CFDB - Cystic Fibrosis DataBase

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ongoing trials trial from other registries

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

  • Year: 2023
  • Date:
  • Author:
Source

https://www.clinicaltrialsregister.eu/ctr-search/trial/2023-000946-41/3rd/

Study design (if review, criteria of inclusion for studies)

Controlled: No.1 Randomised: No.2 Open: Yes.3 Single blind: No.4 Double blind: No.5 Parallel group: No.6 Cross over: No

Participants

Principal inclusion criteria: 1.Subjects From Study VX16-809-122 Part B (Study 122) - Completed the 24-week Treatment Period and the Safety Followup Visit in Study 122B 2.Subjects Not From Study 122 - Subjects will be 1 to less than 2 years of age - Homozygous for the F508del mutation (F/F)

Interventions

Long-term lumacaftor/ivacaftor (LUM/IVA) treatment

Outcome measures

Primary end point(s): Safety and tolerability as assessed by the number of adverse events (AEs) and serious adverse events (SAEs)

Related topics

CFTR modulators therapy

Keywords: Child; Aminophenols; CFTR Modulators; Genetic Predisposition to Disease; Orkambi; pharmacological_intervention; VX-770; ivacaftor; lumacaftor; VX-809;