Study of Ataluren (PTC124™) in Cystic Fibrosis
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ClinicalTrials.gov Identifier: NCT00803205 |
Recruitment Status
:
Completed
First Posted
: December 5, 2008
Last Update Posted
: June 6, 2017
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Cystic Fibrosis | Drug: Ataluren (PTC124) Drug: Placebo | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 238 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis |
Actual Study Start Date : | July 2009 |
Actual Primary Completion Date : | November 2011 |
Actual Study Completion Date : | October 2012 |

Arm | Intervention/treatment |
---|---|
Experimental: Ataluren (PTC124)
10-,10-,20-mg/kg TID at morning, midday and evening doses for 48 weeks.
|
Drug: Ataluren (PTC124)
Ataluren PO
Other Name: PTC124
|
Placebo Comparator: Placebo
10-,10-,20-mg/kg TID at morning, midday and evening doses for 48 weeks.
|
Drug: Placebo
Placebo PO
|
- Forced expiratory volume in 1 second (FEV1) [ Time Frame: 48 weeks ]
- Pulmonary exacerbation frequency [ Time Frame: 48 weeks ]
- Cough frequency [ Time Frame: 48 weeks ]
- Respiratory HRQL as assessed by the CFQ-R respiratory domain [ Time Frame: 48 weeks ]
- Forced vital capacity (FVC) [ Time Frame: 48 weeks ]
- Safety profile [ Time Frame: 48 weeks ]
- Compliance with study drug administration [ Time Frame: 48 weeks ]
- Ataluren (PTC124) pharmacokinetics [ Time Frame: 48 weeks ]
- Antibiotic use and hospitalization due to CF-related symptoms [ Time Frame: 48 weeks ]
- Disruptions to school or work due to CF-related symptoms [ Time Frame: 48 weeks ]
- Body weight [ Time Frame: 48 weeks ]
- Markers of lung inflammation [ Time Frame: 48 weeks ]
- Lung computerized tomography CF score [ Time Frame: 48 weeks ]
- Nasal transepithelial potential difference (TEPD) [ Time Frame: 48 weeks ]
- Sweat chloride concentration [ Time Frame: 48 weeks ]

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Ages Eligible for Study: | 6 Years and older (Child, Adult, Senior) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Ability to provide written informed consent (parental/guardian consent and subject assent if <18 years of age)
- Age ≥6 years
- Body weight ≥16 kg
- Abnormal nasal TEPD total chloride conductance (a less electrically negative value than -5 mV for total chloride conductance [Δchloride-free+isoproterenol])
- Sweat chloride >40 mEq/L
- Documentation of the simultaneous presence of a nonsense mutation in at least 1 allele of the CFTR gene and a CF-causing mutation in the other CFTR allele, as determined by gene sequencing from 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 confirmation of the presence of a nonsense mutation in 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 for age, gender, and height
- Resting oxygen saturation (as measured by pulse oximetry) ≥92% on room air
- Documentation by VivoMetrics that the subject has satisfactorily completed a 24-hour LifeShirt® cough frequency assessment
- Confirmed screening laboratory values within the central laboratory ranges (hepatic, adrenal, renal, serum electrolytes, and reproduction [women only] parameters)
- 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 4-week follow-up period
- Willingness and ability to comply with scheduled visits, drug administration plan, study restrictions, and study procedures
Exclusion Criteria:
- Known hypersensitivity to any of the ingredients or excipients of the study drug
- Any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation, or reinitiation) in a chronic treatment/prophylaxis regimen for CF or for CF-related conditions within 4 weeks prior to start of study treatment
- Exposure to another investigational drug within 4 weeks prior to start of study treatment
- Treatment with systemic aminoglycoside antibiotics at the time of the baseline TEPD assessment
- Treatment with intravenous antibiotics within 3 weeks prior to start of study treatment
- History of solid organ or hematological transplantation
- Ongoing immunosuppressive therapy (other than corticosteroids)
- Ongoing warfarin, phenytoin, or tolbutamide therapy
- Ongoing participation in any other therapeutic clinical trial
- Major complications of lung disease (including massive hemoptysis, pneumothorax, or pleural effusion) within 8 weeks prior to start of study treatment
- Evidence of pulmonary exacerbation or acute upper or lower respiratory tract infection (including viral illnesses) within 3 weeks prior to randomization
- 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, 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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00803205

Study Director: | Temitayo Ajayi, MD | PTC Therapeutics |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | PTC Therapeutics |
ClinicalTrials.gov Identifier: | NCT00803205 History of Changes |
Other Study ID Numbers: |
PTC124-GD-009-CF Orphan Product Grant #FD003715 ( Other Grant/Funding Number: Funding Source - FDA OOPD ) |
First Posted: | December 5, 2008 Key Record Dates |
Last Update Posted: | June 6, 2017 |
Last Verified: | May 2017 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Keywords provided by PTC Therapeutics:
Cystic fibrosis Nonsense mutation Premature stop codon PTC124 Ataluren |
Additional relevant MeSH terms:
Fibrosis Cystic Fibrosis Pathologic Processes Pancreatic Diseases Digestive System Diseases |
Lung Diseases Respiratory Tract Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases |