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

Cystic Fibrosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03601637
• Other study ID #: VX16-809-122
• Secondary ID: 2017-004794-13
• Status: Completed
• Phase: Phase 3
• Start date: September 7, 2018
• Est. completion date: October 29, 2021

Study information

• Verified date: December 2022
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 61
• Est. completion date: October 29, 2021
• Est. primary completion date: October 29, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Months to 23 Months

Eligibility

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.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• LUM
Fixed Dose Combination (FDC) granules (LUM/IVA).
• IVA
FDC granules (LUM/IVA).

Locations

Country	Name	City	State
Canada	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
United States	Children's Healthcare of Atlanta	Atlanta	Georgia
United States	Children's Hospital Colorado	Aurora	Colorado
United States	Johns Hopkins Hospital	Baltimore	Maryland
United States	University of Alabama at Birmingham	Birmingham	Alabama
United States	Boston Children's Hospital	Boston	Massachusetts
United States	University of North Carolina Hospitals	Chapel Hill	North Carolina
United States	Ann & Robert Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Children's Medical Center of Dallas	Dallas	Texas
United States	Cook Children's Medical Center	Fort Worth	Texas
United States	Riley Hospital for Children at Indiana University Health	Indianapolis	Indiana
United States	The Children's Mercy Hospital	Kansas City	Missouri
United States	Arkansas Children's Hospital	Little Rock	Arkansas
United States	University of Wisconsin Hospital and Clinics	Madison	Wisconsin
United States	Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota	Minneapolis	Minnesota
United States	Yale New Haven Medical Center	New Haven	Connecticut
United States	Stanford University	Palo Alto	California
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	University of Rochester Medical Center	Rochester	New York
United States	Cardinal Glennon Children's Hospital - St. Louis University	Saint Louis	Missouri
United States	University of Utah / Primary Children's Medical Center	Salt Lake City	Utah
United States	Seattle Children's Hospital	Seattle	Washington
United States	Nemours / Alfred I. duPont Hospital for Children	Wilmington	Delaware
United States	Wake Forest University School of Medicine - Brenner Children's Hospital	Winston-Salem	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Part A: Observed Plasma Concentrations From 3-4 Hours (C3-4hr) of LUM and IVA		Day 1 and Day 15
Primary	Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA		Pre-dose at Day 8 and Day 15
Primary	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	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
Secondary	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
Secondary	Part B: Absolute Change in Sweat Chloride		From Baseline at Week 24
Secondary	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