Subcutaneous ALXN1830 in Adult Participants With Warm Autoimmune Hemolytic Anemia
Study Details
- Warm Autoimmune Hemolytic Anemia
- Drug: ALXN1830
- Drug: Placebo
- 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
- 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.
Study Details
- Warm Autoimmune Hemolytic Anemia
- Drug: ALXN1830
- Drug: Placebo
- 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
- 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.
Protocol 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.