A multicenter, open-label, non-randomized, Phase 1b/2 study to evaluate the safety, pharmacokinetics, and efficacy of subcutaneous isatuximab in adults with warm autoimmune hemolytic anemia

Phase 2

Completed

• Conditions: autoimmune anemia; warm AIHA; 10003816

• Registration Number: NL-OMON51964
• Lead Sponsor: Genzyme Europe BV

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Last Update Posted: 9 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 2

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 <10 g/dL at screening.
b) Hemolysis (haptoglobin <=40 mg/dL and total or indirect/unconjugated
bilirubin above the upper limit of normal).
c) Positive direct antiglobulin test (DAT) (IgG or IgG + complement C3d pattern
or IgM warm autoantibodies (positive dual DAT)).
- Participants who have previously failed to maintain a sustained response
after treatment with corticosteroids (corticosteroid-refractory or
corticosteroid-dependent primary wAIHA).
- Part A only: Participants who have previously failed to maintain a sustained
response after treatment with rituximab (or other anti-CD20 monoclonal
antibodies). The last dose of the anti-CD20 antibody must have been
administered at least 12 weeks before enrollment.
- Part B: Participants who have had an insufficient response to at least 1
prior therapy in addition to corticosteroids (splenectomy is regarded as a
prior therapy).
- Contraceptive use by men and women.

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 <3 g/L.
- Females who are pregnant, lactating, or considered unreliable with respect to
contraceptive practice.
- Concurrent treatment with corticosteroids, unless the participant has been on
a stable daily dose for >= 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.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Part A:<br /><br>- To assess safety and tolerability<br /><br><br /><br>Part B:<br /><br>- To evaluate overall response rate (R) or complete response (CR) at Day 85</p><br>