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Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis

NCT00237380 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 24

Last updated 2020-06-11

No results posted yet for this study

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.

Sponsors & Collaborators

Principal Investigators

  • Eiten Kerem, MD · Hadassah University Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2005-11-30
Primary Completion
2006-05-31
Completion
2006-05-31

Countries

  • Israel

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 NCT00237380 on ClinicalTrials.gov