Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older

Part of paid clinical trials in Orange, California.

Sponsor
Vertex Pharmaceuticals Incorporated
Study ID
NCT05844449
Phase
PHASE3
Status
Enrolling By Invitation

Conditions

  • Cystic Fibrosis

Eligibility Criteria

Sex
ALL
Age
1 Year - N/A
Healthy Volunteers
Not accepted

Interventions

  • VNZ/TEZ/D-IVA — DRUG
    Fixed-dose combination tablets or granules for oral administration.

Study Details

The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) in participants with cystic fibrosis (CF).

Key Dates

Start date
Aug 11, 2023
Status verified
Apr 2026
Primary completion
Jul 30, 2029
Completion
Jul 30, 2029

Study Design

Enrollment
174 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: VNZ/TEZ/D-IVA
    Part A: Participants (Cohort 1: 6 through 11 years of age (inclusive); Cohort 2: 2 through 5 years of age (inclusive) and Cohort 3: 1 to less than (\<) 2 years of age) will receive VNZ/TEZ/D-IVA once daily at doses determined based on their age and weight. The age range is based on date of informed consent in parent study. Part B: Participants in Cohort 1 who meet the eligibility criteria will receive VNZ/TEZ/D-IVA for an additional 96 weeks.

Primary Outcome Measure

Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) [ Time Frame: From Baseline up to Week 100 ]

Locations (18)

FacilityCityStateZIPSite coordinators
Children's Hospital of Orange CountyOrangeCalifornia92868-
Stanford University - Palo Alto - PulmonologyPalo AltoCalifornia94304-
Children's Hospital of Colorado - PulmonologyAuroraColorado80045-
The Emory Clinic - Clifton Road - PulmonologyAtlantaGeorgia30322-
Ann & Robert H. Lurie Children's Hospital of Chicago - HematologyChicagoIllinois60611-
JW Riley Hospital for Children - PulmonologyIndianapolisIndiana46202-
Boston Children's HospitalBostonMassachusetts02115-
Children's Health Care d/b/a Children's Hospitals and Clinics of MinnesotaMinneapolisMinnesota55404-
The Children's Mercy HospitalKansas CityMissouri64108-
St. Louis Children's Hospital - PulmonologySt LouisMissouri63110-
Cohen Children's Medical Center - Lakeville RoadLake SuccessNew York11042-
Cincinnati Children's Hospital Medical Center - PulmonologyCincinnatiOhio45229-
UH Cleveland Medical Center - PulmonologyClevelandOhio44106-
Nationwide Children's HospitalColumbusOhio43205-
Children's Hospital of Pittsburgh - PulmonologyPittsburghPennsylvania15224-
Texas Children's Hospital - Wallace Tower - PulmonologyHoustonTexas77030-
Vermont Lung CenterColchesterVermont05446-
American Family Childrens HospitalMadisonWisconsin53792-

Find similar trials in Orange, CA

By research site
Children's Hospital of Orange County· Orange, CAStanford University - Palo Alto - Pulmonology· Palo Alto, CAChildren's Hospital of Colorado - Pulmonology· Aurora, COThe Emory Clinic - Clifton Road - Pulmonology· Atlanta, GAAnn & Robert H. Lurie Children's Hospital of Chicago - Hematology· Chicago, ILJW Riley Hospital for Children - Pulmonology· Indianapolis, IN

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