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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/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) - 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.
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 - 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.
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 - 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.
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 - 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.
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 - 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.
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 - 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.
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
Gender
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
N/A
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
Terminated
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)
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
Pennsylvania
Country [13] 0 0
United States of America
State/province [13] 0 0
Texas
Country [14] 0 0
United States of America
State/province [14] 0 0
Wisconsin
Country [15] 0 0
Argentina
State/province [15] 0 0
Buenos Aires
Country [16] 0 0
Belgium
State/province [16] 0 0
Brussels
Country [17] 0 0
Belgium
State/province [17] 0 0
Leuven
Country [18] 0 0
Brazil
State/province [18] 0 0
Porto Alegre
Country [19] 0 0
Bulgaria
State/province [19] 0 0
Plovdiv
Country [20] 0 0
Bulgaria
State/province [20] 0 0
Sofia
Country [21] 0 0
Canada
State/province [21] 0 0
Montreal
Country [22] 0 0
Canada
State/province [22] 0 0
Toronto
Country [23] 0 0
Canada
State/province [23] 0 0
Vancouver
Country [24] 0 0
France
State/province [24] 0 0
Montpellier
Country [25] 0 0
France
State/province [25] 0 0
Paris
Country [26] 0 0
France
State/province [26] 0 0
Roscoff
Country [27] 0 0
France
State/province [27] 0 0
Saint-Pierre
Country [28] 0 0
Germany
State/province [28] 0 0
Berlin
Country [29] 0 0
Germany
State/province [29] 0 0
Bochum
Country [30] 0 0
Germany
State/province [30] 0 0
Cologne
Country [31] 0 0
Germany
State/province [31] 0 0
Frankfurt am Main
Country [32] 0 0
Germany
State/province [32] 0 0
Jena
Country [33] 0 0
Germany
State/province [33] 0 0
Munchen
Country [34] 0 0
Germany
State/province [34] 0 0
Munich
Country [35] 0 0
Greece
State/province [35] 0 0
Thessaloniki
Country [36] 0 0
Israel
State/province [36] 0 0
Haifa
Country [37] 0 0
Israel
State/province [37] 0 0
Jerusalem
Country [38] 0 0
Italy
State/province [38] 0 0
Ancona
Country [39] 0 0
Italy
State/province [39] 0 0
Firenze
Country [40] 0 0
Italy
State/province [40] 0 0
Milan
Country [41] 0 0
Italy
State/province [41] 0 0
Rome
Country [42] 0 0
Italy
State/province [42] 0 0
Verona
Country [43] 0 0
Netherlands
State/province [43] 0 0
Zuid-Holland
Country [44] 0 0
Netherlands
State/province [44] 0 0
Nijmegen
Country [45] 0 0
Poland
State/province [45] 0 0
Gdansk
Country [46] 0 0
Poland
State/province [46] 0 0
Warsaw
Country [47] 0 0
Spain
State/province [47] 0 0
Barcelona
Country [48] 0 0
Spain
State/province [48] 0 0
Esplugues De Llobregat
Country [49] 0 0
Spain
State/province [49] 0 0
Malaga
Country [50] 0 0
Spain
State/province [50] 0 0
Sabadell
Country [51] 0 0
Spain
State/province [51] 0 0
Sevilla
Country [52] 0 0
United Kingdom
State/province [52] 0 0
Leeds
Country [53] 0 0
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/show/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/show/NCT02456103