Clinical trial NCT05844449: Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older
Official title: A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Year 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: 2023-05-06
Enrollment: 174
Sex: ALL
Minimum age (years): 1
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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).


Eligibility criteria: Key Inclusion Criteria:

Parts A and B:

• Participants who have completed study drug treatment in the parent study (VX21-121-105; NCT Number: NCT05422222)

Part B:

-Meets at least 1 of the following criteria:

* Completed study drug treatment in Part A
* Had study drug interruption(s) in Part A, but did not permanently discontinue study drug and completed study visits up to the last scheduled visit of the treatment period of Part A

Key Exclusion Criteria:

* Hepatic cirrhosis with portal hypertension, moderate hepatic impairment, or severe hepatic impairment that might pose an additional risk in administering study drug
* History of solid organ, hematological transplantation, or cancer
* History of drug intolerance in the parent study

Other protocol defined Inclusion/Exclusion criteria may apply.

Primary outcomes:
measure: Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
timeFrame: From Baseline up to Week 100
measure: Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
timeFrame: From Baseline up to Week 196
Secondary outcomes:
measure: Part A (All Cohorts): Absolute Change in Sweat Chloride (SwCl)
timeFrame: From Baseline Through Week 96
measure: Part B: Absolute Change in Sweat Chloride (SwCl)
timeFrame: From Baseline Through Week 192
measure: Part A (Cohort 1): Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)
timeFrame: From Baseline Through Week 100
measure: Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)
timeFrame: From Baseline Through Week 196
measure: Part A (All Cohorts): Number of Pulmonary Exacerbation (PEx)
timeFrame: From Baseline Through Week 100
measure: Part B: Number of Pulmonary Exacerbation (PEx)
timeFrame: From Baseline Through Week 196
measure: Part A (All Cohorts): Number of CF- Related Hospitalizations
timeFrame: From Baseline Through Week 100
measure: Part B: Number of CF- Related Hospitalizations
timeFrame: From Baseline Through Week 196
measure: Part A (Cohort 1): Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score
timeFrame: From Baseline Through Week 100
measure: Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score
timeFrame: From Baseline Through Week 196
measure: Part A (Cohorts 1 and 2): Absolute Change in Body Mass Index (BMI)
timeFrame: From Baseline Through Week 100
measure: Part B: Absolute Change in Body Mass Index (BMI)
timeFrame: From Baseline Through Week 196
measure: Part A (Cohorts 1 and 2): Absolute Change in BMI-for-age Z-score
timeFrame: From Baseline Through Week 100
measure: Part B: Absolute Change in BMI-for-age Z-score
timeFrame: From Baseline Through Week 196
measure: Part A (Cohort 3): Absolute Change in Weight-for-length
timeFrame: From Baseline Through Week 100
measure: Part B: Absolute Change in Weight-for-length
timeFrame: From Baseline Through Week 196
measure: Part A (Cohort 3): Absolute Change in Weight-for-length Z-score
timeFrame: From Baseline Through Week 100
measure: Part B: Absolute Change in Weight-for-length Z-score
timeFrame: From Baseline Through Week 196
measure: Part A (All Cohorts): Absolute Change in Weight
timeFrame: From Baseline Through Week 100
measure: Part B: Absolute Change in Weight
timeFrame: From Baseline Through Week 196
measure: Part A (All Cohorts): Change in Weight-for-age Z-score
timeFrame: From Baseline Through Week 100
measure: Part B: Change in Weight-for-age Z-score
timeFrame: From Baseline Through Week 196
measure: Part A (Cohorts 1 and 2): Absolute Change in Height
timeFrame: From Baseline Through Week 100
measure: Part B: Absolute Change in Height
timeFrame: From Baseline Through Week 196
measure: Part A (Cohorts 1 and 2): Absolute Change in Height-for-age Z-score
timeFrame: From Baseline Through Week 100
measure: Part B: Absolute Change in Height-for-age Z-score
timeFrame: From Baseline Through Week 196
measure: Part A (Cohort 3): Absolute Change in Length
timeFrame: From Baseline Through Week 100
measure: Part B: Absolute Change in Length
timeFrame: From Baseline Through Week 196
measure: Part A (Cohort 3): Absolute Change in Length-for-age Z-score
timeFrame: From Baseline Through Week 100
measure: Part B: Absolute Change in Length-for-age Z-score
timeFrame: From Baseline Through Week 196
Other outcomes:
Locations:
city: Orange
country: United States
facility: Children's Hospital of Orange County
state: California
city: Palo Alto
country: United States
facility: Stanford University
state: California
city: Aurora
country: United States
facility: Children's Hospital of Colorado
state: Colorado
city: Atlanta
country: United States
facility: The Emory Clinic / Children's Healthcare of Atlanta at Egleston
state: Georgia
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Indianapolis
country: United States
facility: Riley Hospital for Children at Indiana University Health
state: Indiana
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Minneapolis
country: United States
facility: Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota
state: Minnesota
city: Kansas City
country: United States
facility: The Children's Mercy Hospital
state: Missouri
city: Saint Louis
country: United States
facility: Washington University School of Medicine / St. Louis Children's Hospital
state: Missouri
city: Lake Success
country: United States
facility: Cohen Children's Medical Center
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Cleveland
country: United States
facility: Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Pittsburgh
country: United States
facility: UPMC Children's Hospital of Pittsburgh
state: Pennsylvania
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Colchester
country: United States
facility: Vermont Lung Center
state: Vermont
city: Madison
country: United States
facility: University of Wisconsin Hospital and Clinics
state: Wisconsin
city: North Adelaide
country: Australia
facility: Women & Children's Hospital
city: Parkville
country: Australia
facility: The Royal Childrens Hospital
city: South Brisbane
country: Australia
facility: Queensland Children's Hospital
city: Bron Cedex
country: France
facility: CHU Lyon - Hopital Femme Mere-Enfant
city: Paris Cedex 15
country: France
facility: Hopital Necker, Enfants Malades
city: Berlin
country: Germany
facility: Charite Paediatric Pulmonology Department
city: Essen
country: Germany
facility: Kinderklinik III, Abt. fur Pneumologie
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover
city: Rotterdam
country: Netherlands
facility: Erasmus Medical Center / Sophia Children's Hospital
city: Göteborg
country: Sweden
facility: Sahlgrenska Universitetssjukhuset
city: Bern
country: Switzerland
facility: Inselspital - Universitaetsspital Bern
city: Zürich
country: Switzerland
facility: Kinderspital Zuerich
city: Cardiff
country: United Kingdom
facility: Children and Young Adults Research Unit
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children
Study officials:
References: