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ALXN1830 in Patients With Warm Autoimmune Hemolytic Anemia

Primary Purpose

Warm Autoimmune Hemolytic Anemia

Status

Withdrawn

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

ALXN1830

Placebo

About this trial

This is an interventional treatment trial for Warm Autoimmune Hemolytic Anemia focused on measuring Warm autoimmune hemolytic anemia, WAIHA, Immunoglobulin G (IgG)-mediated autoimmune disorder, Pathogenesis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

Alexion Study Site

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Active Comparator

Arm Label

ALXN1830 Dosing Regimen 1

ALXN1830 Dosing Regimen 2

ALXN1830 Dosing Regimen 3

Placebo

Arm Description

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Total Number Of Units Of Packed Red Blood Cells (pRBCs) Transfused

Mean Change From Baseline To Day 92 In Quality Of Life, Assessed Via EuroQoL 5 Dimension 5 Level (EQ-5D-5L) Questionnaire

Mean Change From Baseline To Day 92 In Quality Of Life, Assessed Via Functional Assessment Of Cancer Therapy Anemia (FACT-AN) Questionnaire

Number Of Weekly Hgb Measurements With Change From Baseline ≥ 2 g/dL

Number Of Participants Needing New WAIHA Rescue Medication Or Increase In Dose Of WAIHA Medication

Full Information

NCT ID

NCT04256148

First Posted

January 27, 2020

Last Updated

July 9, 2020

Sponsor

Alexion

1. Study Identification

Unique Protocol Identification Number

NCT04256148

Brief Title

ALXN1830 in Patients With Warm Autoimmune Hemolytic Anemia

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

July 2020

Overall Recruitment Status

Withdrawn

Why Stopped

Study is withdrawn due to the global impact of the COVID-19 pandemic.

Study Start Date

July 2021 (Anticipated)

Primary Completion Date

October 30, 2022 (Anticipated)

Study Completion Date

April 30, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Alexion

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. 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).

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Warm Autoimmune Hemolytic Anemia

Keywords

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Model Description

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

Masking

ParticipantCare ProviderInvestigator

Masking Description

Double-blind

Allocation

Randomized

Enrollment

0 (Actual)

8. Arms, Groups, and Interventions

Arm Title

ALXN1830 Dosing Regimen 1

Arm Type

Experimental

Arm Title

ALXN1830 Dosing Regimen 2

Arm Type

Experimental

Arm Title

ALXN1830 Dosing Regimen 3

Arm Type

Experimental

Arm Title

Placebo

Arm Type

Active Comparator

Intervention Type

Biological

Intervention Name(s)

ALXN1830

Other Intervention Name(s)

SYNT001

Intervention Description

Administered via intravenous (IV) infusion

Intervention Type

Other

Intervention Name(s)

Placebo

Intervention Description

Matching placebo (sterile liquid diluent) administered via IV infusion

Primary Outcome Measure Information:

Title

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

Time Frame

Baseline (Day 1) through Day 92

Secondary Outcome Measure Information:

Title

Total Number Of Units Of Packed Red Blood Cells (pRBCs) Transfused

Time Frame

Day 15 through Day 92

Title

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

Title

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

Title

Number Of Weekly Hgb Measurements With Change From Baseline ≥ 2 g/dL

Time Frame

Day 1 through Day 92

Title

Number Of Participants Needing New WAIHA Rescue Medication Or Increase In Dose Of WAIHA Medication

Time Frame

Day 15 through Day 92

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility 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

Facility Information:

Facility Name

Alexion Study Site

City

Whittier

State/Province

California

ZIP/Postal Code

90602

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

ALXN1830 in Patients With Warm Autoimmune Hemolytic Anemia

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