ALXN1830 in Patients With Warm Autoimmune Hemolytic Anemia

Alexion Pharmaceuticals

Status and phase: Withdrawn; Phase 2

Conditions: Warm Autoimmune Hemolytic Anemia

Treatments: Other: Placebo; Biological: ALXN1830

Study type: Interventional

Funder types: Industry

Identifiers

NCT04256148

ALXN1830-WAI-201

Details and patient eligibility

About

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).

Full 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).

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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0 participants in 4 patient groups

ALXN1830 Dosing Regimen 1

Experimental group

Treatment:

Biological: ALXN1830

ALXN1830 Dosing Regimen 2

Experimental group

Treatment:

Biological: ALXN1830

ALXN1830 Dosing Regimen 3

Experimental group

Treatment:

Biological: ALXN1830

Placebo

Active Comparator group

Treatment:

Other: Placebo