Subcutaneous ALXN1830 in Adult Participants With Warm Autoimmune Hemolytic Anemia

• Status: Not yet recruiting
• Trial end date: 2024-07-01
• Target enrollment: 0
• Participant gender: All

Summary

This is a Phase 2, multiple ascending, dose-finding, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, health-related quality of life, tolerability, pharmacokinetic, pharmacodynamic, and immunogenicity, of up to 3 dose regimens of ALXN1830 administered subcutaneous(ly) (SC) in the treatment of WAIHA.

This study will include 2 randomized, double-blind, placebo-controlled cohorts (Cohorts 1 and 2) to evaluate an 8-week treatment regimen, and an optional third open-label cohort (Cohort 3) to evaluate an alternative 12-week dosing regimen. Participants may continue participation in this study at the participant's and investigator's discretion in an open-label extension (OLE) period, consisting of monthly visits to observe participants for relapse, which will require going back on active treatment.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Alexion Pharmaceuticals

Criteria

Key Inclusion Criteria:

• Diagnosed with primary or secondary WAIHA at least 6 weeks prior to Screening.

• Failed or have not tolerated at least one prior WAIHA treatment regimen, for example, corticosteroids, rituximab, azathioprine, cyclophosphamide, cyclosporine, mycophenolate mofetil, danazol, or vincristine.

• Hemoglobin < 10 g/dL and ≥ 6 g/dL at Screening.

• Positive direct antiglobulin test (Coombs) (IgG positive who are positive or negative for the presence of complement C3) at Screening.

• Evidence of active hemolysis including any one of the below:

• LDH > upper limit of normal (ULN) or

• Haptoglobin < lower limit of normal or

• Indirect bilirubin > ULN

• Total IgG > 500 mg/dL at Screening

• Platelet count ≥ 75 x 10^9/liter (L)

• Absolute neutrophil count greater than 1.0 x 10^9/L

Key Exclusion Criteria:

• Participants with Evan's syndrome.

• Human immunodeficiency virus (HIV) infection (positive HIV 1 or HIV 2 antibody test).

• Positive hepatitis B surface antigen or hepatitis C antibody test.

• Inability to travel to the clinic for specified visits during the Primary Treatment Period or fulfill the logistical requirements of study intervention administration.