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CompletedNCT02139306Updated May 14, 2020Results posted

Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)

A Phase 3 interventional study of Ataluren (PTC124®) and Placebo in Cystic Fibrosis, sponsored by PTC Therapeutics. Completed at 88 sites in 16 countries. Open to participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2020-05-14.

Sponsored by PTC Therapeutics · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
279
Allocation
Randomized
Ages
6 Years and older
Sex
All
01

Study summary

This is a Phase 3, international, multicenter, randomized, double-blind, placebo-controlled, efficacy and safety study of ataluren in patients with nonsense mutation cystic fibrosis (nmCF) not receiving chronic inhaled aminoglycosides.

Read the detailed description

This study is to enroll 208 subjects (184 fully evaluable) with nonsense-mutation-mediated CF who are at least 6 years of age and have an forced expiratory volume in 1 second (FEV1) >= 40% and \<= 90% of predicted. Subjects will be stratified based on age, inhaled antibiotic use, and baseline FEV1, and will be randomized in a 1:1 ratio to receive oral ataluren administered 3 times per day (TID) at respective morning, midday, and evening doses of 10-, 10-, and 20-mg/kg or placebo. Based on the results of a previously conducted study, patients treated with chronic inhaled aminoglycosides (including TOBI) will not be eligible for participation. Spirometry measurement at the screening visit will establish patient eligibility for inclusion based on lung function. FEV1 stability will be assessed during the approximately 4-week screening period, at the conclusion of which patients will be required to demonstrate a relative change in %-predicted FEV1 of less than 15% when compared to the screening value. Assessments will be performed every 8 weeks, depending upon the outcome measure.

02

Conditions studied

  • Cystic Fibrosis
03

In context

Cystic Fibrosis

1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.

This study's enrollment of 279 is above the median of 36 across 1,034 interventional studies indexed under Cystic Fibrosis.

Browse Cystic Fibrosis studies →

Lead sponsor

PTC Therapeutics is the lead sponsor of 65 studies on the registry; 3 are open to participants now.

Of its 18 completed or terminated interventional studies of FDA-regulated products, 14 (78%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
6 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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

Exclusion criteria

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
05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
279 participants (actual)

Study arms

  • 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.

    Drug: Ataluren (PTC124®)

  • Placebo comparator
    Placebo

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

    Drug: Placebo

Interventions

  • DrugAtaluren (PTC124®)

    Oral Ataluren TID

  • DrugPlacebo

    Oral Placebo TID

06

What researchers measure

Primary outcomes

  1. Absolute Change From Baseline in Percent-predicted Forced Expiratory Volume in One Second (ppFEV1) at Week 48

    The FEV1 is the volume of air forcibly exhaled in one second and is measured using forced expiratory air spirometry. Change in ppFEV1 at Week 48 was defined as the average between the change from baseline at Week 40 and that at Week 48. Baseline for ppFEV1 was defined as an average of ppFEV1 at Screening (Weeks -4 to -1) and Baseline (Day 1) visits.

    Time frame: From Baseline to Week 48

Secondary outcomes

  1. 48-week Rate of Pulmonary Exacerbations

    Pulmonary exacerbations were assessed using expanded Fuchs criteria. The expanded Fuchs exacerbation is defined as the presence of at least 4 of 12 Fuchs' signs and symptoms requiring treatment with any form of antibiotic treatment (inhaled, oral, or intravenous). Fuchs' signs and symptoms included increased cough; change in sputum volume, color, or consistency; new or increased hemoptysis; increased dyspnea during moderate or mild exertion, or at rest; sinus pain or tenderness; change in sinus discharge; malaise, fatigue, or lethargy; anorexia or weight loss; temperature above 38 degrees Celsius; change in findings on chest examination; relative 10% decrease in ppFEV1, and chest radiography results consistent with pulmonary infection. The 48-week rate was calculated as: 48-week rate = total number of events /treatment duration by week\*48.

