This study is currently not recruiting participants.

A Phase 3 Efficacy and Safety Study of Ataluren (PTC124?) in Patients with Nonsense Mutation Cystic Fibrosis (ACT CF) PTC124-GD-021-CF

Study of an Investigational Medication for Cystic Fibrosis

Not Recruiting
100 years or below
All
Phase N/A
1 Location

Brief description of study.

The purpose of this study is to test a new drug called ataluren (also sometimes called PTC124), which is an experimental drug that is not yet available for sale and has not received approval from health authorities for use in CF.

Detailed description of study

The purpose of this study is to test a new drug called ataluren (also sometimes called PTC124), which is an experimental drug that is not yet available for sale and has not received approval from health authorities for use in CF.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: cystic fibrosis
  • Age: 100 years or below
  • Gender: All

This study investigates an experimental medication for cystic fibrosis (CF). Cystic fibrosis is a genetic disorder that affects the lungs and digestive system. The investigational medication is not yet approved for use and is being tested to understand its effects on CF.

Participants in the study will receive either the investigational medication or a placebo. A placebo is an inactive substance that looks like the investigational medication but does not contain any medicine.

  • Who can participate: Participants must be individuals diagnosed with cystic fibrosis, aged 12 to 40 years. Key eligibility criteria include stable lung function and no recent hospitalizations for pulmonary exacerbations.
  • Study details: Participants in the study will receive either the investigational medication or a placebo.
Updated on 19 Feb 2024. Study ID: 1410322095

Interested in the study?

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