Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Drug: RO7239361Drug: Placebo for RO7239361
- Registration Number
- NCT03039686
- Lead Sponsor
- Hoffmann-La Roche
- 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).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Male
- Target Recruitment
- 166
- 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
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description RO7239361 High Dose RO7239361 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. Placebo Placebo for RO7239361 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. RO7239361 Low Dose RO7239361 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.
- Primary Outcome Measures
Name Time Method Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48 Baseline, 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 for the North Star Ambulatory Assessment (NSAA) Total Score Baseline 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.
- Secondary Outcome Measures
Name Time Method Baseline Time for 4 Stair Climb Baseline The time to complete the 4 stair climb was measured at baseline.
Change From Baseline at Week 48 in Stand From Supine Velocity Baseline, Week 48 The time required for a participant to stand from supine position. A longer time reflects a worse outcome. A negative change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV) Baseline, Week 48 4SCV was calculated as the ratio of the number of stairs climbed (4) divided by the number of seconds taken to complete the 4-stair climb. The results were converted into velocity (distance/time). A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
Baseline for the Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale Baseline The PODCI is designed to be completed by the parent/guardian of a child who has knowledge of the child's conditions. The Transfer and Basic Mobility scale is one of the subscales of the PODCI. The results are standardized into a scale of 0-100 with a higher score reflecting better performance.
Baseline for the 6 Minute Walk Distance (6MWD) Baseline The 6MWD measured the distance a participant was able to traverse while walking for 6 minutes. A longer distance reflects a better outcome.
Percentage of Participants for Each Clinical Global Impression of Change (CGI-C) Assessment Status at Week 48 Baseline, Week 48 The CGI-C was used to assess the participant's overall condition on a 7-point scale, using the status markers "very much improved, much improved, slightly improved, no change, slightly worse, much worse or very much worse" at Week 48 as compared to baseline.
Baseline for the Time to Stand From Supine Baseline The time required for a participant to stand from supine position. A longer time reflects a worse outcome.
Change From Baseline at Week 48 in 10 M Walk/Run Velocity Baseline, Week 48 The time required for a participant to run or walk a distance of 10 meters as quickly as possible calculated as velocity (distance/time). A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
Change From Baseline at Week 48 in Proximal Lower Extremity Flexor Strength Baseline, Week 48 Proximal lower extremity flexor (knee extension and knee flexion) strength was measured using manual myometry. A higher score reflects a better outcome. A positive change from baseline indicates an improvement.
Change From Baseline at Week 48 in 6 Minute Walk Distance (6MWD) Baseline, Week 48 The 6MWD measured the distance a participant was able to traverse while walking for 6 minutes. A longer distance reflects a better outcome. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
Number of Participants With Adverse Events (AEs) During DB period (48 weeks) and Whole study (up to approximately 34 months) An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events.
Number of Participants With AEs Leading to Discontinuation During DB period (48 weeks) and Whole study (up to approximately 34 months) An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events. Reported here is the number of participants with AEs that led to study discontinuation.
Baseline Time for 10 Meter Walk/Run Baseline The time required for a participant to run or walk a distance of 10 meters as quickly as possible. A longer time reflects a worse outcome.
Change From Baseline at Week 48 in 95th Percentile Stride Velocity Baseline, Week 48 Stride velocity was recorded with the ActiMyo device in a subset of the overall study population. The ActiMyo device measures the daily movement and activity levels of the participant. The device consists of two sensors worn on each ankle. A higher velocity reflects a better outcome. A positive change from baseline indicates an improvement.
Change From Baseline at Week 48 in Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale Baseline, Week 48 The PODCI is designed to be completed by the parent/guardian of a child who has knowledge of the child's conditions. The Transfer and Basic Mobility scale is one of the subscales of the PODCI. The results are standardized into a scale of 0-100 with a higher score reflecting better performance. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
Trial Locations
- Locations (44)
University of Massachusetts Memorial Childrens Medical Center; Department of Neurology
🇺🇸Worcester, Massachusetts, United States
Rush University Medical Center - PPDS
🇺🇸Chicago, Illinois, United States
Nationwide Childrens Hospital; Research Institute at Nationwide Childrens Hospital
🇺🇸Columbus, Ohio, United States
Instituto centenario
🇦🇷Buenos Aires, Argentina
Hospices Civils de Lyon
🇫🇷Lyon, France
Yale University School of Medicine ; Pulmonary & Critical Care
🇺🇸New Haven, Connecticut, United States
University of California Davis Medical Center
🇺🇸Sacramento, California, United States
Neuromuscular Research Center
🇺🇸Phoenix, Arizona, United States
Stanford University
🇺🇸Palo Alto, California, United States
Rare Disease Research, LLC
🇺🇸Atlanta, Georgia, United States
Nemours Children's Hospital
🇺🇸Orlando, Florida, United States
University of Iowa
🇺🇸Iowa City, Iowa, United States
Saint Louis Children's Hospital
🇺🇸Saint Louis, Missouri, United States
Kennedy Krieger Institute
🇺🇸Baltimore, Maryland, United States
Cincinnati Childrens Hospital Medical Center;Investigational Pharmacy
🇺🇸Cincinnati, Ohio, United States
Lady Cilento Children's Hospital; Neurosciences Department
🇦🇺South Brisbane, Queensland, Australia
Las Vegas Clinic
🇺🇸Las Vegas, Nevada, United States
Royal Children's Hospital
🇦🇺Parkville, Victoria, Australia
Children's Hospital Westmead; Paediatrics & Child Health
🇦🇺Westmead, New South Wales, Australia
UZ Gent
🇧🇪Gent, Belgium
London Health Sciences Centre; Children's Hospital; Pediatrics
🇨🇦London, Ontario, Canada
Children'S Hospital of Eastern Ontario
🇨🇦Ottawa, Ontario, Canada
Hopitaux Universitaires de Strasbourg
🇫🇷Strasbourg, France
Fondazione Policlinico Universitario A Gemelli; Servizio di Farmacia
🇮🇹Rome, Lazio, Italy
Fondazione Serena Onlus - CENTRO CLINICO NEMO
🇮🇹Milano, Emilia-Romagna, Italy
Universitatsklinikum Essen; Innere Klinik
🇩🇪Essen, Germany
Hotel Dieu; Service Pharmacie Essais Cliniques
🇫🇷Nantes, France
Hopital Armand Trousseau; centre reference Maladies Neuro-musculaires Est parisien Neuropediatrie
🇫🇷Paris Cedex 12, France
Hyogo College of Medicine Hospital
🇯🇵Hyogo, Japan
Az. Osp. Universitaria Pol. G. Martino; Dip. Neuroscienze, Scienze Psichiatriche e Anest.
🇮🇹Messina, Sicilia, Italy
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
Radboud University Nijmegen Medical Centre; Ophthalmology
🇳🇱Nijmegen, Netherlands
Leids Universitair Medisch Centrum
🇳🇱Leiden, Netherlands
Alder Hey Children s Hospital; Department of Pediatrics
🇬🇧Liverpool, United Kingdom
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
🇸🇪Goeteborg, Sweden
UCL Institute of Child Health & Great Ormond Street Hospital for Children
🇬🇧London, United Kingdom
University of Florida
🇺🇸Gainesville, Florida, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
University of Kansas Medical Center
🇺🇸Kansas City, Kansas, United States
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