Study Stopped
A pre-planned futility analysis indicated lack of efficacy in study NCT03039686 and led to discontinuation of both ongoing studies in DMD.
Study of an Investigational Drug, RO7239361 (BMS-986089), in Ambulatory Boys With DMD
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
2 other identifiers
interventional
43
2 countries
12
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_1
Started Dec 2015
Longer than P75 for phase_1
12 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 29, 2015
CompletedFirst Posted
Study publicly available on registry
August 5, 2015
CompletedStudy Start
First participant enrolled
December 2, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
February 8, 2018
CompletedResults Posted
Study results publicly available
May 8, 2019
CompletedStudy Completion
Last participant's last visit for all outcomes
April 15, 2020
CompletedNovember 4, 2020
October 1, 2020
2.2 years
July 29, 2015
February 7, 2019
October 13, 2020
Conditions
Outcome Measures
Primary Outcomes (2)
Safety Summary for the 24 Week Double-Blind Phase
Percentage of participants with fatalities, adverse event (AEs) and serious adverse events (SAEs) up to Week 24. Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight \>45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight \>45 kg) RO7239361.
Baseline to Week 24
Safety Summary up to Week 72
Percentage of participants with fatalities, adverse event (AEs) and serious adverse events (SAEs) up to Week 72. Participants in Panel 1 received 4 mg RO7239361; participants in Panel 2 received either 12.5 mg (weight between 15 and 45 kg) or 20 mg (weight \>45 kg) RO7239361, and participants in Panel 3 and the Expansion Panel received either 35 mg (weight between 15 and 45 kg) or 50 mg (weight \>45 kg) RO7239361.
Baseline to Week 72
Secondary Outcomes (18)
Maximum Observed Serum Concentrations (Cmax) of RO7239361 at Steady State for 4 mg QW, 12.5 mg QW and 35 mg QW Doses.
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
Maximum Observed Serum Concentrations (Cmax) of RO7239361 at Steady State for 50 mg QW Dose.
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.
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 50 mg QW Dose.
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.
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
- +13 more secondary outcomes
Study Arms (2)
RO7239361
ACTIVE COMPARATORRO7239361 subcutaneous injections on specified days
Placebo
PLACEBO COMPARATORPlacebo subcutaneous injections on specified days
Interventions
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.
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.
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (12)
David Geffen School of Medicine at UCLA
Los Angeles, California, 90095-6984, United States
Stanford University
Palo Alto, California, 94305, United States
University of Florida
Gainesville, Florida, 32607, United States
Nemours Children's Hospital
Orlando, Florida, 32827, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, 30324, United States
Rush University Medical Center
Chicago, Illinois, 60612, United States
University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
Saint Louis Children's Hospital
St Louis, Missouri, 63110, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Alberta Children's Hospital
Calgary, Alberta, T3B 6A8, Canada
Children'S Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1, Canada
Related Publications (1)
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.
PMID: 38190001DERIVED
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Communications
- Organization
- Hoffmann- LaRoche
Study Officials
- STUDY DIRECTOR
Clinical Trials
Hoffmann-La Roche
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- BASIC SCIENCE
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 29, 2015
First Posted
August 5, 2015
Study Start
December 2, 2015
Primary Completion
February 8, 2018
Study Completion
April 15, 2020
Last Updated
November 4, 2020
Results First Posted
May 8, 2019
Record last verified: 2020-10