Cystic FibrosisPhase 3NCT02107859
Study of Ataluren (PTC124) in Cystic Fibrosis
A Phase 3 study testing Ataluren in people with Cystic Fibrosis, run by PTC Therapeutics at 17 sites in 7 countries.
Trial summary
The primary objective of this study is to determine the long-term safety and tolerability of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) who completed participation in the double-blind study PTC124-GD-009-CF (NCT00803205), as assessed by adverse events and laboratory abnormalities. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Ataluren
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 61 participants
- Where
- 17 sitesin 7 countries
- Study dates
- 23 May 2014to 5 Jun 2017
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Ability to provide written informed consent (parental/guardian consent and participant assent if less than [<] 18 years of age).
- Evidence of completed participation in the double-blind study, PTC124-GD-009-CF (Study 009).
- Body weight greater than or equal to (≥) 16 kilograms (kg).
- Performance of a valid, reproducible spirometry test using the study-specific spirometer during the screening period.
- Confirmed laboratory values within the central laboratory ranges at screening.
- In male and female participants 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, laboratory tests, and study restrictions.
Who cannot join
- Chronic use of systemic tobramycin within 4 weeks prior to screening.
- Evidence of pulmonary exacerbation or acute upper or lower respiratory tract infection (including viral illnesses) within 3 weeks prior to screening or between screening and randomization.
- Any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation, or re-initiation) in a chronic treatment/prophylaxis regimen for CF or for CF-related conditions within 4 weeks prior to screening and randomization.
- Known hypersensitivity to any of the ingredients or excipients of the study drug.
- Exposure to another investigational drug within 4 weeks prior to screening.
- Treatment with intravenous antibiotics within 3 weeks prior to screening.
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- History of solid organ or hematological transplantation.
- Ongoing immunosuppressive therapy (other than corticosteroids).
- Positive hepatitis B surface antigen, hepatitis C antibody test or human immunodeficiency virus (HIV) test.
- Known portal hypertension.
- Pregnancy or breast-feeding.
Trial locations and contacts
17 sites in 7 countries.
17 sites in 7 countries
United States7 sites
Denver Children's Hospital
Aurora, Colorado, 80045
University of Alabama-Birmingham
Birmingham, Alabama, 35233
Children's Hospital Boston
Boston, Massachusetts, 02115
Children's Hospital Chicago
Chicago, Illinois, 60614
Rainbow Babies & Children's Hospital
Cleveland, Ohio, 44106
Miller Children's Hospital Long Beach
Long Beach, California, 90806
Beth Israel Medical Center
New York, New York, 10003
Belgium3 sites
Hôpital Universitaire des Enfants Reine Fabiola
Brussels
University Hospital Brussels
Brussels
University Hospital Leuven
Leuven
France2 sites
Hôpital Necker - Enfants Malades
Paris
Hôpital des Enfants
Toulouse, 31059
About this study
Official title: An Open-Label Safety and Efficacy Study for Patients With Nonsense Mutation Cystic Fibrosis Previously Treated With Ataluren (PTC124)
The primary objective of this study is to determine the long-term safety and tolerability of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) who completed participation in the double-blind study PTC124-GD-009-CF (NCT00803205), as assessed by adverse events and laboratory abnormalities. The secondary objective of this study includes the assessment of the efficacy of ataluren, as measured by forced expiratory volume in 1 second (FEV1) and pulmonary exacerbation rate, and other safety parameters (for example, 12-lead electrocardiogram [ECG] measurements, vital signs).
- Conditions
- Cystic Fibrosis
- Being tested
- Ataluren
- Keywords
- Cystic fibrosis, Nonsense mutation, Premature stop codon, PTC124, Ataluren
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AtalurenDrug
Also called PTC124
Ataluren will be administered per dose and schedule specified in the arm.
| Study group | Role | What they receive |
|---|---|---|
| Ataluren | Experimental | Participants will receive ataluren suspension orally 3 times a day (TID), 10 milligrams/kilogram (mg/kg) at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks. Receives: Drug: Ataluren |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
AE: any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Severity of an AE was classified as: mild (does not interfere with usual function), moderate (interferes to some extent with usual function), severe (interferes significantly with usual function), life threatening (results in potential threat to life), and fatal AEs. Drug-related AEs: AEs with a possible or probable relationship to study drug. Serious AEs: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention. TEAE: AE that occurred or worsened from first dose of study drug to 4 weeks after last dose of study drug. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.
