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NCT03601637 Clinical Trial

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
First received
Last updated
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
Health authority
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.

Study Design

Related Conditions & MeSH terms

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
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