Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD

NCT ID: NCT02515669

Last Updated: 2020-11-04

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

43 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-12-02

Study Completion Date

2020-04-15

Brief Summary

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The purpose of this study is to determine the safety and tolerability of RO7239361 in boys with Duchenne Muscular Dystrophy with any genetic mutation.

Detailed Description

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Conditions

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Muscular Dystrophy (DMD)

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

BASIC_SCIENCE

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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RO7239361

RO7239361 subcutaneous injections on specified days

Group Type ACTIVE_COMPARATOR

RO7239361

Intervention Type DRUG

Colorless to slightly yellow, clear to opalescent solution, essentially free of particulate matter packaged in a 1 cc glass syringe equipped with a safety syringe device.

Placebo

Placebo subcutaneous injections on specified days

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Colorless to slightly yellow, clear to opalescent solution, essentially free of particulate matter packaged in a 1 cc glass syringe equipped with a safety syringe device.

Interventions

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RO7239361

Colorless to slightly yellow, clear to opalescent solution, essentially free of particulate matter packaged in a 1 cc glass syringe equipped with a safety syringe device.

Intervention Type DRUG

Placebo

Colorless to slightly yellow, clear to opalescent solution, essentially free of particulate matter packaged in a 1 cc glass syringe equipped with a safety syringe device.

Intervention Type DRUG

Other Intervention Names

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Anti-Myostatin Adnectin taldefgrobep alfa

Eligibility Criteria

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Inclusion Criteria

* Diagnosed with DMD
* Able to walk without assistance
* Able to walk up 4 stairs in 8 seconds or less
* Weigh at least 15 kg
* Taking corticosteroids for DMD

Exclusion Criteria

* Ejection fraction \< 55% on echocardiogram, based on central read
* Any behavior or mental issue that will affect the ability to complete the required study procedures
* Previously or currently taking medications like androgens or human growth hormone
* Use of a ventilator during the day
* Unable to have blood samples collected or receive an injection under the skin
* Treatment with exon skipping therapies 6 months prior to study start
* Treatment with ataluren or any investigational drug currently or within 5 half-lives prior to study start
Minimum Eligible Age

5 Years

Maximum Eligible Age

10 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Hoffmann-La Roche

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Trials

Role: STUDY_DIRECTOR

Hoffmann-La Roche

Locations

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David Geffen School of Medicine at UCLA

Los Angeles, California, United States

Site Status

Stanford University

Palo Alto, California, United States

Site Status

University of Florida

Gainesville, Florida, United States

Site Status

Nemours Children's Hospital

Orlando, Florida, United States

Site Status

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Site Status

Rush University Medical Center

Chicago, Illinois, United States

Site Status

University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

Kennedy Krieger Institute

Baltimore, Maryland, United States

Site Status

Saint Louis Children's Hospital

St Louis, Missouri, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Alberta Children's Hospital

Calgary, Alberta, Canada

Site Status

Children'S Hospital of Eastern Ontario

Ottawa, Ontario, Canada

Site Status

Countries

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United States Canada

References

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Muntoni F, Byrne BJ, McMillan HJ, Ryan MM, Wong BL, Dukart J, Bansal A, Cosson V, Dreghici R, Guridi M, Rabbia M, Staunton H, Tirucherai GS, Yen K, Yuan X, Wagner KR; Taldefgrobep Alfa Study Group. The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy. Neurol Ther. 2024 Feb;13(1):183-219. doi: 10.1007/s40120-023-00570-w. Epub 2024 Jan 8.

Reference Type DERIVED
PMID: 38190001 (View on PubMed)

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Related Links

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Other Identifiers

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WN40226

Identifier Type: OTHER

Identifier Source: secondary_id

CN001-006

Identifier Type: -

Identifier Source: org_study_id