Extension study of ataluren in patients with Cystic Fibrosis

• Conditions: onsense-Mutation-Mediated Cystic Fibrosis; MedDRA version: 14.1Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

• Registration Number: EUCTR2010-019692-30-DE
• Lead Sponsor: PTC Therapeutics, Inc

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2011-04-13
• Last Update Posted: 26 October 2015

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 200

Inclusion Criteria

1. Completion of ~48 weeks of blinded study drug treatment in the previous Phase 3 study (Study PTC124-GD-009-CF).
2. Evidence of signed and dated informed consent/assent document(s) indicating that the subject (and/or his/her parent/legal guardian) has been informed of all pertinent aspects of the study. Note: If the study candidate is considered a child under local regulation, a parent or legal guardian must provide written consent prior to initiation of study screening procedures and the study candidate may be required to provide written assent.
The rules of the responsible Institutional Review Board/Institutional Ethics Committee (IRB/IEC) regarding whether one or both parents must provide consent and the appropriate ages for obtaining consent and assent from the subject should be followed.
3. In subjects who are sexually active, willingness to abstain from sexual intercourse or employ a highly effective barrier or medical method of contraception during ataluren administration and within 60 days of the last administration of the study drug.
4. Willingness and ability to comply with scheduled visits, ataluren administration plan, study procedures, laboratory tests, and study restrictions.
Are the trial subjects under 18? yes
Number of subjects for this age range: 62
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 151
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

1. Known hypersensitivity to any of the ingredients or excipients of the study drug (polydextrose, polyethylene glycol 3350, poloxamer 407, mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, vanilla, colloidal silica, magnesium stearate).
2. Current pregnancy or lactating, or pregnancy or lactating during the preceding Phase 3 study (Study PTC124-GD-009-CF).
3. Ongoing participation in any other therapeutic clinical trial.
4. 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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified