institute of Health (NIH) consensus criteria and scoring of response was relative to the organ stage at the start of study treatment.
At Cycle 7 Day 1 (Day 168); Cycle = 28 Days
Secondary Outcomes
Measure
Description
Time Frame
Ruxolitinib Concentrations by Timepoint
Pharmacokinetics (PK) of ruxolitinib by age groups (and formulation tablet vs liquid).
Cycle 1 Day 1: 0.5, 2 and 6 hours post-dose; Pre-dose on Cycle 1 Day 8, Cycle 1 Day 15, Cycle 1 Day 22, Cycle 3 Day 1, Cycle 5 Day 1 and Cycle 7 Day 1; Cycle = 28 Days
Duration of Response (DOR)
Other Outcomes
Not provided
Eligibility Module
Eligibility Criteria
Inclusion Criteria:
Male or female subjects age ≥28 days and <18 years at the time of informed consent.
Subjects who have undergone a successful alloSCT from any donor source (matched unrelated donor, sibling, haplo-identical) using bone marrow, peripheral blood stem cells, or cord blood. Recipients of myeloablative or reduced intensity conditioning are eligible.
Subjects with diagnosed moderate to severe cGvHD according to NIH 2014 Consensus Criteria prior to Cycle 1 Day 1. Other possible diagnoses for clinical symptoms supporting cGvHD diagnoses must be excluded (e.g., infection, drug side effects, malignancy). Subjects must be either:
Treatment-naive cGvHD subjects that have not received any prior systemic treatment for cGvHD except for a maximum 72h of prior systemic corticosteroid therapy of methylprednisolone or equivalent after the onset of chronic GvHD. Subjects are allowed to have received prior systemic treatment for cGvHD prophylaxis (as long as the prophylaxis was started prior to the diagnosis of cGvHD).
OR o Steroid-refractory moderate to severe cGvHD as per institutional criteria, or per physician decision in case institutional criteria are not available, and still receiving systemic corticosteroids for the treatment of cGvHD for a duration of <18 months prior to Cycle 1 Day 1. In case the corticosteroids were previously interrupted due to response, the duration of < 18 months applies to the last period of corticosteroid use.
Exclusion Criteria:
SR-cGvHD subjects with a prior cGvHD treatment with a JAK1- or a JAK2- or a JAK1/2-inhibitor, except when the subject achieved complete or partial response and has been off JAK inhibitor treatment for at least 4 weeks prior to Cycle Day 1 or up to 5 times the half-life of the prior JAK inhibitor, whichever is longer.
* Subjects who initiated systemic calcineurin inhibitors (CNI; cyclosporine or tacrolimus) within 3 weeks prior to start of ruxolitinib on Cycle 1 Day 1. Note: systemic CNI are allowed when initiated > 3 weeks from start of ruxolitinib.
Failed prior alloSCT within the past 6 months
Significant respiratory disease including subjects who are on mechanical ventilation or who have a resting oxygen saturation < 90% by pulse-oximetry on room-air.
Impairment of gastrointestinal (GI) function (unrelated to GvHD) or GI disease (unrelated to GvHD) that may significantly alter the absorption of oral ruxolitinib (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome or small bowel resection),
Cholestatic disorders, or unresolved sinusoidal obstructive syndrome/veno-occlusive disease of the liver (defined as persistent bilirubin abnormalities not attributable to cGvHD and ongoing organ dysfunction)
Presence of clinically active uncontrolled infection including significant bacterial, fungal, viral or parasitic infection requiring treatment.
Known human immunodeficiency virus (HIV) infection.
Evidence of uncontrolled hepatitis B virus (HBV) or hepatitis C virus (HCV) based on assessment done by Investigator or delegate.
Known allergies, hypersensitivity, or intolerance to any of the study medications, excipients, or similar compounds.
History of bone disorders such as osteogenesis imperfecta, rickets, renal osteodystrophy, osteomyelitis, osteopenia, fibrous dysplasia, osteomalacia etc. prior to the underlying diagnosis which resulted in the alloSCT.
History of endocrine or kidney related growth retardation prior to the underlying diagnosis which resulted in the alloSCT.
Evidence of clinically active tuberculosis (clinical diagnosis per local practice)
Any corticosteroid therapy for indications other than cGvHD at doses > 1 mg/kg/daymethylprednisolone (or equivalent prednisone dose 1.25 mg/kg/day) within 7 days of the screening visit.
History of progressive multifocal leuko-encephalopathy (PML).
Locatelli F, Antmen B, Kang HJ, Koh K, Takahashi Y, Kupesiz A, Dias Matos MGA, Chopra Y, Bhat S, Im HJ, Gungor T, Lu MY, Stefanelli T, Rosko C, St Pierre A, Burock K, Smith Y, Sinclair K, Diaz-de-Heredia C. Ruxolitinib in treatment-naive or corticosteroid-refractory paediatric patients with chronic graft-versus-host disease (REACH5): interim analysis of a single-arm, multicentre, phase 2 study. Lancet Haematol. 2024 Aug;11(8):e580-e592. doi: 10.1016/S2352-3026(24)00174-1. Epub 2024 Jul 10.