    Time frame: Week 48

  2. Change From Baseline in the Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain at Week 48

    The teen/adult CFQ-R was used for this study. It was developed specifically for participants with cystic fibrosis. It is a disease-specific instrument designed to measure impact on overall health, daily life, perceived well-being, and symptoms. The respiratory domain assessed respiratory symptoms like coughing, congestion, wheezing etc. Scaling of each item is done via 4-point Likert scales. Scores for each item are summed up to generate a domain score. Scores ranges from 0 to 100, with higher scores indicating better health and lower scores indicating worse health.

    Time frame: Baseline (Day 1) and Week 48

  3. Change From Baseline in Body Mass Index (BMI) at Week 48

    Malnutrition is common in participants with cystic fibrosis. The BMI is an important clinical measure of nutritional status.

    Time frame: Baseline (Day 1) and Week 48

  4. Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs)

    An adverse event (AE) is any unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a study treatment, whether or not considered related to the study treatment. A TEAE is defined as an AE that occurs or worsens in the period extending from first dose of study drug to 4 weeks after last dose of study drug. An SAE is an untoward medical occurrence or effect associated with the use of a study drug at any dose, regardless of whether it is considered to be related to the study drug, which results in one of the following: death; inpatient hospitalization or prolongation of existing hospitalization; life threatening adverse event; persistent or significant disability or incapacity or substantial disruption of the ability to conduct normal life functions; any other medically important event; or a pregnancy resulting in spontaneous abortion, stillbirth, neonatal death, or congenital anomaly.

    Time frame: From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)

  5. Number of Participants With TEAEs by Severity and Relationship to Study Drugs

    The relationship of TEAEs to the study drugs were assessed as: probable related, possibly related, unlikely related, and unrelated. The severity of TEAEs were graded using the Common Terminology Criteria for Adverse Events, Version 3.0 as: Grade 1 (mild), Grade 2 (moderate), Grade 3 (severe), Grade 4 (life threatening), or Grade 5 (fatal).

    Time frame: From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)

  6. Number of Participants With SAEs by Severity and Relationship to Study Drugs

    The relationship of SAEs to the study drugs were assessed as: probable related, possibly related, unlikely related, and unrelated. The severity of SAEs were graded using the Common Terminology Criteria for Adverse Events, Version 3.0 as: Grade 1 (mild), Grade 2 (moderate), Grade 3 (severe), Grade 4 (life threatening), or Grade 5 (fatal).

    Time frame: From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)

  7. Number of Participants With Abnormal Vital Signs Reported as TEAEs

    Vital signs included systolic and diastolic blood pressure, pulse rate, pulse oximetry, and body temperature. Participants with abnormal vital signs who required clinical intervention or further investigation (beyond ordering a repeat \[confirmatory\] test) unless they are associated with an already reported clinical event are reported.

    Time frame: From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)

  8. Number of Participants With Abnormal Clinical Laboratory Parameters Reported as TEAEs

    Clinical laboratory tests included haematology, biochemistry, and urinalysis. Participants with abnormal laboratory parameters who required clinical intervention or further investigation (beyond ordering a repeat \[confirmatory\] test) unless they are associated with an already reported clinical event are reported.

    Time frame: From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)

  9. Number of Participants With Abnormal Electrocardiogram Reported as TEAEs

    Participants with abnormal electrocardiogram who required clinical intervention or further investigation (beyond ordering a repeat \[confirmatory\] test) unless they are associated with an already reported clinical event are reported.

    Time frame: From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)

07

Results

Posted May 14, 2020

Participant flow

This study was conducted from 15 August 2014 to 02 November 2016.

Participant flow — Overall Study
MilestoneAtalurenPlacebo
Started140139
Completed127125
Not completed1314
Withdrew: Withdrawal by subject46
Withdrew: Adverse event34
Withdrew: Physician decision01
Withdrew: Other unspecified23
Withdrew: Protocol violation40

Outcome measures

PrimaryAbsolute Change From Baseline in Percent-predicted Forced Expiratory Volume in One Second (ppFEV1) at Week 48

The FEV1 is the volume of air forcibly exhaled in one second and is measured using forced expiratory air spirometry. Change in ppFEV1 at Week 48 was defined as the average between the change from baseline at Week 40 and that at Week 48. Baseline for ppFEV1 was defined as an average of ppFEV1 at Screening (Weeks -4 to -1) and Baseline (Day 1) visits.

Time frame:
From Baseline to Week 48
Reported as:
Least squares mean · Percentage of predicted FEV1
Absolute Change From Baseline in Percent-predicted Forced Expiratory Volume in One Second (ppFEV1) at Week 48
Percentage of predicted FEV1AtalurenPlacebo
Absolute Change From Baseline in Percent-predicted Forced Expiratory Volume in One Second (ppFEV1) at Week 48-1.396 (-2.7735 to -0.0180)-1.992 (-3.3271 to -0.6576)
Statistical analysis
  • Ataluren vs Placebo · Mixed-model, repeated-measures · p = 0.5336 · Mean difference (net): 0.597 · 95% CI -1.2881 to 2.4813
Secondary48-week Rate of Pulmonary Exacerbations

Pulmonary exacerbations were assessed using expanded Fuchs criteria. The expanded Fuchs exacerbation is defined as the presence of at least 4 of 12 Fuchs' signs and symptoms requiring treatment with any form of antibiotic treatment (inhaled, oral, or intravenous). Fuchs' signs and symptoms included increased cough; change in sputum volume, color, or consistency; new or increased hemoptysis; increased dyspnea during moderate or mild exertion, or at rest; sinus pain or tenderness; change in sinus discharge; malaise, fatigue, or lethargy; anorexia or weight loss; temperature above 38 degrees Celsius; change in findings on chest examination; relative 10% decrease in ppFEV1, and chest radiography results consistent with pulmonary infection. The 48-week rate was calculated as: 48-week rate = total number of events /treatment duration by week\*48.

Time frame:
Week 48
Reported as:
Mean · number of exacerbations per 48 weeks
48-week Rate of Pulmonary Exacerbations
number of exacerbations per 48 weeksAtalurenPlacebo
48-week Rate of Pulmonary Exacerbations0.950 ± 1.40381.127 ± 2.5241
Statistical analysis
  • Ataluren vs Placebo · Negative binomial regression · p = 0.4008 · Rate ratio: 0.8567 · 95% CI 0.5973 to 1.2288
SecondaryChange From Baseline in the Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain at Week 48

The teen/adult CFQ-R was used for this study. It was developed specifically for participants with cystic fibrosis. It is a disease-specific instrument designed to measure impact on overall health, daily life, perceived well-being, and symptoms. The respiratory domain assessed respiratory symptoms like coughing, congestion, wheezing etc. Scaling of each item is done via 4-point Likert scales. Scores for each item are summed up to generate a domain score. Scores ranges from 0 to 100, with higher scores indicating better health and lower scores indicating worse health.

Time frame:
Baseline (Day 1) and Week 48
Reported as:
Least squares mean · units on a scale
Change From Baseline in the Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain at Week 48
units on a scaleAtalurenPlacebo
Change From Baseline in the Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain at Week 48-0.760 (-3.4566 to 1.9364)-1.032 (-3.7368 to 1.6728)
Statistical analysis
  • Ataluren vs Placebo · Mixed-model, repeated measures · p = 0.8881 · Mean difference (net): 0.272 · 95% CI -3.5292 to 4.0731
SecondaryChange From Baseline in Body Mass Index (BMI) at Week 48

Malnutrition is common in participants with cystic fibrosis. The BMI is an important clinical measure of nutritional status.

Time frame:
Baseline (Day 1) and Week 48
Reported as:
Least squares mean · kilogram per meter square (kg/m^2)
Change From Baseline in Body Mass Index (BMI) at Week 48
kilogram per meter square (kg/m^2)AtalurenPlacebo
Change From Baseline in Body Mass Index (BMI) at Week 480.296 (0.1126 to 0.4789)0.361 (0.1759 to 0.5455)
Statistical analysis
  • Ataluren vs Placebo · Mixed-model, repeated measures · p = 0.6208 · Mean difference (net): -0.065 · 95% CI -0.3233 to 0.1934
SecondaryNumber of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs)

An adverse event (AE) is any unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a study treatment, whether or not considered related to the study treatment. A TEAE is defined as an AE that occurs or worsens in the period extending from first dose of study drug to 4 weeks after last dose of study drug. An SAE is an untoward medical occurrence or effect associated with the use of a study drug at any dose, regardless of whether it is considered to be related to the study drug, which results in one of the following: death; inpatient hospitalization or prolongation of existing hospitalization; life threatening adverse event; persistent or significant disability or incapacity or substantial disruption of the ability to conduct normal life functions; any other medically important event; or a pregnancy resulting in spontaneous abortion, stillbirth, neonatal death, or congenital anomaly.

Time frame:
From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)
Reported as:
Number · participants
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (SAEs)
participantsAtalurenPlacebo
TEAEs133135
SAEs4046
SecondaryNumber of Participants With TEAEs by Severity and Relationship to Study Drugs

The relationship of TEAEs to the study drugs were assessed as: probable related, possibly related, unlikely related, and unrelated. The severity of TEAEs were graded using the Common Terminology Criteria for Adverse Events, Version 3.0 as: Grade 1 (mild), Grade 2 (moderate), Grade 3 (severe), Grade 4 (life threatening), or Grade 5 (fatal).

Time frame:
From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)
Reported as:
Number · participants
Number of Participants With TEAEs by Severity and Relationship to Study Drugs
participantsAtalurenPlacebo
Severity: Grade 12618
Severity: Grade 28888
Severity: Grade 31929
Severity: Grade 400
Severity: Grade 500
Relationship to study drug: Unrelated7472
Relationship to study drug: Unlikely related3734
Relationship to study drug: Possible related2125
Relationship to study drug: Probable related14
SecondaryNumber of Participants With SAEs by Severity and Relationship to Study Drugs

The relationship of SAEs to the study drugs were assessed as: probable related, possibly related, unlikely related, and unrelated. The severity of SAEs were graded using the Common Terminology Criteria for Adverse Events, Version 3.0 as: Grade 1 (mild), Grade 2 (moderate), Grade 3 (severe), Grade 4 (life threatening), or Grade 5 (fatal).

Time frame:
From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)
Reported as:
Number · participants
Number of Participants With SAEs by Severity and Relationship to Study Drugs
participantsAtalurenPlacebo
Severity: Grade 121
Severity: Grade 22320
Severity: Grade 31525
Severity: Grade 400
Severity: Grade 500
Relationship to study drug: Unrelated2631
Relationship to study drug: Unlikely related1315
Relationship to study drug: Possible related10
Relationship to study drug: Probable related00
SecondaryNumber of Participants With Abnormal Vital Signs Reported as TEAEs

Vital signs included systolic and diastolic blood pressure, pulse rate, pulse oximetry, and body temperature. Participants with abnormal vital signs who required clinical intervention or further investigation (beyond ordering a repeat \[confirmatory\] test) unless they are associated with an already reported clinical event are reported.

Time frame:
From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)
Reported as:
Number · participants
Number of Participants With Abnormal Vital Signs Reported as TEAEs
participantsAtalurenPlacebo
Number of Participants With Abnormal Vital Signs Reported as TEAEs01
SecondaryNumber of Participants With Abnormal Clinical Laboratory Parameters Reported as TEAEs

Clinical laboratory tests included haematology, biochemistry, and urinalysis. Participants with abnormal laboratory parameters who required clinical intervention or further investigation (beyond ordering a repeat \[confirmatory\] test) unless they are associated with an already reported clinical event are reported.

