A Phase 2 interventional study of Rilzabrutinib in Immune Thrombocytopenia, sponsored by Principia Biopharma, a Sanofi Company. Completed at 31 sites in 8 countries. Open to participants aged 18 Years to 80 Years. Per ClinicalTrials.gov, last updated 2026-02-23.
Sponsored by Principia Biopharma, a Sanofi Company (part of Sanofi) · Phase 2, Interventional, and Treatment
This was a 2 part (Part A and B) adaptive, open-label, dose-finding study of PRN1008 in patients with ITP who are refractory or relapsed with no available and approved therapeutic options, with a platelet count \<30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. The dose-finding portion of the study was completed. Part B treatment dose was 400 mg twice daily.
This was a 2 part (Part A and B) adaptive, open-label, dose-finding study of PRN1008 in approximately 60 patients in Part A and approximately 25 patients in Part B.
Part A enrolled patients with ITP who were refractory or relapsed with no available and approved therapeutic options. Eligible patients had a platelet count \<30,000/μL on two counts no sooner than 7 days apart in the 15 days before treatment begins. The active treatment period was 24 weeks and the post-treatment follow-up period is 4 weeks. In the dose-finding part of the study, each patient enrolled in the study was allowed to up-titrate their dose after 28 days of PRN1008 therapy, if they did not experience a platelet response or a dose-limiting toxicity (DLT) at the last dose level. Patients who responded to PRN1008 per protocol may enter a long term-extension.
Part B of the study included approximately 25 patients with ITP who had relapsed or had an insufficient response to prior therapies. Eligible patients had a platelet count \<30,000/µL on two occasions no less than 7 days apart, within 15 days before treatment began and a platelet count of ≤35,000/µL on Study Day 1 (SD1). The study consisted of a 28-day screening period, 24-week active treatment period, and a long-term extension. After the last dose of PRN1008 there was a 4-week safety follow-up period.
503 studies on the registry are indexed under Purpura, Thrombocytopenic, Idiopathic; 158 are open to participants now.
This study's enrollment of 86 is above the median of 58 across 376 interventional studies indexed under Purpura, Thrombocytopenic, Idiopathic.
Browse Purpura, Thrombocytopenic, Idiopathic studies →Principia Biopharma, a Sanofi Company is the lead sponsor of 10 studies on the registry; none are open to participants now.
Of its 8 completed or terminated interventional studies of FDA-regulated products, 5 (63%) have results posted.
First submitted before Principia Biopharma became part of Sanofi.
Counted across the registry records on this site, refreshed daily.
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Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Part A approximately 60 patients: Up to 24 weeks open-label treatment with PRN1008 400mg BID; safety and dose evaluation. Patients who respond to PRN1008 per protocol may enter a long-term extension. Part B approximately 25 patients: Up to 24 weeks open-label treatment with PRN1008 400mg BID; safety and dose evaluation. Patients who respond to PRN1008 per protocol may enter a long-term extension
Drug: Rilzabrutinib
BTK inhibitor
Also known as: PRN1008
Part A: Percentage of Participants Who Achieved 2 or More Consecutive Platelet Counts by Starting Dose Level and Overall
The percentage of participants who achieved 2 or more consecutive platelet counts, separated by at least 5 days, of \>=50,000/ microliter (μL) and an increase of platelet count of \>=20,000/μL from baseline, by starting dose level and overall, without use of rescue medication in the 4 weeks prior to the latest elevated platelet count. 95% confidence interval (CI) was based on the Clopper-Pearson method. The average of the 2 screening results and the Cycle 1 Day 1 result were used as the baseline value.
Time frame: Up to 24 Weeks
Part B: Percentage of Participants Who Achieved Platelet Counts >=50,000/μL
The percentage of participants who achieved platelet counts \>=50,000/μL on at least 8 out of the last 12 weeks of the 24-week treatment period without the use of rescue medication after 10 weeks of active treatment. 95% CI was based on the Clopper-Pearson exact method.
Time frame: Up to 24 Weeks
Part A: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment Related Treatment-Emergent Adverse Events
Adverse event (AE): any untoward medical occurrence in participant or clinical study participant, temporally associated with the use of rilzabrutinib, whether or not considered related to rilzabrutinib. TEAEs: AEs that developed or worsened or became serious on or after the first dose administration of rilzabrutinib (Day 1). Any TEAEs are considered treatment-related TEAEs as per Investigator's evaluation of participant's circumstances surrounding the event, and an evaluation of any potential alternative causes to determine whether an TEAE can be considered as related to the rilzabrutinib.
Time frame: From first dose of rilzabrutinib (Day 1) up to last dose + 1 (up to 294 days)
Part B: Number of Participants With Treatment-Emergent Adverse Events and Treatment Related Treatment-Emergent Adverse Events
AE any untoward medical occurrence in participant or clinical study participant, temporally associated with the use of rilzabrutinib, whether or not considered related to rilzabrutinib. TEAEs: AEs that developed or worsened or became serious during the treatment-emergent period, defined as any time after the first dose administration of rilzabrutinib (Day 1). Any TEAEs are considered treatment-related TEAEs as per Investigator's evaluation of participant's circumstances surrounding the event, and an evaluation of any potential alternative causes to determine whether an TEAE can be considered as related to rilzabrutinib.
Time frame: From first dose of rilzabrutinib (Day 1) up to last dose + 1 (approximately 170 days)
Part A: Percentage of Weeks With Platelet Counts >=50,000/μL by Starting Dose Level and Overall
The percentage of weeks in which participants achieved platelet counts \>=50,000/μL in the treatment period are summarized here.
Time frame: Up to 24 Weeks
Part A: Percentage of Participants With 4 Out of the Final 8 Platelet Counts >=50,000/μL by Starting Dose Level and Overall
The percentage of participants who had at least 4 out of the final 8 platelet counts \>=50,000/μL are summarized here. The final 8 scheduled platelet counts are the measurements performed in the last 8 weeks of rilzabrutinib (depending on treatment duration, not necessarily from Week 19 to Week 24) in the treatment period. 95% CI was based on the Clopper-Pearson method.
Time frame: Up to 24 Weeks
Part A: Change From Baseline to the Average of Post Day 1 Platelet Counts by Dose Level and Overall
Average of post Day 1 platelet count is equivalent to average of (average of each participant's post-Day 1 platelet counts), included platelet counts up to 1 day after the date of last dose of rilzabrutinib and excluded platelet counts on or after date of rescue, if applicable. The average of the 2 screening results and the Cycle 1 Day 1 result measured on different date were used as the baseline value.
Time frame: Baseline and up to 24 Weeks
Part A: Number of Weeks With Platelet Counts >=50,000/μL by Starting Dose Level and Overall
The number of weeks in which participant achieved platelet counts \>=50,000/μL in the treatment period are summarized here. The number of weeks is based on the number of scheduled weekly assessments, by study day.
Time frame: Up to 24 Weeks
Part A: Number of Weeks With Platelet Counts >=30,000/μL by Starting Dose Level and Overall
The number of weeks in which participant achieved platelet counts \>=30,000/μL in the treatment period are summarized here. The number of weeks is based on the number of scheduled weekly assessments, by study day.
Time frame: Up to 24 Weeks
Part A: Time to First Platelet Count >=50,000/μL Across All Dose Levels
Time to first platelet count \>=50,000/μL during the treatment period in days was calculated as: (date of first occurrence of platelet count \>=50,000/μL - date of first rilzabrutinib dosing) +1.
Time frame: Up to 24 Weeks
Part B: Number of Weeks With Platelet Counts >= 50,000/μL or >= 30,000/μL and Doubling the Baseline
The number of weeks in which participant achieved platelet counts with thresholds as: \>=50,000/μL or \>=30,000/μL and doubling the baseline in the absence of rescue therapy (platelet counts will be censored for 4 weeks after the use of rescue medication, if given) are summarized here. Baseline is defined as the average of 3 platelet counts: 2 qualified screening platelet counts collected in electronic case report form (eCRF) and Week 1 (study day 1) platelet count.
Time frame: Up to 24 Weeks
Part B: Percentage of Participants Who Achieved 2 or More Consecutive Platelet Counts
Percentage of participants who achieved 2 or more consecutive platelet counts, separated by at least 5 days, of \>=50,000/μL and an increase of platelet count of \>=20,000/μL from baseline without use of rescue medication in the 4 weeks prior to the latest elevated platelet count are summarized here. Baseline is defined as the average of 3 platelet counts: 2 qualified screening platelet counts collected in eCRF and Week 1 (study day 1) platelet count. 95% CI was based on the Clopper-Pearson exact method.
Time frame: Up to 24 Weeks
Part B: Number of Weeks With Platelet Counts >=30,000/μL and Doubling the Baseline
The number of weeks in which participant achieved platelet counts \>=30,000/μL and doubling the baseline in the absence of rescue therapy (platelet counts will be censored for 4 weeks after the use of rescue medication, if given) are summarized here. Baseline is defined as the average of 3 platelet counts: 2 qualified screening platelet counts collected in eCRF and Week 1 (study day 1) platelet count.
Time frame: Up to 24 Weeks
Part B: Percentage of Participants Who Received Rescue Medication
Rescue medication is defined as any therapy used to rescue a participant (1 of intravenous immunoglobulin \[IVIG\], high-dose steroids, platelet infusion or anti-D immunoglobulin infusion). Percentage of participants who received rescue medication are summarized here. 95% CI was based on the Clopper-Pearson exact method.
Time frame: Up to 24 Weeks
Part B: Change From Baseline in Idiopathic Thrombocytopenic Purpura Bleeding Scale (IBLS)
The IBLS is a bleeding assessment score. IBLS comprises of 11 sites for female and 10 sites for male, and each site is scored from 0 (none) to 2 (marked bleeding). The total overall score ranges from 0-22, with higher scores indicating higher presence of marked bleeding. For each participant, an IBLS score at each visit was calculated by taking the average across 11 items (10 for male and postmenopausal women) at 9 anatomical sites (8 for male and postmenopausal women). For each participant, a mean IBLS score was also calculated by taking the average across all post-baseline visits during the 24-week treatment period. IBLS average value ranges from 0 to 2. The smaller the IBLS average value is, the healthier the participants are. For change from baseline, negative value indicates an improvement. The baseline value is defined as the last available value before the first dose rilzabrutinib.
Time frame: Baseline and up to 24 weeks
Part A: Percentage of Participants Who Received Rescue Medication by Dose Levels and Overall
Rescue medication is defined as any therapy used to rescue a participant (1 of IVIG, high-dose steroids, platelet infusion or anti-D immunoglobulin infusion). The percentage of participants who received rescue medication for each dose level and overall are summarized here. 95% CI was based on the Clopper-Pearson method.
Time frame: Up to 24 Weeks
Part A: Percentage of Participants With Grade 2 or Higher Bleeding Event by Dose Level and Overall
The percentage of participants with intensity grade 2 or higher bleeding event are summarized for each dose level and overall. The TEAEs with standardized medical dictionary for regulatory activities (MedDRA) query (SMQ) hemorrhages were medically determined for analysis of bleeding events. 95% CI was based on the Clopper-Pearson method.
