Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

Roche

Status and phase: Completed; Phase 3; Phase 2

Conditions: Duchenne Muscular Dystrophy

Treatments: Drug: RO7239361; Drug: Placebo for RO7239361

Study type: Interventional

Funder types: Industry

Identifiers

NCT03039686

2016-001654-18 (EudraCT Number)

WN40227 (Other Identifier)

CN001-016

Details and patient eligibility

About

This is a multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of two different weekly doses of RO7239361 in ambulatory boys with Duchenne Muscular Dystrophy (DMD).

Enrollment: 166 patients

Sex: Male

Ages: 6 to 11 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Diagnosed with DMD by confirmed medical history and genetic testing
• Able to walk without assistance
• Minimum North Star Ambulatory Assessment score of 15 at screening
• Able to walk up 4 stairs in 8 seconds or less
• Weigh at least 15 kg (33 lbs)
• Taking corticosteroids for DMD

Exclusion criteria

• 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
• Concomitant or previous participation at any time in a gene therapy study

Other protocol defined Inclusion/Exclusion Criteria could apply.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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166 participants in 3 patient groups, including a placebo group

RO7239361 Low Dose

Experimental group

Description:

Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.

Treatment:

Drug: RO7239361

RO7239361 High Dose

Experimental group

Description:

Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.

Treatment:

Drug: RO7239361

Placebo

Placebo Comparator group

Description:

Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.

Treatment:

Drug: Placebo for RO7239361