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

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

ArmDescriptionAssigned 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.
  • Drug: LUM
    Fixed Dose Combination (FDC) granule (LUM/IVA)
    Other names:
    • lumacaftor
    • VX-809
  • Drug: IVA
    FDC granule (LUM/IVA)
    Other names:
    • ivacaftor
    • VX-770
Experimental
Part A Cohort 2 [12 to <18months]
Subjects will receive LUM/IVA as FDC granules dependent upon weight at Day 1.
  • Drug: LUM
    Fixed Dose Combination (FDC) granule (LUM/IVA)
    Other names:
    • lumacaftor
    • VX-809
  • Drug: IVA
    FDC granule (LUM/IVA)
    Other names:
    • ivacaftor
    • VX-770
Experimental
Part B
Subjects will receive LUM/IVA as FDC granules dependent upon weight at Day 1.
  • Drug: LUM
    Fixed Dose Combination (FDC) granule (LUM/IVA)
    Other names:
    • lumacaftor
    • VX-809
  • Drug: IVA
    FDC granule (LUM/IVA)
    Other names:
    • ivacaftor
    • VX-770

Recruiting Locations

University of Alabama at Birmingham
Birmingham, Alabama 35233

More Details

NCT ID
NCT03601637
Status
Recruiting
Sponsor
Vertex Pharmaceuticals Incorporated

Study Contact

Medical Information
617-341-6777
medicalinfo@vrtx.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.