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PTC124 for Cystic Fibrosis

NCT00234663 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 24

Last updated 2009-01-13

No results posted yet for this study

Summary

In some patients 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. PTC124 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 PTC124 can safely increase functional CFTR protein in the cells of patients with CF due to a nonsense mutation.

Conditions

Interventions

DRUG

PTC124

Sponsors & Collaborators

  • Cystic Fibrosis Foundation

    collaborator OTHER

  • FDA Office of Orphan Products Development

    collaborator FED

  • PTC Therapeutics

    lead INDUSTRY

Principal Investigators

  • John P Clancy, MD · University of Alabama at Birmingham

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2005-09-30
Primary Completion
2006-08-31
Completion
2006-08-31

Countries

  • United States

Study Locations

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Entities

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 NCT00234663 on ClinicalTrials.gov