Trial · NCT04562766
Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)
NCT04562766, Phase 3, Completed, Principia Biopharma, a Sanofi Company; results first posted 2026-02-23
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP). Trials, record NCT04562766. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04562766
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Evaluate Rilzabrutinib in Adults and Adolescents With Persistent or Chronic Immune Thrombocytopenia (ITP)
- NCT number
- NCT04562766
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 232 participants, actual
- Lead sponsor
- Principia Biopharma, a Sanofi Company
- Conditions
- Immune Thrombocytopenia
- Interventions
- Rilzabrutinib, Placebo
- First posted
- 2020-09-24
- Last updated
- 2026-10-07
- Results first posted
- 2026-02-23
Source ClinicalTrials.gov
Registered outcomes15
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in Regions Except European Union and United Kingdom | Up to 24 weeks | 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. |
| Primary | DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in European Union and United Kingdom | Up to 24 weeks | 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. |
| 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 | Up to 24 weeks | 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. |
| Secondary | DB Period: Number of Weeks With Platelet Counts >=30,000/mcL and at Least Doubled From Baseline in the Absence of Rescue Therapy | Up to 24 weeks | 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. |
| 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 | Up to 24 weeks | 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. |
| Secondary | DB Period: Percentage of Participants Who Required Rescue Therapy | Up to 24 weeks | 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. |
| 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 | Baseline (Day 1) and 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. |
| 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 | Baseline (Day 1) and Week 25 | 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. |
| 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 | Baseline (Day 1) and Week 25 | 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. |
| Secondary | DB-OL Period: Percentage of Participants With Stability of Response in Adult Participants | Up to 52 weeks | 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. |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 registered outcomesOutcomes15
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in Regions Except European Union and United Kingdom | Up to 24 weeks | Adult and adolescent intent-to-treat (ITT) population included all randomized participants. | percentage of participants | NUMBER |
| PRIMARY | DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in European Union and United Kingdom | Up to 24 weeks | Adult and adolescent ITT population included all randomized participants. | percentage of participants | NUMBER |
| 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 | Up to 24 weeks | Adult and adolescent ITT population included all randomized participants. | weeks | MEAN |
| SECONDARY | DB Period: Number of Weeks With Platelet Counts >=30,000/mcL and at Least Doubled From Baseline in the Absence of Rescue Therapy | Up to 24 weeks | Adult and adolescent ITT population included all randomized participants. | weeks | MEAN |
| 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 | Up to 24 weeks | Adult and adolescent ITT population included all randomized participants. Only those participants who achieved platelet response are reported. | days | MEDIAN |
| SECONDARY | DB Period: Percentage of Participants Who Required Rescue Therapy | Up to 24 weeks | Adult and adolescent ITT population included all randomized participants. | percentage of participants | NUMBER |
| 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 | Baseline (Day 1) and Week 13 | Adult ITT population included all randomized participants. | score on a scale | LEAST_SQUARES_MEAN |
| 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 | Baseline (Day 1) and Week 25 | Adult ITT population included all randomized participants. | score on a scale | LEAST_SQUARES_MEAN |
| 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 | Baseline (Day 1) and Week 25 | Adolescent ITT population included all randomized participants. Only those participants with data collected at specified timepoints are reported. | score on a scale | MEAN |
| SECONDARY | DB-OL Period: Percentage of Participants With Stability of Response in Adult Participants | Up to 52 weeks | 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. | percentage of participants | NUMBER |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 outcomesResults by group69
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in Regions Except European Union and United Kingdom | DB Period: Adults: Placebo | 0.0 | 95% Confidence Interval 0.00 to 0.00 | percentage of participants | |
| DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in Regions Except European Union and United Kingdom | DB Period: Adults: Rilzabrutinib 400 mg BID | 23.3 | 95% Confidence Interval 16.12 to 30.49 | percentage of participants | |
| DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in Regions Except European Union and United Kingdom | DB Period: Adolescent: Placebo | 0.0 | 95% Confidence Interval 0.00 to 26.46 | percentage of participants | |
| DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in Regions Except European Union and United Kingdom | DB Period: Adolescent: Rilzabrutinib 400 mg BID | 11.1 | 95% Confidence Interval 1.38 to 34.71 | percentage of participants | |
| DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in European Union and United Kingdom | DB Period: Adults: Placebo | 0.0 | 95% Confidence Interval 0.00 to 0.00 | percentage of participants | |
| DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in European Union and United Kingdom | DB Period: Adults: Rilzabrutinib 400 mg BID | 23.3 | 95% Confidence Interval 16.12 to 30.49 | percentage of participants | |
| DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in European Union and United Kingdom | DB Period: Adolescent: Placebo | 0.0 | 95% Confidence Interval 0.00 to 26.46 | percentage of participants | |
| DB Period: Percentage of Participants With Durable Platelet Response Per Guidance in European Union and United Kingdom | DB Period: Adolescent: Rilzabrutinib 400 mg BID | 11.1 | 95% Confidence Interval 1.38 to 34.71 | percentage of participants | |
| 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 | DB Period: Adults: Placebo | 1.0 | Standard Deviation 2.14 | weeks | |
| 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 | DB Period: Adults: Rilzabrutinib 400 mg BID | 7.5 | Standard Deviation 8.74 | weeks |
1 to 10 of 69Source ClinicalTrials.gov
See all 69 results by groupParticipant flow80
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| DB Period (Weeks 1 to 24) | STARTED | DB Period: Adults: Placebo | 69 | |
| DB Period (Weeks 1 to 24) | STARTED | DB Period: Adults: Rilzabrutinib 400 mg BID | 133 | |
| DB Period (Weeks 1 to 24) | STARTED | DB Period: Adolescent: Placebo | 12 | |
| DB Period (Weeks 1 to 24) | STARTED | DB Period: Adolescent: Rilzabrutinib 400 mg BID | 18 | |
| DB Period (Weeks 1 to 24) | STARTED | OL Period: Adults: Rilzabrutinib 400 mg BID | 0 | |
| DB Period (Weeks 1 to 24) | STARTED | OL Period: Adolescent: Rilzabrutinib 400 mg BID | 0 | |
| DB Period (Weeks 1 to 24) | STARTED | LTE Period: Adults: Rilzabrutinib 400 mg BID | 0 | |
| DB Period (Weeks 1 to 24) | STARTED | LTE Period: Adolescent: Rilzabrutinib 400 mg BID | 0 | |
| DB Period (Weeks 1 to 24) | Completed 12-week DB Period | DB Period: Adults: Placebo | 66 | |
| DB Period (Weeks 1 to 24) | Completed 12-week DB Period | DB Period: Adults: Rilzabrutinib 400 mg BID | 121 |
1 to 10 of 80Source ClinicalTrials.gov
See all 80 participant flowAdverse events400
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Covid-19 | Infections and infestations | DB Period: Adults: Placebo | 3 | 69 | 3 |
| other | Covid-19 | Infections and infestations | DB Period: Adults: Rilzabrutinib 400 mg BID | 17 | 133 | 17 |
| other | Covid-19 | Infections and infestations | DB Period: Adolescent: Placebo | 0 | 12 | 0 |
| other | Covid-19 | Infections and infestations | DB Period: Adolescent: Rilzabrutinib 400 mg BID | 1 | 18 | 1 |
| other | Covid-19 | Infections and infestations | OL Period: Adults: Rilzabrutinib 400 mg BID | 12 | 180 | 13 |
| other | Hordeolum | Infections and infestations | DB Period: Adults: Placebo | 0 | 69 | 0 |
| other | Hordeolum | Infections and infestations | DB Period: Adults: Rilzabrutinib 400 mg BID | 0 | 133 | 0 |
| other | Hordeolum | Infections and infestations | DB Period: Adolescent: Placebo | 0 | 12 | 0 |
| other | Hordeolum | Infections and infestations | DB Period: Adolescent: Rilzabrutinib 400 mg BID | 1 | 18 | 1 |
| other | Hordeolum | Infections and infestations | OL Period: Adults: Rilzabrutinib 400 mg BID | 0 | 180 | 0 |
1 to 10 of 400Source ClinicalTrials.gov
See all 400 adverse eventsAdverse event totals15
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | DB Period: Adults: Placebo | 0 | 69 |
| Total, all-cause mortality | DB Period: Adults: Rilzabrutinib 400 mg BID | 1 | 133 |
| Total, all-cause mortality | DB Period: Adolescent: Placebo | 0 | 12 |
| Total, all-cause mortality | DB Period: Adolescent: Rilzabrutinib 400 mg BID | 0 | 18 |
| Total, all-cause mortality | OL Period: Adults: Rilzabrutinib 400 mg BID | 0 | 180 |
| Total, other adverse events | DB Period: Adults: Placebo | 33 | 69 |
| Total, other adverse events | DB Period: Adults: Rilzabrutinib 400 mg BID | 90 | 133 |
| Total, other adverse events | DB Period: Adolescent: Placebo | 10 | 12 |
| Total, other adverse events | DB Period: Adolescent: Rilzabrutinib 400 mg BID | 15 | 18 |
| Total, other adverse events | OL Period: Adults: Rilzabrutinib 400 mg BID | 94 | 180 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 adverse event totalsPapers4
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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