Status:
COMPLETED
Orkambi Treatment in 2 to 5 Year Old Children With CF
Lead Sponsor:
Children's Hospital of Philadelphia
Collaborating Sponsors:
Vertex Pharmaceuticals Incorporated
Conditions:
Cystic Fibrosis
Eligibility:
All Genders
2-5 years
Brief Summary
The purpose of this observational research study is to determine the effects of clinically prescribed Orkambi treatment on 2 to 5 year old children homozygous for the F508del Mutations in the Cystic f...
Detailed Description
Orkambi is a novel FDA approved (August, 2018) therapy for use in patients with cystic fibrosis (CF) who are 2 to 5 years of age and homozygous for F508del mutations in the CFTR gene. It is a combinat...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
April 10 2019
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
June 16 2021
Estimated Enrollment :
28 Patients enrolled
Trial Details
Trial ID
NCT03795363
Start Date
April 10 2019
End Date
June 16 2021
Last Update
December 2 2021
Active Locations (1)
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1
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19146