Time frame: Up to 24 Weeks
Part A: Number of Participants With Idiopathic Thrombocytopenic Purpura/Immune Thrombocytopenia (ITP) Bleeding Assessment Tool (ITP-BAT) Scale by Dose Level
The ITP-BAT scale comprises of 11 grades from 0 (none) to 2 (marked bleeding), with higher scores indicating higher presence of marked bleeding, assessed at 9 anatomical sites (skin, oral, epistaxis, gastrointestinal \[GI\], urinary, gynecological \[GYN\], pulmonary, intracranial hemorrhage \[HEM\], subconjunctival HEM) by history over the previous week (Hx). In addition, 2 sites (skin and oral), were also assessed by physical examination (PE). The 'worst ever' bleeding experienced at each site was graded using the same system. Here, 0 indicates none; 1 indicates 1-5 bruises and/or scattered petechiae and 2 indicates \>5 bruises with size \>2 centimeter (cm) and/or diffuse petechiae. Each participant summed up the transformed scores across all 11 sites per visit assessment. The total overall score ranges from 0-22 with the higher score indicating worst outcome.
Time frame: Up to 24 Weeks
Part A: Maximum Observed Plasma Concentration (Cmax) of Rilzabrutinib
Plasma samples were collected at specified timepoints to determine Cmax of rilzabrutinib.
Time frame: Day 1 of Cycles 1, 2, 3, and 5 (each cycle 28 days)
Part A: Time of Observed Maximum Plasma Concentration (Tmax) of Rilzabrutinib
Plasma samples were collected at specified timepoints to determine tmax of rilzabrutinib.
Time frame: Day 1 of Cycles 1, 2, 3, and 5 (each cycle 28 days)
Part A: Area Under the Plasma Concentration-Time Curve From Time Zero to Last Quantifiable Concentration (AUClast) of Rilzabrutinib
Plasma samples were collected at specified timepoints to determine AUClast of rilzabrutinib.
Time frame: Day 1 of Cycles 1, 2, 3, and 5 (each cycle 28 days)
Part A: Area Under the Plasma Concentration-Time Curve From Time Zero to Infinity (AUCinf) of Rilzabrutinib
Plasma samples were collected at specified timepoints to determine AUCinf of rilzabrutinib.
Time frame: Day 1 of Cycles 1, 2, and 3 (each cycle 28 days)
Part A: Elimination Half-Life (t1/2) of Rilzabrutinib
Plasma samples were collected at specified timepoints to determine t1/2 of rilzabrutinib.
Time frame: Day 1 of Cycles 1, 2, and 3 (each cycle 28 days)
Part A: Apparent Volume of Distribution of the Drug After Oral Administration (Vz/F) of Rilzabrutinib
Plasma samples were collected at specified timepoints to determine Vz/F of rilzabrutinib.
Time frame: Day 1 of Cycles 1, 2, and 3 (each cycle 28 days)
Part A: Apparent Total Clearance of the Drug From Plasma After Oral Administration (CL/F) of Rilzabrutinib
Plasma samples were collected at specified timepoints to determine CL/F of rilzabrutinib.
Time frame: Day 1 of Cycles 1, 2, and 3 (each cycle 28 days)
Part B: Plasma Concentration of Rilzabrutinib
Plasma samples were collected at specified timepoints for evaluation of rilzabrutinib pharmacokinetic (PK) concentrations.
Time frame: Pre-dose and 2 hours post-dose on Days 1, 29, and 57
The study was conducted at 31 active centers in 8 countries. As prespecified in statistical analysis plan (SAP), Part A results were presented by either of the of the treatment group labels: Starting dose level (each participant was assigned to only one of the groups), dose level (dose received during the defined period. As most patients received more than one dose, a given patient could be assigned to multiple dose levels) and overall.
| Milestone | Part A: Starting Dose 200 mg QD | Part A: Starting Dose 400 mg QD | Part A: Starting Dose 300 mg BID | Part A: Starting Dose 400 mg BID | Part B: Rilzabrutinib 400 mg BID |
|---|---|---|---|---|---|
| Started | 9 | 1 | 5 | 45 | 26 |
| Completed | 4 | 0 | 0 | 15 | 4 |
| Not completed | 5 | 1 | 5 | 30 | 22 |
| Withdrew: Other | 0 | 0 | 0 | 0 | 1 |
| Withdrew: Participant uncooperative or noncompliant | 0 | 0 | 0 | 0 | 1 |
| Withdrew: Adverse event | 1 | 0 | 2 | 4 | 3 |
| Withdrew: Need of rescue medication | 1 | 0 | 1 | 4 | 0 |
| Withdrew: Lack of response | 0 | 0 | 0 | 5 | 5 |
| Withdrew: Participant was erroneously enrolled in the study | 1 | 0 | 0 | 0 | 1 |
| Withdrew: Participant decision | 1 | 1 | 0 | 4 | 0 |
| Withdrew: Ongoing at the time of primary completion date | 1 | 0 | 2 | 13 | 11 |
The percentage of participants who achieved 2 or more consecutive platelet counts, separated by at least 5 days, of \>=50,000/ microliter (μL) and an increase of platelet count of \>=20,000/μL from baseline, by starting dose level and overall, without use of rescue medication in the 4 weeks prior to the latest elevated platelet count. 95% confidence interval (CI) was based on the Clopper-Pearson method. The average of the 2 screening results and the Cycle 1 Day 1 result were used as the baseline value.
