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| ID | Type | Description | Link |
|---|---|---|---|
| PRN1008-018 | Other Identifier | Sanofi Identifier | |
| 2023-509401-71 | Registry Identifier | CTIS |
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This was a randomized, double-blind study of rilzabrutinib in patients with persistent or chronic ITP, with an average platelet count of <30,000/μL (and no single platelet count >35,000/μL) on two counts at least 5 days apart in the 14 days before treatment begins. Patients received rilzabrutinib or placebo 400mg twice daily.
For each patient, the study lasted up to 60 weeks from the start of the Screening Period to the End of Study (EOS) visit. This included Screening (up to 4 weeks) through a 12 to 24-week Blinded Treatment Period followed by a 28-week Open-Label Period. Followed by a 4-week post dose follow-up.
For adult participants, the maximum duration of the long-term extension (LTE) period was 12 months from the date of the last adult participant to enter the LTE.
For pediatric participants, the maximum duration of the LTE period was 12 months from the date of the last pediatric participant to enter the LTE.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Rilzabrutinib | Experimental | Patients receive rilzabrutinib 400mg orally twice daily for up to 24 weeks followed by 28 weeks of open label period |
|
| Placebo | Placebo Comparator | Patients receive matching placebo 400mg orally twice daily for up to 24 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Rilzabrutinib | Drug | 400mg Caplet |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in Regions Except European Union and United Kingdom | Durable platelet response per guidance in regions except European Union and United Kingdom was defined as platelet counts at or above 50,000/mcL for >=two-thirds of at least 8 non-missing weekly scheduled platelet measurements during the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy, provided that at least 2 non-missing weekly scheduled platelet measurements were at or above 50,000/mcL during the last 6 weeks of the 24-week blinded treatment period. | Up to 24 weeks |
| DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in European Union and United Kingdom | Durable platelet response per guidance in European Union and United Kingdom was defined as platelet counts at or above 50,000/mcL for at least 8 out of the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy. | Up to 24 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| DB Period: Number of Weeks With Platelet Count >=50,000/mcL or Between >=30,000/mcL and <50,000/mcL and at Least Doubled From Baseline in the Absence of Rescue Therapy | Number of weeks with platelet response was assessed using clinically meaningful threshold of platelet count >=50,000/mcL or between >=30,000/mcL and <50,000/mcL and at least doubled from baseline over the 24-week blinded treatment period in the absence of rescue therapy. |
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Inclusion Criteria:
Male and female with primary ITP with duration of >6 months in pediatric participants aged 12 to <18 years (pediatric participants aged 10 to <12 years will be enrolled in the EU [EEA countries] only) and duration of >3 months in ages 18 years and above
Patients who had a response (achievement of platelet count ≥50,000/µL) to IVIg/anti-D or CSs that was not sustained and who have documented intolerance, insufficient response or any contra-indication to any appropriate courses of standard of care ITP therapy
An average of 2 platelet counts at least 5 days apart of <30,000/µL during the Screening period and no single platelet count >35,000/µL, within 14 days prior to the first dose of study drug.
- Pediatric patients must additionally be determined to need treatment for ITP as per clinical assessment by the Investigator.
Adequate hematologic, hepatic, and renal function (absolute neutrophil count ≥1.5 X 10^9/L, AST/ALT ≤1.5 x upper limit of normal [ULN], albumin ≥3 g/dL, total bilirubin ≤1.5 x ULN [unless the patient has documented Gilbert syndrome], glomerular filtration rate >50 [Cockcroft and Gault method for adult and Bedside Schwartz Equation for Pediatric participants])
Hemoglobin >9 g/dL within 1 week prior to Study Day 1
All contraceptive use by men and women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
Patients must be able to provide written informed consent or informed assent with corresponding informed consent obtained from the patient's guardian and agree to the schedule of assessments
Exclusion Criteria:
Patients with secondary ITP
Pregnant or lactating women
History (within 5 years of Study Day 1) or current, active malignancy requiring or likely to require chemotherapeutic or surgical treatment during the study, with the exception of non melanoma skin cancer
Transfusion with blood, blood products, plasmapheresis, or use of any other rescue medications with intent to increase platelet count within 14 days before Study Day 1
Change in CS and/or TPO-RA dose within 14 days prior to Study Day 1 (more than 10% variation from current doses)
Immunosuppressant drugs other than CSs within 5 times the elimination half-life of the drug or 14 days of Study Day 1, whichever is longer
Treatment with rituximab or splenectomy within the 3 months prior to Study Day 1
- Patients treated with rituximab will have normal B-cell counts prior to enrollment
Had received any investigational drug within the 30 days before receiving the first dose of study medication, or at least 5 times elimination half-life of the drug (whichever is longer); patient should not be using an investigational device at the time of dosing
History of solid organ transplant
Myelodysplastic syndrome
Live vaccine within 28 days prior to Study Day 1 or plan to receive one during the study
Planned surgery in the time frame of the dosing period
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Southern California_Investigational Site Number 84024 | Los Angeles | California | 90033 | United States | ||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 40090011 | Derived | Kuter DJ, Ghanima W, Cooper N, Liebman HA, Zhang L, Hu Y, Miyakawa Y, Homenda W, Galindo LEM, Basquiera AL, Tan CW, Saydam G, Hutter-Kronke ML, Chai-Adisaksopha C, Gomez-Almaguer D, Tran H, Shin HJ, Dantas da Cunha Junior A, Lazar Z, Izquierdo CP, Kirgner I, Lucchini E, Kuzmina G, Fillitz M, Audia S, Taparia M, Cordoba M, Diab R, Yao M, Gouia I, Lee M, Daak A. Safety and efficacy of rilzabrutinib vs placebo in adults with immune thrombocytopenia: the phase 3 LUNA3 study. Blood. 2025 Jun 12;145(24):2914-2926. doi: 10.1182/blood.2024027336. | |
| 39899371 |
| Label | URL |
|---|---|
| EFC17093 Plain Language Results Summary | View source |
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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
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202 adult and 30 adolescent participants were randomized in the study. The study consisted of 3 periods: double-blind (DB) period, open-label (OL) period and safety follow-up or long-term extension (LTE) period. Interim results have been reported up to the database lock (DBL) date of 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period).
The study was conducted at 92 centers in 25 countries for adult participants and 20 centers in 11 countries for adolescent participants. 393 adult and 51 adolescent participants were screened between 14 December 2020 to 30 September 2024, of which 191 adult and 21 adolescent participants were screen failures. Screen failures were mainly due to not meeting eligibility criteria.
