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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02515669
• Date of registration: 29/07/2015
• Prospective Registration: Yes
• Primary sponsor: Hoffmann-La Roche
• Public title: Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD
• Scientific title: A Multi-Site, Randomized, Placebo-Controlled, Double-Blind, Multiple Ascending Subcutaneous Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of RO7239361 (BMS-986089) in Ambulatory Boys With Duchenne Muscular Dystrophy
• Date of first enrolment: December 2, 2015
• Target sample size: 43
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT02515669
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Basic Science. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 1/Phase 2

Countries of recruitment

Canada, United States

Contacts

• Name: Clinical Trials
• Affiliation: Hoffmann-La Roche

Key inclusion & exclusion criteria

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



Age minimum: 5 Years
Age maximum: 10 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Muscular Dystrophy (DMD)

Intervention(s)

Drug: Placebo

Drug: RO7239361

Primary Outcome(s)

Safety Summary for the 24 Week Double-Blind Phase [Time Frame: Baseline to Week 24]

Safety Summary up to Week 72 [Time Frame: Baseline to Week 72]

Secondary Outcome(s)

Area Under the Concentration-Time Curve From Time Zero to Time of Next Dosing (AUCtau) of RO7239361 at Steady State for 50 mg QW Dose. [Time Frame: Day 1: predose, 3, 6, 72 and 96 h postdose; Days 8, 15 and 22: predose; Day 29: predose and 96 h postdose; Weeks 12 and 24: predose]

Fold Change From Baseline in Contractile Versus Non-contractile Content for Muscles in the Right Thigh in the Double-Blind Phase [Time Frame: Baseline through Week 24]

Percentage of Participants With Positive Anti-RO7239361 Antibodies (ADA) Assessment, Double-Blind Phase [Time Frame: Day 8 through Week 24, baseline and on-study information represented in table.]

RO7239361 Trough Concentrations [Time Frame: Day 1: predose, 3, 6, 72 and 96 h postdose; Days 8, 15 and 22: predose; Day 29: predose and 96 h postdose; Weeks 12 and 24: predose]

Maximum Observed Serum Concentrations (Cmax) of RO7239361 at Steady State for 50 mg QW Dose. [Time Frame: Day 1: predose, 3, 6, 72 and 96 h postdose; Days 8, 15 and 22: predose; Day 29: predose and 96 h postdose; Weeks 12 and 24: predose]

Time of Maximum Observed Serum Concentrations (Tmax) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW Doses. [Time Frame: Day 1: predose, 3, 6, 72 and 96 h postdose; Days 8, 15 and 22: predose; Day 29: predose and 96 h postdose; Weeks 12 and 24: predose]

Area Under the Concentration-Time Curve From Time Zero to Time of Next Dosing (AUCtau) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW Doses. [Time Frame: Day 1: predose, 3, 6, 72 and 96 h postdose; Days 8, 15 and 22: predose; Day 29: predose and 96 h postdose; Weeks 12 and 24: predose]

Percent Inhibition of Free Myostatin in the Double-Blind Phase [Time Frame: Baseline through Week 24]

Percent Inhibition of Free Myostatin, Whole Study [Time Frame: Baseline through Week 252]

Percentage of Participants With Positive Anti-RO7239361 Antibodies (ADA) Assessment up to Week 72 [Time Frame: Day 8 through Week 72, baseline and on-study information represented in table.]

Time of Maximum Observed Serum Concentrations (Tmax) of RO7239361 at Steady State for 50 mg QW Dose. [Time Frame: Day 1: predose, 3, 6, 72 and 96 h postdose; Days 8, 15 and 22: predose; Day 29: predose and 96 h postdose; Weeks 12 and 24: predose]

Change From Baseline in Thigh Muscle Maximal Cross Sectional Area (CSAmax) in the Double-Blind Phase [Time Frame: Baseline through Week 24]

Percentage of Participants With Positive Anti-RO7239361 Antibodies (ADA) Assessment, Whole Study [Time Frame: Day 8 through Week 228, baseline and on-study information represented in table.]

Serum Concentration of Drug-Myostatin Complex in the Double-Blind Phase [Time Frame: Baseline through Week 24]

Serum Concentration of Drug-Myostatin Complex, Whole Study [Time Frame: Baseline through Week 252]

Maximum Observed Serum Concentrations (Cmax) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW Doses. [Time Frame: Day 1: predose, 3, 6, 72 and 96 hours (h) postdose; Days 8, 15 and 22: predose; Day 29: predose and 96 h postdose; Weeks 12 and 24: predose]

Serum Concentration of Free Myostatin in the Double-Blind Phase [Time Frame: Baseline through Week 24]

Serum Concentration of Free Myostatin, Whole Study [Time Frame: Baseline through Week 252]

Secondary ID(s)

WN40226

CN001-006

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 08/05/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02515669