Efficacy, Safety and Pharmacokinetics of Rilzabrutinib in Patients With Warm Autoimmune Hemolytic Anemia (wAIHA)
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|ClinicalTrials.gov Identifier: NCT05002777|
Recruitment Status : Not yet recruiting
First Posted : August 12, 2021
Last Update Posted : September 29, 2021
This is a single group treatment, Phase 2, open-label, study to evaluate the efficacy, safety and pharmacokinetics of rilzabrutinib in adult patients with wAIHA.
All participants will receive rilzabrutinib orally. The screening period is up to 28 days, followed by a treatment period of 24 weeks for Part A. Participants who complete Part A and are deemed eligible for Part B will continue to receive the study medication for 52 weeks following the Last Patient In (LPI-Part B). There will be a 7-day safety follow-up period after receiving the last dose of study drug either in Part A (for those not eligible for Part B or early terminated) or Part B. The estimated total duration of the study is approximately 137 weeks (Parts A and B), including the follow-up period. For participants deemed ineligible for Part B, the total length of the study will be 29 weeks (Part A only), including screening and the follow-up period. In Part B, participants who temporarily stop rilzabrutinib treatment and maintain a durable response from W50 to W74, will have their EOS visit at Week 75. In this case, participation will be for 79 weeks including the screening period.
|Condition or disease||Intervention/treatment||Phase|
|Warm Autoimmune Hemolytic Anemia (wAIHA)||Drug: rilzabrutinib||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Multicenter, Open-label, Phase IIb Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Rilzabrutinib in Patients With Warm Autoimmune Hemolytic Anemia|
|Estimated Study Start Date :||October 4, 2021|
|Estimated Primary Completion Date :||July 20, 2023|
|Estimated Study Completion Date :||June 10, 2024|
Oral rilzabrutinib 400 mg BID
Pharmaceutical form: Tablet Route of administration: Oral
Other Name: PRN1008/SAR444671
- Part A: Proportion of participants with overall hemoglobin response [ Time Frame: By Week 24 in Part A ]
Response is defined as an increase in hemoglobin (Hb) by ≥2 g/dL from baseline and an absence of transfusion in the last 7 days, without biochemical resolution of hemolysis at the time response is achieved and an absence of rescue medications during the last 4 weeks.
Complete Response is defined as hemoglobin ≥11 g/dL (women) or ≥12 g/dL (men), no evidence of hemolysis (normal bilirubin, lactate dehydrogenase (LDH) , haptoglobin, and reticulocytes), and absence of transfusion in the last 7 days and an absence of rescue medications during the last 4 weeks.
- Part B: Proportion of participants who maintain durable response achieved during Part A or achieve a durable response during Part B and have a hemoglobin response [ Time Frame: At Week 50 in Part B ]Durable response (Part B) is defined as Hb level ≥10 g/dL with an increase from baseline (Part A) of ≥2 g/dL on three consecutive scheduled visits during Week 24 to Week 50; with absence of transfusion and no rescue medication during the period of 3 consecutive visits and for at least 7 days (transfusions) and 4 weeks (rescue medication) prior to the first consecutive visit.
- Proportion of participants with durable hemoglobin response [ Time Frame: By Week 24 in Part A ]Durable response (Part A) is defined as Hb level ≥10 g/dL with an increase from baseline of ≥2 g/dL on three consecutive evaluable visits during the 24-week treatment period; with absence of transfusion and no rescue medication during the period of 3 consecutive visits and for at least 7 days (transfusions) and 4 weeks (rescue medication) prior to the first consecutive visit.
- Median time from baseline to first hemoglobin response [ Time Frame: From Day 1 to Week 24 in Part A ]
- Frequency of rescue therapy (any wAIHA-directed therapy other than predniso[lo]ne or transfusion) received [ Time Frame: After Week 1 of treatment to Week 24 in Part A and Week 75 in Part B ]
- Change from baseline in FACIT-Fatigue scale score [ Time Frame: Until Week 24 in Part A and Week 75 in Part B ]The Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) scale is a 13-item questionnaire used to assesses self-reported fatigue and its impact on daily activities and function.
- Incidence of treatment emergent adverse events (TEAEs), serious TEAEs, adverse events of special interest (AESIs) [ Time Frame: Until Week 24 in Part A and Week 75 in Part B ]Safety assessment including clinical laboratory evaluations, vital sign measurements and ECG
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05002777
|Contact: Trial Transparency email recommended (Toll free number for US & Canada)||800-633-1610 ext ext option 6||Contact-US@sanofi.com|
|United States, District of Columbia|
|Investigational Site Number 8400003|
|Washington, District of Columbia, United States, 20007|
|Contact: Jennifer Steffen 202-687-0116 firstname.lastname@example.org|
|Principal Investigator: Catherine Broome|
|United States, Florida|
|Investigational Site Number 8400002|
|Tamarac, Florida, United States, 33321|
|Contact: Nima Nagarajan 954-302-3163 email@example.com|
|Principal Investigator: Sumit Sawhney|
|United States, Massachusetts|
|Investigational Site Number 8400001|
|Boston, Massachusetts, United States, 02114|
|Principal Investigator: David Kuter|
|Investigational Site Number 1560002|
|Beijing, China, 100005|
|Contact: Dr. Bing Han 13601059938 firstname.lastname@example.org|
|Principal Investigator: Dr. Bing Han|
|Investigational Site Number 7240001|
|Barcelona, Spain, 08036|
|Contact: Dr. Joan Cid 0034932275400 email@example.com|
|Principal Investigator: Dr. Joan Cid|
|Investigational Site Number 7240003|
|Madrid, Spain, 28046|
|Contact: Dr. Marta Morado 0034917277000 firstname.lastname@example.org|
|Principal Investigator: Dr. Marta Morado|
|Investigational Site Number 7240002|
|Sevilla, Spain, 41013|
|Contact: Dr. Maria Eva Mingot 0034955012000 email@example.com|
|Principal Investigator: Dr. Maria Eva Mingot|