ALXN1830 in Patients With Warm Autoimmune Hemolytic Anemia

NCT04256148 | Withdrawn Phase 2 DMC FDA Drug

• 1 other identifier: ALXN1830-WAI-201
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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).

Conditions

Warm Autoimmune Hemolytic Anemia

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Percentage Of Participants With ≥ 2 Grams (g)/Deciliter (dL) Increase In Hemoglobin (Hgb) From Baseline

  Baseline (Day 1) through Day 92

Secondary Outcomes (5)

• 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

Study Arms (4)

ALXN1830 Dosing Regimen 1

EXPERIMENTAL

Biological: ALXN1830

ALXN1830 Dosing Regimen 2

EXPERIMENTAL

Biological: ALXN1830

ALXN1830 Dosing Regimen 3

EXPERIMENTAL

Biological: ALXN1830

Placebo

ACTIVE COMPARATOR

Other: Placebo

Interventions

ALXN1830 BIOLOGICAL

Administered via intravenous (IV) infusion

Also known as: SYNT001

ALXN1830 Dosing Regimen 1; ALXN1830 Dosing Regimen 2; ALXN1830 Dosing Regimen 3

Placebo OTHER

Matching placebo (sterile liquid diluent) administered via IV infusion

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• Human immunodeficiency virus (HIV) infection (positive HIV-1 or HIV-2 antibody test)
• Positive hepatitis B surface antigen (HBsAg) or hepatitis C antibody test

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (1)

Alexion Study Site

Whittier, California, 90602, United States

Location

MeSH Terms

Interventions

orilanolimab

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: Double-blind
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

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

Study Record Dates

• First Submitted: January 27, 2020
• First Posted: February 5, 2020
• Study Start: July 1, 2021
• Primary Completion: October 30, 2022
• Study Completion: April 30, 2023
• Last Updated: July 13, 2020

Record last verified: 2020-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)