See Also Links
Label
URL
A Plain Language Trial Summary is available on www.novctrd.com
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.
Enrollment initiation into the youngest age group, Group 4, was subject to the availability of data in this age group from study CINC424F12201. However, no participants were enrolled in group 4 (aged ≥ 28 days to < 2y) due to the completion of the overall study enrolment with participants in the older age groups (groups 1, 2, & 3).
Recruitment Details
At least 5 evaluable participants per group were needed for the primary analysis in Groups 1, 2 and 3. No minimum number of evaluable participants were needed in Group 4.
Disposition and Demographics are presented by age group.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
FG000
≥ 12y - < 18y RUX 10mg BID (Group 1)
Participants received ruxolitinib 10mg orally twice a day (BID).
FG001
≥ 6y - < 12y RUX 5mg BID (Group 2)
Participants received ruxolitinib 5mg orally twice a day (BID).
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
Sep 9, 2022
Feb 25, 2025
Derived Section
Miscellaneous Info Module
Version Holder
Jul 10, 2026
Removed Countries
Argentina
Lithuania
Slovenia
Submission Tracking
No data available
No data is available for this block.
Condition Browse Module
MeSH Terms
Intervention Browse Module
MeSH Terms
N/A
Intervention Model
Single Group 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
INC424 (ruxolitinib)
Ruxolitinib
Time from first response until chronic Graft vs. host disease (cGvHD) progression, death, or the date of addition of systemic therapies for cGvHD assessed for responders only based on BOR up to Cycle 7 Day 1. Presented as percentage of participants to still be in response at different time points in months (per Kaplan-Meier estimates). Participants without event will be censored at the date of their last response assessment prior to or at the analysis cut-off date if no events occurred on or before 12 weeks (84 days) after the last GvHD assessment. As planned in the SAP, this outcome measure is provided for all participants instead of per age groups.
From baseline up to 39 cycles; Cycle = 28 Days
Overall Response Rate (ORR) at Cycle 4 Day 1
ORR is defined as the percentage of participants demonstrating a complete response (CR) or partial response (PR) without the requirement of additional systemic therapies for an earlier progression, mixed response or non-response. The response is assessed per National Institute of Health (NIH) consensus criteria and scoring of response will be relative to the organ stage at the start of study treatment at Cycle 4 Day 1.
At Cycle 4 Day 1 (Day 84); Cycle = 28 Days
Best Overall Response (BOR)
Percentage of participants who achieved overall response (complete response (CR) or partial response (PR)) at any time point until Cycle 7 Day 1 or the start of additional systemic therapy for chronic GvHD.
Until Cycle 7 Day 1 (Day 168) or the start of additional systemic therapy for cGvHD; Cycle = 28 Days
Failure Free Survival (FFS)
Failure-free survival was defined as the time from date of treatment to any of the following events: i) relapse or recurrence of underlying disease or death due to underlying disease, ii) non-relapse mortality, or iii) addition or initiation of another systemic therapy for cGvHD per Kaplan-Meier estimates. As planned in the SAP, this outcome measure is provided for all participants instead of per age groups.
From baseline up to 39 cycles; Cycle = 28 Days
Cumulative Incidence of Malignancy Relapse/Recurrence (MR)
MR was defined as the time from date of treatment assignment to the date of hematologic malignancy relapse/recurrence. Calculated for subjects with underlying hematologic malignant disease. The cumulative incidence (CI) of malignancy relapse/recurrence at 1, 2, 6, 12, 18, 24, 30 and 36 months after start of treatment has been reported. As planned in the SAP, this outcome measure is provided for all participants instead of per age groups.
From baseline up to 39 cycles; Cycle = 28 Days
Non-relapse Mortality (NRM)
NRM is defined as the time from date of treatment assignment to date of death not preceded by underlying disease relapse/recurrence calculated for all participants. The cumulative incidence (CI) of non-relapse mortality at 1, 2, 6, 12, 18, 24, 30 and 36 months after start of treatment has been reported. As planned in the SAP, this outcome measure is provided for all participants instead of per age groups.
From baseline up to 39 cycles; Cycle = 28 Days
Overall Survival (OS)
OS is defined as the time from the date of treatment assignment to the date of death due to any cause. As planned in the SAP, this outcome measure is provided for all participants instead of per age groups.
From baseline up to 39 cycles; Cycle = 28 Days
Percentage of Participants With ≥50% Reduction From Baseline in Daily Corticosteroid Dose
Reduction of at least ≥50% from baseline in daily corticosteroid use by Cycle 7 Day 1 (regardless of reason). As planned in the SAP, this outcome measure is provided for all participants instead of per age groups, for those who received corticosteroids at baseline.
Baseline to Cycle 7 Day 1 (Day 168); Cycle = 28 Days
Percentage of Participants With a Reduction to a Low Dose Corticosteriod
Reduction to low dose corticosteroids, is defined as the percentage of participants with reduction from baseline in daily corticosteroid dose to methylprednisolone-equivalent steroid dose of ≤ 0.2 mg/kg/day (or equivalent dose of ≤ 0.25 mg/kg/day prednisone or prednisolone). As planned in the SAP, this outcome measure is provided for all participants instead of per age groups, for those who received corticosteroids at baseline.
