Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Subjects 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del
Purpose
This study will evaluate the safety and pharmacokinetics (PK) of lumacaftor (LUM) and ivacaftor (IVA) in subjects 1 to less than 2 years of age with cystic fibrosis (CF), homozygous for F508del (F/F).
Condition
- Cystic Fibrosis
Eligibility
- Eligible Ages
- Between 12 Months and 23 Months
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- Subjects will be 1 to less than 2 years of age on Day 1 of the relevant part of the study.
- Homozygous for F508del (F/F).
Exclusion Criteria
- Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject.
- Solid organ or hematological transplantation.
Other protocol defined Inclusion/Exclusion criteria may apply.
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Non-Randomized
- Intervention Model
- Single Group Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Part A Cohort 1 [aged 18 to <24 months] |
Subjects will receive LUM/IVA as FDC granules dependent upon weight at Day 1. |
|
|
Experimental Part A Cohort 2 [12 to <18months] |
Subjects will receive LUM/IVA as FDC granules dependent upon weight at Day 1. |
|
|
Experimental Part B |
Subjects will receive LUM/IVA as FDC granules dependent upon weight at Day 1. |
|
Recruiting Locations
University of Alabama at Birmingham
Birmingham, Alabama 35294
Birmingham, Alabama 35294
More Details
- NCT ID
- NCT03601637
- Status
- Recruiting
- Sponsor
- Vertex Pharmaceuticals Incorporated