Subcutaneous ALXN1830 in Adult Participants With Warm Autoimmune Hemolytic Anemia

NCT ID: NCT04956276

Last Updated: 2022-02-11

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

PHASE2

Study Classification

INTERVENTIONAL

Study Start Date

2022-01-01

Study Completion Date

2024-07-31

Brief Summary

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This is a Phase 2, multiple ascending, dose-finding, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, health-related quality of life, tolerability, pharmacokinetic, pharmacodynamic, and immunogenicity, of up to 3 dose regimens of ALXN1830 administered subcutaneous(ly) (SC) in the treatment of WAIHA.

This study will include 2 randomized, double-blind, placebo-controlled cohorts (Cohorts 1 and 2) to evaluate an 8-week treatment regimen, and an optional third open-label cohort (Cohort 3) to evaluate an alternative 12-week dosing regimen. Participants may continue participation in this study at the participant's and investigator's discretion in an open-label extension (OLE) period, consisting of monthly visits to observe participants for relapse, which will require going back on active treatment.

Detailed Description

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Conditions

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Warm Autoimmune Hemolytic Anemia

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators
Cohorts 1 and 2 will be participant and investigator blinded, Cohort 3 will be open label (if initiated).

Study Groups

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Cohort 1: ALXN1830/Placebo

Participants will be randomized 3:1 to receive ALXN1830 or placebo. Treatment will be received for 8 weeks followed by a follow-up period (no treatment) for 8 weeks. Once complete, participants may continue participation in the study at the participant's and investigator's discretion during the OLE period for up to 2 years inclusive of primary treatment period.

Group Type EXPERIMENTAL

ALXN1830

Intervention Type DRUG

Administered as an SC infusion.

Placebo

Intervention Type DRUG

Administered as an SC infusion.

Cohort 2: ALXN1830/Placebo

Participants will be randomized 3:1 to receive ALXN1830 or placebo. Treatment will be received for 8 weeks followed by a follow-up period (no treatment) for 8 weeks. Once complete, participants may continue participation in the study at the participant's and investigator's discretion during the OLE period for up to 2 years inclusive of primary treatment period.

Group Type EXPERIMENTAL

ALXN1830

Intervention Type DRUG

Administered as an SC infusion.

Placebo

Intervention Type DRUG

Administered as an SC infusion.

Cohort 3: ALXN1830

If initiated, participants will receive ALXN1830. Treatment will be received for 12 weeks followed by a follow-up period (no treatment) for 8 weeks. Once complete, participants may continue participation in the study at the participant's and investigator's discretion during the OLE period for up to 2 years inclusive of primary treatment period.

Group Type EXPERIMENTAL

ALXN1830

Intervention Type DRUG

Administered as an SC infusion.

Interventions

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ALXN1830

Administered as an SC infusion.

Intervention Type DRUG

Placebo

Administered as an SC infusion.

Intervention Type DRUG

Other Intervention Names

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SYNT001 (formerly)

Eligibility Criteria

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Inclusion Criteria

* Diagnosed with primary or secondary WAIHA at least 6 weeks prior to Screening.
* Failed or have not tolerated at least one prior WAIHA treatment regimen, for example, corticosteroids, rituximab, azathioprine, cyclophosphamide, cyclosporine, mycophenolate mofetil, danazol, or vincristine.
* Hemoglobin \< 10 g/dL and ≥ 6 g/dL at Screening.
* Positive direct antiglobulin test (Coombs) (IgG positive who are positive or negative for the presence of complement C3) at Screening.
* Evidence of active hemolysis including any one of the below:

* LDH \> upper limit of normal (ULN) or
* Haptoglobin \< lower limit of normal or
* Indirect bilirubin \> ULN
* Total IgG \> 500 mg/dL at Screening
* Platelet count ≥ 75 x 10\^9/liter (L)
* Absolute neutrophil count greater than 1.0 x 10\^9/L

Exclusion Criteria

* Participants with Evan's syndrome.
* Human immunodeficiency virus (HIV) infection (positive HIV 1 or HIV 2 antibody test).
* Positive hepatitis B surface antigen or hepatitis C antibody test.
* Inability to travel to the clinic for specified visits during the Primary Treatment Period or fulfill the logistical requirements of study intervention administration.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Alexion Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Clinical Study Site

Riverside, California, United States

Site Status

Countries

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United States

Other Identifiers

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ALXN1830-WAI-202

Identifier Type: -

Identifier Source: org_study_id

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