ALXN1830 in Patients With Warm Autoimmune Hemolytic Anemia

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

Status

Withdrawn

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT04256148

Enrollment

Registered

2020-02-05

Start date

2021-07-31

Completion date

2023-04-30

Last updated

2020-07-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Warm Autoimmune Hemolytic Anemia

Keywords

Warm autoimmune hemolytic anemia, WAIHA, Immunoglobulin G (IgG)-mediated autoimmune disorder, Pathogenesis

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).

Interventions

BIOLOGICALALXN1830

Administered via intravenous (IV) infusion

OTHERPlacebo

Matching placebo (sterile liquid diluent) administered via IV infusion

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

TRIPLE (Subject, Caregiver, Investigator)

Masking description

Double-blind

Intervention model description

Participants randomized in 1:1:1:1 ratio to 1 of 4 study arms.

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion 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

Design outcomes

Primary

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

Secondary

Measure	Time frame
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 EuroQoL 5 Dimension 5 Level (EQ-5D-5L) Questionnaire	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	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

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026