Measured: Baseline (Day 1) up to end of study (Week 196)
Number of Participants With Clinically Significant Laboratory Abnormalities
Laboratory parameters tests included hematology, biochemistry assay (hepatic, renal, and serum electrolyte values), adrenal assays, and urinalysis. Clinical significance was defined as per investigator's judgement.
Measured: Baseline (Day 1) up to end of study (Week 196)
Secondary outcomes (7)
Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at the End of Treatment (Week 192), as Assessed by Spirometry
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Percent of predicted FEV1 = (observed value)/(predicted value) * 100%. Change from baseline in percent predicted FEV1 at the end of treatment was reported.
Measured: Baseline, Week 192
Percentage of Participants With Pulmonary Exacerbation, As Assessed by Modified Fuchs Criteria
The modified Fuchs' criteria defined exacerbation as the presence of at least 4 of the following 12 Fuchs' signs and symptoms without the requirement for treatment with antibiotics: change in sputum; new or increased hemoptysis; increased cough; increased dyspnea; fatigue; temperature greater than (>) 38 degrees celsius (°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.
Measured: Baseline up to Week 192
Percentage of Participants With Pulmonary Exacerbation, As Assessed by Expanded Fuchs' Criteria
The expanded Fuchs' criteria defined exacerbation as the presence of at least 4 of the following 12 Fuchs' signs and symptoms requiring any form of antibiotic treatment (inhaled, oral, or intravenous): 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.
Measured: Baseline up to Week 192
Percentage of Participants With Pulmonary Exacerbation, As Assessed by Classic Fuchs' Criteria
The Classic Fuchs' criteria defined exacerbation as the presence of at least 4 of the following 12 Fuchs' signs and symptoms requiring treatment with parenteral antibiotics: 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.
Measured: Baseline up to Week 192
Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Final Visit (Week 196)
ECG parameters included RR duration, PR duration, QRS duration, QT duration, QTCB (Bazett's correction formula) duration, QTCF (Fridericia's correction formula) duration.
Measured: Baseline, Week 196
Change From Baseline in Heart Rate at Final Visit (Week 196), as Assessed by 12-Lead ECG
Heart rate was measured using 12-lead ECG.
Measured: Baseline, Week 196
Change From Baseline in Vital Signs at Final Visit (Week 196)
Vital Signs included systolic blood pressure (SBP) and diastolic blood pressure (DBP).
Measured: Baseline, Week 196
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Apr 2014 (estimated)
- Study start23 May 2014
- Primary completion5 Jun 2017
- Study completion5 Jun 2017
Why it stopped: Based on the results of study PTC124-GD-021-CF (NCT02139306), clinical development of ataluren in cystic fibrosis was discontinued and this study was closed.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 61 participants (actual)
Sponsor and investigators
- Lead sponsor
- PTC Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Joseph McIntosh, MD, Study director, PTC Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02107859
- Sponsor's protocol ID
- PTC124-GD-023-CF
- Other IDs
- 2013-005449-35
- Status last verified
- Mar 2020
- First submitted
- 4 Apr 2014
- Last updated
- 25 Mar 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Aslam AA, Sinha IP, Southern KW. Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic fibrosis. Cochrane Database Syst Rev. 2023 Mar 3;3(3):CD012040. doi: 10.1002/14651858.CD012040.pub3.
PubMed 36866921
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Safety and Efficacy Study for Patients With Nonsense Mutation Cystic Fibrosis Previously Treated With Ataluren (PTC124)
- ClinicalTrials.gov ID
- NCT02107859
- Acronym / sponsor ID
- PTC124-GD-023-CF
- Conditions
- Cystic Fibrosis
- Keywords
- Cystic fibrosis, Nonsense mutation, Premature stop codon, PTC124, Ataluren
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Based on the results of study PTC124-GD-021-CF (NCT02139306), clinical development of ataluren in cystic fibrosis was discontinued and this study was closed.
