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OpenTrials
Completed

NCT Number: NCT04235140

Long-term Safety of Lumacaftor/Ivacaftor in Participants With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation

This is a Phase 3, multicenter, open-label and roll-over study in participants who are 12 to <24 months of age at initiation of Lumacaftor/Ivacaftor (LUM/IVA) treatment.

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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 and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

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

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

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

Key Inclusion Criteria:

  • Participants From Study VX16-809-122 Part B (Study 122)
  • Completed the 24-week Treatment Period and the Safety Follow-up Visit in Study 122B
  • Participants Not From Study 122
  • Subjects will be 1 to less than 2 years of age
  • Homozygous for the F508del mutation (F/F)

Key Exclusion Criteria:

  • Any clinically significant laboratory abnormalities that would interfere with the study assessments or pose an undue risk for the subject
  • Solid organ or hematological transplantation

Other protocol defined Inclusion/Exclusion criteria may apply.

Treatment and study plan

LUM/IVA

Drug

LUM/IVA granules for oral administration

Other names: lumacaftor/ivacaftor, VX-809/VX-770

Primary outcomes

  1. Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

    Time frame: Day 1 up to Week 120

Secondary outcomes

  1. Absolute Change in Sweat Chloride (SwCl)

    Time frame: From Baseline at Week 96

    Sweat samples were collected using an approved collection device.

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study title

A Phase 3, Open-label, and Rollover Study to Evaluate the Long-term Safety and Tolerability of Lumacaftor/Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Homozygous for F508del and 12 to <24 Months of Age at Treatment Initiation

Important dates

Study start
2020
Primary completion
2023
Study completion
2023
First posted
Jan 21, 2020
Registry last updated
Sep 19, 2024

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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