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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02139306




Registration number
NCT02139306
Ethics application status
Date submitted
13/05/2014
Date registered
15/05/2014

Titles & IDs
Public title
Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)
Scientific title
A Phase 3 Efficacy and Safety Study of Ataluren (PTC124®) in Patients With Nonsense Mutation Cystic Fibrosis
Secondary ID [1] 0 0
2013-004581-34
Secondary ID [2] 0 0
PTC124-GD-021-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 (PTC124®)
Treatment: Drugs - Placebo

Experimental: Ataluren (PTC124®) - Participants received ataluren as oral powder for suspension at the dosages of 10, 10, and 20-mg/kg at morning, midday and evening, respectively for 48 weeks of treatment duration or until treatment discontinuation.

Placebo comparator: Placebo - Participants received matching placebo orally at morning, midday and evening for 48 weeks of treatment duration or until treatment discontinuation.


Treatment: Drugs: Ataluren (PTC124®)
Oral Ataluren TID

Treatment: Drugs: Placebo
Oral Placebo TID
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Absolute Change From Baseline in Percent-predicted Forced Expiratory Volume in One Second (ppFEV1) at Week 48
Timepoint [1] 0 0
From Baseline to Week 48
Secondary outcome [1] 0 0
48-week Rate of Pulmonary Exacerbations
Timepoint [1] 0 0
Week 48
Secondary outcome [2] 0 0
Change From Baseline in the Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain at Week 48
Timepoint [2] 0 0
Baseline (Day 1) and Week 48
Secondary outcome [3] 0 0
Change From Baseline in Body Mass Index (BMI) at Week 48
Timepoint [3] 0 0
Baseline (Day 1) and Week 48
Secondary outcome [4] 0 0
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs)
Timepoint [4] 0 0
From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)
Secondary outcome [5] 0 0
Number of Participants With TEAEs by Severity and Relationship to Study Drugs
Timepoint [5] 0 0
From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)
Secondary outcome [6] 0 0
Number of Participants With SAEs by Severity and Relationship to Study Drugs
Timepoint [6] 0 0
From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)
Secondary outcome [7] 0 0
Number of Participants With Abnormal Vital Signs Reported as TEAEs
Timepoint [7] 0 0
From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)
Secondary outcome [8] 0 0
Number of Participants With Abnormal Clinical Laboratory Parameters Reported as TEAEs
Timepoint [8] 0 0
From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)
Secondary outcome [9] 0 0
Number of Participants With Abnormal Electrocardiogram Reported as TEAEs
Timepoint [9] 0 0
From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)

Eligibility
Key inclusion criteria
* Evidence of signed and dated informed consent/assent document(s) indicating that the subject (and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial
* Age >=6 years.
* Body weight >=16 kg.
* Sweat chloride >60 milliequivalent per liter (mEq/L)
* Documentation of the presence of a nonsense mutation in at least 1 allele of the cystic fibrosis transmembrane conductance regulator (CFTR) gene, as determined by genotyping performed at a laboratory certified by the College of American Pathologists (CAP), or under the Clinical Laboratory Improvement Act/Amendment (CLIA), or by an equivalent organization
* Verification that a blood sample has been drawn for sequencing of the CFTR gene
* Ability to perform a valid, reproducible spirometry test using the study-specific spirometer with demonstration of an FEV1 >=40% and <=90% of predicted
* Demonstration at Visit 2 of a valid %-predicted FEV1 within 15% of the Screening % predicted FEV1 value
* Resting oxygen saturation (as measured by pulse oximetry) >=92% on room air.
* Confirmed screening laboratory values within pre-specified ranges
* In subjects who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during the study drug administration and 60-day follow-up period
* Willingness and ability to comply with all study procedures and assessments, including scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions
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
* Previous participation in the Phase 3 trial of ataluren (PTC124-GD-009-CF).
* Any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation, or re-initiation) in a chronic treatment/prophylaxis regimen for Cystic Fibrosis (CF) or for CF-related conditions within 4 weeks prior to screening
* Chronic use of inhaled aminoglycosides (eg, tobramycin) or use of inhaled aminoglycosides within 4 weeks prior to screening.
* Exposure to another investigational drug within 4 weeks prior to screening
* Ongoing participation in any other therapeutic clinical trial
* Evidence of pulmonary exacerbation or acute upper or lower respiratory tract infection (including viral illnesses) within 3 weeks prior to screening
* Treatment with intravenous antibiotics within 3 weeks prior to screening
* Ongoing immunosuppressive therapy (other than corticosteroids)
* Ongoing warfarin, phenytoin, or tolbutamide therapy
* History of solid organ or hematological transplantation
* Major complications of lung disease (including massive hemoptysis, pneumothorax, or pleural effusion) within 8 weeks prior to screening
* Known portal hypertension
* Positive hepatitis B surface antigen, hepatitis C antibody test, or human immunodeficiency virus (HIV) test
* Pregnancy or breast-feeding
* Current smoker or a smoking history of >=10 pack-years (number of cigarette packs/day x number of years smoked).
* Prior or ongoing medical condition (eg, concomitant illness, alcoholism, drug abuse, psychiatric condition), medical history, physical findings, electrocardiogram (ECG) findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the subject, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results

