ALXN1830 in Patients With Warm Autoimmune Hemolytic Anemia

July 9, 2020 updated by: Alexion Pharmaceuticals

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

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).

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

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 Type

Interventional

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Whittier, California, United States, 90602
        • Alexion Study Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: Placebo
Other: Placebo
Matching placebo (sterile liquid diluent) administered via IV infusion
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

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Time Frame
Total Number Of Units Of Packed Red Blood Cells (pRBCs) Transfused
Time Frame: Day 15 through Day 92
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
Time Frame: Baseline, Day 92
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
Time Frame: Baseline, Day 92
Baseline, Day 92
Number Of Weekly Hgb Measurements With Change From Baseline ≥ 2 g/dL
Time Frame: Day 1 through Day 92
Day 1 through Day 92
Number Of Participants Needing New WAIHA Rescue Medication Or Increase In Dose Of WAIHA Medication
Time Frame: Day 15 through Day 92
Day 15 through Day 92

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alexion Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Anticipated)

July 1, 2021

Primary Completion (Anticipated)

October 30, 2022

Study Completion (Anticipated)

April 30, 2023

Study Registration Dates

First Submitted

January 27, 2020

First Submitted That Met QC Criteria

February 3, 2020

First Posted (Actual)

February 5, 2020

Study Record Updates

Last Update Posted (Actual)

July 13, 2020

Last Update Submitted That Met QC Criteria

July 9, 2020

Last Verified

July 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALXN1830-WAI-201

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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