Extension Study of Ataluren (PTC124) in Cystic Fibrosis
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Purpose
Cystic fibrosis (CF) is a genetic disorder caused by a mutation in the gene that makes the cystic fibrosis transmembrane conductance regulator (CFTR) protein. A specific type of mutation called a nonsense (premature stop codon) mutation is the cause of CF in approximately 10% of subjects with the disease. Ataluren (PTC124) is an orally delivered investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 3 extension trial that will evaluate the long-term safety of ataluren (PTC124) in adult and pediatric patients with nonsense mutation CF, as determined by adverse events and laboratory abnormalities. The study will also assess changes in pulmonary function, CF pulmonary exacerbations, health-related quality of life, antibiotic use for CF-related infections, CF-related disruptions to daily living, body weight, and CF pathophysiology.
| Condition | Intervention | Phase |
|---|---|---|
|
Cystic Fibrosis |
Drug: Ataluren (PTC124) |
Phase 3 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | A Phase 3 Extension Study of Ataluren (PTC124) in Subjects With Nonsense-Mutation-Mediated Cystic Fibrosis |
- Long-term safety of ataluren in patients with nonsense mutation CF, as determined by adverse events and laboratory abnormalities [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
- Forced expiratory volume in 1 second (FEV1) [ Time Frame: 1 year ] [ Designated as safety issue: No ]
- Forced vital capacity (FVC) [ Time Frame: 1 year ] [ Designated as safety issue: No ]
- Incidence, rate, severity, and duration of pulmonary exacerbations [ Time Frame: 1 year ] [ Designated as safety issue: No ]
- Respiratory HRQL as assessed by the CFQ-R respiratory domain [ Time Frame: 1 year ] [ Designated as safety issue: No ]
- Compliance with study drug treatment [ Time Frame: 1 year ] [ Designated as safety issue: No ]
- Ataluren plasma exposure [ Time Frame: 1 year ] [ Designated as safety issue: No ]
- Antibiotic use and hospitalization due to CF-related symptoms [ Time Frame: 1 year ] [ Designated as safety issue: No ]
- Disruptions to school or work due to CF-related symptoms [ Time Frame: 1 year ] [ Designated as safety issue: No ]
- Body weight [ Time Frame: 1 year ] [ Designated as safety issue: No ]
- Lung computerized tomography CF score [ Time Frame: 1 year ] [ Designated as safety issue: No ]
- Nasal transepithelial potential difference (TEPD) [ Time Frame: 1 year ] [ Designated as safety issue: No ]
- Sweat chloride concentration [ Time Frame: 1 year ] [ Designated as safety issue: No ]
| Enrollment: | 191 |
| Study Start Date: | August 2010 |
| Estimated Study Completion Date: | October 2012 |
| Estimated Primary Completion Date: | October 2012 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: Ataluren (PTC124)
Ataluren (PTC124)
|
Drug: Ataluren (PTC124)
Oral powder for suspension taken 3 times per day (10 mg/kg with breakfast, 10 mg/kg with lunch, and 20 mg/kg with dinner) for up to 48 weeks.
|
Detailed Description:
This Phase 3, open-label, safety and efficacy study will be performed at sites in North America, Europe, and Israel. The study will enroll up to approximately 208 patients with nonsense mutation CF who participated in a previous Phase 3 study of ataluren (PTC124) (PTC124-GD-009-CF, NCT00803205). Subjects will receive study drug 3 times per day (at breakfast, lunch, and dinner) for approximately 48 weeks (approximately 1 year). Study assessments will be performed at clinic visits every 8 weeks.
Eligibility| Ages Eligible for Study: | 6 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Completion of blinded study drug treatment in the previous Phase 3 study (PTC124-GD-009-CF).
- Ability to provide written informed consent (parental/guardian consent if applicable)/assent (if <18 years of age).
- In subjects who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during ataluren administration and the 4-week follow up period.
- Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions.
Exclusion Criteria:
- Known hypersensitivity to any of the ingredients or excipients of the study drug (list provided at study sites).
- Current pregnancy or lactating, or pregnancy or lactating during the previous Phase 3 study.
- Ongoing participation in any other therapeutic clinical trial.
- Prior or ongoing medical condition (eg, concomitant illness, psychiatric condition, behavioral disorder, alcoholism, drug abuse), 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.
Contacts and Locations
Show 31 Study Locations| Study Director: | Temitayo Ajayi, MD | PTC Therapeutics, Inc. |
More Information
Additional Information:
No publications provided
| Responsible Party: | PTC Therapeutics |
| ClinicalTrials.gov Identifier: | NCT01140451 History of Changes |
| Other Study ID Numbers: | PTC124-GD-009e-CF |
| Study First Received: | June 7, 2010 |
| Last Updated: | August 6, 2012 |
| Health Authority: | Belgium: Directorate general for the protection of Public health: Medicines Canada: Health Canada France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis) Germany: Federal Institute for Drugs and Medical Devices Israel: Ministry of Health Italy: The Italian Medicines Agency Netherlands: Medicines Evaluation Board (MEB) Spain: Spanish Agency of Medicines Sweden: Medical Products Agency United Kingdom: Medicines and Healthcare Products Regulatory Agency United States: Food and Drug Administration |
Additional relevant MeSH terms:
|
Cystic Fibrosis Fibrosis Pancreatic Diseases Digestive System Diseases Lung Diseases |
Respiratory Tract Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Pathologic Processes |
ClinicalTrials.gov processed this record on May 23, 2013