Ignite Creation Date:
2024-05-05 @ 12:04 PM
Last Modification Date:
2024-10-26 @ 9:20 AM
Study NCT ID:
NCT00234663
Status:
COMPLETED
Last Update Posted:
2009-01-13
First Post:
2005-10-05
Brief Title:
PTC124 for Cystic Fibrosis
Sponsor:
PTC Therapeutics
Organization:
PTC Therapeutics
Study Overview
Official Title:
A Phase 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis
Status:
COMPLETED
Status Verified Date:
2009-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
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 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
Detailed Description:
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 mgkg breakfast 4 mgkg lunch and 8 mgkg 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 mgkg breakfast 10 mgkg lunch and 20 mgkg 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
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 1R01FD003009-01 | FDA | None | httpsreporternihgovquickSearch1R01FD003009-01 |