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Drug & research development » Clinical trials » NCT01140451

Extension Study of Ataluren (PTC124) in Cystic Fibrosis

Official title: A Phase 3 Extension Study of Ataluren (PTC124) in Subjects With Nonsense-Mutation-Mediated Cystic Fibrosis

Clinical Trials gov number: NCT01140451

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Purpose: A Phase 3 extension trial to evaluate the long-term safety of ataluren (PTC124) in adult and paediatric 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.

Phase: 3

Type: Interventional, open-label, single group assignment

Study sponsor: PTC Therapeutics; Cystic Fibrosis Foundation Therapeutics

  • Completed 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


Geographical Location
Belgium, Canada, France, Israel, Germany, Italy, Netherlands, Spain, Sweden, United Kingdom, United States

Number of Participants
101-1000 (≥6 years)

Primary Endpoint

Adverse events and laboratory abnormalities over 1 year

Secondary Endpoint

Over 1 year

  • Forced expiratory volume in 1 second (FEV1)
  • Forced vital capacity (FVC)
  • Incidence, rate, severity, and duration of pulmonary exacerbations
  • Respiratory HRQL as assessed by the CFQ-R respiratory domain
  • Compliance with study drug treatment

– Ataluren plasma exposure

  • Antibiotic use and hospitalization due to CF-related symptoms
  • Disruptions to school or work due to CF-related symptoms
  • Body weight
  • Lung computerized tomography CF score
  • Nasal transepithelial potential difference
  • Sweat chloride concentration

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  • Experimental: 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.

View Trial Results