Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
NCT ID: NCT03039686
Last Updated: 2020-12-21
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2/PHASE3
166 participants
INTERVENTIONAL
2017-07-06
2020-04-28
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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RO7239361 Low Dose
Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
RO7239361
Take RO7239361 subcutaneously on specified days over a 48 week blinded period
RO7239361 High Dose
Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
RO7239361
Take RO7239361 subcutaneously on specified days over a 48 week blinded period
Placebo
Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.
Placebo for RO7239361
Take placebo subcutaneously on specified days over a 48 week blinded period
Interventions
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RO7239361
Take RO7239361 subcutaneously on specified days over a 48 week blinded period
Placebo for RO7239361
Take placebo subcutaneously on specified days over a 48 week blinded period
Eligibility Criteria
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Inclusion Criteria
* Able to walk without assistance
* Minimum North Star Ambulatory Assessment score of 15 at screening
* Able to walk up 4 stairs in 8 seconds or less
* Weigh at least 15 kg (33 lbs)
* Taking corticosteroids for DMD
Exclusion Criteria
* 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
* Concomitant or previous participation at any time in a gene therapy study
6 Years
11 Years
MALE
No
Sponsors
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Hoffmann-La Roche
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Trials
Role: STUDY_DIRECTOR
Hoffmann-La Roche
Locations
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Neuromuscular Research Center
Phoenix, Arizona, United States
Stanford University
Palo Alto, California, United States
University of California Davis Medical Center
Sacramento, California, United States
Yale University School of Medicine ; Pulmonary & Critical Care
New Haven, Connecticut, United States
University of Florida
Gainesville, Florida, United States
Nemours Children's Hospital
Orlando, Florida, United States
Rare Disease Research, LLC
Atlanta, Georgia, United States
Rush University Medical Center - PPDS
Chicago, Illinois, United States
University of Iowa
Iowa City, Iowa, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Kennedy Krieger Institute
Baltimore, Maryland, United States
University of Massachusetts Memorial Childrens Medical Center; Department of Neurology
Worcester, Massachusetts, United States
Saint Louis Children's Hospital
St Louis, Missouri, United States
Las Vegas Clinic
Las Vegas, Nevada, United States
Cincinnati Childrens Hospital Medical Center;Investigational Pharmacy
Cincinnati, Ohio, United States
Nationwide Childrens Hospital; Research Institute at Nationwide Childrens Hospital
Columbus, Ohio, United States
Seattle Children's Hospital
Seattle, Washington, United States
Instituto centenario
Buenos Aires, , Argentina
Children's Hospital Westmead; Paediatrics & Child Health
Westmead, New South Wales, Australia
Lady Cilento Children's Hospital; Neurosciences Department
South Brisbane, Queensland, Australia
Royal Children's Hospital
Parkville, Victoria, Australia
UZ Gent
Ghent, , Belgium
London Health Sciences Centre; Children's Hospital; Pediatrics
London, Ontario, Canada
Children'S Hospital of Eastern Ontario
Ottawa, Ontario, Canada
Hospices Civils de Lyon
Lyon, , France
Hotel Dieu; Service Pharmacie Essais Cliniques
Nantes, , France
Hopital Armand Trousseau; centre reference Maladies Neuro-musculaires Est parisien Neuropediatrie
Paris, , France
Hopitaux Universitaires de Strasbourg
Strasbourg, , France
Universitatsklinikum Essen; Innere Klinik
Essen, , Germany
Fondazione Serena Onlus - CENTRO CLINICO NEMO
Milano, Emilia-Romagna, Italy
Fondazione Policlinico Universitario A Gemelli; Servizio di Farmacia
Rome, Lazio, Italy
Az. Osp. Universitaria Pol. G. Martino; Dip. Neuroscienze, Scienze Psichiatriche e Anest.
Messina, Sicily, Italy
Hyogo College of Medicine Hospital
Hyōgo, , Japan
Miyagi Children's Hospital
Miyagi, , Japan
Shinshu University Hospital
Nagano, , Japan
National Hospital Organization Osaka Toneyama Medical Center
Osaka, , Japan
National Center of Neurology and Psychiatry
Tokyo, , Japan
Leids Universitair Medisch Centrum
Leiden, , Netherlands
Radboud University Nijmegen Medical Centre; Ophthalmology
Nijmegen, , Netherlands
Hospital Sant Joan De Deu
Esplugues de Llobregas, Barcelona, Spain
Hospital Universitari i Politecnic La Fe de Valencia; Servicio de Farmacia
Valencia, , Spain
Drottning Silvias Barn- och ungdomssjukhus; Kliniken for barnmedicin
Gothenburg, , Sweden
Alder Hey Children s Hospital; Department of Pediatrics
Liverpool, , United Kingdom
UCL Institute of Child Health & Great Ormond Street Hospital for Children
London, , United Kingdom
Countries
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References
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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.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Related Links
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FDA Safety Alerts and Recalls
Provides information about the Roche clinical trial NCT03039686 and molecule being investigated in Duchenne
Other Identifiers
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2016-001654-18
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
WN40227
Identifier Type: OTHER
Identifier Source: secondary_id
CN001-016
Identifier Type: -
Identifier Source: org_study_id