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| ID | Title | Description |
|---|---|---|
| FG000 | DB Period: Adults: Placebo | Adult participants received placebo matched to rilzabrutinib tablet twice daily (BID) orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000 per microliter (/mcL) or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| DB Period (Weeks 1 to 24) |
|
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| 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 | Dec 24, 2025 |
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Double-Blind
| Placebo | Drug | 400mg Caplet |
|
|
| Up to 24 weeks |
| DB Period: Number of Weeks With Platelet Counts >=30,000/mcL and at Least Doubled From Baseline in the Absence of Rescue Therapy | Number of weeks with platelet response was assessed using clinically meaningful threshold of platelet counts >=30,000/mcL and at least doubled from baseline over the 24-week blinded treatment period in the absence of rescue therapy. | Up to 24 weeks |
| DB Period: Time to First Platelet Count of >=50,000/mcL or Between >=30,000/mcL and <50,000/mcL and Doubled From Baseline | Time to first platelet count of >=50,000/mcL or between >=30,000/mcL and <50,000/mcL and doubled from baseline was calculated as: (date of first occurrence of platelet response - date of first study drug intake) + 1. | Up to 24 weeks |
| DB Period: Percentage of Participants Who Required Rescue Therapy | Resue therapy included intravenous immunoglobulin (Ig) or high-dose corticosteroids, platelet infusion, or anti-D Ig infusion. Percentage of participants who required rescue therapy are presented. | Up to 24 weeks |
| DB Period: Change From Baseline on Item 10 (Physical Fatigue) of the Immune Thrombocytopenia-Patient Assessment Questionnaire (ITP-PAQ) in Adult Participants at Week 13 | ITP-PAQ:38 items and 44 items completed by male and female respondents respectively.It included 10 scales:symptoms(6 items:1-6),fatigue/sleep(4 items:7-10),bother-physical health(3 items:11-13),activity(2 items:14-15),psychological health(5 items:16-20),fear(5 items:21-25),overall quality of life(QoL)(5 items:26-30),social activity(4 items:31-34),women's reproductive health(6 items:35-40),work(4 items:41-44).Responses were recorded on 4-point(1:"strongly disagree" to 4:"strongly agree"),5-point(1:"never/not at all" to 5:"all the time/extremely") or 7-point(1:"not at all" to 7:"extremely") Likert scales.Each item score:100x([possible maximal item score - item score]/range).All item scores:transformed to 0 to 100 continuum and were weighted equally to derive scale scores.Total score:0(worst) to 100(best);higher scores:better QoL.Change of positive value:improvement.Change from baseline in physical fatigue is presented.Baseline:last available value before first dose of DB study drug. | Baseline (Day 1) and Week 13 |
| DB Period: Change From Baseline in Idiopathic Thrombocytopenic Purpura Bleeding Scale (IBLS) Assessment in Adult Participants at Week 25 Per Guidance in European Union and United Kingdom | Per guidance in European Union and United Kingdom, ITP IBLS was a bleeding assessment system which comprised of 11 (10 for male) site-specific grades assessed at 9 anatomical sites by history (Hx) over previous period. In addition, 2 of these sites, skin and oral, were also assessed by physical examination (PE). These 11 grades included: skin (PE), skin (Hx), oral (PE), oral (Hx), epistaxis, gastrointestinal, urinary, gynecological, pulmonary, intracranial hemorrhage, and subconjunctival hemorrhage; scores ranged from 0 (none) to 2 (marked bleeding). For each participant, an IBLS score at each visit was calculated by taking average across 11 items (10 for male and postmenopausal female) at 9 sites (8 for male and postmenopausal female). Total score was calculated as mean value for all 11 grades ranging from 0 (best) to 22 (worst); higher scores indicated worse outcomes. Change of negative value indicates an improvement.Baseline:last available value before first dose of DB study drug. | Baseline (Day 1) and Week 25 |
| DB Period: Change From Baseline in Idiopathic Thrombocytopenic Purpura Bleeding Scale Assessment in Adolescent Participants at Week 25 Per Guidance in European Union and United Kingdom | Per guidance in European Union and United Kingdom, ITP IBLS was a bleeding assessment system which comprised of 11 (10 for male) site-specific grades assessed at 9 anatomical sites by Hx over previous period. In addition, 2 of these sites, skin and oral, were also assessed by PE. These 11 grades included: skin (PE), skin (Hx), oral (PE), oral (Hx), epistaxis, gastrointestinal, urinary, gynecological, pulmonary, intracranial hemorrhage, and subconjunctival hemorrhage; scores ranged from 0 (none) to 2 (marked bleeding). For each participant, an IBLS score at each visit was calculated by taking average across 11 items (10 for male) at 9 sites (8 for male). Total score was calculated as mean value for all 11 grades ranging from 0 (best) to 22 (worst); higher scores indicated worse outcomes. Change of negative value indicates an improvement. Baseline:last available value before first dose of DB study drug. | Baseline (Day 1) and Week 25 |
| DB-OL Period: Percentage of Participants With Stability of Response in Adult Participants | Stability of response was defined as the percentage of participants who were able to achieve stable platelet response defined as no 2 scheduled visits, at least 4 weeks apart, with a platelet count <50,000/mcL, without an intervening visit with a platelet count >=50,000/mcL, within a period of 24 weeks following initial achievement of the platelet response (initial platelet response defined as platelet count >=50,000/mcL within 12 weeks of initiation of treatment with rilzabrutinib during the study). This endpoint was assessed from start of DB period through OL period. | Up to 52 weeks |
| DB-OL Period: Percentage of Participants With Stability of Response in Adolescent Participants | Up to 52 weeks |
| DB Period: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs) and Bleeding Treatment-Emergent Adverse Events >=Grade 2 | An AE was any untoward medical occurrence in a participant or clinical investigation participant, administered a study drug and which did not necessarily had a causal relationship with the study drug. An SAE was any AE that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect or was an important medical event. TEAEs were defined as AEs that developed, worsened or became serious during the TE period. Bleeding TEAEs Grade >=2 (criteria mentioned in statistical analysis plan) are also presented. | From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days (adults) and up to 175 days (adolescent) |
