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Drug & research development » Clinical trials » NCT00803205

Study of Ataluren (PTC124TM) in Cystic Fibrosis

Official title: A Phase 3 Efficacy and Safety Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis

Clinical Trials gov number: NCT00803205

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Purpose: To evaluate the clinical benefit of ataluren (PTC124) in adult and paediatric CF patients due to a nonsense mutation. The main goals of the study are to understand whether ataluren (PTC124) can improve pulmonary function and whether the drug can safely be given for a long period of time. The study will also assess the effects of ataluren on CF pulmonary exacerbation frequency, cough frequency, 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, randomized, double-blind, parallel assignment

Study sponsor: PTC Therapeutics; Cystic Fibrosis Foundation Therapeutics

  • Ability to provide written informed consent (parental/guardian consent and subject assent if <18 years of age)
  • 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

Intervention
Ataluren

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

Number of Participants
101-1000 (≥6 years)

Primary Endpoint

Forced expiratory volume in 1 second (FEV1) over 48 weeks

Secondary Endpoint

Over 48 weeks:

  • Pulmonary exacerbation frequency
  • Cough frequency
  • Respiratory HRQL as assessed by the CFQ-R respiratory domain
  • Forced vital capacity (FVC)

– Safety profile

  • Compliance with study drug administration
  • Ataluren (PTC124) pharmacokinetics
  • Antibiotic use and hospitalization due to CF-related symptoms
  • Disruptions to school or work due to CF-related symptoms
  • Body weight
  • Markers of lung inflammation
  • Lung computerized tomography CF score
  • Nasal transepithelial potential difference
  • Sweat chloride concentration

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  • Experimental: Ataluren (PTC124)

10-, 10-, 20-mg/kg TID at morning, midday, and evening doses

  • Placebo Comparator: Placebo

Link:

http://www.ncbi.nlm.nih.gov/pubmed/24836205

Reference:

Kerem E, Konstan MW, De Boeck K, Accurso FJ, Sermet-Gaudelus I, Wilschanski M, Elborn JS, Melotti P, Bronsveld I, Fajac I, Malfroot A, Rosenbluth DB, Walker PA, McColley SA, Knoop C, Quattrucci S, Rietschel E, Zeitlin PL, Barth J, Elfring GL, Welch EM, Branstrom A, Spiegel RJ, Peltz SW, Ajayi T, Rowe SM; Cystic Fibrosis Ataluren Study Group. Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial. Lancet Respir Med. 2014 Jul;2(7):539-47. doi: 10.1016/S2213-2600(14)70100-6. Epub 2014 May 15. PubMed PMID: 24836205; PubMed Central PMCID: PMC4154311.

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