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This study examines Orkambi treatment in children aged 2 to 5 with cystic fibrosis who are homozygous for the F508del mutation. Children in generally good health for whom a clinical decision had been made to begin Orkambi were able to take part. This was a phase-not-applicable study and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * Cystic fibrosis and homozygous for F508del mutations, approved for treatment * Age: 2.0 to 5.9 years * In usual state of good health * A clinical decision has been made for subject to begin Orkambi treatment * Family committed to the 6 to 8 month study protocol with visits to the Children's Hospital of Philadelphia (CHOP) that will last 2-3 days for the baseline visit (Visit 1) prior to Orkambi and the 24 week visit (Visit 3) after clinically prescribed Orkambi treatment has begun, and will last up to 2 days for the 12 week visit (Visit 2) after Orkambi treatment has begun. Exclusion Criteria: * On parenteral nutrition * Use of any medications that inhibit or induce cytochrome P450 (CYP) 3A * Liver function tests elevated above 3x the reference range for age and sex * Lung disease considered severe based on clinical impression by home CF center. * Other illness affecting growth or nutritional status * Other contraindications described for Orkambi therapy