| DB Period: Plasma Concentrations of Rilzabrutinib | Blood samples were collected at specified timepoints for the analysis of plasma concentration of rilzabrutinib. | Pre-dose at Weeks 1, 13 and 25 and 2 hours post-dose at Week 1 and 25 (adults); Pre-dose at Weeks 1, 13 and 25, 0.5, 2, 4 and 6 hours post-dose at Weeks 1 and 25 (adolescent) |
| DB Period: Change From Baseline in the Symptoms, Bother and Activity Domains of the Immune Thrombocytopenia-Patient Assessment Questionnaire in Adult Participants | ITP-PAQ:38 items and 44 items completed by male and female respondents respectively.It included 10 scales:symptoms(6 items:1-6),fatigue/sleep(4 items:7-10),bother-physical health(3 items:11-13),activity(2 items:14-15),psychological health(5 items:16-20),fear(5 items:21-25),overall QoL(5 items:26-30),social activity(4 items:31-34),women's reproductive health(6 items:35-40),work(4 items:41-44).Responses were recorded on 4-point(1:"strongly disagree" to 4:"strongly agree"),5-point(1:"never/not at all" to 5:"all the time/extremely") or 7-point(1:"not at all" to 7:"extremely") Likert scales.Each item score:100x([possible maximal item score - item score]/range).All item scores:transformed to 0 to 100 continuum and were weighted equally to derive scale scores.Total score:0(worst) to 100(best);higher scores:better QoL.Change of positive value:improvement.Change from baseline in symptoms,bother and activity domains is presented.Baseline:last available value before first dose of DB study drug. | Baseline (Day 1) and Week 25 |
| DB Period: Change From Baseline in Disease-Specific Quality Of Life as Measured by the Kids' Immune Thrombocytopenia Tools (ITP-KIT) Score in Adolescent Participants | ITP-KIT was a disease-specific instrument and child self-report form designed to be completed by children >=7 years. It comprised of total of 27 items among which 26 items were structured as Likert scales with 5 response options 1: "never", 2: "rarely", 3: "sometimes", 4: "often" and 5: "always". An additional "not applicable" option was available for items 18 to 26, which was scored as 1: the same as "never". Item 27 was a descriptive question answered "yes" or "no" which was not included in the calculation of the summary score. Instrument yielded a summary KIT score which was the summation of the items calculated as: 100 x (1- [{sum of all valid responses - number of valid responses}/{number of valid responses*4}]). Scores were converted to a 0 to 100 with higher scores indicating better disease-specific QoL. Change from baseline of positive value indicated improvement. Baseline was defined as the last available value before first dose of DB study drug. | Baseline (Day 1) and Week 25 |
| UCSF Benioff Children's Hospital San Francisco_Investigational Site Number 84020 |
| San Francisco |
| California |
| 94158 |
| United States |
| Lundquist Institute for Biomedical Innovation at Harbor UCLA Medical Center_Investigational Site Number 84037 | Torrance | California | 90502 | United States |
| The Oncology Institute of Hope and Innovation_Investigational Site Number 84031 | Whittier | California | 90602 | United States |
| Children's Hospital Colorado_Investigational Site Number 84025 | Aurora | Colorado | 80045 | United States |
| IMMUNOe International Research Centers_Investigational Site Number 84028 | Centennial | Colorado | 80112 | United States |
| ASCLEPES Research Centers_Investigational Site Number 84023 | Weeki Wachee | Florida | 34607 | United States |
| Children's Healthcare of Atlanta_Investigational Site Number 84034 | Atlanta | Georgia | 30322 | United States |
| Rush University Medical Center_Investigational Site Number 84029 | Chicago | Illinois | 60612 | United States |
| University of Louisville - James Graham Brown Cancer Center_Investigational Site Number 84033 | Louisville | Kentucky | 40202 | United States |
| Massachusetts General Hospital Site Number : 84038 | Boston | Massachusetts | 02114 | United States |
| Montefiore Medical Center_Investigational Site Number 84032 | The Bronx | New York | 10461 | United States |
| University Hospitals Cleveland Medical Center Site Number : 84036 | Cleveland | Ohio | 44106 | United States |
| Cleveland Clinic_Investigational Site Number 84026 | Cleveland | Ohio | 44195 | United States |
| The Children's Hospital of Philadelphia (CHOP)_Investigational Site Number 84027 | Philadelphia | Pennsylvania | 19104 | United States |
| University of Utah-Huntsman Cancer Institute_Investigational Site Number 84035 | Salt Lake City | Utah | 84112 | United States |
| University of Washington Medical Centre Site Number : 84041 | Seattle | Washington | 98195 | United States |
| Investigational Site Number : 3206 | Capital Federal | Buenos Aires | C1280AEB | Argentina |
| Investigational Site Number : 3211 | La Plata | Buenos Aires | B1900 | Argentina |
| Investigational Site Number : 3205 | Córdoba | Córdoba Province | X5003DCE | Argentina |
| Investigational Site Number : 3209 | Corrientes | W3410 FND | Argentina |
| Investigational Site Number : 3208 | San Juan | 5400 | Argentina |
| Investigational Site Number : 3607 | Kogarah | New South Wales | 2217 | Australia |
| Investigational Site Number : 3608 | Westmead | New South Wales | 2145 | Australia |
| Investigational Site Number : 3611 | Brisbane | Queensland | 4101 | Australia |
| Investigational Site Number : 3609 | Adelaide | South Australia | 5000 | Australia |
| Investigational Site Number : 3606 | Frankston | Victoria | 3199 | Australia |
| Investigational Site Number : 3610 | Perth | Western Australia | 6000 | Australia |
| Investigational Site Number : 4005 | Graz | 8036 | Austria |
| Investigational Site Number : 4004 | Leoben | 8700 | Austria |
| Investigational Site Number : 4001 | Linz | A4020 | Austria |
| Investigational Site Number : 4003 | Steyr | 4400 | Austria |
| Investigational Site Number : 4002 | Vienna | 1140 | Austria |
| Hospital Sao Rafael Instituto D'Or da Bahia Site Number : 7608 | Salvador | Estado de Bahia | 41253190 | Brazil |
| Uniao Oeste Paranaense de Estudos e Combates ao Cancer Site Number : 7610 | Cascavel | Paraná | 85806-300 | Brazil |
| Hospital De Clinicas De Porto Alegre Site Number : 7605 | Porto Alegre | Rio Grande do Sul | 90035 003 | Brazil |
| Hospital Santa Marcelina Site Number : 7611 | São Paulo | São Paulo | 08270-070 | Brazil |
| CEMEC Oncologica do Brasil Site Number : 7606 | Belém | 66053-000 | Brazil |
| HEMORIO - Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti Site Number : 7609 | Rio de Janeiro | 20211030 | Brazil |
| Hospital do Servidor Publico Estadual de Sao Paulo Site Number : 7607 | São Paulo | 04039-004 | Brazil |
| Investigational Site Number : 12404 | Edmonton | Alberta | T6G 2P4 | Canada |
