Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del

Vertex Pharmaceuticals Incorporated61 enrolled

Overview

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).

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Cystic Fibrosis

Interventions

LUMDRUG

Fixed Dose Combination (FDC) granules (LUM/IVA).

IVADRUG

FDC granules (LUM/IVA).

Eligibility

Sex: ALLMin age: 12 MonthsMax age: 23 Months

Medical Language ↔ Plain English

Key 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) Key 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.

Locations (27)

Outcomes

Primary Outcomes

Part A: Observed Plasma Concentrations From 3-4 Hours (C3-4hr) of LUM and IVA

Time frame: Day 1 and Day 15

Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA

Time frame: Pre-dose at Day 8 and Day 15

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

Time frame: From Day 1 up to Week 26

Secondary Outcomes

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

Time frame: From Day 1 up to Day 25

Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)

Time frame: Pre-dose at Day 8 and Day 15

Part B: Absolute Change in Sweat Chloride

Time frame: From Baseline at Week 24

Part B: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA and Their Respective Metabolites (M28-LUM, M1-IVA and M6-IVA)

Time frame: Pre-dose at Day 15, Week 4, Week 12 and Week 24

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.

University of Alabama at Birmingham

Birmingham, Alabama, United States

Arkansas Children's Hospital

Little Rock, Arkansas, United States

Stanford University

Palo Alto, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Yale New Haven Medical Center

New Haven, Connecticut, United States

Nemours / Alfred I. duPont Hospital for Children

Wilmington, Delaware, United States

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Ann & Robert Lurie Children's Hospital of Chicago

Chicago, Illinois, United States

Riley Hospital for Children at Indiana University Health

Indianapolis, Indiana, United States

Johns Hopkins Hospital

Baltimore, Maryland, United States

...and 17 more locations