A Study to Explore the Impact of Lumacaftor/Ivacaftor on Disease Progression in Subjects Aged 2 Through 5 Years With Cystic Fibrosis, Homozygous for F508del

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

51 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-08-10

Study Completion Date

2021-10-07

Brief Summary

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This study will explore the impact of lumacaftor/ivacaftor (LUM/IVA) on disease progression in subjects aged 2 through 5 years with cystic fibrosis (CF), homozygous for F508del (F/F).

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Detailed Description

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Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Double blinded in Part 1, and Open label in Part 2

Study Groups

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Part 1: Placebo

Participants received placebo matched to LUM/IVA in placebo-controlled period for 48 weeks.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo matched to LUM/IVA for oral administration.

Part 1: LUM/IVA

Participants weighing less than (\<)14 kilograms (kg) at screening received LUM 100 milligrams (mg)/IVA 125 mg fixed-dose combination (FDC) every 12 hours (q12h) in placebo-controlled period for 48 weeks. Participants weighing greater than or equals to (\>=)14 kg at screening received LUM 150 mg/IVA 188 mg FDC q12h in placebo-controlled period for 48 weeks.

Group Type EXPERIMENTAL

LUM/IVA

Intervention Type DRUG

FDC granules for oral administration.

Part 2: Overall LUM/IVA

Participants who received either placebo or LUM/IVA in placebo-controlled period administered LUM/IVA (either LUM 100 mg/IVA 125 mg FDC q12h or LUM 150 mg/IVA 188 mg FDC q12h as per their body weight for participants \<6 years of age at week 48 and LUM 200 mg/IVA 250 mg FDC q12h regardless of their body weight for participants \>=6 years of age at week 48) in open-label period for 48 weeks.

Group Type EXPERIMENTAL

LUM/IVA

Intervention Type DRUG

FDC tablets or granules for oral administration.

Interventions

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LUM/IVA

FDC tablets or granules for oral administration.

Intervention Type DRUG

LUM/IVA

FDC granules for oral administration.

Intervention Type DRUG

Placebo

Placebo matched to LUM/IVA for oral administration.

Intervention Type DRUG

Other Intervention Names

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Lumacaftor/Ivacaftor VX-809/VX-770 Lumacaftor/Ivacaftor VX-809/VX-770

Eligibility Criteria

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Inclusion Criteria

* Subjects with confirmed diagnosis of CF.
* Homozygous for F508del (F/F).
* Subjects who weigh ≥8 kg without shoes and wearing light clothing at the Screening Visit.

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.
* History of any illness or comorbidity reviewed at the Screening Visit that, in the opinion of the investigator, might confound the results of the study.

Minimum Eligible Age

2 Years

Maximum Eligible Age

5 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No