Offered by: Nemours Children's
Location: Jacksonville, Fla.
A Phase 3 Efficacy and Safety Study of Ataluren (PTC124) in Patients With Nonsense Mutation CF
What is the trial about?
The main goal of this study is to find out whether ataluren can help maintain and/or improve lung function. We also want to see if it:
- Reduces CF symptoms
- Decreases the number of hospitalizations and antibiotic use for CF lung infections
- Helps improve quality of life
- Is safe to use long term
Who can participate?
To participate, you must weigh at least 16 kg (35 pounds) and have CF that is due to a nonsense mutation in the CFTR gene. In addition, you:
- Will need to be able to reliably perform the tests required by the study
- Must have adequate lung function
- Have normal or nearly normal blood tests for bone marrow, liver, adrenal gland, and kidney function.
If you are taking certain medications for CF, you must be on a stable dose and medication schedule and must be willing to remain on the same dose and medication schedule throughout the study, unless there is a medical need to change such treatment. The doctor will carefully review your health status to make sure that you meet all of the necessary conditions for participation.
What is involved?
This study will last for about one year. During this study, you will receive study drug or a placebo. A placebo is a powder that looks and tastes the same as ataluren but does not have any medicine in it. You will take the drug/placebo for approximately 48 weeks, during which time you will need to return to the clinic about every eight weeks. You will also need to return to the clinic about four weeks after you stop taking study drug/placebo.
You will go through a screening period of one to four weeks before starting study drug or placebo. During this time, the study staff will confirm the nonsense mutation gene through blood work. They will also collect other data about your health to establish your individual baseline, or normal values in things such as PFT’s, ECG’s, blood work, etc.