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Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT03039686
Recruitment Status : Recruiting
First Posted : February 1, 2017
Last Update Posted : November 16, 2018
Sponsor:
Information provided by (Responsible Party):
Hoffmann-La Roche

Study Description
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Brief Summary:
This is a multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of two different weekly doses of RO7239361 in ambulatory boys with Duchenne Muscular Dystrophy (DMD).

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: RO7239361 Drug: Placebo for RO7239361 Phase 2 Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 159 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
Actual Study Start Date : July 6, 2017
Estimated Primary Completion Date : December 30, 2020
Estimated Study Completion Date : December 17, 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: RO7239361, dose 1
Take RO7239361 subcutaneously on specified days over a 48 week blinded period
 Drug: RO7239361
Take RO7239361 subcutaneously on specified days over a 48 week blinded period
Experimental: RO7239361, dose 2
Take RO7239361 subcutaneously on specified days over a 48 week blinded period
 Drug: RO7239361
Take RO7239361 subcutaneously on specified days over a 48 week blinded period
Placebo Comparator: Placebo
Placebo solution taken subcutaneously on specified days over a 48 week blinded period
 Drug: Placebo for RO7239361
Take placebo subcutaneously on specified days over a 48 week blinded period


Outcome Measures
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Primary Outcome Measures :
  1. Change from baseline in the North Star Ambulatory Assessment (NSAA) total score in RO7239361 treated participants. [ Time Frame: 48 Week ]
  2. Change from baseline in the in the North Star Ambulatory Assessment (NSAA) total score in placebo treated participants. [ Time Frame: 48 Week ]

Secondary Outcome Measures :
  1. Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in the 4 Stair Climb Velocity (4SCV) Assessment [ Time Frame: 48 weeks ]
  2. Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in Stand from supine velocity [ Time Frame: 48 weeks ]
  3. Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in 10 M walk/run velocity [ Time Frame: 48 weeks ]
  4. Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in PODCI transfers and basic mobility subscale [ Time Frame: 48 weeks ]
  5. Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in Proximal lower extremity flexor (knee extension and knee flexion) strength, measured using manual myometry [ Time Frame: 48 weeks ]
  6. Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in 6 Minute Walk Distance (6MWD) [ Time Frame: 48 weeks ]
  7. Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in Clinical Global Impression of Change (CGI-C) rating. [ Time Frame: 48 weeks ]
  8. Change from baseline at Week 48 in RO7239361 treated participants compared to placebo treated participants in 95th percentile stride velocity [ Time Frame: 48 weeks ]
    As recorded with the ActiMyo device in a subset of the overall study population.


Eligibility Criteria
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Ages Eligible for Study:   6 Years to 11 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosed with DMD by confirmed medical history and genetic testing
  • 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:

  • 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
  • Concomitant or previous participation at any time in a gene therapy study

Other protocol defined Inclusion/Exclusion Criteria could apply.

Contacts and Locations
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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 ClinicalTrials.gov identifier (NCT number): NCT03039686


Contacts
Contact: Reference Study ID Number: WN40227 www.roche.com/about_roche/roche_worldwide.htm 888-662-6728 (U.S. and Canada) global-roche-genentech-trials@gene.com

