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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02456103




Registration number
NCT02456103
Ethics application status
Date submitted
26/05/2015
Date registered
28/05/2015
Date last updated
27/04/2020

Titles & IDs
Public title
Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
Scientific title
Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Cystic Fibrosis
Secondary ID [1] 0 0
PTC124-GD-021e-CF
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Cystic fibrosis
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

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

Experimental: Ataluren - Participants will be administered ataluren orally at a dose of 10 milligrams/grams (mg/kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for up to 96 weeks.


Treatment: Drugs: Ataluren
Ataluren will be provided as a vanilla-flavored powder to be mixed with water or milk.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Timepoint [1] 0 0
Baseline up to Week 100
Primary outcome [2] 0 0
Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Serum Biochemistry, Hematology, and Urinalysis) Parameter
Timepoint [2] 0 0
Baseline up to Week 100
Secondary outcome [1] 0 0
Change From Baseline in Percent-Predicted Forced Expiratory Volume in 1 Second (FEV1) as Measured by Spirometry at Week 24
Timepoint [1] 0 0
Baseline, Week 24
Secondary outcome [2] 0 0
Change From Baseline in Percent-Predicted of Forced Vital Capacity (FVC) as Measured by Spirometry at Week 24
Timepoint [2] 0 0
Baseline, Week 24
Secondary outcome [3] 0 0
Change From Baseline in Forced Expiratory Flow Between 25% and 75% of Expiration (FEF25-75) as Measured by Spirometry at Week 24
Timepoint [3] 0 0
Baseline, Week 24
Secondary outcome [4] 0 0
Rate of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria Over 48 Weeks
Timepoint [4] 0 0
Baseline up to Week 48

Eligibility
Key inclusion criteria
* Completion of study treatment (placebo or active) in the previous Phase 3, double-blind study protocol (Protocol PTC124-GD-021-CF)
* Evidence of signed and dated informed consent/assent document(s) indicating that the participant (and/or the participant's parent/legal guardian) has been informed of all pertinent aspects of the trial.
Minimum age
6 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Known hypersensitivity to any of the ingredients or excipients of the study drug.
* Ongoing participation in any other therapeutic clinical trial.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
31/08/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
2/06/2017
Sample size
Target
Accrual to date
Final
246
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [2] 0 0
Prince Charles Hospital - Chermside
Recruitment hospital [3] 0 0
Princess Margaret Hospital - Perth
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment postcode(s) [2] 0 0
4032 - Chermside
Recruitment postcode(s) [3] 0 0
6840 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
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
Colorado
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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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Illinois
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Indiana
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United States of America
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Massachusetts
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United States of America
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Missouri
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New Jersey
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New York
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Ohio
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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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Wisconsin
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Argentina
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Buenos Aires
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Belgium
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Brussels
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Belgium
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Leuven
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Brazil
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Porto Alegre
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Bulgaria
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Plovdiv
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Bulgaria
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Sofia
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Canada
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Montreal
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Canada
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Toronto
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Canada
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Vancouver
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France
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Montpellier
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France
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Paris
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France
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Roscoff
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France
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Saint-Pierre
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Germany
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Berlin
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Germany
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Bochum
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Germany
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Cologne
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Germany
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Frankfurt am Main
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Germany
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Jena
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Germany
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Munchen
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Germany
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Munich
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Greece
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Thessaloniki
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Israel
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Haifa
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Israel
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Jerusalem
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Italy
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Ancona
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Italy
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Firenze
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Italy
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Milan
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Italy
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Rome
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Italy
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Verona
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Netherlands
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Zuid-Holland
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Netherlands
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Nijmegen
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Poland
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Gdansk
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Poland
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Warsaw
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Spain
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Barcelona
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Spain
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Esplugues De Llobregat
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Spain
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Malaga
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Spain
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Sabadell
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Spain
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Sevilla
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United Kingdom
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Leeds
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United Kingdom
State/province [53] 0 0
London

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

Ethics approval
Ethics application status

Summary
Brief summary
This is an open-label extension study for participants who completed a Phase 3, placebo-controlled study of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) not receiving chronic inhaled aminoglycosides.
Trial website
https://clinicaltrials.gov/study/NCT02456103
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Joseph McIntosh, MD
Address 0 0
PTC Therapeutics
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT02456103