Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
3 other identifiers
interventional
166
13 countries
44
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Jul 2017
Typical duration for phase_2
44 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
January 27, 2017
CompletedFirst Posted
Study publicly available on registry
February 1, 2017
CompletedStudy Start
First participant enrolled
July 6, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 28, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
April 28, 2020
CompletedResults Posted
Study results publicly available
December 21, 2020
CompletedDecember 21, 2020
November 1, 2020
2.8 years
January 27, 2017
October 21, 2020
November 25, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Baseline for the North Star Ambulatory Assessment (NSAA) Total Score
The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance.
Baseline
Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48
The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
Baseline, Week 48
Secondary Outcomes (15)
Baseline Time for 4 Stair Climb
Baseline
Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV)
Baseline, Week 48
Baseline for the Time to Stand From Supine
Baseline
Change From Baseline at Week 48 in Stand From Supine Velocity
Baseline, Week 48
Baseline Time for 10 Meter Walk/Run
Baseline
- +10 more secondary outcomes
Study Arms (3)
RO7239361 Low Dose
EXPERIMENTALParticipants 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 High Dose
EXPERIMENTALParticipants 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.
Placebo
PLACEBO COMPARATORParticipants 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.
Interventions
Take RO7239361 subcutaneously on specified days over a 48 week blinded period
Take placebo subcutaneously on specified days over a 48 week blinded period
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (44)
Neuromuscular Research Center
Phoenix, Arizona, 85028, United States
Stanford University
Palo Alto, California, 94304, United States
University of California Davis Medical Center
Sacramento, California, 95817, United States
Yale University School of Medicine ; Pulmonary & Critical Care
New Haven, Connecticut, 06510, United States
University of Florida
Gainesville, Florida, 32607, United States
Nemours Children's Hospital
Orlando, Florida, 32827, United States
Rare Disease Research, LLC
Atlanta, Georgia, 30318, United States
Rush University Medical Center - PPDS
Chicago, Illinois, 60612, United States
University of Iowa
Iowa City, Iowa, 52242, United States
University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
University of Massachusetts Memorial Childrens Medical Center; Department of Neurology
Worcester, Massachusetts, 01655, United States
Saint Louis Children's Hospital
St Louis, Missouri, 63110, United States
Las Vegas Clinic
Las Vegas, Nevada, 89145, United States
Cincinnati Childrens Hospital Medical Center;Investigational Pharmacy
Cincinnati, Ohio, 45229, United States
Nationwide Childrens Hospital; Research Institute at Nationwide Childrens Hospital
Columbus, Ohio, 43205, United States
Seattle Children's Hospital
Seattle, Washington, 98105, United States
Instituto centenario
Buenos Aires, C1204AAD, Argentina
Children's Hospital Westmead; Paediatrics & Child Health
Westmead, New South Wales, 2145, Australia
Lady Cilento Children's Hospital; Neurosciences Department
South Brisbane, Queensland, 4101, Australia
Royal Children's Hospital
Parkville, Victoria, 3052, Australia
UZ Gent
Ghent, 9000, Belgium
London Health Sciences Centre; Children's Hospital; Pediatrics
London, Ontario, N6A 5W9, Canada
Children'S Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1, Canada
Hospices Civils de Lyon
Lyon, 69004, France
Hotel Dieu; Service Pharmacie Essais Cliniques
Nantes, 44093, France
Hopital Armand Trousseau; centre reference Maladies Neuro-musculaires Est parisien Neuropediatrie
Paris, 75571, France
Hopitaux Universitaires de Strasbourg
Strasbourg, 67091, France
Universitatsklinikum Essen; Innere Klinik
Essen, 45122, Germany
Fondazione Serena Onlus - CENTRO CLINICO NEMO
Milano, Emilia-Romagna, 20162, Italy
Fondazione Policlinico Universitario A Gemelli; Servizio di Farmacia
Rome, Lazio, 00168, Italy
Az. Osp. Universitaria Pol. G. Martino; Dip. Neuroscienze, Scienze Psichiatriche e Anest.
Messina, Sicily, 98125, Italy
Hyogo College of Medicine Hospital
Hyōgo, 663-8501, Japan
Miyagi Children's Hospital
Miyagi, 989-3126, Japan
Shinshu University Hospital
Nagano, 390-8621, Japan
National Hospital Organization Osaka Toneyama Medical Center
Osaka, 560-8552, Japan
National Center of Neurology and Psychiatry
Tokyo, 187-8551, Japan
Leids Universitair Medisch Centrum
Leiden, 2333 ZA, Netherlands
Radboud University Nijmegen Medical Centre; Ophthalmology
Nijmegen, 6525 EX, Netherlands
Hospital Sant Joan De Deu
Esplugues de Llobregas, Barcelona, 08950, Spain
Hospital Universitari i Politecnic La Fe de Valencia; Servicio de Farmacia
Valencia, 46026, Spain
Drottning Silvias Barn- och ungdomssjukhus; Kliniken for barnmedicin
Gothenburg, 41685, Sweden
Alder Hey Children s Hospital; Department of Pediatrics
Liverpool, L12 2AP, United Kingdom
UCL Institute of Child Health & Great Ormond Street Hospital for Children
London, WC1N 1EH, United Kingdom
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-La Roche
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 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 27, 2017
First Posted
February 1, 2017
Study Start
July 6, 2017
Primary Completion
April 28, 2020
Study Completion
April 28, 2020
Last Updated
December 21, 2020
Results First Posted
December 21, 2020
Record last verified: 2020-11