Time frame:
From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)
Reported as:
Number · participants
Number of Participants With Abnormal Clinical Laboratory Parameters Reported as TEAEs
participantsAtalurenPlacebo
Number of Participants With Abnormal Clinical Laboratory Parameters Reported as TEAEs3230
SecondaryNumber of Participants With Abnormal Electrocardiogram Reported as TEAEs

Participants with abnormal electrocardiogram who required clinical intervention or further investigation (beyond ordering a repeat \[confirmatory\] test) unless they are associated with an already reported clinical event are reported.

Time frame:
From study drug administration to 4-week post treatment follow-up visit (approximately 52 weeks)
Reported as:
Number · participants
Number of Participants With Abnormal Electrocardiogram Reported as TEAEs
participantsAtalurenPlacebo
Number of Participants With Abnormal Electrocardiogram Reported as TEAEs10

Adverse events

Collected over From study drug administration to 4-week post treatment follow-up visit (approximately 52 Weeks). Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Ataluren0/140 (0%)40/140 (28.6%)123/140 (87.9%)
Placebo0/139 (0%)46/139 (33.1%)120/139 (86.3%)
Most frequent serious events
Showing 10 of 37
Most frequent serious events
EventAtalurenPlacebo
Infective pulmonary exacerbation of cystic fibrosisInfections and infestations25/14034/139
HaemoptysisRespiratory, thoracic and mediastinal disorders4/1401/139
NephrolithiasisRenal and urinary disorders3/1400/139
PneumoniaInfections and infestations1/1402/139
SinusitisInfections and infestations0/1402/139
Pseudomonas infectionInfections and infestations2/1400/139
BronchopneumoniaInfections and infestations1/1401/139
GastroenteritisInfections and infestations0/1401/139
Mycobacterium abscessus infectionInfections and infestations0/1401/139
Pneumonia staphylococcalInfections and infestations0/1401/139
Most frequent other events
Showing 10 of 19
Most frequent other events
EventAtalurenPlacebo
Infective pulmonary exacerbation of cystic fibrosisInfections and infestations75/14078/139
Viral upper respiratory tract infectionInfections and infestations27/14021/139
CoughRespiratory, thoracic and mediastinal disorders25/14025/139
Upper respiratory tract infectionInfections and infestations21/14021/139
HeadacheNervous system disorders12/14016/139
SinusitisInfections and infestations16/14011/139
DiarrhoeaGastrointestinal disorders13/14012/139
HaemoptysisRespiratory, thoracic and mediastinal disorders12/14010/139
NauseaGastrointestinal disorders11/14010/139
Abdominal painGastrointestinal disorders11/1405/139

Baseline characteristics

The as-treated population included all randomized participants who actually received any study treatment.

Age, Continuous
Age, Continuous(years)AtalurenPlaceboTotal
Mean22.0 ± 11.022.0 ± 10.4422.0 ± 10.70
Sex: Female, Male
Sex: Female, Male(Participants)AtalurenPlaceboTotal
Female5974133
Male8165146
08

