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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT03179631
Ethics application status
Date submitted
1/06/2017
Date registered
7/06/2017
Date last updated
19/06/2020

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
Gender
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
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
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
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California
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United States of America
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Connecticut
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United States of America
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Florida
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United States of America
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Indiana
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United States of America
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Kansas
Country [7] 0 0
United States of America
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Massachusetts
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United States of America
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Michigan
Country [9] 0 0
United States of America
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Minnesota
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United States of America
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New York
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United States of America
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Ohio
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United States of America
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Oregon
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United States of America
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Pennsylvania
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United States of America
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Texas
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United States of America
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Utah
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United States of America
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Virginia
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United States of America
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Washington
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United States of America
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Wisconsin
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Brazil
State/province [19] 0 0
Belo Horizonte
Country [20] 0 0
Brazil
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Rio De Janeiro
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Brazil
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São Paulo
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Bulgaria
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Sofia
Country [23] 0 0
Canada
State/province [23] 0 0
British Columbia
Country [24] 0 0
Canada
State/province [24] 0 0
Ontario
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China
State/province [25] 0 0
Beijing
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China
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Fuzhou
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China
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Hunan
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China
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Shanghai
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China
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Shenzhen
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Hong Kong
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Hong Kong
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India
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Gujarat
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India
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Karnataka
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India
State/province [33] 0 0
Maharashtra
Country [34] 0 0
India
State/province [34] 0 0
Tamil Nadu
Country [35] 0 0
India
State/province [35] 0 0
Telangana
Country [36] 0 0
India
State/province [36] 0 0
West Bengal
Country [37] 0 0
India
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Chandigarh
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India
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New Delhi
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Japan
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Hasuda-city
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Japan
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Kodaira
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Japan
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Kumamoto-shi
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Japan
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Kyoto
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Japan
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Nagoya-shi
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Japan
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Nishinomiya
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Japan
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Sendai
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Japan
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Toyonaka-shi
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Japan
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Yonago
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Jordan
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Amman
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Jordan
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Irbid
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Korea, Republic of
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Gyeongsangnamdo
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Korea, Republic of
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Seoul
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Malaysia
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Kuala Lumpur
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Malaysia
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Pantai
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Mexico
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Chihuahua
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Mexico
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Ciudad de mexico
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Mexico
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Tlalpan
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Poland
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Warszawa
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Puerto Rico
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San Juan
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Russian Federation
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Moscow
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Russian Federation
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Saint Petersburg
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Sri Lanka
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Colombo
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Taiwan
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Kaohsiung
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Taiwan
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Taipei
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Thailand
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Bangkok
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Turkey
State/province [65] 0 0
Istanbul

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

Ethics approval
Ethics application status

Summary
Brief summary
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne
muscular dystrophy.
Trial website
https://clinicaltrials.gov/show/NCT03179631
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Francesco Bibbiani, 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
Patient Advocacy Corporate Relations
Address 0 0
Country 0 0
Phone 0 0
1-866-562-4620
Fax 0 0
Email 0 0
medinfo@ptcbio.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT03179631