Clinical trial NCT02515669: Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD
Official 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
PHASE2
Conditions: Muscular Dystrophy (DMD)
Lead sponsor: Hoffmann-La Roche
Sponsor class: INDUSTRY
First posted: 2015-08-05
Enrollment: 43
Sex: MALE
Minimum age (years): 5
Maximum age (years): 10
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: BASIC_SCIENCE

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


Eligibility 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

Primary outcomes:
measure: Safety Summary for the 24 Week Double-Blind Phase
timeFrame: Baseline to Week 24
measure: Safety Summary up to Week 72
timeFrame: Baseline to Week 72
Secondary outcomes:
measure: Maximum Observed Serum Concentrations (Cmax) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW Doses.
timeFrame: 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
measure: Maximum Observed Serum Concentrations (Cmax) of RO7239361 at Steady State for 50 mg QW Dose.
timeFrame: 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
measure: 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.
timeFrame: 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
measure: Time of Maximum Observed Serum Concentrations (Tmax) of RO7239361 at Steady State for 50 mg QW Dose.
timeFrame: 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
measure: 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.
timeFrame: 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
measure: 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.
timeFrame: 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
measure: RO7239361 Trough Concentrations
timeFrame: 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
measure: Percentage of Participants With Positive Anti-RO7239361 Antibodies (ADA) Assessment, Double-Blind Phase
timeFrame: Day 8 through Week 24, baseline and on-study information represented in table.
measure: Percentage of Participants With Positive Anti-RO7239361 Antibodies (ADA) Assessment up to Week 72
timeFrame: Day 8 through Week 72, baseline and on-study information represented in table.
measure: Serum Concentration of Free Myostatin in the Double-Blind Phase
timeFrame: Baseline through Week 24
measure: Percent Inhibition of Free Myostatin in the Double-Blind Phase
timeFrame: Baseline through Week 24
measure: Serum Concentration of Drug-Myostatin Complex in the Double-Blind Phase
timeFrame: Baseline through Week 24
measure: Fold Change From Baseline in Contractile Versus Non-contractile Content for Muscles in the Right Thigh in the Double-Blind Phase
timeFrame: Baseline through Week 24
measure: Change From Baseline in Thigh Muscle Maximal Cross Sectional Area (CSAmax) in the Double-Blind Phase
timeFrame: Baseline through Week 24
measure: Percentage of Participants With Positive Anti-RO7239361 Antibodies (ADA) Assessment, Whole Study
timeFrame: Day 8 through Week 228, baseline and on-study information represented in table.
measure: Serum Concentration of Free Myostatin, Whole Study
timeFrame: Baseline through Week 252
measure: Percent Inhibition of Free Myostatin, Whole Study
timeFrame: Baseline through Week 252
measure: Serum Concentration of Drug-Myostatin Complex, Whole Study
timeFrame: Baseline through Week 252
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: David Geffen School of Medicine at UCLA
state: California
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Orlando
country: United States
facility: Nemours Children's Hospital
state: Florida
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Baltimore
country: United States
facility: Kennedy Krieger Institute
state: Maryland
city: Saint Louis
country: United States
facility: Saint Louis Children's Hospital
state: Missouri
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Calgary
country: Canada
facility: Alberta Children's Hospital
state: Alberta
city: Ottawa
country: Canada
facility: Children'S Hospital of Eastern Ontario
state: Ontario
Study officials:
affiliation: Hoffmann-La Roche
name: Clinical Trials
role: STUDY_DIRECTOR
References: