Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis

PTC Therapeutics

Status and phase: Terminated; Phase 3

Conditions: Cystic Fibrosis

Treatments: Drug: Ataluren

Study type: Interventional

Funder types: Industry

Identifiers

NCT02456103

PTC124-GD-021e-CF

Details and patient eligibility

About

This is an open-label extension study for participants who completed a Phase 3, placebo-controlled study of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) not receiving chronic inhaled aminoglycosides.

Full description

The primary objective of this Phase 3 extension study will be to obtain long-term safety data to augment the overall safety database. The secondary objectives will be to augment the efficacy data collected in the double-blind study (PTC124-GD-021-CF; NCT02139306).

Enrollment: 246 patients

Sex: All

Ages: 6+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Completion of study treatment (placebo or active) in the previous Phase 3, double-blind study protocol (Protocol PTC124-GD-021-CF)
• Evidence of signed and dated informed consent/assent document(s) indicating that the participant (and/or the participant's parent/legal guardian) has been informed of all pertinent aspects of the trial.

Exclusion criteria

• Known hypersensitivity to any of the ingredients or excipients of the study drug.
• Ongoing participation in any other therapeutic clinical trial.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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246 participants in 1 patient group

Ataluren

Experimental group

Description:

Participants will be administered ataluren orally at a dose of 10 milligrams/grams (mg/kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening for up to 96 weeks.

Treatment:

Drug: Ataluren