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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03179631




Registration number
NCT03179631
Ethics application status
Date submitted
1/06/2017
Date registered
7/06/2017

Titles & IDs
Public title
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
Scientific 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
Secondary ID [1] 0 0
PTC124-GD-041-DMD
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Muscular Dystrophy, Duchenne 0 0
Muscular Dystrophies 0 0
Muscular Disorders, Atrophic 0 0
Muscular Diseases 0 0
Musculoskeletal Disease 0 0
Neuromuscular Diseases 0 0
Nervous System Diseases 0 0
Genetic Diseases, X-Linked 0 0
Genetic Diseases, Inborn 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Ataluren
Treatment: Drugs - PLACEBO

Experimental: Ataluren - 10, 20 milligrams per kilogram (mg/kg)

Placebo comparator: Placebo - 10, 20 mg/kg


Treatment: Drugs: Ataluren
10, 20 mg/kg

Treatment: Drugs: PLACEBO
10, 20 mg/kg
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Slope of Change in 6-Minute Walk Distance (6MWD) Over 72 Weeks
Timepoint [1] 0 0
72 weeks
Secondary outcome [1] 0 0
Change from Baseline to Week 72 in 6MWD
Timepoint [1] 0 0
Baseline, Week 72
Secondary outcome [2] 0 0
Change from Baseline to Week 72 in Time to Run/Walk 10 Meters
Timepoint [2] 0 0
Baseline, Week 72
Secondary outcome [3] 0 0
Change from Baseline to Week 72 in Time to Climb 4 Stairs
Timepoint [3] 0 0
Baseline, Week 72
Secondary outcome [4] 0 0
Change from Baseline to Week 72 in Time to Descend 4 Stairs
Timepoint [4] 0 0
Baseline, Week 72
Secondary outcome [5] 0 0
Change from Baseline to Week 72 in North Start Ambulatory Assessment (NSAA) Total Score
Timepoint [5] 0 0
Baseline, Week 72
Secondary outcome [6] 0 0
Time to Loss of Ambulation Over 72 Weeks
Timepoint [6] 0 0
72 weeks
Secondary outcome [7] 0 0
Time to Loss of Stair-Climbing Over 72 Weeks
Timepoint [7] 0 0
72 Weeks
Secondary outcome [8] 0 0
Time to Loss of Stair-Descending Over 72 Weeks
Timepoint [8] 0 0
72 weeks
Secondary outcome [9] 0 0
Risk of Loss of NSAA Items Over 72 weeks
Timepoint [9] 0 0
72 weels
Secondary outcome [10] 0 0
Number of Treatment-Emergent Adverse Events Considered Related to Study Drug
Timepoint [10] 0 0
72 weeks

Eligibility
Key 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.
Minimum age
5 Years
Maximum age
No limit
Sex
Males
Can healthy volunteers participate?
No
Key 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
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
6/07/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
25/07/2023
Sample size
Target
Accrual to date
Final
360
Recruitment in Australia
Recruitment state(s)
NSW,VIC,WA
Recruitment hospital [1] 0 0
The Childrens Hospital at Westmead - Westmead
Recruitment hospital [2] 0 0
The Royal Childrens Hospital - Parkville
Recruitment hospital [3] 0 0
Perth Children's Hospital - Nedlands
Recruitment hospital [4] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment postcode(s) [3] 0 0
6009 - Nedlands
Recruitment postcode(s) [4] 0 0
Q4101 - South Brisbane
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Indiana
Country [5] 0 0
United States of America
State/province [5] 0 0
Kansas
Country [6] 0 0
United States of America
State/province [6] 0 0
Michigan
Country [7] 0 0
United States of America
State/province [7] 0 0
Minnesota
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
Ohio
Country [10] 0 0
United States of America
State/province [10] 0 0
Oregon
Country [11] 0 0
United States of America
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Pennsylvania
Country [12] 0 0
United States of America
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Texas
Country [13] 0 0
United States of America
State/province [13] 0 0
Utah
Country [14] 0 0
United States of America
State/province [14] 0 0
Virginia
Country [15] 0 0
United States of America
State/province [15] 0 0
Washington
Country [16] 0 0
Brazil
State/province [16] 0 0
Belo Horizonte
Country [17] 0 0
Brazil
State/province [17] 0 0
Rio De Janeiro
Country [18] 0 0
Brazil
State/province [18] 0 0
São Paulo
Country [19] 0 0
Bulgaria
State/province [19] 0 0
Sofia
Country [20] 0 0
Canada
State/province [20] 0 0
Ontario
Country [21] 0 0
China
State/province [21] 0 0
Beijing
Country [22] 0 0
China
State/province [22] 0 0
Fuzhou
Country [23] 0 0
China
State/province [23] 0 0
Hunan
Country [24] 0 0
China
State/province [24] 0 0
Shanghai
Country [25] 0 0
China
State/province [25] 0 0
Shenzhen
Country [26] 0 0
Hong Kong
State/province [26] 0 0
Hong Kong
Country [27] 0 0
India
State/province [27] 0 0
Gujarat
Country [28] 0 0
India
State/province [28] 0 0
Karnataka
Country [29] 0 0
India
State/province [29] 0 0
Maharashtra
Country [30] 0 0
India
State/province [30] 0 0
Tamil Nadu
Country [31] 0 0
India
State/province [31] 0 0
Telangana
Country [32] 0 0
India
State/province [32] 0 0
West Bengal
Country [33] 0 0
India
State/province [33] 0 0
Chandigarh
Country [34] 0 0
India
State/province [34] 0 0
New Delhi
Country [35] 0 0
Japan
State/province [35] 0 0
Multiple Locations
Country [36] 0 0
Korea, Republic of
State/province [36] 0 0
Gyeongsangnamdo
Country [37] 0 0
Korea, Republic of
State/province [37] 0 0
Seoul
Country [38] 0 0
Malaysia
State/province [38] 0 0
Kuala Lumpur
Country [39] 0 0
Malaysia
State/province [39] 0 0
Pantai
Country [40] 0 0
Mexico
State/province [40] 0 0
Chihuahua
Country [41] 0 0
Mexico
State/province [41] 0 0
Ciudad de mexico
Country [42] 0 0
Mexico
State/province [42] 0 0
Tlalpan
Country [43] 0 0
Poland
State/province [43] 0 0
Warszawa
Country [44] 0 0
Puerto Rico
State/province [44] 0 0
San Juan
Country [45] 0 0
Russian Federation
State/province [45] 0 0
Moscow
Country [46] 0 0
Russian Federation
State/province [46] 0 0
Saint Petersburg
Country [47] 0 0
Taiwan
State/province [47] 0 0
Kaohsiung
Country [48] 0 0
Taiwan
State/province [48] 0 0
Taipei
Country [49] 0 0
Thailand
State/province [49] 0 0
Bangkok
Country [50] 0 0
Turkey
State/province [50] 0 0
Istanbul

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
PTC Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Vinay Penematsa, MD
Address 0 0
PTC Therapeutics, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT03179631