Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
NCT ID: NCT03179631
Last Updated: 2024-01-18
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE3
360 participants
INTERVENTIONAL
2017-07-06
2023-07-25
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Ataluren
10, 20 milligrams per kilogram (mg/kg)
Ataluren
10, 20 mg/kg
Placebo
10, 20 mg/kg
PLACEBO
10, 20 mg/kg
Interventions
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Ataluren
10, 20 mg/kg
PLACEBO
10, 20 mg/kg
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Age ≥5 years
* Phenotypic evidence of Duchenne Muscular Dystrophy
* Nonsense point mutation in the dystrophin gene
* Use of systemic corticosteroids (prednisone/prednisolone or deflazacort)for a minimum of 12 months immediately prior to start of study treatment, with no significant change in dosage or dosing regimen for a minimum of 3 months immediately prior to start of study treatment
* 6MWD ≥150 meters
* Ability to perform timed function tests within 30 seconds
* Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions.
Exclusion Criteria
* Ongoing intravenous (IV) aminoglycoside or IV vancomycin therapy.
* Prior or ongoing therapy with ataluren.
* Known hypersensitivity to any of the ingredients or excipients of the study drug
* Exposure to another investigational drug within 6 months prior to start of study treatment, or ongoing participation in any interventional clinical trial.
* History of major surgical procedure within 12 weeks prior to start of study treatment, or expectation of major surgical procedure during the 72-week placebo-controlled treatment period.
* Requirement for daytime ventilator assistance or any use of invasive mechanical ventilation via tracheostomy.
* Uncontrolled clinical symptoms and signs of congestive heart failure
* Elevated serum creatinine or cystatin C at screening.
5 Years
MALE
No
Sponsors
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PTC Therapeutics
INDUSTRY
Responsible Party
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Principal Investigators
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Vinay Penematsa, MD
Role: STUDY_DIRECTOR
PTC Therapeutics, Inc.
Locations
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Phoenix Childrens Hospital
Phoenix, Arizona, United States
Children's Hospital of Los Angeles
Los Angeles, California, United States
University of California, San Francisco (UCSF) - Benioff Children's Hospital - Oakland
Oakland, California, United States
Stanford University Medical Center
Palo Alto, California, United States
University of California (UC) Davis Medical Center
Sacramento, California, United States
Loma Linda University Children's Hospital
San Bernardino, California, United States
Northwest Florida Clinical Research Group, LLC
Gulf Breeze, Florida, United States
Indiana University Health - Riley Child Neurology
Indianapolis, Indiana, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
University of Michigan - CS Mott Children's Hospital
Ann Arbor, Michigan, United States
Children's Hospital of Michigan
Detroit, Michigan, United States
University of Minnesota
Minneapolis, Minnesota, United States
Columbia University College of Physicians & Surgeons
New York, New York, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Shriners Hospital for Children
Portland, Oregon, United States
University of Pittsburgh School of Medicine
Pittsburgh, Pennsylvania, United States
Cook Childrens Medical Center
Fort Worth, Texas, United States
Texas Children's Hospital
Houston, Texas, United States
University of Texas Health Science Center at San Antonio
San Antonio, Texas, United States
University Of Utah
Salt Lake City, Utah, United States
Children's Hospital of the King's Daughters
Norfolk, Virginia, United States
Seattle Children's Hospital
Seattle, Washington, United States
The Childrens Hospital at Westmead
Westmead, New South Wales, Australia
The Royal Childrens Hospital
Parkville, Victoria, Australia
Perth Children's Hospital
Nedlands, Western Australia, Australia
Queensland Children's Hospital
South Brisbane, , Australia
Hospital de Clínicas da Universidade Federal de Minas Gerais
Belo Horizonte, , Brazil
Universidade Federal do Rio de Janeiro
Rio de Janeiro, , Brazil
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo - FMUSP
São Paulo, , Brazil
UMHAT Sofiamed
Sofia, , Bulgaria
Childrens Hospital London Health Sciences Centre
London, Ontario, Canada
Childrens Hospital of Eastern Ontario
Ottawa, Ontario, Canada
General Hospital of Chinese Armed Police Forces
Beijing, , China
The First Affiliated Hospital of Fujian Medical University
Fuzhou, , China
Xiangya Hospital Central South University
Hunan, , China
Children's Hospital of Fudan University
Shanghai, , China
Shenzhen Children's Hospital
Shenzhen, , China
Queen Mary Hospital
Hong Kong, , Hong Kong
Panchshil Hospital
Ahmedabad, Gujarat, India
National Institute of Mental Health and Neurosciences
Bengaluru, Karnataka, India
P.D. Hinduja Hospital
Māhīm, Maharashtra, India
Apollo Children's Hospital Chennai
Chennai, Tamil Nadu, India
Christian Medical College Hospital Vellore
Vellore, Tamil Nadu, India
Nizam's Institute of Medical Sciences (NIMS)
Hyderabad, Telangana, India
Apollo Gleneagles Hospital
Kolkata, West Bengal, India
Postgraduate Institute of Medical Education and Research
Chandigarh, , India
All India Institute of Medical Sciences
New Delhi, , India
PTC Clinical Site
Multiple Locations, , Japan
Hospital Tunku Azizah Kuala Lumpur
Kuala Lumpur, , Malaysia
University Malaya Medical Centre (UMMC)
Pantai, , Malaysia
Hospital Angeles Chihuahua
Chihuahua City, , Mexico
Instituto Nacional de Pediatría
Mexico City, , Mexico
Instituto Nacional de Rehabilitacion
Tlalpan, , Mexico
Samodzielny Publiczny Centralny Szpital Kliniczny w Warszawie
Warsaw, , Poland
University of Puerto Rico - School of Medicine
San Juan, , Puerto Rico
Russian National Research Medical University n.a. N.I.Pirogov, structural branch - Research Clinical Institute of Pediatrics n.a. Academician Yu. E. Veltishchev
Moscow, , Russia
"Saint Petersburg State Paediatric Medical University" based at Consultative and Diagnostic Centre
Saint Petersburg, , Russia
Pusan National University Yangsan Hospital
Yangsan, Gyeongsangnam-do, South Korea
Samsung Medical Center
Seoul, , South Korea
Seoul National University Hospital
Seoul, , South Korea
Kaohsiung Medical University Chung-Ho Memorial Hospital
Kaohsiung City, , Taiwan
National Taiwan University Hospital
Taipei, , Taiwan
Siriraj Hospital
Bangkok, , Thailand
Istanbul University- Instanbul Medical Faculty
Istanbul, , Turkey (Türkiye)
Countries
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Related Links
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References
Other Identifiers
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PTC124-GD-041-DMD
Identifier Type: -
Identifier Source: org_study_id
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