Ignite Creation Date:
2024-05-05 @ 4:58 PM
Last Modification Date:
2024-10-26 @ 9:26 AM
Study NCT ID:
NCT00351078
Status:
COMPLETED
Last Update Posted:
2017-12-13
First Post:
2006-07-11
Brief Title:
PTC124 for the Treatment of Cystic Fibrosis
Sponsor:
PTC Therapeutics
Organization:
PTC Therapeutics
Study Overview
Official Title:
A Phase 2b Extension Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis
Status:
COMPLETED
Status Verified Date:
2017-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 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
Detailed Description:
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
PTC124 given 3 times per day TID with a regimen of 4 mgkg at breakfast 4 mgkg at lunch and 8 mgkg at dinner or
PTC124 given 3 times per day TID with a regimen of 10 mgkg at breakfast 10 mgkg at lunch and 20 mgkg at dinner
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
PTC124 given 3 times per day TID with a regimen of 4 mgkg at breakfast 4 mgkg at lunch and 8 mgkg at dinner or
PTC124 given 3 times per day TID with a regimen of 10 mgkg at breakfast 10 mgkg at lunch and 20 mgkg at dinner
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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None