Baseline to Cycle 7 Day 1 (Day 168); Cycle = 28 Days
Graft Failure
Reported are the number of participants with graft failure from all age groups together. Graft failure was assessed by donor cell chimerism, defined as initial whole blood or marrow donor chimerism for those who had ≥ 5% donor cell chimerism at baseline. If donor cell chimerism declined to < 5% on subsequent measurements, graft failure was declared. As planned in the SAP, this outcome measure is provided for all participants instead of per age groups.
From baseline up to 39 cycles; Cycle = 28 Days
Toronto
Ontario
M5G 1X8
Canada
Novartis Investigative Site
Prague
150 06
Czechia
Novartis Investigative Site
Tamil Nadu
Chennai
600035
India
Novartis Investigative Site
Pune
Maharashtra
411004
India
Novartis Investigative Site
Bangalore
560099
India
Novartis Investigative Site
Delhi
110 085
India
Novartis Investigative Site
Roma
RM
00165
Italy
Novartis Investigative Site
Nagoya
Aichi-ken
466 8560
Japan
Novartis Investigative Site
Saitama
330 8777
Japan
Novartis Investigative Site
Saint Petersburg
197022
Russia
Novartis Investigative Site
Bratislava
833 40
Slovakia
Novartis Investigative Site
Seoul
03080
South Korea
Novartis Investigative Site
Seoul
05505
South Korea
Novartis Investigative Site
Barcelona
Catalonia
08035
Spain
Novartis Investigative Site
Zurich
CH - 8032
Switzerland
Novartis Investigative Site
Taipei
10002
Taiwan
Novartis Investigative Site
Bangkok
10400
Thailand
Novartis Investigative Site
Adana
1330
Turkey (Türkiye)
Novartis Investigative Site
Antalya
07000
Turkey (Türkiye)
Novartis Investigative Site
Antalya
07070
Turkey (Türkiye)
FG002
≥ 2y - < 6y RUX 4mg/m2 BID (Group 3)
Participants received ruxolitinib 4mg/m2 orally twice a day (BID).
FG00022 subjects
FG00117 subjects
FG0027 subjects
Full Analysis Set
Full Analysis Set comprised all subjects to whom study treatment has been assigned and who received at least one dose of study treatment.
FG00022 subjects
FG00116 subjects
FG0027 subjects
Completed Treatment
FG0003 subjects
FG0015 subjects
FG0023 subjects
Discontinued From Treatment
FG00019 subjects
FG00111 subjects
FG0024 subjects
Participants Who Received Tablets
FG00022 subjects
FG00114 subjects
FG0020 subjects
Participants Who Received Liquid
FG0000 subjects
FG0012 subjects
FG0027 subjects
COMPLETED
FG00014 subjects
FG00110 subjects
FG0024 subjects
NOT COMPLETED
FG0008 subjects
FG0017 subjects
FG0023 subjects
Type
Comment
Reasons
Death
FG0006 subjects
FG0013 subjects
FG0022 subjects
Subject decision
FG0000 subjects
FG0013 subjects
FG0020 subjects
Lost to Follow-up
FG0002 subjects
FG0010 subjects
FG0020 subjects
Guardian decision
FG0000 subjects
FG0010 subjects
FG0021 subjects
Enrolled but received treatment beyond protocol requirement
FG0000 subjects
FG0011 subjects
FG0020 subjects
Full Analysis Set (FAS) comprised all subjects to whom study treatment has been assigned and who received at least one dose of study treatment.
Type of Units Analyzed
Not provided
Arm/Group Information
ID
Title
Description
BG000
≥ 12y - < 18y RUX 10mg BID (Group 1)
Participants received ruxolitinib 10mg orally twice a day (BID).
BG001
≥ 6y - < 12y RUX 5mg BID (Group 2)
Participants received ruxolitinib 5mg orally twice a day (BID).
BG002
≥ 2y - < 6y RUX 4mg/m2 BID (Group 3)
Participants received ruxolitinib 4mg/m2 orally twice a day (BID).
BG003
Total
Total of all reporting groups
Denominators
Units
Counts
Participants
BG00022
BG00116
BG0027
BG00345
Baseline Measures
Title
Description
Population Description
Parameter Type
Dispersion Type
Unit of Measure
Calculate Percentage
Denominator Units Selected
Denominators
Classes
Age, Continuous
Mean
Standard Deviation
months
Title
Denominators
Categories
Title
Measurements
BG000175.2± 20.05
BG00199.3± 18.39
BG00247.1± 15.08
BG003
Sex: Female, Male
Count of Participants
Participants
Title
Denominators
Categories
Title
Measurements
Female
BG0007
BG0017
BG002
Race/Ethnicity, Customized
Number
Participants
Title
Denominators
Categories
White
Title
Measurements
BG0008
BG00110
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
Overall Response Rate (ORR) at Cycle 7 Day 1
ORR is defined as the percentage of participants demonstrating a complete response (CR) or partial response (PR) without the requirement of additional systemic therapies for an earlier progression, mixed response or non-response. The response is assessed per National Institute of Health (NIH) consensus criteria and scoring of response was relative to the organ stage at the start of study treatment.
