Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 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 participants 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

  • 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