ALXN1830 in Patients With Warm Autoimmune Hemolytic Anemia

Sponsor

Alexion Pharmaceuticals (Industry)

Overall Status

Withdrawn

CT.gov ID

NCT04256148

Collaborator

(none)

Enrollment

Location

Arms

21.9

Anticipated Duration (Months)

Patients Per Site Per Month

Study Details

Study Description

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).

Condition or Disease	Intervention/Treatment	Phase
Warm Autoimmune Hemolytic Anemia	Biological: ALXN1830 Other: Placebo	Phase 2

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).

Study Design

Study Type:

Interventional

Actual Enrollment :

0 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Intervention Model Description:

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

Masking:

Triple (Participant, Care Provider, Investigator)

Masking Description:

Double-blind

Primary Purpose:

Treatment

Official Title:

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

Anticipated Study Start Date :

Jul 1, 2021

Anticipated Primary Completion Date :

Oct 30, 2022

Anticipated Study Completion Date :

Apr 30, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: ALXN1830 Dosing Regimen 1	Biological: ALXN1830 Administered via intravenous (IV) infusion Other Names: SYNT001
Experimental: ALXN1830 Dosing Regimen 2	Biological: ALXN1830 Administered via intravenous (IV) infusion Other Names: SYNT001
Experimental: ALXN1830 Dosing Regimen 3	Biological: ALXN1830 Administered via intravenous (IV) infusion Other Names: SYNT001
Active Comparator: Placebo	Other: Placebo Matching placebo (sterile liquid diluent) administered via IV infusion

Outcome Measures

Primary Outcome Measures

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

Secondary Outcome Measures

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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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