Full Analysis Set (FAS) comprised all subjects to whom study treatment has been assigned and who received at least one dose of study treatment.
Posted
Number
90% Confidence Interval
Percentage of participants
At Cycle 7 Day 1 (Day 168); Cycle = 28 Days
ID
Title
Description
OG000
≥ 12y - < 18y RUX 10mg BID (Group 1)
Participants received ruxolitinib 10mg orally twice a day (BID).
OG001
≥ 6y - < 12y RUX 5mg BID (Group 2)
Participants received ruxolitinib 5mg orally twice a day (BID).
OG002
≥ 2y - < 6y RUX 4mg/m2 BID (Group 3)
Participants received ruxolitinib 4mg/m2 orally twice a day (BID).
OG003
All Participants
All participants from Group 1, Group 2 and Group 3.
Units
Counts
Participants
OG00022
OG00116
OG0027
OG003
Title
Denominators
Categories
Title
Measurements
OG00036.4(19.6 to 56.1)
OG00150.0(27.9 to 72.1)
OG00228.6(5.3 to 65.9)
OG003
Secondary
Ruxolitinib Concentrations by Timepoint
Pharmacokinetics (PK) of ruxolitinib by age groups (and formulation tablet vs liquid).
The Pharmacokinetic Analysis Set (PAS) included all subjects who provided at least one evaluable PK concentration. Evaluable PK concentration: Participants took the dose of ruxolitinib prior to PK sample; For post-dose samples on Cycle 1 Day 1: participants did not vomit within 2 hours after dosing with ruxolitinib; For pre-dose samples: participant samples were collected before the next dose administration of ruxolitinib.
Posted
Geometric Mean
Geometric Coefficient of Variation
ug/ml
Cycle 1 Day 1: 0.5, 2 and 6 hours post-dose; Pre-dose on Cycle 1 Day 8, Cycle 1 Day 15, Cycle 1 Day 22, Cycle 3 Day 1, Cycle 5 Day 1 and Cycle 7 Day 1; Cycle = 28 Days
ID
Title
Description
OG000
≥ 12y - < 18y RUX 10mg BID (Tablet)
Participants in this group were administered 10mg ruxolitinib tablet twice a day.
OG001
≥ 6y - < 12y RUX 5mg BID (Tablet)
Participants in this group were administered 5mg ruxolitinib tablets twice a day.
OG002
≥ 6y - < 12y RUX 5mg BID (Liquid)
Participants in this group were administered ruxolitinib oral pediatric formulation twice a day.
Secondary
Duration of Response (DOR)
Time from first response until chronic Graft vs. host disease (cGvHD) progression, death, or the date of addition of systemic therapies for cGvHD assessed for responders only based on BOR up to Cycle 7 Day 1. Presented as percentage of participants to still be in response at different time points in months (per Kaplan-Meier estimates). Participants without event will be censored at the date of their last response assessment prior to or at the analysis cut-off date if no events occurred on or before 12 weeks (84 days) after the last GvHD assessment. As planned in the SAP, this outcome measure is provided for all participants instead of per age groups.
FAS comprised all subjects to whom study treatment has been assigned and who received at least one dose of study treatment. Duration of response (DOR) is assessed for responders only.
Posted
Number
95% Confidence Interval
Percentage of participants
From baseline up to 39 cycles; Cycle = 28 Days
ID
Title
Description
OG000
All Participants
DOR was analyzed for all responders from the three age groups (Groups 1, 2 and 3)
Units
Counts
Participants
OG000
Secondary
Overall Response Rate (ORR) at Cycle 4 Day 1
ORR is defined as the percentage of participants demonstrating a complete response (CR) or partial response (PR) without the requirement of additional systemic therapies for an earlier progression, mixed response or non-response. The response is assessed per National Institute of Health (NIH) consensus criteria and scoring of response will be relative to the organ stage at the start of study treatment at Cycle 4 Day 1.
FAS comprised all subjects to whom study treatment has been assigned and who received at least one dose of study treatment.
Posted
Number
90% Confidence Interval
Percentage of participants
At Cycle 4 Day 1 (Day 84); Cycle = 28 Days
ID
Title
Description
OG000
≥ 12y - < 18y RUX 10mg BID (Group 1)
Participants received ruxolitinib 10mg orally twice a day (BID).
OG001
≥ 6y - < 12y RUX 5mg BID (Group 2)
Participants received ruxolitinib 5mg orally twice a day (BID).
OG002
≥ 2y - < 6y RUX 4mg/m2 BID (Group 3)
Participants received ruxolitinib 4mg/m2 orally twice a day (BID).
OG003
Secondary
Best Overall Response (BOR)
Percentage of participants who achieved overall response (complete response (CR) or partial response (PR)) at any time point until Cycle 7 Day 1 or the start of additional systemic therapy for chronic GvHD.
FAS comprised all subjects to whom study treatment has been assigned and who received at least one dose of study treatment.
Posted
Number
90% Confidence Interval
Percentage of participants
Until Cycle 7 Day 1 (Day 168) or the start of additional systemic therapy for cGvHD; Cycle = 28 Days
ID
Title
Description
OG000
≥ 12y - < 18y RUX 10mg BID (Group 1)
Participants received ruxolitinib 10mg orally twice a day (BID).