Study locations

88 sites
  • University of Alabama at Birmingham
    Birmingham, Alabama 35233, United States
  • Pulmonary Associates of Mobile PC
    Mobile, Alabama 36608, United States
  • Miller Children's Hospital Long Beach
    Long Beach, California 90806, United States
  • Children's Hospital Los Angeles
    Los Angeles, California 90027, United States
  • Children's Hospital and Research Center at Oakland
    Oakland, California 94609, United States
  • Stanford University-Children's Hospital
    Palo Alto, California 94304, United States
  • Children's Hospital Colorado
    Aurora, Colorado 80045, United States
  • Nemours Children's Clinic
    Jacksonville, Florida 32207, United States
  • University of Miami
    Miami, Florida 33136, United States
  • Miami Children's Hospital
    Miami, Florida 33155, United States
  • All Children's Hospital
    Saint Petersburg, Florida 33701, United States
  • Ann and Robert H. Lurie Children's Hospital of Chicago
    Chicago, Illinois 60611, United States
  • Indiana University
    Indianapolis, Indiana 46202, United States
  • Children's Hospital Boston
    Boston, Massachusetts 02115, United States
  • Washington University
    Saint Louis, Missouri 63110, United States
  • Monmouth Medical Center
    Long Branch, New Jersey 07740, United States
  • Morristown Medical Center
    Morristown, New Jersey 07960, United States
  • Beth Israel Medical Center
    New York, New York 10003, United States
  • New York University Langone Medical Center
    New York, New York 10016, United States
  • Columbia University Medical Center
    New York, New York 10032, United States
  • SUNY Upstate Medical University
    Syracuse, New York 13210, United States
  • University of Cincinnati
    Cincinnati, Ohio 45221, United States
  • Rainbow Babies & Children's Hospital
    Cleveland, Ohio 44106, United States
  • Santiago Reyes, MD
    Oklahoma City, Oklahoma 73112, United States
  • Penn State Milton S. Hershey Medical Center
    Hershey, Pennsylvania 17033, United States
  • Children's Hospital of Philadelphia
    Philadelphia, Pennsylvania 19104, United States
  • Drexel University College of Medicine
    Philadelphia, Pennsylvania 19129, United States
  • Texas Children's Hospital
    Houston, Texas 77094, United States
  • University of Texas Health Science Center
    Tyler, Texas 75708, United States
  • Childrens Hospital of Wisconsin
    Milwaukee, Wisconsin 53226, United States
  • Hospital Universitario Austral
    Buenos Aires, B1629ODT, Argentina
  • Hospital de Niños Dr. Ricardo Gutiérrez
    Buenos Aires, C1425EFD, Argentina
  • Hospital de Niños Superiora Sor Maria Ludovica
    La Plata, 1900, Argentina
  • Royal Adelaide Hospital
    Adelaide, 5000, Australia
  • Prince Charles Hospital
    Chermside, 4032, Australia
  • Princess Margaret Hospital
    Perth, 6840, Australia
  • University Hospital Brussels
    Brussels, 1090, Belgium
  • Hôpital Universitaire des Enfants Reine Fabiola
    Bruxelles, 1020, Belgium
  • University Hospital Leuven
    Leuven, 3000, Belgium
  • Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul
    Porto Alegre, Brazil
  • Instituto da Criança - Hospital das Clínicas
    São Paulo, 05403-000, Brazil
  • University Mulitiprofile Hospital for Active Treatment Sveti Georgi EAD
    Plovdiv, Bulgaria
  • University Multiprofile Hospital for Active Treatment Aleksandrovska EAD
    Sofia, Bulgaria
  • Clinical Research Institute of Montreal
    Montreal, H2W 1R7, Canada
  • CHU de Quebec - Hopital CHUL
    Québec City, G1V 4G2, Canada
  • University of Toronto Hospital for Sick Children
    Toronto, M5G 1X8, Canada
  • British Columbia Children's Hospital
    Vancouver, V6H 3V4, Canada
  • Hôpital Femme-Mère-Enfant
    Bron, 69677, France
  • Hôpital Arnaud de Villeneuve
    Montpellier, 34295, France
  • Hôpital Necker-Enfants Malades
    Paris, 75015, France
  • Centre de Perharidy
    Roscoff, 29684, France
  • Centre Hospitalier Regional Sud Reunion
    Saint-Pierre, 97448, France
  • Charité Universitätsmedizin Berlin
    Berlin, 13353, Germany
  • St. Josef Hospital GmbH
    Bochum, 44791, Germany
  • Universitätsklinikum Köln
    Cologne, 50937, Germany
  • Christiane Herzog CF-Zentrum
    Frankfurt am Main, 60590, Germany
  • Universitätsklinikum Jena
    Jena, 07745, Germany
  • LMU Klinikum der Universität München
    München, 80336, Germany
  • Dr. Von Haunersches Kinderspital
    München, 80337, Germany
  • General Hospital of Thessaloniki Ippokration
    Thessaloniki, Greece
  • Meyer Children's Hospital
    Haifa, 31096, Israel
  • Hadassah University Hospital
    Jerusalem, 91240, Israel
  • Ospedali Riuniti di Ancona
    Ancona, 60123, Italy
  • Azienda Ospedaliera A Meyer
    Firenze, 50139, Italy
  • Lombardia Cystic Fibrosis Center
    Milan, 20122, Italy
  • Ospedale Pediatrico Bambino Gesù IRCCS
    Roma, Italy
  • Azienda Policlinico Umberto I
    Rome, 00161, Italy
  • University of Verona
    Verona, 37126, Italy
  • Hagaziekenhuis
    Den Haag, 2491, Netherlands
  • Radboud University
    Nijmegen, 6525 GA, Netherlands
  • Erasmus MC
    Rotterdam, Netherlands
  • Szpital Dzieciecy Polanki im Macieja Plazynskiego w Gdansku
    Gdansk, Poland
  • NZOZ Sanatorium Cassia-Villa Medica
    Rabka-Zdrój, 34-700, Poland
  • NZOZ Podkarpacki Osrodek Pulmonologii i Alergologii
    Rzeszow, 35-612, Poland
  • Instytut Matki I Dziecka
    Warsaw, 01-211, Poland
  • Hospital Universitario Vall d'Hebron
    Barcelona, 08035, Spain
  • Hospital University
    Barcelona, 08035, Spain
  • Hospital Sant Joan de Deu
    Esplugues De Llobregat, 08950, Spain
  • Hospital Regional Universitario de Malaga
    Málaga, Spain
  • Hospital de Sabadell, Consorci Sanitari Parc Tauli
    Sabadell, 08208, Spain
  • Hospital Universitario Virgen del Rocio
    Sevilla, Spain
  • Birmingham Children's Hospital NHS Foundation Trust
    Birmingham, United Kingdom
  • Heart of England NHS Foundation Trust
    Birmingham, United Kingdom
  • Southern General Hospital
    Glasgow, G120YN, United Kingdom
  • St James's University Hospital
    Leeds, United Kingdom
  • Royal Brompton Hospital
    London, United Kingdom
  • Llandough Hospital
    Penarth, CF64 2XX, United Kingdom
  • Southampton University Hospitals NHS Trust
    Southampton, United Kingdom
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References and documents