| Investigational Site Number : 12406 | Toronto | Ontario | M5G 1X8 | Canada |
| CHU Sainte-Justine_Investigational site number 12405 | Montreal | Quebec | H3T 1C5 | Canada |
| Investigational Site Number : 15201 | La Serena | Coquimbo Region | 1720430 | Chile |
| Investigational Site Number : 15204 | Santiago | Reg Metropolitana de Santiago | 7500653 | Chile |
| Investigational Site Number : 15202 | Viña del Mar | Valparaiso | 322000 | Chile |
| Wuhan Union Hospital of Tongji Medical College of HUST - Investigational Site Number: 15601 | Wuhan | Hubei | 430022 | China |
| Shengjing Hospital of China Medical University - Investigational Site Number: 15603 | Shenyang | Liaoning | 110022 | China |
| Shaanxi Provincial People's Hospital - Investigational Site Number: 15607 | Xi'an | Shaanxi | 710068 | China |
| Qilu Hospital of Shandong University - Investigational Site Number: 15605 | Jinan | Shandong | 250012 | China |
| Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences - Investigational Site Number: 15602 | Tianjin | Tianjin Municipality | 300020 | China |
| Second Affiliated Hospital of Kunming Medical University - Investigational Site Number: 15604 | Kunming | Yunnan | 650101 | China |
| Investigational Site Number : 15611 | Hangzhou | 310018 | China |
| Investigational Site Number : 15608 | Hefei | China |
| Investigational Site Number : 15610 | Nanchang | 330006 | China |
| Investigational Site Number : 15613 | Tangshan | 63000 | China |
| Investigational Site Number : 15609 | Wuxi | 214023 | China |
| Investigational Site Number : 15614 | Zhenjiang | 212001 | China |
| Investigational Site Number : 25014 | Angers | 49933 | France |
| Investigational Site Number : 25011 | Créteil | 94000 | France |
| Investigational Site Number : 25010 | Dijon | 21079 | France |
| Investigational Site Number : 25009 | Nantes | 44093 | France |
| Investigational Site Number : 25008 | Paris | 75012 | France |
| Investigational Site Number : 25012 | Paris | 75019 | France |
| Investigational Site Number : 25007 | Pessac | 33600 | France |
| Investigational Site Number : 27610 | Berlin | 10117 | Germany |
| Investigational Site Number 27612 | Düsseldorf | 40497 | Germany |
| Investigational Site Number : 27613 | Frankfurt am Main | 60596 | Germany |
| Investigational Site Number : 27611 | Recklinghausen | 45659 | Germany |
| Investigational Site Number : 34803 | Budapest | 1083 | Hungary |
| Investigational Site Number : 34805 | Debrecen | 4032 | Hungary |
| Investigational Site Number : 34801 | Győr | 9024 | Hungary |
| Investigational Site Number : 34804 | Nyíregyháza | 4405 | Hungary |
| Investigational Site Number : 34802 | Székesfehérvár | 8000 | Hungary |
| Investigational Site Number : 37605 | Haifa | 3109601 | Israel |
| Investigational Site Number : 37606 | Kfar Saba | 4428164 | Israel |
| Investigational Site Number : 37607 | Tel Aviv | 6423906 | Israel |
| Investigational Site Number : 37608 | Tel Litwinsky | 52621 | Israel |
| Investigational Site Number : 37609 | Ẕerifin | 70300 | Israel |
| Investigational Site Number : 38014 | Florence | Tuscany | 50134 | Italy |
| Investigational Site Number : 38012 | Bologna | 40138 | Italy |
| Investigational Site Number : 38015 | Milan | 20123 | Italy |
| Investigational Site Number : 38013 | Milan | 20142 | Italy |
| Investigational Site Number : 38010 | Trieste | 34125 | Italy |
| Investigational Site Number : 38011 | Vicenza | 36100 | Italy |
| Investigational Site Number : 39214 | Tsuchiura-shi | Ibaraki | Japan |
| Investigational Site Number : 39205 | Kanazawa | Ishikawa-ken | 920-8530 | Japan |
| Investigational Site Number : 39207 | Sagamihara-shi | Kanagawa | 252-0375 | Japan |
| Investigational Site Number : 39202 | Suita-shi | Osaka | 565-0871 | Japan |
| Investigational Site Number : 39201 | Iruma-gun | Saitama | 350-0495 | Japan |
| Investigational Site Number : 39208 | Bunkyo-ku | Tokyo | 113-8655 | Japan |
| Investigational Site Number : 39210 | Meguro-ku | Tokyo | 152-8902 | Japan |
| Investigational Site Number : 39204 | Setagaya City | Tokyo | 157-0074 | Japan |
| Investigational Site Number : 39209 | Sumida-ku | Tokyo | 130-8575 | Japan |
| Investigational Site Number : 39212 | Chiba | 60-0852 | Japan |
| Investigational Site Number : 39203 | Hiroshima | 730-8619 | Japan |
| Investigational Site Number : 39206 | Saitama-shi | 330-8777 | Japan |
| Investigational Site Number : 48401 | Monterrey | Nuevo León | 64460 | Mexico |
| Investigational Site Number : 48402 | Chihuahua City | 31200 | Mexico |
| Investigational Site Number : 48405 | Delegacion Benito Juarez | 03720 | Mexico |
| Investigational Site Number : 48404 | Durango | 34000 | Mexico |
| Investigational Site Number : 48406 | Mexico City | 06760 | Mexico |
| Investigational Site Number : 48403 | Zapopan | 45030 | Mexico |
| Erasmus MC_Investigational Site Number 52801 | Rotterdam | 3015 GD | Netherlands |
| Investigational Site Number : 57802 | Bergen | 5021 | Norway |
| Investigational Site Number : 57801 | Grålum | 1714 | Norway |
| Investigational Site Number : 61617 | Piła | Greater Poland Voivodeship | 64-920 | Poland |
| Investigational Site Number : 61609 | Słupsk | Pomeranian Voivodeship | 76-200 | Poland |
| Investigational Site Number : 61615 | Gdynia | 81-519 | Poland |
| Investigational Site Number : 61614 | Poznan | 61 696 | Poland |
| Investigational Site Number : 61612 | Warsaw | 02-776 | Poland |
| Investigational Site Number : 61613 | Wroclaw | 50-556 | Poland |
| Investigational Site Number : 64307 | Moscow | 119049 | Russia |
| Investigational Site Number : 64305 | Moscow | 125167 | Russia |
| Investigational Site Number : 64304 | Novosibirsk | 630090 | Russia |
| Investigational Site Number : 64301 | Pyatigorsk | 357502 | Russia |
| Investigational Site Number : 64302 | Saint Petersburg | 191024 | Russia |
| Investigational Site Number : 64306 | Samara | 443099 | Russia |
| Investigational Site Number : 64303 | Tula | 300053 | Russia |
| Investigational Site Number : 70201 | Singapore | 119228 | Singapore |
| Investigational Site Number : 70202 | Singapore | 169608 | Singapore |
| Investigational Site Number : 70203 | Singapore | 308433 | Singapore |
| Investigational Site Number : 41004 | Busan | Busan | 49241 | South Korea |
| Investigational Site Number : 41001 | Suwon | Gyeonggi-do | 16499 | South Korea |
| Investigational Site Number : 41003 | Seoul | Seoul-teukbyeolsi | 02841 | South Korea |
| Investigational Site Number : 41005 | Seoul | Seoul-teukbyeolsi | 03080 | South Korea |
| Investigational Site Number : 41006 | Seoul | Seoul-teukbyeolsi | 03722 | South Korea |
| Investigational Site Number : 72408 | Málaga | Málaga | 29010 | Spain |
