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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02515669
Recruitment Status : Active, not recruiting
First Posted : August 5, 2015
Last Update Posted : November 5, 2018
Information provided by (Responsible Party):
Hoffmann-La Roche

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.

Condition or disease Intervention/treatment Phase
Muscular Dystrophy (DMD) Drug: RO7239361 Drug: Placebo Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 43 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Basic Science
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
Actual Study Start Date : December 2, 2015
Actual Primary Completion Date : February 8, 2018
Estimated Study Completion Date : June 29, 2022

Arm Intervention/treatment
Active Comparator: RO7239361
RO7239361 subcutaneous injections on specified days
Drug: RO7239361
Other Name: Anti-Myostatin Adnectin

Placebo Comparator: Placebo
Placebo subcutaneous injections on specified days
Drug: Placebo

Primary Outcome Measures :
  1. Safety and Tolerability based on number of incidences of AEs, serious AEs, AEs leading to discontinuation and death, as well as marked treatment emergent abnormalities in clinical laboratory tests [ Time Frame: 24 weeks ]
    Adverse event (AEs)

  2. Safety and Tolerability based on number of incidences of vital sign measurements, ECGs, echocardiograms, and physical examinations [ Time Frame: 24 weeks ]

Secondary Outcome Measures :
  1. Trough concentrations of RO7239361 [ Time Frame: At pre-defined intervals from day 8 through week 24 ]
  2. Maximum observed concentration (Cmax) of RO7239361 [ Time Frame: Day 1 and 29 ]
  3. Frequency of subjects with positive anti-RO7239361 antibodies (ADA) assessment [ Time Frame: Day 8 through Week 24 ]
  4. Frequency of subjects who develop positive ADA following a negative baseline [ Time Frame: Day 8 through Week 24 ]
  5. Anti-RO7239361 antibodies on selected days [ Time Frame: Day 8 through Week 24 ]
  6. Serum concentration of free myostatin [ Time Frame: Baseline through week 24 ]
  7. Change from baseline in volume of thigh muscle contractile and non-contractile tissue [ Time Frame: Day 8 through Week 24 ]
  8. Change from baseline in thigh muscle maximal cross sectional area [ Time Frame: Day 8 through Week 24 ]
  9. Serum concentration of drug-myostatin complex [ Time Frame: Baseline through week 24 ]
  10. Serum concentration of percent inhibition of free myostatin at trough [ Time Frame: At pre-defined intervals from day 8 through week 24 ]

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 10 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02515669

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United States, California
David Geffen School of Medicine at UCLA
Los Angeles, California, United States, 90095-6984
Stanford University
Palo Alto, California, United States, 94305
United States, Florida
University of Florida
Gainesville, Florida, United States, 32607
Nemours Children's Hospital
Orlando, Florida, United States, 32827
United States, Georgia
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30324
United States, Illinois
Rush University Medical Center
Chicago, Illinois, United States, 60612
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
United States, Maryland
Kennedy Krieger Institute
Baltimore, Maryland, United States, 21205
United States, Missouri
Saint Louis Children's Hospital
Saint Louis, Missouri, United States, 63110
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Canada, Alberta
Alberta Children's Hospital
Calgary, Alberta, Canada, T3B 6A8
Canada, Ontario
Children'S Hospital of Eastern Ontario
Ottawa, Ontario, Canada, K1H 8L1
Sponsors and Collaborators
Hoffmann-La Roche
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Study Director: Clinical Trials Hoffmann-La Roche

Additional Information:
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Responsible Party: Hoffmann-La Roche Identifier: NCT02515669     History of Changes
Other Study ID Numbers: CN001-006
WN40226 ( Other Identifier: Hoffman-La Roche )
First Posted: August 5, 2015    Key Record Dates
Last Update Posted: November 5, 2018
Last Verified: November 2018

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn