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Study of Ataluren (PTC124™) in Cystic Fibrosis

NCT00803205 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 238

Last updated 2020-05-14

Study results available
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Summary

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 patients with the disease. Ataluren is an orally delivered investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 3 trial that will evaluate the clinical benefit of ataluren in adult and pediatric participants with CF due to a nonsense mutation. The main goals of the study are to understand whether ataluren 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.

Conditions

Interventions

DRUG

Ataluren

Ataluren will be provided as a vanilla-flavored powder to be mixed with water.

DRUG

Placebo

Placebo matching to ataluren will be provided.

Sponsors & Collaborators

Principal Investigators

  • Temitayo Ajayi, MD · PTC Therapeutics

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2009-09-08
Primary Completion
2011-11-12
Completion
2011-11-12

Countries

  • United States
  • Belgium
  • Canada
  • France
  • Germany
  • Israel
  • Italy
  • Netherlands
  • Spain
  • Sweden
  • United Kingdom

Study Locations

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Entities

Drugs
Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00803205 on ClinicalTrials.gov