| Investigational Site Number : 72407 | Valencia | Valenciana, Comunidad | 46010 | Spain |
| Investigational Site Number : 72412 | Barcelona | 08035 | Spain |
| Investigational Site Number : 72414 | Barcelona | 08035 | Spain |
| Investigational Site Number : 72409 | Barcelona | 08041 | Spain |
| Investigational Site Number : 72416 | Burgos | Spain |
| Investigational Site Number : 72410 | Madrid | 28007 | Spain |
| Investigational Site Number : 72411 | Murcia | 30008 | Spain |
| Investigational Site Number : 72413 | Seville | 41013 | Spain |
| Investigational Site Number : 76405 | Bangkok | 10330 | Thailand |
| Investigational Site Number : 76404 | Bangkok | 10400 | Thailand |
| Investigational Site Number : 76402 | Chiang Mai | 50200 | Thailand |
| Investigational Site Number : 76401 | Khon Kaen | 40002 | Thailand |
| Investigational Site Number : 76403 | Songkhla | 90110 | Thailand |
| Investigational Site Number 79208 | Ankara | 06620 | Turkey (Türkiye) |
| Investigational Site Number 79210 | Istanbul | 34093 | Turkey (Türkiye) |
| Investigational Site Number 79206 | Izmir | 35100 | Turkey (Türkiye) |
| Investigational Site Number 79209 | Kayseri | 38039 | Turkey (Türkiye) |
| Investigational Site Number : 80408 | Dnipropetrovsk | 49102 | Ukraine |
| Investigational Site Number : 80409 | Kryvyi Rih | 50025 | Ukraine |
| Investigational Site Number : 80410 | Kyiv | 03143 | Ukraine |
| Investigational Site Number : 82607 | London | London, City of | W12 0HS | United Kingdom |
| Investigational Site Number : 82604 | Harrow | HA1 3UJ | United Kingdom |
| Investigational Site Number : 82606 | London | SE5 9PJ | United Kingdom |
| Investigational Site Number : 82609 | London | W2 1NY | United Kingdom |
| Investigational Site Number : 82603 | Manchester | M13 9WL | United Kingdom |
| Investigational Site Number : 82605 | Norfolk | NR31 6LA | United Kingdom |
| Investigational Site Number : 82608 | Southampton | SO16 6YD | United Kingdom |
| Derived |
| Lin J, Radhakrishnan J. What Are Baskets, Umbrellas, and Platforms Doing in Nephrology Clinical Trials? J Am Soc Nephrol. 2025 Feb 3;36(8):1652-1654. doi: 10.1681/ASN.0000000648. No abstract available. |
| 37869360 | Derived | Kuter DJ, Bussel JB, Ghanima W, Cooper N, Gernsheimer T, Lambert MP, Liebman HA, Tarantino MD, Lee M, Guo H, Daak A. Rilzabrutinib versus placebo in adults and adolescents with persistent or chronic immune thrombocytopenia: LUNA 3 phase III study. Ther Adv Hematol. 2023 Oct 18;14:20406207231205431. doi: 10.1177/20406207231205431. eCollection 2023. |
| FG001 | DB Period: Adults: Rilzabrutinib 400 mg BID | Adult participants received rilzabrutinib 400 milligrams (mg) tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
| FG002 | DB Period: Adolescent: Placebo | Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
| FG003 | DB Period: Adolescent: Rilzabrutinib 400 mg BID | Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
| FG004 | OL Period: Adults: Rilzabrutinib 400 mg BID | Eligible adult participants (responders after 24 weeks of DB period and non-responders after 12 weeks of DB period) entered the OL period to receive rilzabrutinib 400 mg tablet BID orally for 28 weeks. |
| FG005 | OL Period: Adolescent: Rilzabrutinib 400 mg BID | Eligible adolescent participants (responders after 24 weeks of DB period and non-responders after 12 weeks of DB period) entered the OL period to receive rilzabrutinib 400 mg tablet BID orally for 28 weeks. |
| FG006 | LTE Period: Adults: Rilzabrutinib 400 mg BID | Adult participants who completed the OL period and demonstrated a platelet response defined as: platelet counts >=50,000/mcL or >=30,000/mcL and at least doubled from baseline at >=50% of the visits without receiving rescue therapy while on treatment during the last 8 weeks of the OL period were allowed to enter the LTE period and received rilzabrutinib 400 mg tablet BID orally in OL period. The maximum duration of the LTE period will be 12 months from the date of the last adult participant to enter the LTE. |
| FG007 | LTE Period: Adolescent: Rilzabrutinib 400 mg BID | Adolescent participants who completed the OL period and demonstrated a platelet response defined as: platelet counts >=50,000/mcL or >=30,000/mcL and at least doubled from baseline at >=50% of the visits without receiving rescue therapy while on treatment during the last 8 weeks of the OL period were allowed to enter the LTE period and received rilzabrutinib 400 mg tablet BID orally in OL period. The maximum duration of the LTE period will be 12 months from the date of the last adolescent participant to enter the LTE. |
| Completed 12-week DB Period |
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| COMPLETED | 24-week DB period |
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| NOT COMPLETED |
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| OL Period (From Week 25 to Week 53) |
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| LTE(Weeks 53-183[Adult]&147[Adolescent]) |
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Adult and adolescent randomized population included all participants from screened population who were allocated to a randomized study drug by interactive response technology regardless of whether the study drug was received or not.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | DB Period: Adults: Placebo | Adult participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
| BG001 | DB Period: Adults: Rilzabrutinib 400 mg BID | Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
| BG002 | DB Period: Adolescent: Placebo | Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
| BG003 | DB Period: Adolescent: Rilzabrutinib 400 mg BID | Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
| BG004 | Total | Total of all reporting groups |
| Units | Counts |
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| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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| 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 | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in Regions Except European Union and United Kingdom | Durable platelet response per guidance in regions except European Union and United Kingdom was defined as platelet counts at or above 50,000/mcL for >=two-thirds of at least 8 non-missing weekly scheduled platelet measurements during the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy, provided that at least 2 non-missing weekly scheduled platelet measurements were at or above 50,000/mcL during the last 6 weeks of the 24-week blinded treatment period. | Adult and adolescent intent-to-treat (ITT) population included all randomized participants. | Posted | Number | 95% Confidence Interval | percentage of participants | Up to 24 weeks |
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| Primary | DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in European Union and United Kingdom | Durable platelet response per guidance in European Union and United Kingdom was defined as platelet counts at or above 50,000/mcL for at least 8 out of the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy. | Adult and adolescent ITT population included all randomized participants. | Posted | Number | 95% Confidence Interval | percentage of participants | Up to 24 weeks |
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| Secondary | DB Period: Number of Weeks With Platelet Count >=50,000/mcL or Between >=30,000/mcL and <50,000/mcL and at Least Doubled From Baseline in the Absence of Rescue Therapy | Number of weeks with platelet response was assessed using clinically meaningful threshold of platelet count >=50,000/mcL or between >=30,000/mcL and <50,000/mcL and at least doubled from baseline over the 24-week blinded treatment period in the absence of rescue therapy. | Adult and adolescent ITT population included all randomized participants. | Posted | Mean | Standard Deviation | weeks | Up to 24 weeks |
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| Secondary | DB Period: Number of Weeks With Platelet Counts >=30,000/mcL and at Least Doubled From Baseline in the Absence of Rescue Therapy | Number of weeks with platelet response was assessed using clinically meaningful threshold of platelet counts >=30,000/mcL and at least doubled from baseline over the 24-week blinded treatment period in the absence of rescue therapy. | Adult and adolescent ITT population included all randomized participants. | Posted | Mean | Standard Deviation | weeks | Up to 24 weeks |
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| Secondary | DB Period: Time to First Platelet Count of >=50,000/mcL or Between >=30,000/mcL and <50,000/mcL and Doubled From Baseline | Time to first platelet count of >=50,000/mcL or between >=30,000/mcL and <50,000/mcL and doubled from baseline was calculated as: (date of first occurrence of platelet response - date of first study drug intake) + 1. | Adult and adolescent ITT population included all randomized participants. Only those participants who achieved platelet response are reported. | Posted | Median | 95% Confidence Interval | days | Up to 24 weeks |
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| Secondary | DB Period: Percentage of Participants Who Required Rescue Therapy | Resue therapy included intravenous immunoglobulin (Ig) or high-dose corticosteroids, platelet infusion, or anti-D Ig infusion. Percentage of participants who required rescue therapy are presented. | Adult and adolescent ITT population included all randomized participants. | Posted | Number | percentage of participants | Up to 24 weeks |
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| Secondary | DB Period: Change From Baseline on Item 10 (Physical Fatigue) of the Immune Thrombocytopenia-Patient Assessment Questionnaire (ITP-PAQ) in Adult Participants at Week 13 | ITP-PAQ:38 items and 44 items completed by male and female respondents respectively.It included 10 scales:symptoms(6 items:1-6),fatigue/sleep(4 items:7-10),bother-physical health(3 items:11-13),activity(2 items:14-15),psychological health(5 items:16-20),fear(5 items:21-25),overall quality of life(QoL)(5 items:26-30),social activity(4 items:31-34),women's reproductive health(6 items:35-40),work(4 items:41-44).Responses were recorded on 4-point(1:"strongly disagree" to 4:"strongly agree"),5-point(1:"never/not at all" to 5:"all the time/extremely") or 7-point(1:"not at all" to 7:"extremely") Likert scales.Each item score:100x([possible maximal item score - item score]/range).All item scores:transformed to 0 to 100 continuum and were weighted equally to derive scale scores.Total score:0(worst) to 100(best);higher scores:better QoL.Change of positive value:improvement.Change from baseline in physical fatigue is presented.Baseline:last available value before first dose of DB study drug. | Adult ITT population included all randomized participants. | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline (Day 1) and Week 13 |
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| Secondary | DB Period: Change From Baseline in Idiopathic Thrombocytopenic Purpura Bleeding Scale (IBLS) Assessment in Adult Participants at Week 25 Per Guidance in European Union and United Kingdom | Per guidance in European Union and United Kingdom, ITP IBLS was a bleeding assessment system which comprised of 11 (10 for male) site-specific grades assessed at 9 anatomical sites by history (Hx) over previous period. In addition, 2 of these sites, skin and oral, were also assessed by physical examination (PE). These 11 grades included: skin (PE), skin (Hx), oral (PE), oral (Hx), epistaxis, gastrointestinal, urinary, gynecological, pulmonary, intracranial hemorrhage, and subconjunctival hemorrhage; scores ranged from 0 (none) to 2 (marked bleeding). For each participant, an IBLS score at each visit was calculated by taking average across 11 items (10 for male and postmenopausal female) at 9 sites (8 for male and postmenopausal female). Total score was calculated as mean value for all 11 grades ranging from 0 (best) to 22 (worst); higher scores indicated worse outcomes. Change of negative value indicates an improvement.Baseline:last available value before first dose of DB study drug. | Adult ITT population included all randomized participants. | Posted | Least Squares Mean | Standard Error | score on a scale | Baseline (Day 1) and Week 25 |
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| Secondary | DB Period: Change From Baseline in Idiopathic Thrombocytopenic Purpura Bleeding Scale Assessment in Adolescent Participants at Week 25 Per Guidance in European Union and United Kingdom | Per guidance in European Union and United Kingdom, ITP IBLS was a bleeding assessment system which comprised of 11 (10 for male) site-specific grades assessed at 9 anatomical sites by Hx over previous period. In addition, 2 of these sites, skin and oral, were also assessed by PE. These 11 grades included: skin (PE), skin (Hx), oral (PE), oral (Hx), epistaxis, gastrointestinal, urinary, gynecological, pulmonary, intracranial hemorrhage, and subconjunctival hemorrhage; scores ranged from 0 (none) to 2 (marked bleeding). For each participant, an IBLS score at each visit was calculated by taking average across 11 items (10 for male) at 9 sites (8 for male). Total score was calculated as mean value for all 11 grades ranging from 0 (best) to 22 (worst); higher scores indicated worse outcomes. Change of negative value indicates an improvement. Baseline:last available value before first dose of DB study drug. | Adolescent ITT population included all randomized participants. Only those participants with data collected at specified timepoints are reported. | Posted | Mean | Standard Deviation | score on a scale | Baseline (Day 1) and Week 25 |