| percentage of participants | Part A: Starting Dose 200 mg QD | Part A: Starting Dose 400 mg QD | Part A: Starting Dose 300 mg BID | Part A: Starting Dose 400 mg BID | Part A: Overall |
|---|---|---|---|---|---|
| Part A: Percentage of Participants Who Achieved 2 or More Consecutive Platelet Counts by Starting Dose Level and Overall | 44.4 (13.70 to 78.80) | 0.0 (0.00 to 97.50) | 40.0 (5.27 to 85.34) | 40.0 (25.70 to 55.67) | 40.0 (27.56 to 53.46) |
The percentage of participants who achieved platelet counts \>=50,000/μL on at least 8 out of the last 12 weeks of the 24-week treatment period without the use of rescue medication after 10 weeks of active treatment. 95% CI was based on the Clopper-Pearson exact method.
| percentage of participants | Part B: Rilzabrutinib 400 mg BID |
|---|---|
| Part B: Percentage of Participants Who Achieved Platelet Counts >=50,000/μL | 34.6 (17.21 to 55.67) |
Adverse event (AE): any untoward medical occurrence in participant or clinical study participant, temporally associated with the use of rilzabrutinib, whether or not considered related to rilzabrutinib. TEAEs: AEs that developed or worsened or became serious on or after the first dose administration of rilzabrutinib (Day 1). Any TEAEs are considered treatment-related TEAEs as per Investigator's evaluation of participant's circumstances surrounding the event, and an evaluation of any potential alternative causes to determine whether an TEAE can be considered as related to the rilzabrutinib.
| Participants | Part A: Dose Level 200 mg QD | Part A: Dose Level 400 mg QD | Part A: Dose Level 300 mg BID | Part A: Dose Level 400 mg BID | Part A: Overall |
|---|---|---|---|---|---|
| TEAEs | 3 | 3 | 5 | 44 | 48 |
| Treatment related TEAEs | 2 | 2 | 2 | 29 | 31 |
AE any untoward medical occurrence in participant or clinical study participant, temporally associated with the use of rilzabrutinib, whether or not considered related to rilzabrutinib. TEAEs: AEs that developed or worsened or became serious during the treatment-emergent period, defined as any time after the first dose administration of rilzabrutinib (Day 1). Any TEAEs are considered treatment-related TEAEs as per Investigator's evaluation of participant's circumstances surrounding the event, and an evaluation of any potential alternative causes to determine whether an TEAE can be considered as related to rilzabrutinib.
| Participants | Part B: Rilzabrutinib 400 mg BID |
|---|---|
| TEAEs | 22 |
| Treatment related TEAEs | 16 |
The percentage of weeks in which participants achieved platelet counts \>=50,000/μL in the treatment period are summarized here.
| percentage of weeks | Part A: Starting Dose 200 mg QD | Part A: Starting Dose 400 mg QD | Part A: Starting Dose 300 mg BID | Part A: Starting Dose 400 mg BID | Part A: Overall |
|---|---|---|---|---|---|
| Part A: Percentage of Weeks With Platelet Counts >=50,000/μL by Starting Dose Level and Overall | 27.92 ± 29.42 | 0.00 ± NA | 39.58 ± 41.30 | 28.14 ± 36.41 | 28.60 ± 35.39 |
The percentage of participants who had at least 4 out of the final 8 platelet counts \>=50,000/μL are summarized here. The final 8 scheduled platelet counts are the measurements performed in the last 8 weeks of rilzabrutinib (depending on treatment duration, not necessarily from Week 19 to Week 24) in the treatment period. 95% CI was based on the Clopper-Pearson method.
| percentage of participants | Part A: Starting Dose 200 mg QD | Part A: Starting Dose 400 mg QD | Part A: Starting Dose 300 mg BID | Part A: Starting Dose 400 mg BID | Part A: Overall |
|---|---|---|---|---|---|
| Part A: Percentage of Participants With 4 Out of the Final 8 Platelet Counts >=50,000/μL by Starting Dose Level and Overall | 11.1 (0.28 to 48.25) | 0.0 (0.00 to 97.50) | 40.0 (5.27 to 85.34) | 31.1 (18.17 to 46.65) | 28.3 (17.45 to 41.44) |
Average of post Day 1 platelet count is equivalent to average of (average of each participant's post-Day 1 platelet counts), included platelet counts up to 1 day after the date of last dose of rilzabrutinib and excluded platelet counts on or after date of rescue, if applicable. The average of the 2 screening results and the Cycle 1 Day 1 result measured on different date were used as the baseline value.
| platelets x10^9/Liter (L) | Part A: Dose Level 200 mg QD | Part A: Dose Level 400 mg QD | Part A: Dose Level 300 mg BID | Part A: Dose Level 400 mg BID | Part A: Overall |
|---|---|---|---|---|---|
| Part A: Change From Baseline to the Average of Post Day 1 Platelet Counts by Dose Level and Overall | 6.92 ± 22.52 | 68.10 ± NA | 37.05 ± 29.10 | 29.92 ± 41.70 | 28.61 ± 39.57 |
The number of weeks in which participant achieved platelet counts \>=50,000/μL in the treatment period are summarized here. The number of weeks is based on the number of scheduled weekly assessments, by study day.
| weeks | Part A: Starting Dose 200 mg QD | Part A: Starting Dose 400 mg QD | Part A: Starting Dose 300 mg BID | Part A: Starting Dose 400 mg BID | Part A: Overall |
|---|---|---|---|---|---|
| Part A: Number of Weeks With Platelet Counts >=50,000/μL by Starting Dose Level and Overall | 5.9 ± 7.8 | 0.0 ± NA | 10.2 ± 12.0 | 5.7 ± 8.1 | 6.0 ± 8.3 |
The number of weeks in which participant achieved platelet counts \>=30,000/μL in the treatment period are summarized here. The number of weeks is based on the number of scheduled weekly assessments, by study day.
