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

Roche

Status and phase: Terminated; Phase 2; Phase 1

Conditions: Muscular Dystrophy (DMD)

Treatments: Drug: Placebo; Drug: RO7239361

Study type: Interventional

Funder types: Industry

Identifiers

NCT02515669

CN001-006

WN40226 (Other Identifier)

Details and patient eligibility

About

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

Enrollment: 43 patients

Sex: Male

Ages: 5 to 10 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Basic Science

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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43 participants in 2 patient groups, including a placebo group

RO7239361

Active Comparator group

Description:

RO7239361 subcutaneous injections on specified days

Treatment:

Drug: RO7239361

Placebo

Placebo Comparator group

Description:

Placebo subcutaneous injections on specified days

Treatment:

Drug: Placebo