Status:
COMPLETED
Extension Study of Ataluren (PTC124) in Cystic Fibrosis
Lead Sponsor:
PTC Therapeutics
Collaborating Sponsors:
Cystic Fibrosis Foundation
Conditions:
Cystic Fibrosis
Eligibility:
All Genders
6+ years
Phase:
PHASE3
Brief Summary
Cystic fibrosis (CF) is a genetic disorder caused by a mutation in the gene that makes the cystic fibrosis transmembrane conductance regulator (CFTR) protein. A specific type of mutation called a nons...
Detailed Description
This Phase 3, open-label, safety and efficacy study will be performed at sites in North America, Europe, and Israel. The study will enroll up to approximately 208 participants with nmCF who participat...
Eligibility Criteria
Inclusion
- Completion of blinded study drug treatment in the previous Phase 3 study (PTC124-GD-009-CF).
- Ability to provide written informed consent (parental/guardian consent if applicable)/assent (if \<18 years of age).
- In participants who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during ataluren administration and the 4-week follow up period.
- Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions.
Exclusion
- Known hypersensitivity to any of the ingredients or excipients of the study drug (list provided at study sites).
- Current pregnancy or lactating, or pregnancy or lactating during the previous Phase 3 study.
- Ongoing participation in any other therapeutic clinical trial.
- Prior or ongoing medical condition (for example, concomitant illness, psychiatric condition, behavioral disorder, alcoholism, drug abuse), medical history, physical findings, ECG findings, or laboratory abnormality that, in the Investigator's opinion, could adversely affect the safety of the participant, makes it unlikely that the course of treatment or follow up would be completed, or could impair the assessment of study results.
Key Trial Info
Start Date :
August 12 2010
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 2 2013
Estimated Enrollment :
191 Patients enrolled
Trial Details
Trial ID
NCT01140451
Start Date
August 12 2010
End Date
December 2 2013
Last Update
October 19 2020
Active Locations (31)
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1
University of Alabama-Birmingham
Birmingham, Alabama, United States, 35233
2
Miller Children's Hospital Long Beach
Long Beach, California, United States, 90806
3
Lucile Packard Children's Hospital
Palo Alto, California, United States, 94304
4
The Children's Hospital
Aurora, Colorado, United States, 80045