| weeks | Part A: Starting Dose 200 mg QD | Part A: Starting Dose 400 mg QD | Part A: Starting Dose 300 mg BID | Part A: Starting Dose 400 mg BID | Part A: Overall |
|---|---|---|---|---|---|
| Part A: Number of Weeks With Platelet Counts >=30,000/μL by Starting Dose Level and Overall | 8.4 ± 11.2 | 0.0 ± NA | 14.0 ± 14.1 | 8.8 ± 9.2 | 9.0 ± 9.8 |
Time to first platelet count \>=50,000/μL during the treatment period in days was calculated as: (date of first occurrence of platelet count \>=50,000/μL - date of first rilzabrutinib dosing) +1.
| days | Part A: Overall Participants |
|---|---|
| Part A: Time to First Platelet Count >=50,000/μL Across All Dose Levels | 27.4 ± 31.86 |
The number of weeks in which participant achieved platelet counts with thresholds as: \>=50,000/μL or \>=30,000/μL and doubling the baseline in the absence of rescue therapy (platelet counts will be censored for 4 weeks after the use of rescue medication, if given) are summarized here. Baseline is defined as the average of 3 platelet counts: 2 qualified screening platelet counts collected in electronic case report form (eCRF) and Week 1 (study day 1) platelet count.
| weeks | Part B: Rilzabrutinib 400 mg BID |
|---|---|
| Part B: Number of Weeks With Platelet Counts >= 50,000/μL or >= 30,000/μL and Doubling the Baseline | 9.3 ± 10.1 |
Percentage of participants who achieved 2 or more consecutive platelet counts, separated by at least 5 days, of \>=50,000/μL and an increase of platelet count of \>=20,000/μL from baseline without use of rescue medication in the 4 weeks prior to the latest elevated platelet count are summarized here. Baseline is defined as the average of 3 platelet counts: 2 qualified screening platelet counts collected in eCRF and Week 1 (study day 1) platelet count. 95% CI was based on the Clopper-Pearson exact method.
| percentage of participants | Part B: Rilzabrutinib 400 mg BID |
|---|---|
| Part B: Percentage of Participants Who Achieved 2 or More Consecutive Platelet Counts | 42.3 (23.35 to 63.08) |
The number of weeks in which participant achieved platelet counts \>=30,000/μL and doubling the baseline in the absence of rescue therapy (platelet counts will be censored for 4 weeks after the use of rescue medication, if given) are summarized here. Baseline is defined as the average of 3 platelet counts: 2 qualified screening platelet counts collected in eCRF and Week 1 (study day 1) platelet count.
| weeks | Part B: Rilzabrutinib 400 mg BID |
|---|---|
| Part B: Number of Weeks With Platelet Counts >=30,000/μL and Doubling the Baseline | 9.3 ± 10.1 |
Rescue medication is defined as any therapy used to rescue a participant (1 of intravenous immunoglobulin \[IVIG\], high-dose steroids, platelet infusion or anti-D immunoglobulin infusion). Percentage of participants who received rescue medication are summarized here. 95% CI was based on the Clopper-Pearson exact method.
| percentage of participants | Part B: Rilzabrutinib 400 mg BID |
|---|---|
| Part B: Percentage of Participants Who Received Rescue Medication | 11.5 (2.45 to 30.15) |
The IBLS is a bleeding assessment score. IBLS comprises of 11 sites for female and 10 sites for male, and each site is scored from 0 (none) to 2 (marked bleeding). The total overall score ranges from 0-22, with higher scores indicating higher presence of marked bleeding. For each participant, an IBLS score at each visit was calculated by taking the average across 11 items (10 for male and postmenopausal women) at 9 anatomical sites (8 for male and postmenopausal women). For each participant, a mean IBLS score was also calculated by taking the average across all post-baseline visits during the 24-week treatment period. IBLS average value ranges from 0 to 2. The smaller the IBLS average value is, the healthier the participants are. For change from baseline, negative value indicates an improvement. The baseline value is defined as the last available value before the first dose rilzabrutinib.
| score on a scale | Part B: Rilzabrutinib 400 mg BID |
|---|---|
| Part B: Change From Baseline in Idiopathic Thrombocytopenic Purpura Bleeding Scale (IBLS) | -0.07 ± 0.13 |
Rescue medication is defined as any therapy used to rescue a participant (1 of IVIG, high-dose steroids, platelet infusion or anti-D immunoglobulin infusion). The percentage of participants who received rescue medication for each dose level and overall are summarized here. 95% CI was based on the Clopper-Pearson method.
| percentage of participants | Part A: Dose Level 200 mg QD | Part A: Dose Level 400 mg QD | Part A: Dose Level 300 mg BID | Part A: Dose Level 400 mg BID | Part A: Overall |
|---|---|---|---|---|---|
| Part A: Percentage of Participants Who Received Rescue Medication by Dose Levels and Overall | 11.1 (0.28 to 48.25) | 0.0 (0.00 to 36.94) | 8.3 (0.21 to 38.48) | 9.6 (3.20 to 21.03) | 11.7 (4.82 to 22.57) |
The percentage of participants with intensity grade 2 or higher bleeding event are summarized for each dose level and overall. The TEAEs with standardized medical dictionary for regulatory activities (MedDRA) query (SMQ) hemorrhages were medically determined for analysis of bleeding events. 95% CI was based on the Clopper-Pearson method.