OG001
≥ 6y - < 12y RUX 5mg BID (Group 2)
Participants received ruxolitinib 5mg orally twice a day (BID).
OG002
≥ 2y - < 6y RUX 4mg/m2 BID (Group 3)
Participants received ruxolitinib 4mg/m2 orally twice a day (BID).
OG003
All Participants
All participants from Group 1, Group 2 and Group 3.
Secondary
Failure Free Survival (FFS)
Failure-free survival was defined as the time from date of treatment to any of the following events: i) relapse or recurrence of underlying disease or death due to underlying disease, ii) non-relapse mortality, or iii) addition or initiation of another systemic therapy for cGvHD per Kaplan-Meier estimates. As planned in the SAP, this outcome measure is provided for all participants instead of per age groups.
FAS comprised all subjects to whom study treatment has been assigned and who received at least one dose of study treatment.
Posted
Number
95% Confidence Interval
Percentage of participants
From baseline up to 39 cycles; Cycle = 28 Days
ID
Title
Description
OG000
All Participants
FFS was analyzed for all participants from the three age groups (Groups 1, 2 and 3)
Units
Counts
Participants
OG000
Secondary
Cumulative Incidence of Malignancy Relapse/Recurrence (MR)
MR was defined as the time from date of treatment assignment to the date of hematologic malignancy relapse/recurrence. Calculated for subjects with underlying hematologic malignant disease. The cumulative incidence (CI) of malignancy relapse/recurrence at 1, 2, 6, 12, 18, 24, 30 and 36 months after start of treatment has been reported. As planned in the SAP, this outcome measure is provided for all participants instead of per age groups.
FAS comprised all subjects to whom study treatment has been assigned and who received at least one dose of study treatment, assessed for subjects with malignancy relapse/occurrence only.
Posted
Number
95% Confidence Interval
Percentage of participants
From baseline up to 39 cycles; Cycle = 28 Days
ID
Title
Description
OG000
All Participants
MR was analyzed for all participants with underlying hematologic malignant disease from the three age groups (Groups 1, 2 and 3).
Units
Counts
Participants
OG000
Secondary
Non-relapse Mortality (NRM)
NRM is defined as the time from date of treatment assignment to date of death not preceded by underlying disease relapse/recurrence calculated for all participants. The cumulative incidence (CI) of non-relapse mortality at 1, 2, 6, 12, 18, 24, 30 and 36 months after start of treatment has been reported. As planned in the SAP, this outcome measure is provided for all participants instead of per age groups.
FAS comprised all subjects to whom study treatment has been assigned and who received at least one dose of study treatment.
Posted
Number
95% Confidence Interval
Percentage of participants
From baseline up to 39 cycles; Cycle = 28 Days
ID
Title
Description
OG000
All Participants
NRM was analyzed for all participants from the three age groups (Groups 1, 2 and 3).
Units
Counts
Participants
OG000
Secondary
Overall Survival (OS)
OS is defined as the time from the date of treatment assignment to the date of death due to any cause. As planned in the SAP, this outcome measure is provided for all participants instead of per age groups.
FAS comprised all subjects to whom study treatment has been assigned and who received at least one dose of study treatment.
Posted
Number
95% Confidence Interval
survival probability in percentage
From baseline up to 39 cycles; Cycle = 28 Days
ID
Title
Description
OG000
All Participants
OS was analyzed for all participants from the three age groups (Groups 1, 2 and 3).
Units
Counts
Participants
OG000
Secondary
Percentage of Participants With ≥50% Reduction From Baseline in Daily Corticosteroid Dose
Reduction of at least ≥50% from baseline in daily corticosteroid use by Cycle 7 Day 1 (regardless of reason). As planned in the SAP, this outcome measure is provided for all participants instead of per age groups, for those who received corticosteroids at baseline.
FAS comprised all subjects to whom study treatment has been assigned and who received at least one dose of study treatment and received corticosteroids at baseline.
Posted
Number
Percentage of participants
Baseline to Cycle 7 Day 1 (Day 168); Cycle = 28 Days
ID
Title
Description
OG000
All Participants
The percentage of participants with ≥50% reduction from baseline in daily corticosteroid dose at least once was analyzed for all participants in Groups 1, 2 and 3 who received corticosteroids at baseline.
Units
Counts
Participants
OG000
Secondary
Percentage of Participants With a Reduction to a Low Dose Corticosteriod
Reduction to low dose corticosteroids, is defined as the percentage of participants with reduction from baseline in daily corticosteroid dose to methylprednisolone-equivalent steroid dose of ≤ 0.2 mg/kg/day (or equivalent dose of ≤ 0.25 mg/kg/day prednisone or prednisolone). As planned in the SAP, this outcome measure is provided for all participants instead of per age groups, for those who received corticosteroids at baseline.
FAS comprised all subjects to whom study treatment has been assigned and who received at least one dose of study treatment and received corticosteroids at baseline.
Posted
Number
Percentage of participants
Baseline to Cycle 7 Day 1 (Day 168); Cycle = 28 Days
ID
Title
Description
OG000
All Participants
The percentage of participants with reduction to <=0.2mg/kg/day from baseline in daily corticosteroid dose at least once was analyzed for all participants in Groups 1, 2 and 3 who received corticosteroids at baseline.