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| Secondary | DB-OL Period: Percentage of Participants With Stability of Response in Adult Participants | Stability of response was defined as the percentage of participants who were able to achieve stable platelet response defined as no 2 scheduled visits, at least 4 weeks apart, with a platelet count <50,000/mcL, without an intervening visit with a platelet count >=50,000/mcL, within a period of 24 weeks following initial achievement of the platelet response (initial platelet response defined as platelet count >=50,000/mcL within 12 weeks of initiation of treatment with rilzabrutinib during the study). This endpoint was assessed from start of DB period through OL period. | Adult rilzabrutinib safety population included all randomized participants who took at least 1 dose of study drug (rilzabrutinib), regardless of the amount of study drug (rilzabrutinib) administrated. The participants were evaluated from the start of DB period up to the end of OL period. | Posted | Number | 95% Confidence Interval | percentage of participants | Up to 52 weeks |
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| Secondary | DB-OL Period: Percentage of Participants With Stability of Response in Adolescent Participants | Not Posted | Aug 2027 | Up to 52 weeks | Participants | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | DB Period: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Treatment-Emergent Serious Adverse Events (TESAEs) and Bleeding Treatment-Emergent Adverse Events >=Grade 2 | An AE was any untoward medical occurrence in a participant or clinical investigation participant, administered a study drug and which did not necessarily had a causal relationship with the study drug. An SAE was any AE that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect or was an important medical event. TEAEs were defined as AEs that developed, worsened or became serious during the TE period. Bleeding TEAEs Grade >=2 (criteria mentioned in statistical analysis plan) are also presented. | Adult and adolescent safety population included all randomized participants who took at least 1 dose of study drug, regardless of the amount of study drug administrated. | Posted | Count of Participants | Participants | From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days (adults) and up to 175 days (adolescent) |
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| Secondary | DB Period: Plasma Concentrations of Rilzabrutinib | Blood samples were collected at specified timepoints for the analysis of plasma concentration of rilzabrutinib. | Adult and adolescent pharmacokinetic (PK) population included all randomized and exposed participants (safety population) with at least 1 post-baseline PK sample. Only those participants with data collected at specified timepoints are reported. | Posted | Mean | Standard Deviation | nanogram per milliliter (ng/mL) | Pre-dose at Weeks 1, 13 and 25 and 2 hours post-dose at Week 1 and 25 (adults); Pre-dose at Weeks 1, 13 and 25, 0.5, 2, 4 and 6 hours post-dose at Weeks 1 and 25 (adolescent) |
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| Secondary | DB Period: Change From Baseline in the Symptoms, Bother and Activity Domains of the Immune Thrombocytopenia-Patient Assessment Questionnaire in Adult Participants | ITP-PAQ:38 items and 44 items completed by male and female respondents respectively.It included 10 scales:symptoms(6 items:1-6),fatigue/sleep(4 items:7-10),bother-physical health(3 items:11-13),activity(2 items:14-15),psychological health(5 items:16-20),fear(5 items:21-25),overall QoL(5 items:26-30),social activity(4 items:31-34),women's reproductive health(6 items:35-40),work(4 items:41-44).Responses were recorded on 4-point(1:"strongly disagree" to 4:"strongly agree"),5-point(1:"never/not at all" to 5:"all the time/extremely") or 7-point(1:"not at all" to 7:"extremely") Likert scales.Each item score:100x([possible maximal item score - item score]/range).All item scores:transformed to 0 to 100 continuum and were weighted equally to derive scale scores.Total score:0(worst) to 100(best);higher scores:better QoL.Change of positive value:improvement.Change from baseline in symptoms,bother and activity domains is presented.Baseline:last available value before first dose of DB study drug. | Adult ITT population included all randomized participants. Only those participants with data collected at specified timepoints are reported. | Posted | Mean | Standard Deviation | score on a scale | Baseline (Day 1) and Week 25 |
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| Secondary | DB Period: Change From Baseline in Disease-Specific Quality Of Life as Measured by the Kids' Immune Thrombocytopenia Tools (ITP-KIT) Score in Adolescent Participants | ITP-KIT was a disease-specific instrument and child self-report form designed to be completed by children >=7 years. It comprised of total of 27 items among which 26 items were structured as Likert scales with 5 response options 1: "never", 2: "rarely", 3: "sometimes", 4: "often" and 5: "always". An additional "not applicable" option was available for items 18 to 26, which was scored as 1: the same as "never". Item 27 was a descriptive question answered "yes" or "no" which was not included in the calculation of the summary score. Instrument yielded a summary KIT score which was the summation of the items calculated as: 100 x (1- [{sum of all valid responses - number of valid responses}/{number of valid responses*4}]). Scores were converted to a 0 to 100 with higher scores indicating better disease-specific QoL. Change from baseline of positive value indicated improvement. Baseline was defined as the last available value before first dose of DB study drug. | Adolescent ITT population included all randomized participants. Only those participants with data collected at specified timepoints are reported. | Posted | Mean | Standard Deviation | score on a scale | Baseline (Day 1) and Week 25 |
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Adverse events and all-cause mortality (deaths) were collected: DB period: From first dose of study drug (Day 1) up to maximum DB exposure, up to 182 days for adults and up to 175 days for adolescent participants; Adult OL period: From first dose of study drug in OL period (Day 1) up to maximum OL exposure, up to 215 days.