| percentage of participants | Part A: Dose Level 200 mg QD | Part A: Dose Level 400 mg QD | Part A: Dose Level 300 mg BID | Part A: Dose Level 400 mg BID | Part A: Overall |
|---|---|---|---|---|---|
| Part A: Percentage of Participants With Grade 2 or Higher Bleeding Event by Dose Level and Overall | 0.0 (0.00 to 33.63) | 0.0 (0.00 to 36.94) | 8.3 (0.21 to 38.48) | 17.3 (8.23 to 30.33) | 16.7 (8.29 to 28.52) |
The ITP-BAT scale comprises of 11 grades from 0 (none) to 2 (marked bleeding), with higher scores indicating higher presence of marked bleeding, assessed at 9 anatomical sites (skin, oral, epistaxis, gastrointestinal \[GI\], urinary, gynecological \[GYN\], pulmonary, intracranial hemorrhage \[HEM\], subconjunctival HEM) by history over the previous week (Hx). In addition, 2 sites (skin and oral), were also assessed by physical examination (PE). The 'worst ever' bleeding experienced at each site was graded using the same system. Here, 0 indicates none; 1 indicates 1-5 bruises and/or scattered petechiae and 2 indicates \>5 bruises with size \>2 centimeter (cm) and/or diffuse petechiae. Each participant summed up the transformed scores across all 11 sites per visit assessment. The total overall score ranges from 0-22 with the higher score indicating worst outcome.
| Participants | Part A: Dose Level 200 mg QD | Part A: Dose Level 400 mg QD | Part A: Dose Level 300 mg BID | Part A: Dose Level 400 mg BID |
|---|---|---|---|---|
| Skin (PE): Score 0 | 7 | 8 | 6 | 32 |
| Skin (PE): Score 1 | 0 | 0 | 3 | 12 |
| Skin (PE): Score 2 | 0 | 0 | 0 | 6 |
| Skin (PE): Scoring not done/missing | 2 | 0 | 3 | 2 |
| Oral (PE): Score 0 | 7 | 8 | 9 | 42 |
| Oral (PE): Score 1 | 0 | 0 | 0 | 4 |
| Oral (PE): Score 2 | 0 | 0 | 0 | 4 |
| Oral (PE): Scoring not done/missing | 2 | 0 | 3 | 2 |
| Skin (Hx): Score 0 | 7 | 8 | 5 | 34 |
| Skin (Hx): Score 1 | 0 | 0 | 4 | 11 |
| Skin (Hx): Score 2 | 0 | 0 | 0 | 5 |
| Skin (Hx): Scoring not done/missing | 2 | 0 | 3 | 2 |
| Oral (Hx): Score 0 | 7 | 8 | 9 | 43 |
| Oral (Hx): Score 1 | 0 | 0 | 0 | 2 |
| Oral (Hx): Score 2 | 0 | 0 | 0 | 5 |
| Oral (Hx): Scoring not done/missing | 2 | 0 | 3 | 2 |
| Epistaxis: Score 0 | 7 | 8 | 9 | 49 |
| Epistaxis: Score 1 | 0 | 0 | 0 | 0 |
| Epistaxis: Score 2 | 0 | 0 | 0 | 1 |
| Epistaxis: Scoring not done/missing | 2 | 0 | 3 | 2 |
| GI: Score 0 | 7 | 8 | 10 | 49 |
| GI: Score 1 | 0 | 0 | 0 | 1 |
| GI: Score 2 | 0 | 0 | 0 | 0 |
| GI: Scoring not done/missing | 2 | 0 | 2 | 2 |
| Urinary: Score 0 | 7 | 8 | 9 | 48 |
| Urinary: Score 1 | 0 | 0 | 0 | 2 |
| Urinary: Score 2 | 0 | 0 | 0 | 0 |
| Urinary: Scoring not done/missing | 2 | 0 | 3 | 2 |
| GYN: Score 0 | 1 | 1 | 4 | 22 |
| GYN: Score 1 | 0 | 0 | 0 | 1 |
| GYN: Score 2 | 0 | 0 | 0 | 1 |
| GYN: Scoring not done/missing | 8 | 7 | 8 | 28 |
| Pulmonary: Score 0 | 6 | 8 | 8 | 49 |
| Pulmonary: Score 1 | 0 | 0 | 0 | 0 |
| Pulmonary: Score 2 | 0 | 0 | 0 | 0 |
| Pulmonary: Scoring not done/missing | 3 | 0 | 4 | 3 |
| Intracranial HEM: Score 0 | 7 | 8 | 8 | 49 |
| Intracranial HEM: Score 1 | 0 | 0 | 0 | 0 |
| Intracranial HEM: Score 2 | 0 | 0 | 0 | 0 |
| Intracranial HEM: Scoring not done/missing | 2 | 0 | 4 | 3 |
| Subconjunctival HEM: Score 0 | 7 | 8 | 8 | 50 |
| Subconjunctival HEM: Score 1 | 0 | 0 | 0 | 0 |
| Subconjunctival HEM: Score 2 | 0 | 0 | 0 | 0 |
| Subconjunctival HEM: Scoring not done/missing | 2 | 0 | 4 | 2 |
Plasma samples were collected at specified timepoints to determine Cmax of rilzabrutinib.
