Extension Study of Ataluren (PTC124) in Cystic Fibrosis

Status:	Completed
Conditions:	Pulmonary
Therapuetic Areas:	Pulmonary / Respiratory Diseases
Healthy:	No
Age Range:	6 - Any
Updated:	12/17/2017
Start Date:	August 2010
End Date:	December 2013

A Phase 3 Extension Study of Ataluren (PTC124) in Subjects With Nonsense-Mutation-Mediated Cystic Fibrosis

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 nonsense (premature stop codon) mutation is the cause of CF in
approximately 10% of subjects with the disease. Ataluren (PTC124) is an orally delivered
investigational drug that has the potential to overcome the effects of the nonsense mutation.
This study is a Phase 3 extension trial that will evaluate the long-term safety of ataluren
(PTC124) in adult and pediatric patients with nonsense mutation CF, as determined by adverse
events and laboratory abnormalities. The study will also assess changes in pulmonary
function, CF pulmonary exacerbations, health-related quality of life, antibiotic use for
CF-related infections, CF-related disruptions to daily living, body weight, and CF
pathophysiology. Funding Source - FDA OOPD.

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 patients with
nonsense mutation CF who participated in a previous Phase 3 study of ataluren (PTC124)
(PTC124-GD-009-CF, NCT00803205). Subjects will receive study drug 3 times per day (at
breakfast, lunch, and dinner) for approximately 48 weeks (approximately 1 year). Study
assessments will be performed at clinic visits every 8 weeks.

Inclusion Criteria:

- 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 subjects 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 Criteria:

- 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 (eg, 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 subject, makes it unlikely that the course of
treatment or follow up would be completed, or could impair the assessment of study
results.

We found this trial at

sites

University of Alabama at Birmingham

1720 2nd Ave S
Birmingham, Alabama 35233

(205) 934-4011