Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
MY TRIALS
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Register a trial
The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
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
Titles & IDs
Public title
Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
Query!
Scientific title
Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Cystic Fibrosis
Query!
Secondary ID [1]
0
0
PTC124-GD-021e-CF
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis
0
0
Query!
Condition category
Condition code
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Cystic fibrosis
Query!
Respiratory
0
0
0
0
Query!
Other respiratory disorders / diseases
Query!
Oral and Gastrointestinal
0
0
0
0
Query!
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Query!
Inflammatory and Immune System
0
0
0
0
Query!
Connective tissue diseases
Query!
Inflammatory and Immune System
0
0
0
0
Query!
Other inflammatory or immune system disorders
Query!
Intervention/exposure
Study type
Interventional
Query!
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.
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Query!
Assessment method [1]
0
0
TEAE: any untoward medical occurrence or undesirable event that begins or worsens following administration of study drug, whether or not considered related to study drug by Investigator. Serious adverse event (SAE): an adverse event (AE) resulting in any of following outcomes or deemed significant for any other reason: death, initial or prolonged inpatient hospitalization, life-threatening experience (immediate risk of dying) or persistent or significant disability/incapacity. Except for cystic fibrosis (CF) pulmonary exacerbations, an event wasn't reported as an SAE, if event was exclusively a relapse or an expected change or progression of baseline CF. AEs included both SAEs and nonserious AEs. AEs classified according to National Cancer Institute Common Terminology Criteria for Adverse Events version 3.0 and coded using Medical Dictionary for Regulatory Activities. A summary of SAEs and all nonserious AEs, regardless of causality, is located in the Reported Adverse Events section.
Query!
Timepoint [1]
0
0
Baseline up to Week 100
Query!
Primary outcome [2]
0
0
Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Serum Biochemistry, Hematology, and Urinalysis) Parameter
Query!
Assessment method [2]
0
0
Clinical laboratory results considered clinically meaningful were determined by Investigator. Serum biochemistry parameters: sodium, potassium, chloride, bicarbonate, blood urea nitrogen, creatinine, magnesium, calcium, phosphorus, uric acid, glucose, total protein, albumin, globulin, bilirubin, creatine kinase, lactate dehydrogenase, alanine aminotransferase, aspartate aminotransferase, gamma glutamyl transferase, alkaline phosphatase, total cholesterol, high-density lipoprotein, low-density lipoprotein, triglycerides, and cystatin C. Hematology parameters: white blood cell count, hemoglobin, hematocrit, mean corpuscular volume, mean corpuscular hemoglobin, red cell count with morphology, and platelet count. Urinalysis parameters: pH, specific gravity, glucose, ketones, blood, protein, creatinine, urobilinogen, bilirubin, nitrite, and leukocyte esterase. A summary of all SAEs/nonserious AEs, regardless of causality, is located in the Reported Adverse Events section.
Query!
Timepoint [2]
0
0
Baseline up to Week 100
Query!
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
Query!
Assessment method [1]
0
0
Pulmonary function of percent-predicted FEV1 was measured using a spirometer. FEV1 is the volume of air that can forcibly be blown out in 1 second. Each percent-predicted FEV1 was based gender, age, and the height value obtained at the same study visit. The percentage of change in percent-predicted of FEV1 was calculated as follows: (percent-predicted FEV1 - Baseline percent-predicted FEV1/Baseline percent-predicted FEV1)\*100.
Query!
Timepoint [1]
0
0
Baseline, Week 24
Query!
Secondary outcome [2]
0
0
Change From Baseline in Percent-Predicted of Forced Vital Capacity (FVC) as Measured by Spirometry at Week 24
Query!
Assessment method [2]
0
0
Pulmonary function of FVC was measured using a spirometer. FVC is the volume of air that can forcibly be blown out. Each percent-predicted FVC was based gender, age, and the height value obtained at the same study visit. The percentage of change in percent-predicted of FVC was calculated as follows: (percent-predicted FVC - Baseline percent-predicted FVC/Baseline percent-predicted FVC)\*100.
Query!
Timepoint [2]
0
0
Baseline, Week 24
Query!
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
Query!
Assessment method [3]
0
0
Pulmonary function of FEF25-75 was measured using a spirometer. FEF25-75 is the forced expiratory flow between 25% and 75% of vital capacity. Each percent-predicted FEF25-75 was based gender, age, and the height value obtained at the same study visit. The percentage of change in percent-predicted of FEF25-75 was calculated as follows: (percent-predicted FEF25-75 - Baseline percent-predicted FEF25-75/Baseline percent-predicted FEF25-75)\*100.
Query!
Timepoint [3]
0
0
Baseline, Week 24
Query!
Secondary outcome [4]
0
0
Rate of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria Over 48 Weeks
Query!
Assessment method [4]
0
0
A modified Fuchs' exacerbation was defined as an event requiring treatment with or without intravenous antibiotics for any 4 of the following 12 symptoms: change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; fatigue; temperature \>38°C; anorexia; sinus pain; change in sinus discharge; change in physical examination of the chest; decrease in pulmonary function by 10 percent or more from a previously recorded value; or radiographic changes indicative of pulmonary function. The 48-week rate = (the total number of events/ treatment duration by week)\*48.
Query!
Timepoint [4]
0
0
Baseline up to Week 48
Query!
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.
Query!
Minimum age
6
Years
Query!
Query!
Maximum age
No limit
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key exclusion criteria
* Known hypersensitivity to any of the ingredients or excipients of the study drug.
* Ongoing participation in any other therapeutic clinical trial.
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
NA
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Open (masking not used)
Query!
Who is / are masked / blinded?
Query!
Query!
Query!
Query!
Intervention assignment
Single group
Query!
