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About
This is a Phase 3, 2-part (Part A and Part B), open label, multicenter study evaluating the pharmacokinetic (PK), safety, and tolerability of multiple doses of tezacaftor (TEZ) in combination with ivacaftor (IVA) in subjects 6 through 11 years of age with CF who are homozygous or heterozygous for the F508del- CF transmembrane conductance regulator protein (CFTR) mutation.
Enrollment
Sex
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Volunteers
Inclusion criteria
Subjects who weigh ≥15 kg without shoes at the Screening Visit.
All genotypes as specified by the study protocol are eligible in Part A.
The following genotypes are eligible in Part B:
Subjects with a confirmed diagnosis of CF defined as a sweat chloride value ≥60 mmol/L or chronic sinopulmonary and/or gastrointestinal disease consistent with a diagnosis of CF. Subjects who are homozygous for the F508del-CFTR mutation must have a sweat chloride value ≥60 mmol/L.
Subjects with ppFEV1 of ≥40 percentage points at the Screening Visit
Subjects with stable CF disease as deemed by the investigator at the Screening Visit.
Subjects who are willing to remain on their stable CF medication regimen through Day 14 (Part A) or through Week 24 (Part B) or, if applicable, through the Safety Follow up Visit.
Subjects who are able to swallow tablets.
Female subjects of childbearing potential must have a negative serum pregnancy test at the Screening Visit and a negative urine pregnancy test at the Day 1 Visit before receiving the first dose of study drug.
Subjects of childbearing potential who are sexually active must meet the contraception requirements
Exclusion criteria
Primary purpose
Allocation
Interventional model
Masking
83 participants in 2 patient groups
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Data sourced from clinicaltrials.gov
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