| nanogram per milliliter (ng/mL) | Part A: Dose Level 200 mg QD | Part A: Dose Level 400 mg QD | Part A: Dose Level 300 mg BID | Part A: Dose Level 400 mg BID |
|---|---|---|---|---|
| Cycle 1 Day 1 | 154 ± 117 | — | 287 ± 149 | 273 ± 203 |
| Cycle 2 Day 1 | — | 451 ± 408 | — | 396 ± 257 |
| Cycle 3 Day 1 | — | — | 319 ± 266 | — |
| Cycle 5 Day 1 | — | — | — | 447 ± 67.2 |
Plasma samples were collected at specified timepoints to determine tmax of rilzabrutinib.
| hours (h) | Part A: Dose Level 200 mg QD | Part A: Dose Level 400 mg QD | Part A: Dose Level 300 mg BID | Part A: Dose Level 400 mg BID |
|---|---|---|---|---|
| Cycle 1 Day 1 | 1.43 (0.500 to 2.00) | — | 1.50 (1.00 to 1.53) | 1.50 (0.500 to 4.00) |
| Cycle 2 Day 1 | — | 2.00 (1.00 to 3.03) | — | 1.05 (0.500 to 2.00) |
| Cycle 3 Day 1 | — | — | 1.50 (0.500 to 3.00) | — |
| Cycle 5 Day 1 | — | — | — | 1.25 (1.00 to 1.50) |
Plasma samples were collected at specified timepoints to determine AUClast of rilzabrutinib.
| h*ng/mL | Part A: Dose Level 200 mg QD | Part A: Dose Level 400 mg QD | Part A: Dose Level 300 mg BID | Part A: Dose Level 400 mg BID |
|---|---|---|---|---|
| Cycle 1 Day 1 | 320 ± 217 | — | 616 ± 334 | 642 ± 435 |
| Cycle 2 Day 1 | — | 986 ± 646 | — | 1150 ± 1030 |
| Cycle 3 Day 1 | — | — | 788 ± 610 | — |
| Cycle 5 Day 1 | — | — | — | 872 ± 412 |
Plasma samples were collected at specified timepoints to determine AUCinf of rilzabrutinib.
| h*ng/mL | Part A: Dose Level 200 mg QD | Part A: Dose Level 400 mg QD | Part A: Dose Level 300 mg BID | Part A: Dose Level 400 mg BID |
|---|---|---|---|---|
| Cycle 1 Day 1 | 331 ± 224 | — | 653 ± 351 | 703 ± 455 |
| Cycle 2 Day 1 | — | 787 ± 446 | — | 827 ± NA |
| Cycle 3 Day 1 | — | — | 818 ± 671 | — |
Plasma samples were collected at specified timepoints to determine t1/2 of rilzabrutinib.
| hours | Part A: Dose Level 200 mg QD | Part A: Dose Level 400 mg QD | Part A: Dose Level 300 mg BID | Part A: Dose Level 400 mg BID |
|---|---|---|---|---|
| Cycle 1 Day 1 | 1.36 (1.09 to 1.50) | — | 1.30 (1.15 to 1.95) | 1.32 (0.780 to 3.24) |
| Cycle 2 Day 1 | — | 1.52 (1.17 to 3.89) | — | 1.34 (1.34 to 1.34) |
| Cycle 3 Day 1 | — | — | 1.60 (1.18 to 1.88) | — |
Plasma samples were collected at specified timepoints to determine Vz/F of rilzabrutinib.
| Liters | Part A: Dose Level 200 mg QD | Part A: Dose Level 400 mg QD | Part A: Dose Level 300 mg BID | Part A: Dose Level 400 mg BID |
|---|---|---|---|---|
| Cycle 1 Day 1 | 1780 ± 1180 | — | 1180 ± 611 | 1720 ± 1460 |
| Cycle 2 Day 1 | — | 2090 ± 2640 | — | 934 ± NA |
| Cycle 3 Day 1 | — | — | 1650 ± 1510 | — |
Plasma samples were collected at specified timepoints to determine CL/F of rilzabrutinib.
| liters/hour | Part A: Dose Level 200 mg QD | Part A: Dose Level 400 mg QD | Part A: Dose Level 300 mg BID | Part A: Dose Level 400 mg BID |
|---|---|---|---|---|
| Cycle 1 Day 1 | 911 ± 569 | — | 592 ± 320 | 854 ± 551 |
| Cycle 2 Day 1 | — | 743 ± 593 | — | 484 ± NA |
| Cycle 3 Day 1 | — | — | 713 ± 621 | — |
Plasma samples were collected at specified timepoints for evaluation of rilzabrutinib pharmacokinetic (PK) concentrations.