Units
Counts
Participants
OG000
Secondary
Graft Failure
Reported are the number of participants with graft failure from all age groups together. Graft failure was assessed by donor cell chimerism, defined as initial whole blood or marrow donor chimerism for those who had ≥ 5% donor cell chimerism at baseline. If donor cell chimerism declined to < 5% on subsequent measurements, graft failure was declared. As planned in the SAP, this outcome measure is provided for all participants instead of per age groups.
FAS comprised all subjects to whom study treatment has been assigned and who received at least one dose of study treatment. As planned in the SAP, this outcome measure is provided for all participants instead of per age groups.
Posted
Number
Participants
From baseline up to 39 cycles; Cycle = 28 Days
ID
Title
Description
OG000
All Participants
Graft Failure was analyzed for all participants from the three age groups (Groups 1, 2 and 3).
Units
Counts
Participants
OG000
Time Frame
Adverse events (AEs) and on-treatment deaths were collected from the first dose of study treatment up to 30 days after last dose of study medication. Post-treatment survival follow-up deaths were collected 31 days after last dose of study medication until the end of the study, up to approx. 36 months/39 cycles.
Description
An Adverse Event (AE) is any sign or symptom that occurs during the conduct of the trial and post-treatment survival follow-up.
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
On-Treatment >=12y - <18y RUX 10mg BID
On-treatment period: from first dose of study treatment up to 30 days post-treatment.
0
22
15
22
22
22
EG001
On-Treatment >=6y - <12y RUX 5mg BID
On-treatment period: from first dose of study treatment up to 30 days post-treatment.
2
16
7
16
15
16
EG002
On-Treatment >=2y - <6y RUX 4mg/m2 BID
On-treatment period: from first dose of study treatment up to 30 days post-treatment.
1
7
4
7
7
7
EG003
On-Treatment - All Participants
All participants from Group 1, Group 2 and Group 3.
3
45
26
45
44
45
EG004
Post-Treatment >=12y - <18y RUX 10mg BID
Events collected in the post- treatment survival follow-up phase (starting from day 31 post- treatment).
6
22
3
22
4
22
EG005
Post-Treatment >=6y - <12y RUX 5mg BID
Events collected in the post- treatment survival follow-up phase (starting from day 31 post- treatment).
1
16
0
16
2
16
EG006
Post-Treatment >=2y - <6y RUX 4mg/m2 BID
Events collected in the post- treatment survival follow-up phase (starting from day 31 post- treatment).
1
7
0
7
0
7
EG007
Post-Treatment All Participants
All participants from Group 1, Group 2 and Group 3.
8
45
3
45
6
45
Serious Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Febrile neutropenia
Blood and lymphatic system disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG0031 affected45 at risk
EG004
Thrombocytopenia
Blood and lymphatic system disorders
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Bradycardia
Cardiac disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Retinal vein occlusion
Eye disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Pneumatosis intestinalis
Gastrointestinal disorders
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Oedema peripheral
General disorders
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Pyrexia
General disorders
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Hepatorenal syndrome
Hepatobiliary disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Haemophagocytic lymphohistiocytosis
Immune system disorders
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Aspergillus infection
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
COVID-19
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Cytomegalovirus infection reactivation
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Dengue fever
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Epstein-Barr virus infection
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Gastroenteritis viral
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Herpes zoster
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Human bocavirus infection
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Infectious pleural effusion
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Parainfluenzae virus infection
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Pneumonia
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Pneumonia fungal
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Pneumonia respiratory syncytial viral
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Pneumonia viral
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Respiratory syncytial virus infection
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Rhinovirus infection
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Sepsis
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Septic shock
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Tooth infection
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Upper respiratory tract infection
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Urinary tract infection
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Transfusion-related acute lung injury
Injury, poisoning and procedural complications
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Transplant failure
Injury, poisoning and procedural complications
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Blood creatinine increased
Investigations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Hyponatraemia
Metabolism and nutrition disorders
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Steroid diabetes
Metabolism and nutrition disorders
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Muscular weakness
Musculoskeletal and connective tissue disorders
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Pain in extremity
Musculoskeletal and connective tissue disorders
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Post transplant lymphoproliferative disorder
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Aura
Nervous system disorders
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Presyncope
Nervous system disorders
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Urinary retention
Renal and urinary disorders
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Acute respiratory distress syndrome
Respiratory, thoracic and mediastinal disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Alveolar proteinosis
Respiratory, thoracic and mediastinal disorders
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Dyspnoea
Respiratory, thoracic and mediastinal disorders
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Pneumothorax
Respiratory, thoracic and mediastinal disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Respiratory distress
Respiratory, thoracic and mediastinal disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Other Adverse Events
Term
Organ System
Source Vocabulary
Assessment Type
Notes
Statistical Information
Anaemia
Blood and lymphatic system disorders
MedDRA (27.0)
Systematic Assessment
EG0007 affected22 at risk
EG0012 affected16 at risk
EG0022 affected7 at risk
EG00311 affected45 at risk
EG0040 affected22 at risk
EG0050 affected16 at risk
EG0060 affected7 at risk
EG0070 affected45 at risk
Coagulopathy
Blood and lymphatic system disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Leukopenia
Blood and lymphatic system disorders
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Lymphopenia
