Home
Search
Study Topics
Glossary
|
|
|
|
|
|
Sponsored by: |
PTC Therapeutics |
---|---|
Information provided by: | PTC Therapeutics |
ClinicalTrials.gov Identifier: | NCT00351078 |
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. In an ongoing Phase 2a study being performed at the Hadassah University Hospital - Mount Scopus in Israel, patients with nonsense-mutation-mediated CF have received PTC124 in two 14-day treatment courses. Preliminary results from that study indicate that PTC124 has pharmacodynamic activity of PTC124 in CF and that PTC124 can be safely administered in this patient population. This Phase 2b extension study is designed to evaluate the activity and safety of 3 months (approximately 84 days) of continuous treatment with PTC124 in the same patients who were enrolled to the Phase 2a study. The main purpose of this study is to understand whether PTC124 can be safely administered and achieve pharmacodynamic activity in 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, Parallel Assignment, Safety/Efficacy Study |
Official Title: | A Phase 2b Extension Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis |
Estimated Enrollment: | 23 |
Study Start Date: | December 2006 |
Study Completion Date: | July 2007 |
In this study, the same patients who were enrolled in a previous study of PTC124 (protocol number PTC124-GD-005-CF) will be treated with the investigational drug PTC124 for a period of 84 days. 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 be assigned to receive PTC124 treatment (based upon TEPD results from the previous study) at 1 of the 2 following dose levels:
During the study, PTC124 efficacy, safety, and pharmacokinetics will be evaluated periodically with measurement of transepithelial potential difference (TEPD), medical history, physical examinations, blood tests, urinalysis, sputum tests, 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:
Patients must meet all of the following conditions to be eligible for enrollment into the study:
Exclusion Criteria:
The presence of any of the following conditions will exclude a patient from enrollment in the study:
Israel | |
Hadassah University Hospital - Mount Scopus | |
Jerusalem, Israel, 91240 |
Principal Investigator: | Eitan Kerem, MD | Hadassah University Hospital - Mount Scopus |
Study ID Numbers: | PTC124-GD-005e-CF |
Study First Received: | July 11, 2006 |
Last Updated: | October 16, 2007 |
ClinicalTrials.gov Identifier: | NCT00351078 |
Health Authority: | Israel: Israeli Health Ministry Pharmaceutical Administration |
Cystic fibrosis Nonsense mutation Premature stop codon CF PTC124 |
Digestive System Diseases Genetic Diseases, Inborn Respiratory Tract Diseases Cystic Fibrosis Fibrosis |
Lung Diseases Infant, Newborn, Diseases Pancreatic Diseases Cystic fibrosis |
Pathologic Processes |