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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche
ClinicalTrials.gov Identifier:
NCT02515669
First received: July 29, 2015
Last updated: August 2, 2017
Last verified: August 2017
  Purpose
The purpose of this study is to determine the safety and tolerability of BMS-986089 in boys with Duchenne Muscular Dystrophy with any genetic mutation.

Condition Intervention Phase
Muscular Dystrophy (DMD) Biological: BMS-986089 Biological: Placebo Phase 1 Phase 2

Study Type: Interventional
Study Design: 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 BMS-986089 in Ambulatory Boys With Duchenne Muscular Dystrophy

Resource links provided by NLM:


Further study details as provided by Hoffmann-La Roche:

Primary Outcome Measures:
  • 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)

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

Secondary Outcome Measures:
  • Trough concentrations of BMS-986089 [ Time Frame: Day 8 through Week 24 ]
  • Maximum observed concentration (Cmax) of BMS-986089 [ Time Frame: Day 1 and 29 ]
  • Frequency of subjects with positive anti-BMS-986089 antibodies (ADA) assessment [ Time Frame: Day 8 through Week 24 ]
  • Frequency of subjects who develop positive ADA following a negative baseline [ Time Frame: Day 8 through Week 24 ]
  • Anti-BMS-986089 antibodies on selected days [ Time Frame: Day 8 through Week 24 ]
  • Serum concentration of free myostatin [ Time Frame: Day 8 through Week 24 ]
  • Change from baseline in volume of thigh muscle contractile and non-contractile tissue [ Time Frame: Day 8 through Week 24 ]
  • Change from baseline in thigh muscle maximal cross sectional area [ Time Frame: Day 8 through Week 24 ]
  • Serum concentration of drug-myostatin complex [ Time Frame: Day 8 through Week 24 ]
  • Serum concentration of percent inhibition of free myostatin at trough [ Time Frame: Day 8 through Week 24 ]

Estimated Enrollment: 40
Actual Study Start Date: December 2, 2015
Estimated Study Completion Date: August 4, 2020
Estimated Primary Completion Date: August 3, 2020 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: BMS-986089
BMS-986089 subcutaneous injections on specified days
Biological: BMS-986089
Other Name: Anti-Myostatin Adnectin
Placebo Comparator: Placebo
Placebo subcutaneous injections on specified days
Biological: Placebo

  Eligibility

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

For more information regarding BMS clinical trial participation, please visit www.BMSStudyConnect.com

Inclusion Criteria:

  • Diagnosed with DMD by a blood test
  • 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:

  • 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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02515669

Locations
United States, California
Ucla Medical Center
Los Angeles, California, United States, 90095
Stanford University Medical Center
Palo Alto, California, United States, 94304
United States, District of Columbia
Childrens National Medical Center
Washington, D.C., District of Columbia, United States, 20010
United States, Florida
University of Florida Board of Trustees
Gainesville, Florida, United States, 32610
Nemours Childrens Hospital
Orlando, Florida, United States, 32827
United States, Georgia
Childrens 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
Hugo W Moser Research Institute at Kennedy Krieger inc
Baltimore, Maryland, United States, 21205
United States, Missouri
Washington University School Of Medicine
Saint Louis, Missouri, United States, 63110
United States, Ohio
Cincinnati Childrens Hospital Medical Center
Cincinnati, Ohio, United States, 45229
United States, Pennsylvania
Children Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15224
Canada, Alberta
Local Institution
Calgary, Alberta, Canada, T3B 6A8
Canada, Ontario
Local Institution
Ottawa, Ontario, Canada, K1H 8L1
Sponsors and Collaborators
Hoffmann-La Roche
Investigators
Study Director: Clinical Trials Hoffmann-La Roche
  More Information

Additional Information:
Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT02515669     History of Changes
Other Study ID Numbers: CN001-006
WN40226 ( Other Identifier: Hoffman-La Roche )
Study First Received: July 29, 2015
Last Updated: August 2, 2017

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

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

ClinicalTrials.gov processed this record on August 18, 2017