This study is currently not recruiting participants.

A Phase 3 Extension Study of Ataluren (PTC124) In Patients with Nonsense Mutation Cystic Fibrosis PTC124-GD-021e-CF

Investigation of an Investigational Medication for Cystic Fibrosis

Not Recruiting
100 years or below
All
Phase N/A

Brief description of study.

The purpose of this study is to test a new drug called ataluren, 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, 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, a genetic disorder that affects the lungs and digestive system. Cystic fibrosis causes thick, sticky mucus to build up in the lungs, leading to breathing problems and infections.

Participants will receive either the investigational medication or a placebo, which is an inactive substance that looks like the investigational medication but does not contain any medicine. The study will involve regular monitoring of participants' health and response to the treatment.

  • Who can participate: Participants aged 18 to 50 years with a confirmed diagnosis of cystic fibrosis, who are not currently on conflicting medications, may be eligible to participate.
  • Study details: Participants will be randomly assigned to receive either the investigational medication or a placebo.
Updated on 19 Feb 2024. Study ID: 1512181074

Interested in the study?

Select a study center that’s convenient for you, and get in touch with the study team.

Connect with the study team