ALXN1830 in Patients With Warm Autoimmune Hemolytic Anemia

Warm Autoimmune Hemolytic Anemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT04256148
• Other study ID #: ALXN1830-WAI-201
• Status: Withdrawn
• Phase: Phase 2
• Start date: July 2021
• Est. completion date: April 30, 2023

Study information

• Verified date: July 2020
• Source: Alexion Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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 information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: April 30, 2023
• Est. primary completion date: October 30, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Anemia
• Anemia, Hemolytic
• Anemia, Hemolytic, Autoimmune
• Hemolysis
• Warm Autoimmune Hemolytic Anemia

Intervention

Biological:
• ALXN1830
Administered via intravenous (IV) infusion

Other:
• Placebo
Matching placebo (sterile liquid diluent) administered via IV infusion

Locations

Country	Name	City	State
United States	Alexion Study Site	Whittier	California

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percentage Of Participants With = 2 Grams (g)/Deciliter (dL) Increase In Hemoglobin (Hgb) From Baseline		Baseline (Day 1) through Day 92
Secondary	Total Number Of Units Of Packed Red Blood Cells (pRBCs) Transfused		Day 15 through Day 92
Secondary	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
Secondary	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
Secondary	Number Of Weekly Hgb Measurements With Change From Baseline = 2 g/dL		Day 1 through Day 92
Secondary	Number Of Participants Needing New WAIHA Rescue Medication Or Increase In Dose Of WAIHA Medication		Day 15 through Day 92