Home
Search
Study Topics
Glossary
|
|
|
|
|
|
Sponsors and Collaborators: |
PTC Therapeutics Cystic Fibrosis Foundation FDA Office of Orphan Products Development |
---|---|
Information provided by: | PTC Therapeutics |
ClinicalTrials.gov Identifier: | NCT00234663 |
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.
Condition | Intervention | Phase |
---|---|---|
Cystic Fibrosis |
Drug: PTC124 |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Dose Comparison, Single Group Assignment, Safety/Efficacy Study |
Official Title: | A Phase 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis |
Estimated Enrollment: | 24 |
Study Start Date: | September 2005 |
Study Completion Date: | August 2006 |
Primary Completion Date: | August 2006 (Final data collection date for primary outcome measure) |
In this study, patients with CF due to a nonsense mutation will be treated with a new investigational drug called PTC124. Evaluation procedures (history, physical examination, blood and urine tests to assess organ function, electrocardiogram (ECG), chest x-ray, and CF-specific tests) to determine if a patient qualifies for the study will be performed within 21 days prior to the start of treatment. Eligible patients who elect to enroll in the study will then participate in two 28-day treatment and follow-up periods (56 days total). Within the first 28-day period, PTC124 treatment will be taken 3 times per day with meals for 14 days at doses of 4 mg/kg (breakfast), 4 mg/kg (lunch) and 8 mg/kg (dinner); there will then be an interval of 14 days without treatment. Within the second 28-day period, PTC124 treatment will be taken 3 times per day with meals for 14 days at doses of 10 mg/kg (breakfast), 10 mg/kg (lunch) and 20 mg/kg (dinner); there will then be an interval of 14 days without treatment. There will be a 2-night stay at the clinical research center at the beginning and at the end of each 14 days of PTC124 treatment, which means that there will be four 2-night stays at the clinical research center during the study. During the study, PTC124 efficacy, safety, and pharmacokinetics will be evaluated periodically with measurement of transepithelial potential difference (TEPD), nasal mucosal brushing to assess for cellular CFTR mRNA and protein, medical history, physical examinations, blood tests, urinalysis, ECGs, chest x-ray, and pulmonary function tests.
Ages Eligible for Study: | 18 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
United States, Alabama | |
University of Alabama at Birmingham | |
Birmingham, Alabama, United States, 35233-1711 | |
United States, California | |
Stanford University Medical Center | |
Palo Alto, California, United States, 94304-5786 | |
United States, Colorado | |
The Children's Hospital | |
Denver, Colorado, United States, 80218 | |
United States, Maryland | |
Johns Hopkins Hospital | |
Baltimore, Maryland, United States, 21287 | |
United States, Ohio | |
Rainbow Babies and Children's Hospital | |
Cleveland, Ohio, United States, 44106 |
Principal Investigator: | John P Clancy, MD | University of Alabama at Birmingham |
Study ID Numbers: | PTC124-GD-003-CF, 1 R01 FD003009-01 |
Study First Received: | October 5, 2005 |
Last Updated: | January 9, 2009 |
ClinicalTrials.gov Identifier: | NCT00234663 |
Health Authority: | United States: Food and Drug Administration |
Cystic fibrosis Nonsense mutation Premature stop codon |
Digestive System Diseases Genetic Diseases, Inborn Respiratory Tract Diseases Cystic Fibrosis Fibrosis |
Lung Diseases Infant, Newborn, Diseases Pancreatic Diseases Cystic fibrosis |
Pathologic Processes |