ALXN1830 in Patients With Warm Autoimmune Hemolytic Anemia

Overview

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 Title of Study: “A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Study of ALXN1830 in Patients With Warm Autoimmune Hemolytic Anemia”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: Triple (Participant, Care Provider, Investigator)
  • Study Primary Completion Date: October 30, 2022

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).

Interventions

  • Biological: ALXN1830
    • Administered via intravenous (IV) infusion
  • Other: Placebo
    • Matching placebo (sterile liquid diluent) administered via IV infusion

Arms, Groups and Cohorts

  • Experimental: ALXN1830 Dosing Regimen 1
  • Experimental: ALXN1830 Dosing Regimen 2
  • Experimental: ALXN1830 Dosing Regimen 3
  • Active Comparator: Placebo

Clinical Trial Outcome Measures

Primary Measures

  • Percentage Of Participants With ≥ 2 Grams (g)/Deciliter (dL) Increase In Hemoglobin (Hgb) From Baseline
    • Time Frame: Baseline (Day 1) through Day 92

Secondary Measures

  • Total Number Of Units Of Packed Red Blood Cells (pRBCs) Transfused
    • Time Frame: Day 15 through Day 92
  • Mean Change From Baseline To Day 92 In Quality Of Life, Assessed Via EuroQoL 5 Dimension 5 Level (EQ-5D-5L) Questionnaire
    • Time Frame: Baseline, Day 92
  • Mean Change From Baseline To Day 92 In Quality Of Life, Assessed Via Functional Assessment Of Cancer Therapy Anemia (FACT-AN) Questionnaire
    • Time Frame: Baseline, Day 92
  • Number Of Weekly Hgb Measurements With Change From Baseline ≥ 2 g/dL
    • Time Frame: Day 1 through Day 92
  • Number Of Participants Needing New WAIHA Rescue Medication Or Increase In Dose Of WAIHA Medication
    • Time Frame: Day 15 through Day 92

Participating in This Clinical Trial

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
  • Gender Eligibility: All

    Minimum Age: 18 Years

    Maximum Age: N/A

    Are Healthy Volunteers Accepted: No

    Investigator Details

    • Lead Sponsor
      • Alexion Pharmaceuticals
    • Provider of Information About this Clinical Study
      • Sponsor

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