Other design features
Query!
Phase
Phase 3
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Stopped early
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
31/08/2015
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
2/06/2017
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
246
Query!
Recruitment in Australia
Recruitment state(s)
Query!
Recruitment hospital [1]
0
0
Royal Adelaide Hospital - Adelaide
Query!
Recruitment hospital [2]
0
0
Prince Charles Hospital - Chermside
Query!
Recruitment hospital [3]
0
0
Princess Margaret Hospital - Perth
Query!
Recruitment postcode(s) [1]
0
0
5000 - Adelaide
Query!
Recruitment postcode(s) [2]
0
0
4032 - Chermside
Query!
Recruitment postcode(s) [3]
0
0
6840 - Perth
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
Alabama
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
California
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Colorado
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Florida
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Illinois
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
Indiana
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
Massachusetts
Query!
Country [8]
0
0
United States of America
Query!
State/province [8]
0
0
Missouri
Query!
Country [9]
0
0
United States of America
Query!
State/province [9]
0
0
New Jersey
Query!
Country [10]
0
0
United States of America
Query!
State/province [10]
0
0
New York
Query!
Country [11]
0
0
United States of America
Query!
State/province [11]
0
0
Ohio
Query!
Country [12]
0
0
United States of America
Query!
State/province [12]
0
0
Pennsylvania
Query!
Country [13]
0
0
United States of America
Query!
State/province [13]
0
0
Texas
Query!
Country [14]
0
0
United States of America
Query!
State/province [14]
0
0
Wisconsin
Query!
Country [15]
0
0
Argentina
Query!
State/province [15]
0
0
Buenos Aires
Query!
Country [16]
0
0
Belgium
Query!
State/province [16]
0
0
Brussels
Query!
Country [17]
0
0
Belgium
Query!
State/province [17]
0
0
Leuven
Query!
Country [18]
0
0
Brazil
Query!
State/province [18]
0
0
Porto Alegre
Query!
Country [19]
0
0
Bulgaria
Query!
State/province [19]
0
0
Plovdiv
Query!
Country [20]
0
0
Bulgaria
Query!
State/province [20]
0
0
Sofia
Query!
Country [21]
0
0
Canada
Query!
State/province [21]
0
0
Montreal
Query!
Country [22]
0
0
Canada
Query!
State/province [22]
0
0
Toronto
Query!
Country [23]
0
0
Canada
Query!
State/province [23]
0
0
Vancouver
Query!
Country [24]
0
0
France
Query!
State/province [24]
0
0
Montpellier
Query!
Country [25]
0
0
France
Query!
State/province [25]
0
0
Paris
Query!
Country [26]
0
0
France
Query!
State/province [26]
0
0
Roscoff
Query!
Country [27]
0
0
France
Query!
State/province [27]
0
0
Saint-Pierre
Query!
Country [28]
0
0
Germany
Query!
State/province [28]
0
0
Berlin
Query!
Country [29]
0
0
Germany
Query!
State/province [29]
0
0
Bochum
Query!
Country [30]
0
0
Germany
Query!
State/province [30]
0
0
Cologne
Query!
Country [31]
0
0
Germany
Query!
State/province [31]
0
0
Frankfurt am Main
Query!
Country [32]
0
0
Germany
Query!
State/province [32]
0
0
Jena
Query!
Country [33]
0
0
Germany
Query!
State/province [33]
0
0
Munchen
Query!
Country [34]
0
0
Germany
Query!
State/province [34]
0
0
Munich
Query!
Country [35]
0
0
Greece
Query!
State/province [35]
0
0
Thessaloniki
Query!
Country [36]
0
0
Israel
Query!
State/province [36]
0
0
Haifa
Query!
Country [37]
0
0
Israel
Query!
State/province [37]
0
0
Jerusalem
Query!
Country [38]
0
0
Italy
Query!
State/province [38]
0
0
Ancona
Query!
Country [39]
0
0
Italy
Query!
State/province [39]
0
0
Firenze
Query!
Country [40]
0
0
Italy
Query!
State/province [40]
0
0
Milan
Query!
Country [41]
0
0
Italy
Query!
State/province [41]
0
0
Rome
Query!
Country [42]
0
0
Italy
Query!
State/province [42]
0
0
Verona
Query!
Country [43]
0
0
Netherlands
Query!
State/province [43]
0
0
Zuid-Holland
Query!
Country [44]
0
0
Netherlands
Query!
State/province [44]
0
0
Nijmegen
Query!
Country [45]
0
0
Poland
Query!
State/province [45]
0
0
Gdansk
Query!
Country [46]
0
0
Poland
Query!
State/province [46]
0
0
Warsaw
Query!
Country [47]
0
0
Spain
Query!
State/province [47]
0
0
Barcelona
Query!
Country [48]
0
0
Spain
Query!
State/province [48]
0
0
Esplugues De Llobregat
Query!
Country [49]
0
0
Spain
Query!
State/province [49]
0
0
Malaga
Query!
Country [50]
0
0
Spain
Query!
State/province [50]
0
0
Sabadell
Query!
Country [51]
0
0
Spain
Query!
State/province [51]
0
0
Sevilla
Query!
Country [52]
0
0
United Kingdom
Query!
State/province [52]
0
0
Leeds
Query!
Country [53]
0
0
United Kingdom
Query!
State/province [53]
0
0
London
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
PTC Therapeutics
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
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.
Query!
Trial website
https://clinicaltrials.gov/study/NCT02456103
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Joseph McIntosh, MD
Query!
Address
0
0
PTC Therapeutics
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
Query!
No/undecided IPD sharing reason/comment
Query!
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/03/NCT02456103/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/03/NCT02456103/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT02456103