Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children
Recruiting
Observational Study
Cystic Fibrosis
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- Up to 10
- Sex
- Any
- Study type
- Observational
- Participants needed
- 210 (estimated)
- Sponsor
- Sonya Heltshe · Other
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
Check Your Eligibility
About This Trial
This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators and their impact on children with cystic fibrosis (CF).
Trial Locations
Loading…
Loading trial locations…
Eligibility Criteria
Inclusion Criteria:
* Part A:
* Less than 10 years of age at the first study visit.
* Documentation of a CF diagnosis.
Part B:
* Participated in Part A OR less than 7 years of age at the first study visit.
* Documentation of a CF diagnosis.
* CFTR mutations consistent with FDA labeled indica…
Contacts