A Phase 3 Study of Obexelimab in Patients with Warm Autoimmune Hemolytic Anemia

JRCT ID: jRCT2021230052

Registered date:18/01/2024

Basic Information

• Recruitment status: Pending
• Health condition(s) or Problem(s) studied: Warm Autoimmune Hemolytic Anemia
• Date of first enrollment: 01/02/2024
• Target sample size: 8
• Countries of recruitment: ITALY,Japan,POLAND,Japan,SPAIN,Japan,TAIWAN,Japan,TURKEY,Japan,UNITED KINGDOM,Japan,UNITED STATES,Japan
• Study type: Interventional
• Intervention(s): Obexelimab is administered as SC injections every 7 days during Dose Confirmation Run-in Period and Open-Label Extension Period. Obexelimab or placebo is administered as SC injections every 7 days during Randomized Control Period.

Outcome(s)

Primary Outcome

1.Safety and Dose Confirmation Run-in Period (SRP) [Time Frame: 24 weeks]: Proportion of participants with hemoglobin (Hgb) 10 g/dL or more and 2 g/dL or more increase from Baseline with no use of blood transfusion or glucocorticoid (GC) rescue therapy.
2.Randomized Control Period (RCP) [Time Frame: 24 weeks]: Proportion of participants who achieve a durable Hgb response (defined as Hgb 10 g/dL or more and 2 g/dL or more increase from Baseline on at least 3 of 4 consecutive available visits), at the earliest on or after Week 12, with no use of blood transfusion or GC rescue therapy prior to attaining durable response through Week 24.

Secondary Outcome

1.SRP [Time Frame: 24 weeks] Change from Baseline in FACIT-F score through Week 24, Proportion of patients with no use of blood transfusion or GC rescue therapy through Week 24, Cumulative dose of GC rescue therapy through Week 24 and other secondary outcomes.
2.RCP [Time Frame: 24 weeks]: Change from Baseline to Week 24 in FACIT-F Score, Proportion of patients with no use of blood transfusion or GC rescue therapy through Week 24, Cumulative dose of GC rescue therapy through Week 24, Proportion of patients with no use of blood transfusions through Week 24 and other secondary outcomes.

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1.Males and females, 18 years of age
2.Clinically diagnosed with wAIHA for at least 3 months and currently receiving treatment for wAIHA or have previously received treatment for wAIHA.
3.Diagnosis of primary or secondary wAIHA documented by a positive direct antiglobulin test specific for anti-IgG or anti-IgA.
4.Failed at least 1 prior wAIHA treatment regimen.
5.At least one sign or symptom of anemia as assessed by the investigator at screening.
6.Other inclusion criteria apply.

Exclude criteria

1.Have cold antibody AIHA, cold agglutinin syndrome, mixed type (i.e., warm, and cold) AIHA, or paroxysmal cold hemoglobinuria.
2.Have any other associated cause of hereditary or acquired hemolytic anemia.
3.For the Randomized Control Period only, patients with secondary wAIHA not due to autoimmune disorders, including lymphoproliferative disease s.
4.Received a transfusion within 2 weeks prior to randomization.
5.Use of B cell-depleting, B cell-targeted, or other biologic immunomodulatory agents within the 6 months prior to randomization.
6.Received IVIg or epoetin alfa within 6 weeks prior to randomization.
7.Receiving more than 2 concomitant medications for the treatment of wAIHA.
8.Other exclusion criteria apply.

Related Information

• Primary Sponsor: Sato Toshiyuki
• Secondary ID(s): NCT05786573