autoimmune anemia warm AIHA
Conditions
Interventions
Part A, Cohort 1: 140 mg every 2 weeks x 2
Part A, Cohort 2: 70 mg, or 140 mg, or 280 mg every 2 weeks x 6
Part A, Cohort 3, and Part B: every 2 weeks x 6
dose level to be determined
based on results from prior Cohorts
the dose will not exceed 560 mg
Sponsors
Genzyme Europe BV
Eligibility
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: Participant must be >=18 to years of age, inclusive, at the time of signing the informed consent. - Males and females with a confirmed diagnosis of primary w AIHA or systemic lupus erythematosus (SLE)-associated wAIHA (without other SLE-related manifestations apart from cutaneous and musculoskeletal manifestations) who meet the following criteria: a) Hemoglobin level
Exclusion criteria
Exclusion criteria: Participants are excluded from the study if any of the following criteria apply: - 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. - Serious infection that required hospitalization within 3 months prior to enrollment. - Secondary wAIHA from any cause including drugs, lymphoproliferative disorders, infectious or autoimmune disease (SLE without other SLE-related manifestations apart from cutaneous and musculoskeletal manifestations is allowed), or active hematologic malignancies. Participants with positive antinuclear antibodies but without a definitive diagnosis of an autoimmune disease are allowed. - History of coagulation or bleeding disorders (Evans Syndrome is allowed). - Uncontrolled or active HBV or HCV infection. - HIV infection. - Serum gammaglobulin levels = 15 days prior to enrollment. - Treatment with cyclophosphamide within 4 weeks prior to enrollment. - Treatment with cytotoxic drugs (other than cyclophosphamide) within 12 weeks prior to enrollment. - Treatment with non-cytotoxic, immunomodulatory drugs (including but not limited to Cyclosporine, Sirolimus, Tacrolimus, Idelalisib, Ibrutinib), excluding biologic agents, within 4 weeks prior to enrollment. - Treatment with any biologic agent within 12 weeks prior to enrollment.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part A: - To assess safety and tolerability Part B: - To evaluate overall response rate (R) or complete response (CR) at Day 85 | — |
Secondary
| Measure | Time frame |
|---|---|
| Part A (Cohorts 2 and 3 Only): - To evaluate overall response rate (R) or complete response (CR) at Day 85 - Proportion of participants with durable hemoglobin response by Day 169 - Overall response rate at Day 169, median time to R or CR, median time to loss of R or CR, proportion of participants requiring rescue therapy (any wAIHA-directed therapy other than prednisone or transfusion) or splenectomy - FACIT-fatigue scale score Part B - To assess safety and tolerability - Proportion of participants with durable hemoglobin response by Day 169 - Overall response rate at Day 169, median time to PR or CR, median time to loss of PR or CR, proportion of participants requiring rescue therapy (any wAIHA-directed therapy other than prednisone or transfusion) or splenectomy - FACIT-fatigue scale score Part A (All cohorts) and B - Change from baseline in LDH, haptoglobin, reticulocytes, and total bilirubin - PK parameters after subcutaneous administrations - Incidence and titer (if relevant) of anti-isatuximab antibodies | — |
Countries
Netherlands
Outcome results
None listed