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Locations
United States, Arizona
Neuromuscular Research Center Recruiting
Phoenix, Arizona, United States, 85028
United States, Arkansas
Arkansas Children's Hospital; Pediatrics Not yet recruiting
Little Rock, Arkansas, United States, 72202-3591
United States, California
David Geffen School of Medicine at UCLA; Clinical Trials Contract Unit Recruiting
Los Angeles, California, United States, 90095
Stanford University Recruiting
Palo Alto, California, United States, 94304
University of California Davis Medical Center Recruiting
Sacramento, California, United States, 95817
United States, Connecticut
Yale University School of Medicine ; Pulmonary & Critical Care Recruiting
New Haven, Connecticut, United States, 06510
United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States, 32607
Nemours Children's Hospital Recruiting
Orlando, Florida, United States, 32827
United States, Georgia
Rare Disease Research, LLC Recruiting
Atlanta, Georgia, United States, 30318
United States, Illinois
Rush University Medical Center - PPDS Recruiting
Chicago, Illinois, United States, 60612
United States, Iowa
University of Iowa Recruiting
Iowa City, Iowa, United States, 52242
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
United States, Maryland
Kennedy Krieger Institute Recruiting
Baltimore, Maryland, United States, 21205
United States, Massachusetts
University of Massachusetts Memorial Childrens Medical Center; Department of Neurology Not yet recruiting
Worcester, Massachusetts, United States, 01655
United States, Missouri
Saint Louis Children's Hospital Recruiting
Saint Louis, Missouri, United States, 63110
United States, Nevada
Las Vegas Clinic Recruiting
Las Vegas, Nevada, United States, 89145
United States, Ohio
Cincinnati Childrens Hospital Medical Center;Investigational Pharmacy Recruiting
Cincinnati, Ohio, United States, 45229
Nationwide Childrens Hospital; Research Institute at Nationwide Childrens Hospital Recruiting
Columbus, Ohio, United States, 43205
United States, Pennsylvania
Children'S Hospital of Philadelphia Not yet recruiting
Philadelphia, Pennsylvania, United States, 19104
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
Argentina
Instituto centenario Recruiting
Buenos Aires, Argentina, C1204AAD
Australia, New South Wales
Children's Hospital Westmead; Paediatrics & Child Health Recruiting
Westmead, New South Wales, Australia, 2145
Australia, Queensland
Lady Cilento Children's Hospital; Neurosciences Department Recruiting
South Brisbane, Queensland, Australia, 4101
Australia, Victoria
Royal Children's Hospital Recruiting
Parkville, Victoria, Australia, 3052
Belgium
UZ Gent Recruiting
Gent, Belgium, 9000
UZ Leuven; Neurology Recruiting
Leuven, Belgium, 3000
Canada, Alberta
Alberta Children's Hospital Recruiting
Calgary, Alberta, Canada, T3B 6A8
Canada, Ontario
McMaster University Health Sciences Center Withdrawn
Hamilton, Ontario, Canada, L8N 3Z5
London Health Sciences Centre; Children's Hospital; Pediatrics Recruiting
London, Ontario, Canada, N6A 5W9
Children'S Hospital of Eastern Ontario Recruiting
Ottawa, Ontario, Canada, K1H 8L1
France
Hospices Civils de Lyon Recruiting
Lyon, France, 69004
Hotel Dieu; Service Pharmacie Essais Cliniques Recruiting
Nantes, France, 44093
Hopital Armand Trousseau; centre reference Maladies Neuro-musculaires Est parisien Neuropediatrie Recruiting
Paris Cedex 12, France, 75571
Hopitaux Universitaires de Strasbourg Recruiting
Strasbourg, France, 67091
Germany
Universitatsklinikum Essen; Innere Klinik Recruiting
Essen, Germany, 45122
Italy
Fondazione Serena Onlus - CENTRO CLINICO NEMO Recruiting
Milano, Emilia-Romagna, Italy, 20162
Fondazione Policlinico Universitario A Gemelli; Servizio di Farmacia Recruiting
Rome, Lazio, Italy, 00168
Istituto G Gaslini Ospedale Pediatrico IRCCS Not yet recruiting
Genova, Liguria, Italy, 16147
Fondazione IRCCS Di Rilievo Nazionale Istituto Nazionale Neurologico Carlo Besta Not yet recruiting
Milano, Lombardia, Italy, 20133
Az. Osp. Universitaria Pol. G. Martino; Dip. Neuroscienze, Scienze Psichiatriche e Anest. Recruiting
Messina, Sicilia, Italy, 98125
Japan
Hyogo College of Medicine Hospital Recruiting
Hyogo, Japan, 663-8501
Miyagi Children's Hospital Recruiting
Miyagi, Japan, 989-3126
Shinshu University Hospital Recruiting
Nagano, Japan, 390-8621
National Hospital Organization Toneyama National Hospital Recruiting
Osaka, Japan, 560-8552
National Center of Neurology and Psychiatry Recruiting
Tokyo, Japan, 187-8551
Netherlands
Leids Universitair Medisch Centrum Recruiting
Leiden, Netherlands, 2300 RC
Radboud University Nijmegen Medical Centre; Ophthalmology Not yet recruiting
Nijmegen, Netherlands, 6525 EX
Spain
Hospital Sant Joan De Deu Recruiting
Esplugues De Llobregas, Barcelona, Spain, 08950
Hospital Universitario Vall d'Hebron - PPDS Not yet recruiting
Barcelona, Spain, 08035
Hospital Universitari i Politecnic La Fe de Valencia; Servicio de Farmacia Not yet recruiting
Valencia, Spain, 46026
Sweden
Drottning Silvias Barn- och ungdomssjukhus; Kliniken for barnmedicin Recruiting
Goeteborg, Sweden, 41685
United Kingdom
Alder Hey Children s Hospital; Department of Pediatrics Recruiting
Liverpool, United Kingdom, L12 2AP
UCL Institute of Child Health & Great Ormond Street Hospital for Children Recruiting
London, United Kingdom, WC1N 1EH
Sponsors and Collaborators
Hoffmann-La Roche
Investigators
Study Director: Clinical Trials Hoffmann-La Roche
More Information
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Additional Information:
FDA Safety Alerts and Recalls  This link exits the ClinicalTrials.gov site Provides information about the Roche clinical trial NCT03039686 and molecule being investigated in Duchenne  This link exits the ClinicalTrials.gov site

Responsible Party: Hoffmann-La Roche
ClinicalTrials.gov Identifier: NCT03039686     History of Changes
Other Study ID Numbers: CN001-016
2016-001654-18 ( EudraCT Number )
WN40227 ( Other Identifier: Hoffman-La Roche )
First Posted: February 1, 2017    Key Record Dates
Last Update Posted: November 16, 2018
Last Verified: November 2018

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

Keywords provided by Hoffmann-La Roche:
muscular dystrophy
Duchenne's Muscular Dystrophy
DMD

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
 Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked