Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del
A Phase 3, 2-part, Open-label Study to Evaluate the Safety and Pharmacokinetics of Lumacaftor/Ivacaftor in Subjects 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del
2 other identifiers
interventional
61
2 countries
27
Brief Summary
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Sep 2018
Typical duration for phase_3
27 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 18, 2018
CompletedFirst Posted
Study publicly available on registry
July 26, 2018
CompletedStudy Start
First participant enrolled
September 7, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 29, 2021
CompletedStudy Completion
Last participant's last visit for all outcomes
October 29, 2021
CompletedResults Posted
Study results publicly available
January 6, 2023
CompletedJanuary 6, 2023
December 1, 2022
3.1 years
July 18, 2018
October 28, 2022
December 14, 2022
Conditions
Outcome Measures
Primary Outcomes (3)
Part A: Observed Plasma Concentrations From 3-4 Hours (C3-4hr) of LUM and IVA
Day 1 and Day 15
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA
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)
From Day 1 up to Week 26
Secondary Outcomes (4)
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
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)
Pre-dose at Day 8 and Day 15
Part B: Absolute Change in Sweat Chloride
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)
Pre-dose at Day 15, Week 4, Week 12 and Week 24
Study Arms (2)
Part A: LUM/IVA
EXPERIMENTALParticipants 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.
Part B: LUM/IVA
EXPERIMENTALParticipants 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.
Interventions
Eligibility Criteria
You may qualify if:
- 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)
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (27)
University of Alabama at Birmingham
Birmingham, Alabama, 35233, United States
Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
Stanford University
Palo Alto, California, 94304, United States
Children's Hospital Colorado
Aurora, Colorado, 80045, United States
Yale New Haven Medical Center
New Haven, Connecticut, 06511, United States
Nemours / Alfred I. duPont Hospital for Children
Wilmington, Delaware, 19803, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, 30329, United States
Ann & Robert Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, 46202, United States
Johns Hopkins Hospital
Baltimore, Maryland, 21287, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, 55404, United States
The Children's Mercy Hospital
Kansas City, Missouri, 64108, United States
Cardinal Glennon Children's Hospital - St. Louis University
St Louis, Missouri, 63104, United States
University of Rochester Medical Center
Rochester, New York, 14642, United States
University of North Carolina Hospitals
Chapel Hill, North Carolina, 27514, United States
Wake Forest University School of Medicine - Brenner Children's Hospital
Winston-Salem, North Carolina, 27157, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Children's Medical Center of Dallas
Dallas, Texas, 75235, United States
Cook Children's Medical Center
Fort Worth, Texas, 76104, United States
University of Utah / Primary Children's Medical Center
Salt Lake City, Utah, 84132, United States
Seattle Children's Hospital
Seattle, Washington, 98105, United States
University of Wisconsin Hospital and Clinics
Madison, Wisconsin, 53792, United States
McGill University Health Centre, Glen Site, Montreal Children's Hospital
Montreal, Canada
The Hospital for Sick Children
Toronto, Canada
British Columbia's Children's Hospital
Vancouver, Canada
Related Publications (3)
Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.
PMID: 37983082DERIVEDRayment JH, Asfour F, Rosenfeld M, Higgins M, Liu L, Mascia M, Paz-Diaz H, Tian S, Zahigian R, McColley SA. A Phase 3, Open-Label Study of Lumacaftor/Ivacaftor in Children 1 to Less Than 2 Years of Age with Cystic Fibrosis Homozygous for F508del-CFTR. Am J Respir Crit Care Med. 2022 Nov 15;206(10):1239-1247. doi: 10.1164/rccm.202204-0734OC.
PMID: 35771568DERIVEDSouthern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
PMID: 33331662DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Monitor
- Organization
- Vertex Pharmaceuticals Incorporated
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 18, 2018
First Posted
July 26, 2018
Study Start
September 7, 2018
Primary Completion
October 29, 2021
Study Completion
October 29, 2021
Last Updated
January 6, 2023
Results First Posted
January 6, 2023
Record last verified: 2022-12
Data Sharing
- IPD Sharing
- Will not share
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing