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Purpose

This study will evaluate the safety and pharmacokinetics (PK) of lumacaftor (LUM) and ivacaftor (IVA) in participants 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

  • Participants 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 participants - 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: LUM/IVA 		Participants weighing 7 to less than (<)10 kilograms (kg) at screening received LUM 75 milligrams (mg)/IVA 94 mg fixed-dose combination (FDC) every 12 hours (q12h) and those weighing 10 to <14 kg at screening received LUM 100 mg/IVA 125 mg q12h for 15 days. Participants weighing greater than or equal to (>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 15 days.
  • Drug: LUM
    Fixed Dose Combination (FDC) granules (LUM/IVA).
    Other names:
    • lumacaftor
    • VX-809
  • Drug: IVA
    FDC granules (LUM/IVA).
    Other names:
    • ivacaftor
    • VX-770
Experimental
Part B: LUM/IVA 		Participants weighing 7 to <9 kg at screening received LUM 75 mg/IVA 94 mg FDC q12h and those weighing 9 to <14 kg received LUM 100 mg/IVA 125 mg q12h for 24 weeks. Participants weighing >=14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h for 24 weeks. Doses were adjusted upwards for changes in weight.
  • Drug: LUM
    Fixed Dose Combination (FDC) granules (LUM/IVA).
    Other names:
    • lumacaftor
    • VX-809
  • Drug: IVA
    FDC granules (LUM/IVA).
    Other names:
    • ivacaftor
    • VX-770

More Details

Status
Completed
Sponsor
Vertex Pharmaceuticals Incorporated

Study Contact

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.