Analysis was performed on the adult and adolescent safety population. Interim results have been reported up to the DBL date 15-Oct-24 (adult OL period) and 15-Jan-2025 (adolescent DB period). AEs data of adult LTE, and adolescent (OL and LTE) periods will be reported after the participants have completed the LTE period. Part A MedDRA 24.0, Part B 25.1
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | DB Period: Adults: Placebo | Adult participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. | 0 | 69 | 8 | 69 | 33 | 69 |
| EG001 | DB Period: Adults: Rilzabrutinib 400 mg BID | Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. | 1 | 133 | 12 | 133 | 90 | 133 |
| EG002 | DB Period: Adolescent: Placebo | Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. | 0 | 12 | 0 | 12 | 10 | 12 |
| EG003 | DB Period: Adolescent: Rilzabrutinib 400 mg BID | Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. | 0 | 18 | 2 | 18 | 15 | 18 |
| EG004 | OL Period: Adults: Rilzabrutinib 400 mg BID | Eligible adult participants (responders after 24 weeks of DB period and non-responders after 12 weeks of DB period) entered the OL period to receive rilzabrutinib 400 mg tablet BID orally for 28 weeks. | 0 | 180 | 17 | 180 | 94 | 180 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Bronchopulmonary Aspergillosis | Infections and infestations | MedDRA 24.0 and 25.1 | Systematic Assessment |
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| Covid-19 | Infections and infestations | MedDRA 24.0 and 25.1 | Systematic Assessment |
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| Cytomegalovirus Viraemia | Infections and infestations | MedDRA 24.0 and 25.1 | Systematic Assessment |
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| Pneumonia | Infections and infestations | MedDRA 24.0 and 25.1 | Systematic Assessment |
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| Renal Abscess | Infections and infestations | MedDRA 24.0 and 25.1 | Systematic Assessment |
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| Sepsis | Infections and infestations | MedDRA 24.0 and 25.1 | Systematic Assessment |
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| Urinary Tract Infection | Infections and infestations | MedDRA 24.0 and 25.1 | Systematic Assessment |
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| Wound Infection | Infections and infestations | MedDRA 24.0 and 25.1 | Systematic Assessment |
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| Immune Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
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| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
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| Paraesthesia | Nervous system disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
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| Conjunctival Haemorrhage | Eye disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
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| Deep Vein Thrombosis | Vascular disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
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| Haematoma | Vascular disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
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| Peripheral Embolism | Vascular disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Interstitial Lung Disease | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Pulmonary Embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Haematemesis | Gastrointestinal disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Mouth Haemorrhage | Gastrointestinal disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Oesophageal Varices Haemorrhage | Gastrointestinal disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Hepatic Cirrhosis | Hepatobiliary disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Purpura | Skin and subcutaneous tissue disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Haematuria | Renal and urinary disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Heavy Menstrual Bleeding | Reproductive system and breast disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Ovarian Cyst Ruptured | Reproductive system and breast disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Vaginal Haemorrhage | Reproductive system and breast disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Hepatic Enzyme Increased | Investigations | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Platelet Count Decreased | Investigations | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Periorbital Haematoma | Injury, poisoning and procedural complications | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Seroma | Injury, poisoning and procedural complications | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Subdural Haematoma | Injury, poisoning and procedural complications | MedDRA 24.0 and 25.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Covid-19 | Infections and infestations | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Hordeolum | Infections and infestations | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Lower Respiratory Tract Infection | Infections and infestations | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Pharyngitis Streptococcal | Infections and infestations | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Pneumonia Influenzal | Infections and infestations | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Anaemia | Blood and lymphatic system disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Blood Loss Anaemia | Blood and lymphatic system disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Coagulopathy | Blood and lymphatic system disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Iron Deficiency Anaemia | Blood and lymphatic system disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Leukopenia | Blood and lymphatic system disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Presyncope | Nervous system disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Ear Haemorrhage | Ear and labyrinth disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Orthostatic Hypotension | Vascular disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Oropharyngeal Pain | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Rhinitis Allergic | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Abdominal Pain | Gastrointestinal disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Abdominal Pain Upper | Gastrointestinal disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Stomatitis | Gastrointestinal disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Rash Erythematous | Skin and subcutaneous tissue disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Musculoskeletal Chest Pain | Musculoskeletal and connective tissue disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Proteinuria | Renal and urinary disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Dysmenorrhoea | Reproductive system and breast disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Oedema Peripheral | General disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Activated Partial Thromboplastin Time Prolonged | Investigations | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Neutrophil Count Decreased | Investigations | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Urobilinogen Urine Increased | Investigations | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Weight Increased | Investigations | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Animal Bite | Injury, poisoning and procedural complications | MedDRA 24.0 and 25.1 | Systematic Assessment |
| |
| Arthropod Bite | Injury, poisoning and procedural complications | MedDRA 24.0 and 25.1 | Systematic Assessment |
|
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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Trial Transparency Team | Sanofi aventis recherche & développement | 800-633-1610 ext 6# | Contact-US@sanofi.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 18, 2024 | Dec 24, 2025 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D057049 | Thrombotic Microangiopathies |
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |
Not provided
Not provided
| Adverse Event |
|
| Lost to Follow-up |
|
| Lack of response |
|
| Other |
|
| Ongoing |
|
| Pregnancy |
|
| Lack of response |
|
| Other |
|
| Sponsor discretion |
|
| Adverse Event |
|
| Ongoing |
|
| Male |
|
| Asian |
|
| Black or African American |
|
| White |
|
| Unknown or Not Reported |
|
| Other |
|
| OG002 | DB Period: Adolescent: Placebo | Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
| OG003 | DB Period: Adolescent: Rilzabrutinib 400 mg BID | Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
|
|
|
Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment.
| OG002 | DB Period: Adolescent: Placebo | Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
| OG003 | DB Period: Adolescent: Rilzabrutinib 400 mg BID | Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
|
|
|
| OG002 | DB Period: Adolescent: Placebo | Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
| OG003 | DB Period: Adolescent: Rilzabrutinib 400 mg BID | Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
|
|
|
| OG002 | DB Period: Adolescent: Placebo | Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
| OG003 | DB Period: Adolescent: Rilzabrutinib 400 mg BID | Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
|
|
|
| OG002 | DB Period: Adolescent: Placebo | Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
| OG003 | DB Period: Adolescent: Rilzabrutinib 400 mg BID | Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
|
|
|
| OG001 | DB Period: Adults: Rilzabrutinib 400 mg BID | Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
|
|
|
| OG001 | DB Period: Adults: Rilzabrutinib 400 mg BID | Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
|
|
|
| OG001 | DB Period: Adolescent: Rilzabrutinib 400 mg BID | Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
|
|
| OG001 | DB-OL Period: Adults: Rilzabrutinib 400 mg BID in DB | Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period and received rilzabrutinib 400 mg tablet BID orally in OL period (if entered OL period). At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
| OG002 | DB-OL Period: Adults: Rilzabrutinib 400 mg BID | Eligible adult participants (responders after 24 weeks of DB period and non-responders after 12 weeks of DB period) entered the OL period to receive rilzabrutinib 400mg tablet BID orally for 28 weeks. Or adult participants received rilzabrutinib 400 mg tablet BID orally in DB period and did not enter OL period. |
|
|
| OG001 | DB Period: Adults: Rilzabrutinib 400 mg BID | Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
| OG002 | DB Period: Adolescent: Placebo | Adolescent participants received placebo matched to rilzabrutinib tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
| OG003 | DB Period: Adolescent: Rilzabrutinib 400 mg BID | Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
|
|
Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
|
|
| OG001 | DB Period: Adults: Rilzabrutinib 400 mg BID | Adult participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
|
|
| OG001 | DB Period: Adolescent: Rilzabrutinib 400 mg BID | Adolescent participants received rilzabrutinib 400 mg tablet BID orally in DB period. At the end of 12 weeks of treatment (at the Week 13 visit), participants were assessed for a platelet response defined as: platelet count of >=50,000/mcL or a platelet count of between >=30,000/mcL and <50,000/mcL and at least doubled from baseline at any time during the first 12 weeks and absence of rescue therapy in the 4 weeks prior to the elevated platelet count that met platelet response criteria. Participants who met the criteria for response, continued in the blinded treatment period for a total of 24 weeks before entering the OL period; the ones who did not meet the criteria discontinued from the study or entered the 28-week OL period at the end of 12 weeks of blinded treatment. |
|
|