| ng/mL | Part B: Rilzabrutinib 400 mg BID |
|---|---|
| Day 1: pre-dose | NA ± NA |
| Day 1: 2 hours post-dose | 154.09 ± 131.79 |
| Day 29: pre-dose | 5.07 ± 8.61 |
| Day 29: 2 hours post-dose | 228.67 ± 173.68 |
| Day 57: pre-dose | 26.42 ± 90.43 |
| Day 57: 2 hours post-dose | 331.81 ± 151.07 |
Collected over Adverse events data was collected from first dose of rilzabrutinib (Day 1) up to last dose + 1, approximately up to 294 days (Part A) and up to 170 days (Part B). All-cause mortality (death) was assessed from the signing of the ICF up to primary completion date (31 January 2023) approximately 253 weeks.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Part A: Dose Level 200 mg QD | 0/9 (0%) | 1/9 (11.1%) | 5/9 (55.6%) |
| Part A: Dose Level 400 mg QD | 0/8 (0%) | 2/8 (25%) | 6/8 (75%) |
| Part A: Dose Level 300 mg BID | 0/12 (0%) | 3/12 (25%) | 9/12 (75%) |
| Part A: Dose Level 400 mg BID | 1/52 (1.9%) | 9/52 (17.3%) | 43/52 (82.7%) |
| Part A: Overall | 1/60 (1.7%) | 11/60 (18.3%) | 48/60 (80%) |
| Part B: 400 mg BID | 0/26 (0%) | 3/26 (11.5%) | 21/26 (80.8%) |
| Event | Part A: Dose Level 200 mg QD | Part A: Dose Level 400 mg QD | Part A: Dose Level 300 mg BID | Part A: Dose Level 400 mg BID | Part A: Overall | Part B: 400 mg BID |
|---|---|---|---|---|---|---|
| Covid-19 PneumoniaInfections and infestations | 1/9 | 1/8 | 0/12 | 0/52 | 1/60 | 0/26 |
| IridocyclitisEye disorders | 0/9 | 1/8 | 1/12 | 1/52 | 1/60 | 0/26 |
| Rectal HaemorrhageGastrointestinal disorders | 0/9 | 0/8 | 1/12 | 1/52 | 1/60 | 0/26 |
| ContusionInjury, poisoning and procedural complications | 0/9 | 0/8 | 1/12 | 0/52 | 1/60 | 0/26 |
| Subcutaneous AbscessInfections and infestations | 0/9 | 0/8 | 0/12 | 0/52 | 0/60 | 1/26 |
| ThrombocytopeniaBlood and lymphatic system disorders | 0/9 | 0/8 | 0/12 | 2/52 | 2/60 | 1/26 |
| SyncopeNervous system disorders | 0/9 | 0/8 | 0/12 | 0/52 | 0/60 | 1/26 |
| Transient Ischaemic AttackNervous system disorders | 0/9 | 0/8 | 0/12 | 0/52 | 0/60 | 1/26 |
| Post Procedural HaemorrhageInjury, poisoning and procedural complications | 0/9 | 0/8 | 0/12 | 0/52 | 0/60 | 1/26 |
| PneumoniaInfections and infestations | 0/9 | 0/8 | 0/12 | 1/52 | 1/60 | 0/26 |
| Event | Part A: Dose Level 200 mg QD | Part A: Dose Level 400 mg QD | Part A: Dose Level 300 mg BID | Part A: Dose Level 400 mg BID | Part A: Overall | Part B: 400 mg BID |
|---|---|---|---|---|---|---|
| DiarrhoeaGastrointestinal disorders | 2/9 | 2/8 | 3/12 | 22/52 | 23/60 | 12/26 |
| ContusionInjury, poisoning and procedural complications | 3/9 | 3/8 | 3/12 | 10/52 | 12/60 | 5/26 |
| NauseaGastrointestinal disorders | 2/9 | 2/8 | 4/12 | 19/52 | 21/60 | 7/26 |
| HeadacheNervous system disorders | 1/9 | 1/8 | 1/12 | 7/52 | 7/60 | 9/26 |
| FatigueGeneral disorders | 2/9 | 2/8 | 2/12 | 12/52 | 13/60 | 3/26 |
| Covid-19Infections and infestations | 0/9 | 0/8 | 0/12 | 1/52 | 1/60 | 6/26 |
| Abdominal Pain UpperGastrointestinal disorders | 1/9 | 1/8 | 1/12 | 3/52 | 3/60 | 5/26 |
| DyspepsiaGastrointestinal disorders | 1/9 | 1/8 | 2/12 | 3/52 | 4/60 | 5/26 |
| Back PainMusculoskeletal and connective tissue disorders | 1/9 | 1/8 | 1/12 | 9/52 | 10/60 | 1/26 |
| Abdominal DistensionGastrointestinal disorders | 1/9 | 1/8 | 2/12 | 6/52 | 6/60 | 0/26 |
Part A: Intent-to-treat population (ITT) consisted of all participants who had enrolled into the study. Part B: The enrolled population consisted of all participants from screened population (who signed informed consent) who had been allocated to rilzabrutinib regardless of whether rilzabrutinib was received or not.
| Age, Customized(Participants) | Part A: Starting Dose 200 mg QD | Part A: Starting Dose 400 mg QD | Part A: Starting Dose 300 mg BID | Part A: Starting Dose 400 mg BID | Part B: Rilzabrutinib 400 mg BID | Total |
|---|---|---|---|---|---|---|
| <65 years | 8 | 1 | 5 | 38 | 19 | 71 |
| >= 65 years | 1 | 0 | 0 | 7 | 7 | 15 |
| Sex: Female, Male(Participants) | Part A: Starting Dose 200 mg QD | Part A: Starting Dose 400 mg QD | Part A: Starting Dose 300 mg BID | Part A: Starting Dose 400 mg BID | Part B: Rilzabrutinib 400 mg BID | Total |
|---|---|---|---|---|---|---|
| Female | 4 | 0 | 3 | 27 | 16 | 50 |
| Male | 5 | 1 | 2 | 18 | 10 | 36 |
| Race/Ethnicity, Customized(Participants) | Part A: Starting Dose 200 mg QD | Part A: Starting Dose 400 mg QD | Part A: Starting Dose 300 mg BID | Part A: Starting Dose 400 mg BID | Part B: Rilzabrutinib 400 mg BID | Total |
|---|---|---|---|---|---|---|
| White | 9 | 1 | 5 | 39 | 21 | 75 |
| Black or African American | 0 | 0 | 0 | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 | 2 | 4 | 6 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 1 | 0 | 1 |
| American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 | 0 |
| Other | 0 | 0 | 0 | 3 | 1 | 4 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — 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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Studies of the drug tested here: Rilzabrutinib
Purpura, Thrombocytopenic, Idiopathic →
Principia Biopharma, a Sanofi Company →