- Enrolment
- 61 participants (actual)
- Lead sponsor
- PTC TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 23 May 2014 (actual)
- Primary completion
- 5 Jun 2017 (actual)
- Study completion
- 5 Jun 2017 (actual)
- First posted
- 8 Apr 2014 (estimated)
- Results
- Posted 25 Mar 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this study is to determine the long-term safety and tolerability of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) who completed participation in the double-blind study PTC124-GD-009-CF (NCT00803205), as assessed by adverse events and laboratory abnormalities. The secondary objective of this study includes the assessment of the efficacy of ataluren, as measured by forced expiratory volume in 1 second (FEV1) and pulmonary exacerbation rate, and other safety parameters (for example, 12-lead electrocardiogram [ECG] measurements, vital signs).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ataluren | Experimental | Drug: AtalurenParticipants will receive ataluren suspension orally 3 times a day (TID), 10 milligrams/kilogram (mg/kg) at morning, 10 mg/kg at midday, and 20 mg/kg at evening (total daily dose 40 mg/kg) for 192 weeks. |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Ability to provide written informed consent (parental/guardian consent and participant assent if less than [<] 18 years of age).
- Evidence of completed participation in the double-blind study, PTC124-GD-009-CF (Study 009).
- Body weight greater than or equal to (≥) 16 kilograms (kg).
- Performance of a valid, reproducible spirometry test using the study-specific spirometer during the screening period.
- Confirmed laboratory values within the central laboratory ranges at screening.
Key exclusion criteria (11 listed)
- Chronic use of systemic tobramycin within 4 weeks prior to screening.
- Evidence of pulmonary exacerbation or acute upper or lower respiratory tract infection (including viral illnesses) within 3 weeks prior to screening or between screening and randomization.
- Any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation, or re-initiation) in a chronic treatment/prophylaxis regimen for CF or for CF-related conditions within 4 weeks prior to screening and randomization.
- Known hypersensitivity to any of the ingredients or excipients of the study drug.
- Exposure to another investigational drug within 4 weeks prior to screening.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AtalurenAlso: PTC124Ataluren will be administered per dose and schedule specified in the arm. | Drug | Ataluren |
Primary outcome
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time frame: Baseline (Day 1) up to end of study (Week 196)
AE: any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Severity of an AE was classified as: mild (does not interfere with usual function), moderate (interferes to some extent with usual function), severe (interferes significantly with usual function), life threatening (results in potential threat to life), and fatal AEs. Drug-related AEs: AEs with a possible or probable relationship to study drug. Serious AEs: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention. TEAE: AE that occurred or worsened from first dose of study drug to 4 weeks after last dose of study drug. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.
Number of Participants With Clinically Significant Laboratory Abnormalities
Time frame: Baseline (Day 1) up to end of study (Week 196)
Laboratory parameters tests included hematology, biochemistry assay (hepatic, renal, and serum electrolyte values), adrenal assays, and urinalysis. Clinical significance was defined as per investigator's judgement.
7 secondary outcomes
Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at the End of Treatment (Week 192), as Assessed by Spirometry
Time frame: Baseline, Week 192
Percentage of Participants With Pulmonary Exacerbation, As Assessed by Modified Fuchs Criteria
Time frame: Baseline up to Week 192
Percentage of Participants With Pulmonary Exacerbation, As Assessed by Expanded Fuchs' Criteria
Time frame: Baseline up to Week 192
Percentage of Participants With Pulmonary Exacerbation, As Assessed by Classic Fuchs' Criteria
Time frame: Baseline up to Week 192
Change From Baseline in 12-Lead Electrocardiogram (ECG) Parameters at Final Visit (Week 196)
Time frame: Baseline, Week 196
Change From Baseline in Heart Rate at Final Visit (Week 196), as Assessed by 12-Lead ECG
Time frame: Baseline, Week 196
Change From Baseline in Vital Signs at Final Visit (Week 196)
Time frame: Baseline, Week 196
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 36866921Aslam AA, Sinha IP, Southern KW. Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic fibrosis. Cochrane Database Syst Rev. 2023 Mar 3;3(3):CD012040. doi: 10.1002/14651858.CD012040.pub3.
- PTC Therapeutics, Inc. websiteLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Joseph McIntosh, MD study director
PTC Therapeutics, Inc.
17 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Ataluren in people with Cystic Fibrosis, run by PTC Therapeutics at 17 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 25 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in United States, Belgium, France, Italy, Israel and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 25 Mar 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) (time frame: Baseline (Day 1) up to end of study (Week 196)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02107859, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Mar 2020; AzTrials last refreshed its copy on 8 Nov 2026.