Status:
COMPLETED
Ivacaftor Treatment in 4 Month to 2 Year Old CF Subjects
Lead Sponsor:
Children's Hospital of Philadelphia
Collaborating Sponsors:
Vertex Pharmaceuticals Incorporated
Conditions:
Cystic Fibrosis
Eligibility:
All Genders
4-2 years
Brief Summary
The purpose of this research study is to determine the effects of clinically prescribed ivacaftor treatment on 4-24 month old children with CF and gating mutations on sleeping energy expenditure, grow...
Detailed Description
Ivacaftor is a novel FDA approved therapy for patients with CF and gating mutations who are 4 months and older. This investigator-Initiated study is designed to evaluate the nutritional, growth and GI...
Eligibility Criteria
Inclusion
- Cystic fibrosis with at least one CFTR gating mutation (E56K, G178R, S549R, S977F, F1074L, 2789+5G→A,P67L, E193K, G551D, F1052V, D1152H, 3272-26A→G, R74W, L206W, G551S, K1060T, G1244E, 3849+10kbC→T, D110E, R347H, D579G, A1067T, S1251N, D110H, R352Q, 711+3A→G, G1069R, S1255P, R117C, A455E, E831X, R1070Q, D1270N, R117H, S549N, S945L, R1070W, G1349D) approved for treatment
- Age: 4-24 months of age
- In their usual state of good health
- A clinical decision has been made for subject to begin ivacaftor treatment
- Family committed to the 4 to 6 month study protocol with visits to CHOP that will last 2 or 3 days for the baseline visit (Visit 1) prior to ivacaftor and the 12 week visit (Visit 3) after clinically prescribed ivacaftor treatment has begun, and will last 2 days for the 6 week visit (Visit 2) after ivacaftor treatment has begun.
Exclusion
- On parenteral nutrition
- Use of any medications which are as inhibitors or inducers of cytochrome P450 (CYP) 3A
- Liver function tests elevated above 3x the reference range for age and sex
- Other illness affecting growth or nutritional status
- Other contraindications described for ivacaftor therapy
Key Trial Info
Start Date :
February 6 2019
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
December 30 2021
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT03783286
Start Date
February 6 2019
End Date
December 30 2021
Last Update
March 11 2022
Active Locations (1)
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1
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19146