Blood and lymphatic system disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Neutropenia
Blood and lymphatic system disorders
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0014 affected16 at risk
EG0022 affected7 at risk
EG003
Thrombocytopenia
Blood and lymphatic system disorders
MedDRA (27.0)
Systematic Assessment
EG0003 affected22 at risk
EG0011 affected16 at risk
EG0021 affected7 at risk
EG003
Tachycardia
Cardiac disorders
MedDRA (27.0)
Systematic Assessment
EG0003 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Ear canal stenosis
Ear and labyrinth disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Ear pain
Ear and labyrinth disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Cushing's syndrome
Endocrine disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Astigmatism
Eye disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Blepharitis
Eye disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Cataract
Eye disorders
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Conjunctivitis allergic
Eye disorders
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0011 affected16 at risk
EG0021 affected7 at risk
EG003
Dry eye
Eye disorders
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Glaucoma
Eye disorders
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Ocular hyperaemia
Eye disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Abdominal pain
Gastrointestinal disorders
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0011 affected16 at risk
EG0021 affected7 at risk
EG003
Constipation
Gastrointestinal disorders
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Dental caries
Gastrointestinal disorders
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0011 affected16 at risk
EG0021 affected7 at risk
EG003
Diarrhoea
Gastrointestinal disorders
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0012 affected16 at risk
EG0021 affected7 at risk
EG003
Lip pain
Gastrointestinal disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Vomiting
Gastrointestinal disorders
MedDRA (27.0)
Systematic Assessment
EG0003 affected22 at risk
EG0012 affected16 at risk
EG0020 affected7 at risk
EG003
Influenza like illness
General disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Oedema peripheral
General disorders
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Pain
General disorders
MedDRA (27.0)
Systematic Assessment
EG0003 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Pyrexia
General disorders
MedDRA (27.0)
Systematic Assessment
EG0003 affected22 at risk
EG0014 affected16 at risk
EG0020 affected7 at risk
EG003
Jaundice
Hepatobiliary disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Liver disorder
Hepatobiliary disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Allergy to chemicals
Immune system disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Acarodermatitis
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Aspergillus infection
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
BK virus infection
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Bronchiolitis
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
COVID-19
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0004 affected22 at risk
EG0013 affected16 at risk
EG0022 affected7 at risk
EG003
Conjunctivitis
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0012 affected16 at risk
EG0021 affected7 at risk
EG003
Cytomegalovirus infection reactivation
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0012 affected16 at risk
EG0020 affected7 at risk
EG003
Epstein-Barr viraemia
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Fungal skin infection
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Gastroenteritis
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Gastroenteritis viral
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Influenza
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0012 affected16 at risk
EG0021 affected7 at risk
EG003
Lower respiratory tract infection
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Nasopharyngitis
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0013 affected16 at risk
EG0021 affected7 at risk
EG003
Onychomycosis
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0021 affected7 at risk
EG003
Oral viral infection
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Otitis media
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0021 affected7 at risk
EG003
Parainfluenzae virus infection
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Pneumonia
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0004 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Rhinovirus infection
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Skin infection
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Streptococcal infection
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Tonsillitis
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Upper respiratory tract infection
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0014 affected16 at risk
EG0024 affected7 at risk
EG003
Urinary tract infection
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Viral infection
Infections and infestations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Arthropod bite
Injury, poisoning and procedural complications
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Extraskeletal ossification
Injury, poisoning and procedural complications
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Fall
Injury, poisoning and procedural complications
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Head injury
Injury, poisoning and procedural complications
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Ligament sprain
Injury, poisoning and procedural complications
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Post procedural haemorrhage
Injury, poisoning and procedural complications
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Procedural pain
Injury, poisoning and procedural complications
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Ulna fracture
Injury, poisoning and procedural complications
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Alanine aminotransferase increased
Investigations
MedDRA (27.0)
Systematic Assessment
EG0003 affected22 at risk
EG0011 affected16 at risk
EG0021 affected7 at risk
EG003
Amylase increased
Investigations
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Aspartate aminotransferase increased
Investigations
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0011 affected16 at risk
EG0021 affected7 at risk
EG003
Blood alkaline phosphatase increased
Investigations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0021 affected7 at risk
EG003
Blood cholesterol increased
Investigations
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Blood creatine phosphokinase increased
Investigations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0012 affected16 at risk
EG0020 affected7 at risk
EG003
Blood lactate dehydrogenase increased
Investigations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Blood magnesium increased
Investigations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
C-reactive protein increased
Investigations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0021 affected7 at risk
EG003
Epstein-Barr virus test positive
Investigations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Gamma-glutamyltransferase increased
Investigations
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Hepatic enzyme increased
Investigations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Herpes simplex test positive
Investigations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Immunoglobulins decreased
Investigations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Lymphocyte count decreased
Investigations
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Neutrophil count decreased
Investigations
MedDRA (27.0)
Systematic Assessment
EG0005 affected22 at risk
EG0012 affected16 at risk
EG0022 affected7 at risk
EG003
Platelet count decreased
Investigations
MedDRA (27.0)
Systematic Assessment
EG0005 affected22 at risk
EG0012 affected16 at risk
EG0020 affected7 at risk
EG003
Serum ferritin increased
Investigations
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Vascular access device culture positive
Investigations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Weight decreased
Investigations
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0012 affected16 at risk
EG0020 affected7 at risk
EG003
Weight increased
Investigations
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
White blood cell count decreased
Investigations
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0012 affected16 at risk
EG0021 affected7 at risk
EG003
Decreased appetite
Metabolism and nutrition disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Hypercholesterolaemia
Metabolism and nutrition disorders
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Hyperkalaemia
Metabolism and nutrition disorders
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0012 affected16 at risk
EG0020 affected7 at risk
EG003
Hyperphosphataemia
Metabolism and nutrition disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Hypertriglyceridaemia
Metabolism and nutrition disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Hypoglycaemia
Metabolism and nutrition disorders
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Hypokalaemia
Metabolism and nutrition disorders
MedDRA (27.0)
Systematic Assessment
EG0003 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Hypomagnesaemia
Metabolism and nutrition disorders
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0012 affected16 at risk
EG0020 affected7 at risk
EG003
Hypophosphataemia
Metabolism and nutrition disorders
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Hypozincaemia
Metabolism and nutrition disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Obesity
Metabolism and nutrition disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Steroid diabetes
Metabolism and nutrition disorders
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Vitamin D deficiency
Metabolism and nutrition disorders
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Arthralgia
Musculoskeletal and connective tissue disorders
MedDRA (27.0)
Systematic Assessment
EG0003 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Back pain
Musculoskeletal and connective tissue disorders
MedDRA (27.0)
Systematic Assessment
EG0003 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Bone pain
Musculoskeletal and connective tissue disorders
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Foot deformity
Musculoskeletal and connective tissue disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Knee deformity
Musculoskeletal and connective tissue disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Osteopenia
Musculoskeletal and connective tissue disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Osteoporosis
Musculoskeletal and connective tissue disorders
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Pain in extremity
Musculoskeletal and connective tissue disorders
MedDRA (27.0)
Systematic Assessment
EG0003 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Skin papilloma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Headache
Nervous system disorders
MedDRA (27.0)
Systematic Assessment
EG0005 affected22 at risk
EG0012 affected16 at risk
EG0020 affected7 at risk
EG003
Polyneuropathy
Nervous system disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Insomnia
Psychiatric disorders
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Acquired phimosis
Reproductive system and breast disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Pruritus genital
Reproductive system and breast disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Acute respiratory failure
Respiratory, thoracic and mediastinal disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Cough
Respiratory, thoracic and mediastinal disorders
MedDRA (27.0)
Systematic Assessment
EG0003 affected22 at risk
EG0013 affected16 at risk
EG0020 affected7 at risk
EG003
Epistaxis
Respiratory, thoracic and mediastinal disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Oropharyngeal pain
Respiratory, thoracic and mediastinal disorders
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Pneumothorax
Respiratory, thoracic and mediastinal disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Productive cough
Respiratory, thoracic and mediastinal disorders
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Rhinorrhoea
Respiratory, thoracic and mediastinal disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Acne
Skin and subcutaneous tissue disorders
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Alopecia
Skin and subcutaneous tissue disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Dermatitis contact
Skin and subcutaneous tissue disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Dry skin
Skin and subcutaneous tissue disorders
MedDRA (27.0)
Systematic Assessment
EG0003 affected22 at risk
EG0010 affected16 at risk
EG0020 affected7 at risk
EG003
Eczema
Skin and subcutaneous tissue disorders
MedDRA (27.0)
Systematic Assessment
EG0002 affected22 at risk
EG0012 affected16 at risk
EG0020 affected7 at risk
EG003
Erythema
Skin and subcutaneous tissue disorders
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0012 affected16 at risk
EG0020 affected7 at risk
EG003
Hypertrichosis
Skin and subcutaneous tissue disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0011 affected16 at risk
EG0021 affected7 at risk
EG003
Pruritus
Skin and subcutaneous tissue disorders
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0011 affected16 at risk
EG0020 affected7 at risk
EG003
Rash
Skin and subcutaneous tissue disorders
MedDRA (27.0)
Systematic Assessment
EG0001 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Rash macular
Skin and subcutaneous tissue disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Skin haemorrhage
Skin and subcutaneous tissue disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Skin irritation
Skin and subcutaneous tissue disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Urticaria
Skin and subcutaneous tissue disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0012 affected16 at risk
EG0020 affected7 at risk
EG003
Hot flush
Vascular disorders
MedDRA (27.0)
Systematic Assessment
EG0000 affected22 at risk
EG0010 affected16 at risk
EG0021 affected7 at risk
EG003
Hypertension
Vascular disorders
MedDRA (27.0)
Systematic Assessment
EG0003 affected22 at risk
EG0013 affected16 at risk
EG0021 affected7 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 terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of pooled data (i.e.,data from all sites) in clinical trial or disclosure of trial results in their entirety.