Publications

  • VanDevanter DR, Hamblett NM, Simon N, McIntosh J, Konstan MW. Evaluating assumptions of definition-based pulmonary exacerbation endpoints in cystic fibrosis clinical trials. J Cyst Fibros. 2021 Jan;20(1):39-45. doi: 10.1016/j.jcf.2020.07.008. Epub 2020 Jul 15. PubMed 32682670 ↗
  • Konstan MW, VanDevanter DR, Rowe SM, Wilschanski M, Kerem E, Sermet-Gaudelus I, DiMango E, Melotti P, McIntosh J, De Boeck K; ACT CF Study Group. Efficacy and safety of ataluren in patients with nonsense-mutation cystic fibrosis not receiving chronic inhaled aminoglycosides: The international, randomized, double-blind, placebo-controlled Ataluren Confirmatory Trial in Cystic Fibrosis (ACT CF). J Cyst Fibros. 2020 Jul;19(4):595-601. doi: 10.1016/j.jcf.2020.01.007. Epub 2020 Jan 23. PubMed 31983658 ↗

Related links

Individual participant data

Plan to share: No

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on May 14, 2020, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
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Registry details

Key details

Study ID
NCT02139306
Lead sponsor
PTC Therapeutics
Collaborators
Cystic Fibrosis Foundation, ECFS-Clinical Trial Network (ECFS-CTN)
Responsible party
Sponsor
First posted
May 15, 2014
Start date
Aug 2014
Primary completion
Nov 2016
Completion
Nov 2016
Results posted
May 14, 2020
Last update
May 14, 2020

Study contacts

Joseph McIntosh, MD
study director · PTC Therapeutics

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in May 2020. You cannot join it, but the record below documents what was studied.

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