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
1/08/2014
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/11/2016
Sample size
Target
Accrual to date
Final
279
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
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Indiana
Country [7] 0 0
United States of America
State/province [7] 0 0
Massachusetts
Country [8] 0 0
United States of America
State/province [8] 0 0
Missouri
Country [9] 0 0
United States of America
State/province [9] 0 0
New Jersey
Country [10] 0 0
United States of America
State/province [10] 0 0
New York
Country [11] 0 0
United States of America
State/province [11] 0 0
Ohio
Country [12] 0 0
United States of America
State/province [12] 0 0
Oklahoma
Country [13] 0 0
United States of America
State/province [13] 0 0
Pennsylvania
Country [14] 0 0
United States of America
State/province [14] 0 0
Texas
Country [15] 0 0
United States of America
State/province [15] 0 0
Wisconsin
Country [16] 0 0
Argentina
State/province [16] 0 0
Buenos Aires
Country [17] 0 0
Argentina
State/province [17] 0 0
La Plata
Country [18] 0 0
Belgium
State/province [18] 0 0
Brussels
Country [19] 0 0
Belgium
State/province [19] 0 0
Bruxelles
Country [20] 0 0
Belgium
State/province [20] 0 0
Leuven
Country [21] 0 0
Brazil
State/province [21] 0 0
Porto Alegre
Country [22] 0 0
Brazil
State/province [22] 0 0
São Paulo
Country [23] 0 0
Bulgaria
State/province [23] 0 0
Plovdiv
Country [24] 0 0
Bulgaria
State/province [24] 0 0
Sofia
Country [25] 0 0
Canada
State/province [25] 0 0
Montreal
Country [26] 0 0
Canada
State/province [26] 0 0
Québec City
Country [27] 0 0
Canada
State/province [27] 0 0
Toronto
Country [28] 0 0
Canada
State/province [28] 0 0
Vancouver
Country [29] 0 0
France
State/province [29] 0 0
Bron
Country [30] 0 0
France
State/province [30] 0 0
Montpellier
Country [31] 0 0
France
State/province [31] 0 0
Paris
Country [32] 0 0
France
State/province [32] 0 0
Roscoff
Country [33] 0 0
France
State/province [33] 0 0
Saint-Pierre
Country [34] 0 0
Germany
State/province [34] 0 0
Berlin
Country [35] 0 0
Germany
State/province [35] 0 0
Bochum
Country [36] 0 0
Germany
State/province [36] 0 0
Cologne
Country [37] 0 0
Germany
State/province [37] 0 0
Frankfurt am Main
Country [38] 0 0
Germany
State/province [38] 0 0
Jena
Country [39] 0 0
Germany
State/province [39] 0 0
München
Country [40] 0 0
Greece
State/province [40] 0 0
Thessaloniki
Country [41] 0 0
Israel
State/province [41] 0 0
Haifa
Country [42] 0 0
Israel
State/province [42] 0 0
Jerusalem
Country [43] 0 0
Italy
State/province [43] 0 0
Ancona
Country [44] 0 0
Italy
State/province [44] 0 0
Firenze
Country [45] 0 0
Italy
State/province [45] 0 0
Milan
Country [46] 0 0
Italy
State/province [46] 0 0
Roma
Country [47] 0 0
Italy
State/province [47] 0 0
Rome
Country [48] 0 0
Italy
State/province [48] 0 0
Verona
Country [49] 0 0
Netherlands
State/province [49] 0 0
Den Haag
Country [50] 0 0
Netherlands
State/province [50] 0 0
Nijmegen
Country [51] 0 0
Netherlands
State/province [51] 0 0
Rotterdam
Country [52] 0 0
Poland
State/province [52] 0 0
Gdansk
Country [53] 0 0
Poland
State/province [53] 0 0
Rabka-Zdrój
Country [54] 0 0
Poland
State/province [54] 0 0
Rzeszow
Country [55] 0 0
Poland
State/province [55] 0 0
Warsaw
Country [56] 0 0
Spain
State/province [56] 0 0
Barcelona
Country [57] 0 0
Spain
State/province [57] 0 0
Esplugues De Llobregat
Country [58] 0 0
Spain
State/province [58] 0 0
Málaga
Country [59] 0 0
Spain
State/province [59] 0 0
Sabadell
Country [60] 0 0
Spain
State/province [60] 0 0
Sevilla
Country [61] 0 0
United Kingdom
State/province [61] 0 0
Birmingham
Country [62] 0 0
United Kingdom
State/province [62] 0 0
Glasgow
Country [63] 0 0
United Kingdom
State/province [63] 0 0
Leeds
Country [64] 0 0
United Kingdom
State/province [64] 0 0
London
Country [65] 0 0
United Kingdom
State/province [65] 0 0
Penarth
Country [66] 0 0
United Kingdom
State/province [66] 0 0
Southampton

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
PTC Therapeutics
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Cystic Fibrosis Foundation
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
ECFS-Clinical Trial Network (ECFS-CTN)
Address [2] 0 0
Country [2] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
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

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 are available at https://clinicaltrials.gov/study/NCT02139306