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Completed

NCT Number: NCT03601637

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

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).

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

12 month–23 month

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

McGill University Health Centre, Glen Site, Montreal Children's Hospital, Montreal, Canada

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

LUM

Drug

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

Other names: lumacaftor, VX-809

IVA

Drug

FDC granules (LUM/IVA).

Other names: ivacaftor, VX-770

Primary outcomes

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

    Time frame: Day 1 and Day 15

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

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

  3. 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

  1. 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

  2. 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

  3. Part B: Absolute Change in Sweat Chloride

    Time frame: From Baseline at Week 24

  4. 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

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study 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

Important dates

Study start
2018
Primary completion
2021
Study completion
2021
First posted
Jul 26, 2018
Registry last updated
Jan 6, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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