Study for children under 5 with CF
Protocol: STUDY00004777
Full Title
A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN)
This study enrolls children with cystic fibrosis (CF). The purpose of the study is to look at growth in infants and young children that have cystic fibrosis (CF).

There are two parts to the study:
Part A: We will collect health information on infants and young children before starting treatment with Trikafta, an FDA approved drug to treat CF in certain teens and adults. Your child will come to the study center for up to 6 study visits over 36 months. At the visits, we will do a variety of tests and exams to learn about your child's health.
Part B: If Trikafta is approved in children less than 6 years old you may be approached to allow your child to join Part B.

Study visits will take place at UBMD Pediatrics offices in Buffalo, NY.

Technical Description

(CSR #200721432724) Part A: To describe and define the natural history of growth, gastrointestinal health, and pulmonary function in infants and young children with CF.Part B: To describe changes in growth, gastrointestinal health, and pulmonary function following initiation of ivacaftor or elex/tez/iva in infants and young children with CF.
Compensation: Yes
Compensation may include cash, checks, gift cards, debit cards, or incentives like gift baskets, technology items, or merchandise.
-Children ages less than 6.
-CF diagnosis (with CFTR mutations consistent with FDA labeled indication of highly effective modulator (HEM) therapy).
-Physician intends to prescribe HEM therapy.
Age Group: Children
Principal Investigator: DANIELLE GOETZ
+1 716-878-7375
Want to Learn More?
Let us know how the study team can reach you. If you do not hear back within 2 business days, reach out to the study team directly at the contact information above or email and someone will assist you.