Clinical trial NCT06460506: Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Participants 12 Months of Age and Older
Official title: A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 12 Months of Age and Older
Study type: INTERVENTIONAL
Overall status: ENROLLING_BY_INVITATION
Phase: PHASE3
Conditions: Cystic Fibrosis
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2024-06-14
Enrollment: 50
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of the study is to evaluate the long-term safety, tolerability, efficacy, and pharmacodynamics (PD) of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA).


Eligibility criteria: Key Inclusion Criteria:

* Completed study drug treatment in the parent study VX22-445-122 Part B (NCT05882357) OR had study drug interruption(s) in the parent study but did not permanently discontinue study drug and completed study visits up to the last scheduled visit of the Treatment Period of the parent study

Key Exclusion Criteria:

* History of drug intolerance in the parent study that would pose an additional risk to the participant
* Current participation in an investigational drug trial other than the parent study

Other protocol defined Inclusion/Exclusion criteria may apply.

Primary outcomes:
measure: Safety and Tolerability as Assessed by Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Day 1 up to Week 100
Secondary outcomes:
measure: Absolute Change in Sweat Chloride (SwCl)
timeFrame: From Baseline through Week 96
Other outcomes:
Locations:
city: Nedlands
country: Australia
facility: The Kids Research Institute Australia
city: Parkville
country: Australia
facility: The Royal Children's Hospital
city: South Brisbane
country: Australia
facility: Queensland Children's Hospital
city: Toronto
country: Canada
facility: The Hospital for Sick Children
city: Vancouver
country: Canada
facility: British Columbia Children's Hospital
city: Copenhagen
country: Denmark
facility: Juliane Marie Center, Rigshospitalet
city: Berlin
country: Germany
facility: Charite Paediatric Pulmonology Department
city: Essen
country: Germany
facility: Kinderklinik III, Abt. fur Pneumologie
city: Rotterdam
country: Netherlands
facility: Erasmus Medical Center
city: Bern
country: Switzerland
facility: Inselspital - Universitaetsspital Bern
city: Zurich
country: Switzerland
facility: Kinderspital Zurich
city: Cardiff
country: United Kingdom
facility: Children and Young Adults Research Unit
city: Leeds
country: United Kingdom
facility: Leeds General Infirmary
city: Liverpool
country: United Kingdom
facility: Alder Hey Children's NHS Foundation Trust
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children
city: London
country: United Kingdom
facility: Royal Brompton Hospital
city: Southampton
country: United Kingdom
facility: Southampton General Hospital
Study officials:
References: