ALXN1830 in Patients With Warm Autoimmune Hemolytic Anemia
Phase 2
Withdrawn
- Conditions
- Warm Autoimmune Hemolytic Anemia
- Registration Number
- NCT04256148
- Lead Sponsor
- Alexion Pharmaceuticals, Inc.
- Brief Summary
The main objective of the study is to evaluate the safety and efficacy of ALXN1830 compared to placebo in adult participants with warm autoimmune hemolytic anemia (WAIHA).
- Detailed Description
This study will consist of a 4-week Screening period, 13-week Primary Treatment period, and optional Extended Treatment period (up to 2 years).
Recruitment & Eligibility
- Status
- WITHDRAWN
- Sex
- All
- Target Recruitment
- Not specified
Inclusion Criteria
- Primary or secondary WAIHA, diagnosed at least 6 weeks prior to Screening
- Failed or not tolerated at least 1 prior WAIHA treatment regimen, for example, corticosteroids, rituximab, azathioprine, cyclophosphamide, cyclosporine, mycophenolate mofetil, danazol, or vincristine
- Hemoglobin < 10 g/dL and ≥ 6 g/dL
- Positive direct antiglobulin test (Coombs) (IgG positive, complement C3 [C3] positive or negative)
- Evidence of active hemolysis including any of the following: a) Lactate dehydrogenase (LDH) > upper limit of normal (ULN), b) Haptoglobin < lower limit of normal (LLN), c) Indirect bilirubin > ULN
- Total IgG > 500 mg/dL
Key
Exclusion Criteria
- Human immunodeficiency virus (HIV) infection (positive HIV-1 or HIV-2 antibody test)
- Positive hepatitis B surface antigen (HBsAg) or hepatitis C antibody test
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Primary Outcome Measures
Name Time Method Percentage Of Participants With ≥ 2 Grams (g)/Deciliter (dL) Increase In Hemoglobin (Hgb) From Baseline Baseline (Day 1) through Day 92
- Secondary Outcome Measures
Name Time Method Total Number Of Units Of Packed Red Blood Cells (pRBCs) Transfused Day 15 through Day 92 Mean Change From Baseline To Day 92 In Quality Of Life, Assessed Via Functional Assessment Of Cancer Therapy Anemia (FACT-AN) Questionnaire Baseline, Day 92 Mean Change From Baseline To Day 92 In Quality Of Life, Assessed Via EuroQoL 5 Dimension 5 Level (EQ-5D-5L) Questionnaire Baseline, Day 92 Number Of Weekly Hgb Measurements With Change From Baseline ≥ 2 g/dL Day 1 through Day 92 Number Of Participants Needing New WAIHA Rescue Medication Or Increase In Dose Of WAIHA Medication Day 15 through Day 92
Trial Locations
- Locations (1)
Alexion Study Site
🇺🇸Whittier, California, United States
Alexion Study Site🇺🇸Whittier, California, United States