Warm autoimmune hemolytic anemia MedDRA version: 20.0 Level: PT Classification code 10073785 Term: Autoimmune haemolytic anaemia System Organ Class: 10005329 - Blood and lymphatic system disorders
Conditions
Sponsors
Sanofi aventis recherche et developpement
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Male and female patients with a confirmed diagnosis of primary wAIHA or systemic lupus erythematosus (SLE)-associated wAIHA (without other SLE-related manifestations apart from cutaneous and musculoskeletal manifestations) - Participants who have previously failed to maintain a sustained response after treatment with corticosteroids. - Eastern Cooperative Oncology Group (ECOG) performance status grade 2 or lower. - Up-to-date vaccination status as per local guidelines. - Body mass index (BMI) >17.5 and =65 years) yes F.1.3.1 Number of subjects for this age range 10
Exclusion criteria
Exclusion criteria: - Clinically significant medical history or ongoing chronic illness that would jeopardize the safety of the participant or compromise the quality of the data derived from his or her participation in the study as determined by the Investigator. - Participants with medical history of lymphoma, leukemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for the past 3 years. - Secondary wAIHA from any cause including drugs, lymphoproliferative disorders (low-count monoclonal B-cell lymphocytosis is allowed), infectious or autoimmune disease, or active hematologic malignancies. Participants with positive antinuclear antibodies but without a definitive diagnosis of an autoimmune disease are allowed. - Myelodysplastic syndrome. - Uncontrolled or active HBV infection: Patients with positive HBsAg and/or HBV DNA. - HIV infection. - Concurrent treatment with other experimental drugs or participation in another clinical trial with any investigational drug within 30 days or 5 half-lives, whichever is greater, prior to treatment start. - Known allergy to any of the study medications, their analogues, or excipients in the various formulations of any agent. Part B only - Participants who receive any therapy during Part A known to be active in wAIHA. - Presence of unacceptable side effect(s) or toxicity associated with rilzabrutinib such that there is an unfavorable risk-benefit assessment for continued treatment with rilzabrutinib in the opinion of the Investigator and/or Sponsor.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Secondary Objective: Part A: • To evaluate the durable hemoglobin response in adult patients with wAIHA • To assess time to response (TTR) Parts A and B: • To assess the effect of treatment with rilzabrutinib on rescue medication requirement in patients with wAIHA • To evaluate the impact of rilzabrutinib treatment on fatigue • To evaluate the safety and tolerability of rilzabrutinib in patients with wAIHA;Main Objective: Part A: To evaluate the efficacy of rilzabrutinib in adult patients with wAIHA Part B: To evaluate the long-term efficacy of rilzabrutinib in patients with wAIHA;Primary end point(s): 1 - Proportion of participants with overall hemoglobin response ; 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, LDH, haptoglobin, and reticulocytes), and absence of transfusion in the last 7 days and an absence of rescue medications during the last 4 weeks. 2 - Proportion of participants who maintain durable response achieved during Part A or achieve a durable response during Part B and have a hemoglobin response ; 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.;Timepoint(s) of evaluation of this end point: 1 - By Week 24 in Part A 2 - At Week 50 in Part B | — |
Secondary
| Measure | Time frame |
|---|---|
| Timepoint(s) of evaluation of this end point: 1 - By Week 24 in Part A 2 - From Day 1 to Week 24 in Part A 3 - After Week 1 of treatment to Week 24 in Part A and Week 75 in Part B 4, 5 - Until Week 24 in Part A and Week 75 in Part B;Secondary end point(s): 1 - Proportion of participants with durable hemoglobin response ; 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. 2 - Median time from baseline to first hemoglobin response 3 - Frequency of rescue therapy (any wAIHA-directed therapy other than predniso[lo]ne or transfusion) received 4 - Change from baseline in FACIT-Fatigue scale score 5 - Safety assessments | — |
Countries
China, Denmark, Italy, Spain, United Kingdom, United States
Contacts
Public ContactUnidad de Estudios Clínicos
Sanofi-Aventis, S.A
Outcome results
None listed