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

Phase 3Enrolling By InvitationNCT05844449

Vertex Pharmaceuticals Incorporated174 enrolled

Overview

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).

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

174

Conditions

Cystic Fibrosis

Interventions

VNZ/TEZ/D-IVADRUG

Fixed-dose combination tablets or granules for oral administration.

Eligibility

Sex: ALLMin age: 1 Year

Medical Language ↔ Plain English

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.

Locations (37)

Outcomes

Primary Outcomes

Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)

Time frame: From Baseline up to Week 100

Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)

Time frame: From Baseline up to Week 196

Secondary Outcomes

Part A (All Cohorts): Absolute Change in Sweat Chloride (SwCl)

Time frame: From Baseline Through Week 96

Part B: Absolute Change in Sweat Chloride (SwCl)

Time frame: From Baseline Through Week 192

Part A (Cohort 1): Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)

Time frame: From Baseline Through Week 100

Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)

Time frame: From Baseline Through Week 196

Part A (All Cohorts): Number of Pulmonary Exacerbation (PEx)

Time frame: From Baseline Through Week 100

Part B: Number of Pulmonary Exacerbation (PEx)

Time frame: From Baseline Through Week 196

Part A (All Cohorts): Number of CF- Related Hospitalizations

Time frame: From Baseline Through Week 100

Part B: Number of CF- Related Hospitalizations

Time frame: From Baseline Through Week 196

Data from ClinicalTrials.gov

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Children's Hospital of Orange County

Orange, California, United States

Stanford University - Palo Alto - Pulmonology

Palo Alto, California, United States

Children's Hospital of Colorado - Pulmonology

Aurora, Colorado, United States

The Emory Clinic - Clifton Road - Pulmonology

Atlanta, Georgia, United States

Ann & Robert H. Lurie Children's Hospital of Chicago - Hematology

Chicago, Illinois, United States

JW Riley Hospital for Children - Pulmonology

Indianapolis, Indiana, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Children's Health Care d/b/a Children's Hospitals and Clinics of Minnesota

Minneapolis, Minnesota, United States

The Children's Mercy Hospital

Kansas City, Missouri, United States

St. Louis Children's Hospital - Pulmonology

St Louis, Missouri, United States

...and 27 more locations

Part A (Cohort 1): Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score

Time frame: From Baseline Through Week 100

Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score

Time frame: From Baseline Through Week 196

Part A (Cohorts 1 and 2): Absolute Change in Body Mass Index (BMI)

Time frame: From Baseline Through Week 100

Part B: Absolute Change in Body Mass Index (BMI)

Time frame: From Baseline Through Week 196

Part A (Cohorts 1 and 2): Absolute Change in BMI-for-age Z-score

Time frame: From Baseline Through Week 100

Part B: Absolute Change in BMI-for-age Z-score

Time frame: From Baseline Through Week 196

Part A (Cohort 3): Absolute Change in Weight-for-length

Time frame: From Baseline Through Week 100

Part B: Absolute Change in Weight-for-length

Time frame: From Baseline Through Week 196

Part A (Cohort 3): Absolute Change in Weight-for-length Z-score

Time frame: From Baseline Through Week 100

Part B: Absolute Change in Weight-for-length Z-score

Time frame: From Baseline Through Week 196

Part A (All Cohorts): Absolute Change in Weight

Time frame: From Baseline Through Week 100

Part B: Absolute Change in Weight

Time frame: From Baseline Through Week 196

Part A (All Cohorts): Change in Weight-for-age Z-score

Time frame: From Baseline Through Week 100

Part B: Change in Weight-for-age Z-score

Time frame: From Baseline Through Week 196

Part A (Cohorts 1 and 2): Absolute Change in Height

Time frame: From Baseline Through Week 100

Part B: Absolute Change in Height

Time frame: From Baseline Through Week 196

Part A (Cohorts 1 and 2): Absolute Change in Height-for-age Z-score

Time frame: From Baseline Through Week 100

Part B: Absolute Change in Height-for-age Z-score

Time frame: From Baseline Through Week 196

Part A (Cohort 3): Absolute Change in Length

Time frame: From Baseline Through Week 100

Part B: Absolute Change in Length

Time frame: From Baseline Through Week 196

Part A (Cohort 3): Absolute Change in Length-for-age Z-score

Time frame: From Baseline Through Week 100

Part B: Absolute Change in Length-for-age Z-score

Time frame: From Baseline Through Week 196