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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT03179631
Other study ID # PTC124-GD-041-DMD
Secondary ID
Status Completed
Phase Phase 3
First received
Last updated
Start date July 6, 2017
Est. completion date July 25, 2023

Study information

Verified date January 2024
Source PTC Therapeutics
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.


Description:

This study is a randomized, double-blind, placebo-controlled, 72-week study, followed by a 72-week open-label period. The purpose is to characterize the long-term effects of ataluren-mediated dystrophin restoration on disease progression. Participants will be randomized in a 1:1 ratio to ataluren or placebo. Participants will receive blinded study drug three times daily (TID) at morning, midday, and evening for 72 weeks, after which all participants will receive open-label ataluren for an additional 72 weeks (144 weeks in total). Study assessments will be performed at clinic visits every 12 weeks during the double-blind period and every 24 weeks during the open-label period. The total sample size of ~250 subjects will include ~160 subjects who meet the criteria for inclusion in the primary analysis population (age 7 to 16 years old, baseline six minute walk distance (6MWD) greater than or equal to (>=) 300 meters, supine to stand >= 5 seconds). The study will be conducted in the United States and other countries around the world.


Recruitment information / eligibility

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.

Study Design


Intervention

Drug:
Ataluren
10, 20 mg/kg
PLACEBO
10, 20 mg/kg

Locations

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

Sponsors (1)

Lead Sponsor Collaborator
PTC Therapeutics

Countries where clinical trial is conducted

United States,  Australia,  Brazil,  Bulgaria,  Canada,  China,  Hong Kong,  India,  Japan,  Korea, Republic of,  Malaysia,  Mexico,  Poland,  Puerto Rico,  Russian Federation,  Taiwan,  Thailand,  Turkey, 

Outcome

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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