Clinical trial NCT03039686: Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Duchenne Muscular Dystrophy
Keywords: muscular dystrophy
Duchenne's Muscular Dystrophy
DMD
Lead sponsor: Hoffmann-La Roche
Sponsor class: INDUSTRY
First posted: 2017-02-01
Enrollment: 166
Sex: MALE
Minimum age (years): 6
Maximum age (years): 11
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).


Eligibility 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.

Primary outcomes:
measure: Baseline for the North Star Ambulatory Assessment (NSAA) Total Score
timeFrame: Baseline
measure: Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48
timeFrame: Baseline, Week 48
Secondary outcomes:
measure: Baseline Time for 4 Stair Climb
timeFrame: Baseline
measure: Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV)
timeFrame: Baseline, Week 48
measure: Baseline for the Time to Stand From Supine
timeFrame: Baseline
measure: Change From Baseline at Week 48 in Stand From Supine Velocity
timeFrame: Baseline, Week 48
measure: Baseline Time for 10 Meter Walk/Run
timeFrame: Baseline
measure: Change From Baseline at Week 48 in 10 M Walk/Run Velocity
timeFrame: Baseline, Week 48
measure: Baseline for the Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
timeFrame: Baseline
measure: Change From Baseline at Week 48 in Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
timeFrame: Baseline, Week 48
measure: Change From Baseline at Week 48 in Proximal Lower Extremity Flexor Strength
timeFrame: Baseline, Week 48
measure: Baseline for the 6 Minute Walk Distance (6MWD)
timeFrame: Baseline
measure: Change From Baseline at Week 48 in 6 Minute Walk Distance (6MWD)
timeFrame: Baseline, Week 48
measure: Percentage of Participants for Each Clinical Global Impression of Change (CGI-C) Assessment Status at Week 48
timeFrame: Baseline, Week 48
measure: Change From Baseline at Week 48 in 95th Percentile Stride Velocity
timeFrame: Baseline, Week 48
measure: Number of Participants With Adverse Events (AEs)
timeFrame: During DB period (48 weeks) and Whole study (up to approximately 34 months)
measure: Number of Participants With AEs Leading to Discontinuation
timeFrame: During DB period (48 weeks) and Whole study (up to approximately 34 months)
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Neuromuscular Research Center
state: Arizona
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: Sacramento
country: United States
facility: University of California Davis Medical Center
state: California
city: New Haven
country: United States
facility: Yale University School of Medicine ; Pulmonary & Critical Care
state: Connecticut
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Orlando
country: United States
facility: Nemours Children's Hospital
state: Florida
city: Atlanta
country: United States
facility: Rare Disease Research, LLC
state: Georgia
city: Chicago
country: United States
facility: Rush University Medical Center - PPDS
state: Illinois
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Baltimore
country: United States
facility: Kennedy Krieger Institute
state: Maryland
city: Worcester
country: United States
facility: University of Massachusetts Memorial Childrens Medical Center; Department of Neurology
state: Massachusetts
city: Saint Louis
country: United States
facility: Saint Louis Children's Hospital
state: Missouri
city: Las Vegas
country: United States
facility: Las Vegas Clinic
state: Nevada
city: Cincinnati
country: United States
facility: Cincinnati Childrens Hospital Medical Center;Investigational Pharmacy
state: Ohio
city: Columbus
country: United States
facility: Nationwide Childrens Hospital; Research Institute at Nationwide Childrens Hospital
state: Ohio
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Buenos Aires
country: Argentina
facility: Instituto centenario
city: Westmead
country: Australia
facility: Children's Hospital Westmead; Paediatrics & Child Health
state: New South Wales
city: South Brisbane
country: Australia
facility: Lady Cilento Children's Hospital; Neurosciences Department
state: Queensland
city: Parkville
country: Australia
facility: Royal Children's Hospital
state: Victoria
city: Gent
country: Belgium
facility: UZ Gent
city: London
country: Canada
facility: London Health Sciences Centre; Children's Hospital; Pediatrics
state: Ontario
city: Ottawa
country: Canada
facility: Children'S Hospital of Eastern Ontario
state: Ontario
city: Lyon
country: France
facility: Hospices Civils de Lyon
city: Nantes
country: France
facility: Hotel Dieu; Service Pharmacie Essais Cliniques
city: Paris Cedex 12
country: France
facility: Hopital Armand Trousseau; centre reference Maladies Neuro-musculaires Est parisien Neuropediatrie
city: Strasbourg
country: France
facility: Hopitaux Universitaires de Strasbourg
city: Essen
country: Germany
facility: Universitatsklinikum Essen; Innere Klinik
city: Milano
country: Italy
facility: Fondazione Serena Onlus - CENTRO CLINICO NEMO
state: Emilia-Romagna
city: Rome
country: Italy
facility: Fondazione Policlinico Universitario A Gemelli; Servizio di Farmacia
state: Lazio
city: Messina
country: Italy
facility: Az. Osp. Universitaria Pol. G. Martino; Dip. Neuroscienze, Scienze Psichiatriche e Anest.
state: Sicilia
city: Hyogo
country: Japan
facility: Hyogo College of Medicine Hospital
city: Miyagi
country: Japan
facility: Miyagi Children's Hospital
city: Nagano
country: Japan
facility: Shinshu University Hospital
city: Osaka
country: Japan
facility: National Hospital Organization Osaka Toneyama Medical Center
city: Tokyo
country: Japan
facility: National Center of Neurology and Psychiatry
city: Leiden
country: Netherlands
facility: Leids Universitair Medisch Centrum
city: Nijmegen
country: Netherlands
facility: Radboud University Nijmegen Medical Centre; Ophthalmology
city: Esplugues De Llobregas
country: Spain
facility: Hospital Sant Joan De Deu
state: Barcelona
city: Valencia
country: Spain
facility: Hospital Universitari i Politecnic La Fe de Valencia; Servicio de Farmacia
city: Goeteborg
country: Sweden
facility: Drottning Silvias Barn- och ungdomssjukhus; Kliniken for barnmedicin
city: Liverpool
country: United Kingdom
facility: Alder Hey Children s Hospital; Department of Pediatrics
city: London
country: United Kingdom
facility: UCL Institute of Child Health & Great Ormond Street Hospital for Children
Study officials:
affiliation: Hoffmann-La Roche
name: Clinical Trials
role: STUDY_DIRECTOR
References: