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Restore CFTR Protein Closed to Enrollment
Study of the triple-combination modulator, elexacaftor/tezacaftor/ivacaftor, in children 2-5 years old with cystic fibrosis (Part A) (Vertex VX20-445-111 Part A)
This study will look at the safety and effectiveness of the triple-combination therapy, elexacaftor/tezacaftor/ivacaftor (Trikafta®), in children ages 2-5 years old with CF. These drugs are intended to help CFTR protein function closer to normal.
This study is open label, which means that all participants will receive the triple-combination study drug. In Part A, participants will receive the study drug in the morning and ivacaftor in the evening. Researchers will evaluate how the body processes the drug by measuring how much of the drug is in the blood. They will also measure safety by monitoring for adverse events.This study may require blood draws, lung function tests, sweat tests, and/or other measures.
Eligibility
See other primary eligibility criteria for more information.
-
Age:
2 Years to 5 Years -
Mutation(s):
Two Copies F508del or One Copy F508del -
FEV1% Predicted:
No FEV1 Limit
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Participants in Part A must weigh at least 14 kg (31 lbs). Participants must have two copies of the F508del CFTR mutation, or one copy of F508del and one copy of a minimal function CFTR mutation. Click here to see a list of eligible minimal function mutations.
Study Design
-
Study Type: ?more info
Interventional -
Randomized Study: ?more info
No -
Placebo Controlled: ?more info
No -
Length of Participation:
53 days -
Number of Study Visits:
5
Additional Information
-
Phase: ?more info
Phase Three -
Study Sponsor: ?more info
Vertex -
Study Drugs:
Study Sites
-
Closed to Enrollment
California
Stanford University Medical Center, Palo Alto, CA 94304
-
Closed to Enrollment
Colorado
Children's Hospital Colorado, Aurora, CO 80045
-
Closed to Enrollment
Illinois
Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL 60611
-
Closed to Enrollment
Indiana
Riley Hospital for Children, Indianapolis, IN 46202
-
Closed to Enrollment
Missouri
St. Louis Children's Hospital, St. Louis, MO 63110
-
Closed to Enrollment
North Carolina
University of North Carolina at Chapel Hill, Chapel Hill, NC 27599
-
Closed to Enrollment
Pennsylvania
Children's Hospital of Pittsburgh of UPMC, Pittsburgh, PA 15224
Eligibility
See other primary eligibility criteria for more information.
-
Age:
2 Years to 5 Years -
Mutation(s):
Two Copies F508del or One Copy F508del -
FEV1% Predicted:
No FEV1 Limit
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Participants in Part A must weigh at least 14 kg (31 lbs). Participants must have two copies of the F508del CFTR mutation, or one copy of F508del and one copy of a minimal function CFTR mutation. Click here to see a list of eligible minimal function mutations.
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