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A Study of Ataluren in Pediatric Participants With Cystic Fibrosis

NCT00458341 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 30

Last updated 2020-03-06

Study results available
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Summary

In some participants with cystic fibrosis (CF), the disease is caused by a nonsense mutation (premature stop codon) in the gene that makes the cystic fibrosis transmembrane regulator (CFTR) protein. Ataluren has been shown to partially restore CFTR production in animals with CF due to a nonsense mutation. The main purpose of this study is to understand whether ataluren can safely increase functional CFTR protein in the cells of participants with CF due to a nonsense mutation.

Conditions

Interventions

DRUG

Ataluren

Ataluren will be provided as a vanilla-flavored powder to be mixed with water, milk, or apple juice. Participants are to receive a total of 42 doses of ataluren during each cycle, for a total of 84 doses of ataluren in both cycles.

Sponsors & Collaborators

Principal Investigators

  • Isabelle Sermet-Gaudelus, MD · Hopital Necker

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
CROSSOVER

Eligibility

Min Age
6 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2007-03-23
Primary Completion
2008-02-29
Completion
2008-02-29

Countries

  • Belgium
  • France

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 NCT00458341 on ClinicalTrials.gov