Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age

Cystic Fibrosis Clinical Trial

Official title:

A Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-121/Tezacaftor/Deutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Through 11 Years of Age

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05422222
• Other study ID #: VX21-121-105
• Secondary ID: 2021-005930-40
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: June 21, 2022
• Est. completion date: June 2030

Study information

• Verified date: June 2023
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to evaluate the pharmacokinetics, safety, tolerability and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants with at least 1 triple combination responsive (TCR) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 210
• Est. completion date: June 2030
• Est. primary completion date: June 2030
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 11 Years

Eligibility

Key Inclusion Criteria:

• Participants with stable CF and at least 1 TCR mutation (including F508del) in the CFTR gene

Key Exclusion Criteria:

• History of solid organ, hematological transplantation, or cancer

• Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)

• Lung infection with organisms associated with a more rapid decline in pulmonary status

Other protocol defined Inclusion/Exclusion criteria may apply.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• VX-121/TEZ/D-IVA
Fixed-dose combination for oral administration.

Locations

Country	Name	City	State
Australia	Telethon Kids Institute	Nedlands
Australia	Women & Children's Hospital	North Adelaide
Australia	The Royal Children's Hospital	Parkville
Australia	Queensland Children's Hospital	South Brisbane
France	CHU Lyon - Hopital Femme Mere-Enfant	Bron Cedex
France	Hopital Necker, Enfants Malades	Paris Cedex 15
Germany	Charite Paediatric Pulmonology Department	Berlin
Germany	Kinderklinik III, Abt. fur Pneumologie	Essen
Germany	Medizinische Hochschule Hannover	Hannover
Netherlands	Erasmus Medical Center / Sophia Children's Hospital	Rotterdam
Sweden	Sahlgrenska Universitetssjukhuset	Göteborg
Switzerland	Inselspital - Universitaetsspital Bern	Bern
Switzerland	Kinderspital Zuerich	Zürich
United Kingdom	Children and Young Adults Research Unit	Cardiff
United Kingdom	Great Ormond Street Hospital for Children	London
United States	The Emory Clinic / Children's Healthcare of Atlanta at Egleston	Atlanta	Georgia
United States	Children's Hospital of Colorado	Aurora	Colorado
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center	Cleveland	Ohio
United States	Vermont Lung Center	Colchester	Vermont
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	Texas Children's Hospital - Baylor College of Medicine	Houston	Texas
United States	Riley Hospital for Children at Indiana University Health	Indianapolis	Indiana
United States	The Children's Mercy Hospital	Kansas City	Missouri
United States	University of Wisconsin Hospital and Clinics	Madison	Wisconsin
United States	Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota	Minneapolis	Minnesota
United States	Cohen Children's Medical Center	New York	New York
United States	Children's Hospital of Orange County	Orange	California
United States	Stanford University	Palo Alto	California
United States	UPMC Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	Oregon Health & Science University	Portland	Oregon
United States	Washington University School of Medicine / St. Louis Children's Hospital	Saint Louis	Missouri

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

United States,  Australia,  France,  Germany,  Netherlands,  Sweden,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Part A: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites		From Day 1 up to Day 22
Primary	Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)		From Day 1 up to Day 50
Primary	Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)		From Day 1 up to Week 28
Secondary	Part B: Absolute Change in Sweat Chloride (SwCl)		From Baseline Through Week 24
Secondary	Part B: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites		From Day 1 up to Week 16
Secondary	Part B: Drug Acceptability Assessment Using Modified Facial Hedonic Scale		At Day 1 and Week 24
Secondary	Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)		From Baseline Through Week 24
Secondary	Part B: Number of Pulmonary Exacerbation (PEx)		From Baseline Through Week 24
Secondary	Part B: Number of CF-Related Hospitalizations		From Baseline Through Week 24
Secondary	Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score		From Baseline Through Week 24
Secondary	Part B: Absolute Change in Body Mass Index (BMI)		From Baseline at Week 24
Secondary	Part B: Absolute Change in BMI-for-age Z-score		From Baseline at Week 24
Secondary	Part B: Absolute Change in Weight		From Baseline at Week 24
Secondary	Part B: Absolute Change in Weight-for-age Z-score		From Baseline at Week 24
Secondary	Part B: Absolute Change in Weight-for-length		From Baseline at Week 24
Secondary	Part B: Absolute Change in Weight-for-length Z-score		From Baseline at Week 24
Secondary	Part B: Absolute Change in Height		From Baseline at Week 24
Secondary	Part B: Absolute Change in Height-for-age Z-score		From Baseline at Week 24
Secondary	Part B: Absolute Change in Length		From Baseline at Week 24
Secondary	Part B: Absolute Change in Length-for-age Z-score		From Baseline at Week 24
Secondary	Part B: Proportion of Participants With SwCl <60 millimole per liter (mmol/L)		From Baseline Through Week 24
Secondary	Part B: Proportion of Participants With SwCl <30 mmol/L		From Baseline Through Week 24