Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del
- Conditions
- Cystic Fibrosis
- Interventions
- Drug: LUMDrug: IVA
- Registration Number
- NCT03601637
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- 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).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 61
- 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
- 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
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Part A: LUM/IVA IVA Participants 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 IVA Participants 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. Part B: LUM/IVA LUM Participants 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. Part A: LUM/IVA LUM Participants 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.
- Primary Outcome Measures
Name Time Method 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 Part A: Observed Pre-dose Plasma Concentration (Ctrough) of LUM and IVA Pre-dose at Day 8 and Day 15 Part A: Observed Plasma Concentrations From 3-4 Hours (C3-4hr) of LUM and IVA Day 1 and Day 15
- Secondary Outcome Measures
Name Time Method 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 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
Trial Locations
- Locations (27)
Children's Medical Center of Dallas
🇺🇸Dallas, Texas, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
Children's Healthcare of Atlanta
🇺🇸Atlanta, Georgia, United States
Johns Hopkins Hospital
🇺🇸Baltimore, Maryland, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
🇺🇸Minneapolis, Minnesota, United States
University of Rochester Medical Center
🇺🇸Rochester, New York, United States
University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
Yale New Haven Medical Center
🇺🇸New Haven, Connecticut, 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
Cardinal Glennon Children's Hospital - St. Louis University
🇺🇸Saint Louis, Missouri, United States
Wake Forest University School of Medicine - Brenner Children's Hospital
🇺🇸Winston-Salem, North Carolina, United States
The Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
University of North Carolina Hospitals
🇺🇸Chapel Hill, North Carolina, United States
University of Utah / Primary Children's Medical Center
🇺🇸Salt Lake City, Utah, United States
McGill University Health Centre, Glen Site, Montreal Children's Hospital
🇨🇦Montreal, Canada
University of Wisconsin Hospital and Clinics
🇺🇸Madison, Wisconsin, United States
British Columbia's Children's Hospital
🇨🇦Vancouver, Canada
The Hospital for Sick Children
🇨🇦Toronto, Canada
Stanford University
🇺🇸Palo Alto, California, United States
Cook Children's Medical Center
🇺🇸Fort Worth, Texas, United States
Nemours / Alfred I. duPont Hospital for Children
🇺🇸Wilmington, Delaware, United States