Nervous System Diseases Clinical Trial
Official title:
A Phase 3, Randomized, Double-blind, Placebo-controlled Efficacy and Safety Study of Ataluren in Patients With Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension
Verified date | January 2024 |
Source | PTC Therapeutics |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
Status | Completed |
Enrollment | 360 |
Est. completion date | July 25, 2023 |
Est. primary completion date | March 5, 2022 |
Accepts healthy volunteers | No |
Gender | Male |
Age group | 5 Years and older |
Eligibility | Inclusion Criteria: - Male sex - 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: - Any change in prophylaxis treatment for cardiomyopathy within 1 month prior to start of study treatment. - 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. |
Country | Name | City | State |
---|---|---|---|
Australia | Perth Children's Hospital | Nedlands | Western Australia |
Australia | The Royal Childrens Hospital | Parkville | Victoria |
Australia | Queensland Children's Hospital | South Brisbane | |
Australia | The Childrens Hospital at Westmead | Westmead | New South Wales |
Brazil | 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 | |
Bulgaria | UMHAT Sofiamed | Sofia | |
Canada | Childrens Hospital London Health Sciences Centre | London | Ontario |
Canada | Childrens Hospital of Eastern Ontario | Ottawa | Ontario |
China | 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 | |
Hong Kong | Queen Mary Hospital | Hong Kong | |
India | Panchshil Hospital | Ahmedabad | Gujarat |
India | National Institute of Mental Health and Neurosciences | Bengaluru | Karnataka |
India | Postgraduate Institute of Medical Education and Research | Chandigarh | |
India | Apollo Children's Hospital Chennai | Chennai | Tamil Nadu |
India | Nizam's Institute of Medical Sciences (NIMS) | Hyderabad | Telangana |
India | Apollo Gleneagles Hospital | Kolkata | West Bengal |
India | P.D. Hinduja Hospital | Mahim | Maharashtra |
India | All India Institute of Medical Sciences | New Delhi | |
India | Christian Medical College Hospital Vellore | Vellore | Tamil Nadu |
Japan | PTC Clinical Site | Multiple Locations | |
Korea, Republic of | Samsung Medical Center | Seoul | |
Korea, Republic of | Seoul National University Hospital | Seoul | |
Korea, Republic of | Pusan National University Yangsan Hospital | Yangsan | Gyeongsangnamdo |
Malaysia | Hospital Tunku Azizah Kuala Lumpur | Kuala Lumpur | |
Malaysia | University Malaya Medical Centre (UMMC) | Pantai | |
Mexico | Hospital Angeles Chihuahua | Chihuahua | |
Mexico | Instituto Nacional de Pediatría | Ciudad de mexico | |
Mexico | Instituto Nacional de Rehabilitacion | Tlalpan | |
Poland | Samodzielny Publiczny Centralny Szpital Kliniczny w Warszawie | Warszawa | |
Puerto Rico | University of Puerto Rico - School of Medicine | San Juan | |
Russian Federation | Russian National Research Medical University n.a. N.I.Pirogov, structural branch - Research Clinical Institute of Pediatrics n.a. Academician Yu. E. Veltishchev | Moscow | |
Russian Federation | "Saint Petersburg State Paediatric Medical University" based at Consultative and Diagnostic Centre | Saint Petersburg | |
Taiwan | Kaohsiung Medical University Chung-Ho Memorial Hospital | Kaohsiung | |
Taiwan | National Taiwan University Hospital | Taipei | |
Thailand | Siriraj Hospital | Bangkok | |
Turkey | Istanbul University- Instanbul Medical Faculty | Istanbul | |
United States | University of Michigan - CS Mott Children's Hospital | Ann Arbor | Michigan |
United States | Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio |
United States | Children's Hospital of Michigan | Detroit | Michigan |
United States | Cook Childrens Medical Center | Fort Worth | Texas |
United States | Northwest Florida Clinical Research Group, LLC | Gulf Breeze | Florida |
United States | Texas Children's Hospital | Houston | Texas |
United States | Indiana University Health - Riley Child Neurology | Indianapolis | Indiana |
United States | University of Kansas Medical Center | Kansas City | Kansas |
United States | Children's Hospital of Los Angeles | Los Angeles | California |
United States | University of Minnesota | Minneapolis | Minnesota |
United States | Columbia University College of Physicians & Surgeons | New York | New York |
United States | Children's Hospital of the King's Daughters | Norfolk | Virginia |
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 | Phoenix Childrens Hospital | Phoenix | Arizona |
United States | University of Pittsburgh School of Medicine | Pittsburgh | Pennsylvania |
United States | Shriners Hospital for Children | Portland | Oregon |
United States | University of California (UC) Davis Medical Center | Sacramento | California |
United States | University Of Utah | Salt Lake City | Utah |
United States | University of Texas Health Science Center at San Antonio | San Antonio | Texas |
United States | Loma Linda University Children's Hospital | San Bernardino | California |
United States | Seattle Children's Hospital | Seattle | Washington |
Lead Sponsor | Collaborator |
---|---|
PTC Therapeutics |
United States, Australia, Brazil, Bulgaria, Canada, China, Hong Kong, India, Japan, Korea, Republic of, Malaysia, Mexico, Poland, Puerto Rico, Russian Federation, Taiwan, Thailand, Turkey,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Slope of Change in 6-Minute Walk Distance (6MWD) Over 72 Weeks | 72 weeks | ||
Secondary | Change from Baseline to Week 72 in 6MWD | Baseline, Week 72 | ||
Secondary | Change from Baseline to Week 72 in Time to Run/Walk 10 Meters | Baseline, Week 72 | ||
Secondary | Change from Baseline to Week 72 in Time to Climb 4 Stairs | Baseline, Week 72 | ||
Secondary | Change from Baseline to Week 72 in Time to Descend 4 Stairs | Baseline, Week 72 | ||
Secondary | Change from Baseline to Week 72 in North Start Ambulatory Assessment (NSAA) Total Score | Baseline, Week 72 | ||
Secondary | Time to Loss of Ambulation Over 72 Weeks | 72 weeks | ||
Secondary | Time to Loss of Stair-Climbing Over 72 Weeks | 72 Weeks | ||
Secondary | Time to Loss of Stair-Descending Over 72 Weeks | 72 weeks | ||
Secondary | Risk of Loss of NSAA Items Over 72 weeks | 72 weels | ||
Secondary | Number of Treatment-Emergent Adverse Events Considered Related to Study Drug | 72 weeks |
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