Long-term Safety of Lumacaftor/Ivacaftor in Participants With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation

Vertex Pharmaceuticals Incorporated52 enrolled

Overview

This is a Phase 3, multicenter, open-label and roll-over study in participants who are 12 to \<24 months of age at initiation of Lumacaftor/Ivacaftor (LUM/IVA) treatment.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Cystic Fibrosis

Interventions

LUM/IVADRUG

LUM/IVA granules for oral administration

Eligibility

Sex: ALLMin age: 12 Months

Medical Language ↔ Plain English

Key Inclusion Criteria: * Participants From Study VX16-809-122 Part B (Study 122) * Completed the 24-week Treatment Period and the Safety Follow-up Visit in Study 122B * Participants Not From Study 122 * Subjects will be 1 to less than 2 years of age * Homozygous for the F508del mutation (F/F) Key Exclusion Criteria: * Any clinically significant laboratory abnormalities 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.

Locations (23)

University of Alabama at Birmingham

Outcomes

Primary Outcomes

Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Time frame: Day 1 up to Week 120

Secondary Outcomes

Absolute Change in Sweat Chloride (SwCl)

Sweat samples were collected using an approved collection device.

Time frame: From Baseline at Week 96

Data from ClinicalTrials.gov

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