Cystic FibrosisPhase 3NCT02456103
Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis
A Phase 3 study testing Ataluren in people with Cystic Fibrosis, run by PTC Therapeutics at 70 sites in 16 countries.
Trial 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. 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
- 246 participants
- Where
- 70 sitesin 16 countries
- Study dates
- 31 Aug 2015to 2 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
- 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.
Who cannot join
- Known hypersensitivity to any of the ingredients or excipients of the study drug.
- Ongoing participation in any other therapeutic clinical trial.
Trial locations and contacts
70 sites in 16 countries.
70 sites in 16 countries
United States24 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
University of Alabama at Birmingham
Birmingham, Alabama, 35233
Children's Hospital Boston
Boston, Massachusetts, 02115
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
University of Cincinnati
Cincinnati, Ohio, 45221
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033
Texas Children's Hospital
Houston, Texas, 77094
Indiana University
Indianapolis, Indiana, 46202
Nemours Children's Clinic
Jacksonville, Florida, 32207
Miller Children's Hospital Long Beach
Long Beach, California, 90806
Children's Hospital Los Angeles
Los Angeles, California, 90027
University of Miami
Miami, Florida, 33136
Miami Children's Hospital
Miami, Florida, 33155
Childrens Hospital of Wisconsin
Milwaukee, Wisconsin, 53226
Morristown Medical Center
Morristown, New Jersey, 07960
Beth Israel Medical Center
New York, New York, 10011
New York University Langone Medical Center
New York, New York, 10016
Columbia University Medical Center
New York, New York, 10032
Children's Hospital and Research Center at Oakland
Oakland, California, 94609
Stanford University-Children's Hospital
Palo Alto, California, 94304
Drexel University College of Medicine
Philadelphia, Pennsylvania, 19129
Washington University
St Louis, Missouri, 63110
All Children's Hospital
St. Petersburg, Florida, 33701
University of Texas Health Science Center
Tyler, Texas, 75708
Germany7 sites
Charite-Universitatsmedizin Berlin
Berlin, 10117
St. Josef Hospital GmbH
Bochum, 44791
University of Cologne Children's Hospital
Cologne, 50937
Christiane Herzog CF-Zentrum
Frankfurt am Main, 60590
Universitatsklinikum Jena
Jena, 1
Dr. Von Haunersches Kinderspital
München, 80337
LMU Klinikum der Universitat Muchen
Munich, 80539
Italy6 sites
Azienda Ospedaliero Universitaria Ospedall Riuniti di Acona-Umberto I G.M. Lancisi G. Salesi
Ancona, 71
Azienda Ospedaliera A Meyer
Florence, 50139
Lombardia Cystic Fibrosis Center
Milan, 20122
Azienda Policlinico Umberto I
Rome, 00161
Ospedale Pediatrico Bambino Gesu
Rome, 4
Azienda Ospedaliera di Verona
Verona
Spain6 sites
Hospital Universitario Vall d'Hebron
Barcelona, 08035
Hospital University
Barcelona, 08035
Hospital San Juan
Esplugues de Llobregat, 08950
Hospital Regional Universitario de Malaga
Málaga, 29001
Hospital de Sabadell, Consorci Sanitari Parc Tauli
Sabadell, 08208
Hospital Universitario Virgen del Rocio
Seville, 41013
France4 sites
Hoptial Arnaud de Villeneuve
Montpellier, 34295
Hopital Necker - Enfants Malades
Paris, 75015
Centre de Perharidy
Roscoff, 29684
Centre Hospitalier Regional Sud Reunion
Saint-Pierre, 97448
Australia3 sites
Royal Adelaide Hospital
Adelaide, 5000
Prince Charles Hospital
Chermside, 4032
Princess Margaret Hospital
Perth, 6840
Belgium3 sites
University Hospital Brussels
Brussels, 1090
Hopital Universitaire des Enfants Reine Fabiola
Brussels, 15
University Hospital Leuven
Leuven, 3000
Canada3 sites
Clinical Research Institute of Montreal
Montreal, H2W 1R7
University of Toronto Hospital for Sick Children
Toronto, M5G 1X8
British Columbia Children's Hospital
Vancouver, V6H 3V4
Argentina2 sites
Hospital de Ninos Ricardo Gutierrez
Buenos Aires, 1330
Hospital Universitario Austral
Buenos Aires
Bulgaria2 sites
University Mulitiprofile Hospital for Active Treatment Sveti Georgi EAD
Plovdiv
University Multiprofile Hospital for Active Treatment Aleksandrovska EAD
Sofia
Israel2 sites
Meyer Children's Hospital
Haifa, 31096
Hadassah University Hospital
Jerusalem, 91240
Netherlands2 sites
Radboud University
Nijmegen, 6525 GA
Hagaziekenhuis
s-Gravenweg, South Holland, 2545 CH
Poland2 sites
Szpital Dzieciecy Polanki im Macieja Plazynskiego w Gdansku
Gdansk
Institute of Mother and Child
Warsaw, 01-211
United Kingdom2 sites
St James University Hospital
Leeds, LS9 7TF
Royal Brompton Hospital
London, SW3 6NP
Brazil1 site
Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul
Porto Alegre
About this study
Official title: Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Cystic Fibrosis
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.
Read the detailed description
The primary objective of this Phase 3 extension study will be to obtain long-term safety data to augment the overall safety database. The secondary objectives will be to augment the efficacy data collected in the double-blind study (PTC124-GD-021-CF; NCT02139306).
- Conditions
- Cystic Fibrosis
- Being tested
- 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, Translarna
Ataluren will be provided as a vanilla-flavored powder to be mixed with water or milk.
| Study group | Role | What they receive |
|---|---|---|
| Ataluren | Experimental | 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. 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)
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.
Measured: Baseline up to Week 100
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.
Measured: Baseline up to Week 100
Secondary outcomes (4)
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.
Measured: Baseline, Week 24
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.
Measured: Baseline, Week 24
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.
Measured: Baseline, Week 24
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.
Measured: Baseline up to Week 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 May 2015 (estimated)
- Study start31 Aug 2015
- Primary completion2 Jun 2017
- Study completion2 Jun 2017
Why it stopped: Cystic Fibrosis (CF) data from the double-blind CF Study PTC124-GD-021-CF did not meet endpoints.
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.
- 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
- 246 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
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02456103
- Sponsor's protocol ID
- PTC124-GD-021e-CF
- Status last verified
- Apr 2020
- First submitted
- 26 May 2015
- Last updated
- 27 Apr 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 3 Extension Study of Ataluren (PTC124) in Patients With Nonsense Mutation Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT02456103
- Acronym / sponsor ID
- PTC124-GD-021e-CF
- Conditions
- Cystic Fibrosis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Cystic Fibrosis (CF) data from the double-blind CF Study PTC124-GD-021-CF did not meet endpoints.
- Enrolment
- 246 participants (actual)
- Lead sponsor
- PTC TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 31 Aug 2015 (actual)
- Primary completion
- 2 Jun 2017 (actual)
- Study completion
- 2 Jun 2017 (actual)
- First posted
- 28 May 2015 (estimated)
- Results
- Posted 15 Apr 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
The primary objective of this Phase 3 extension study will be to obtain long-term safety data to augment the overall safety database. The secondary objectives will be to augment the efficacy data collected in the double-blind study (PTC124-GD-021-CF; NCT02139306).
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 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. |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- 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.
Key exclusion criteria (2 listed)
- Known hypersensitivity to any of the ingredients or excipients of the study drug.
- Ongoing participation in any other therapeutic clinical trial.
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: PTC124, TranslarnaAtaluren will be provided as a vanilla-flavored powder to be mixed with water or milk. | Drug | Ataluren |
Primary outcome
Number of Participants With Treatment Emergent Adverse Events (TEAEs)
Time frame: Baseline up to Week 100
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.
Number of Participants With a Clinically Meaningful Abnormal Clinical Laboratory (Serum Biochemistry, Hematology, and Urinalysis) Parameter
Time frame: Baseline up to Week 100
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.
4 secondary outcomes
Change From Baseline in Percent-Predicted Forced Expiratory Volume in 1 Second (FEV1) as Measured by Spirometry at Week 24
Time frame: Baseline, Week 24
Change From Baseline in Percent-Predicted of Forced Vital Capacity (FVC) as Measured by Spirometry at Week 24
Time frame: Baseline, Week 24
Change From Baseline in Forced Expiratory Flow Between 25% and 75% of Expiration (FEF25-75) as Measured by Spirometry at Week 24
Time frame: Baseline, Week 24
Rate of Pulmonary Exacerbations as Defined by Modified Fuch's Criteria Over 48 Weeks
Time frame: Baseline up to Week 48
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Apr 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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
70 sites in 16 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 70 sites in 16 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 2 inclusion and 2 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 27 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 70 sites in United States, Germany, Italy, Spain, France and 11 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 15 Apr 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 up to Week 100). 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?
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 27 Apr 2020.