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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05422222




Registration number
NCT05422222
Ethics application status
Date submitted
13/06/2022
Date registered
16/06/2022
Date last updated
25/01/2024

Titles & IDs
Public title
Evaluation of VX-121/Tezacaftor/Deutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age
Scientific 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
Secondary ID [1] 0 0
2021-005930-40
Secondary ID [2] 0 0
VX21-121-105
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Cystic Fibrosis 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Cystic fibrosis
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Inflammatory and Immune System 0 0 0 0
Connective tissue diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - VX-121/TEZ/D-IVA

Experimental: Part A: VX-121/TEZ/D-IVA - Participants will receive VX-121/TEZ/D-IVA in the morning.

Experimental: Part B: VX-121/TEZ/D-IVA - Participants will receive VX-121/TEZ/D-IVA in the morning with the dose(s) to be based on the outcome of Part A.


Treatment: Drugs: VX-121/TEZ/D-IVA
Fixed-dose combination for oral administration.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites
Timepoint [1] 0 0
From Day 1 up to Day 22
Primary outcome [2] 0 0
Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [2] 0 0
From Day 1 up to Day 50
Primary outcome [3] 0 0
Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timepoint [3] 0 0
From Day 1 up to Week 28
Secondary outcome [1] 0 0
Part B: Absolute Change in Sweat Chloride (SwCl)
Timepoint [1] 0 0
From Baseline Through Week 24
Secondary outcome [2] 0 0
Part B: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites
Timepoint [2] 0 0
From Day 1 up to Week 16
Secondary outcome [3] 0 0
Part B: Drug Acceptability Assessment Using Modified Facial Hedonic Scale
Timepoint [3] 0 0
At Day 1 and Week 24
Secondary outcome [4] 0 0
Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)
Timepoint [4] 0 0
From Baseline Through Week 24
Secondary outcome [5] 0 0
Part B: Number of Pulmonary Exacerbation (PEx)
Timepoint [5] 0 0
From Baseline Through Week 24
Secondary outcome [6] 0 0
Part B: Number of CF-Related Hospitalizations
Timepoint [6] 0 0
From Baseline Through Week 24
Secondary outcome [7] 0 0
Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score
Timepoint [7] 0 0
From Baseline Through Week 24
Secondary outcome [8] 0 0
Part B: Absolute Change in Body Mass Index (BMI)
Timepoint [8] 0 0
From Baseline at Week 24
Secondary outcome [9] 0 0
Part B: Absolute Change in BMI-for-age Z-score
Timepoint [9] 0 0
From Baseline at Week 24
Secondary outcome [10] 0 0
Part B: Absolute Change in Weight
Timepoint [10] 0 0
From Baseline at Week 24
Secondary outcome [11] 0 0
Part B: Absolute Change in Weight-for-age Z-score
Timepoint [11] 0 0
From Baseline at Week 24
Secondary outcome [12] 0 0
Part B: Absolute Change in Weight-for-length
Timepoint [12] 0 0
From Baseline at Week 24
Secondary outcome [13] 0 0
Part B: Absolute Change in Weight-for-length Z-score
Timepoint [13] 0 0
From Baseline at Week 24
Secondary outcome [14] 0 0
Part B: Absolute Change in Height
Timepoint [14] 0 0
From Baseline at Week 24
Secondary outcome [15] 0 0
Part B: Absolute Change in Height-for-age Z-score
Timepoint [15] 0 0
From Baseline at Week 24
Secondary outcome [16] 0 0
Part B: Absolute Change in Length
Timepoint [16] 0 0
From Baseline at Week 24
Secondary outcome [17] 0 0
Part B: Absolute Change in Length-for-age Z-score
Timepoint [17] 0 0
From Baseline at Week 24
Secondary outcome [18] 0 0
Part B: Proportion of Participants With SwCl <60 millimole per liter (mmol/L)
Timepoint [18] 0 0
From Baseline Through Week 24
Secondary outcome [19] 0 0
Part B: Proportion of Participants With SwCl <30 mmol/L
Timepoint [19] 0 0
From Baseline Through Week 24

Eligibility
Key inclusion criteria
Key

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

Key
Minimum age
1 Year
Maximum age
11 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
21/06/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/06/2030
Actual
Sample size
Target
210
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Telethon Kids Institute - Nedlands
Recruitment hospital [2] 0 0
Women & Children's Hospital - North Adelaide
Recruitment hospital [3] 0 0
The Royal Children's Hospital - Parkville
Recruitment hospital [4] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment postcode(s) [1] 0 0
- Nedlands
Recruitment postcode(s) [2] 0 0
- North Adelaide
Recruitment postcode(s) [3] 0 0
- Parkville
Recruitment postcode(s) [4] 0 0
- South Brisbane
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Indiana
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Minnesota
Country [8] 0 0
United States of America
State/province [8] 0 0
Missouri
Country [9] 0 0
United States of America
State/province [9] 0 0
New York
Country [10] 0 0
United States of America
State/province [10] 0 0
Ohio
Country [11] 0 0
United States of America
State/province [11] 0 0
Oregon
Country [12] 0 0
United States of America
State/province [12] 0 0
Pennsylvania
Country [13] 0 0
United States of America
State/province [13] 0 0
Texas
Country [14] 0 0
United States of America
State/province [14] 0 0
Vermont
Country [15] 0 0
United States of America
State/province [15] 0 0
Wisconsin
Country [16] 0 0
France
State/province [16] 0 0
Bron Cedex
Country [17] 0 0
France
State/province [17] 0 0
Paris Cedex 15
Country [18] 0 0
Germany
State/province [18] 0 0
Berlin
Country [19] 0 0
Germany
State/province [19] 0 0
Essen
Country [20] 0 0
Germany
State/province [20] 0 0
Hannover
Country [21] 0 0
Netherlands
State/province [21] 0 0
Rotterdam
Country [22] 0 0
Sweden
State/province [22] 0 0
Göteborg
Country [23] 0 0
Switzerland
State/province [23] 0 0
Bern
Country [24] 0 0
Switzerland
State/province [24] 0 0
Zürich
Country [25] 0 0
United Kingdom
State/province [25] 0 0
Cardiff
Country [26] 0 0
United Kingdom
State/province [26] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Vertex Pharmaceuticals Incorporated
Address
Country

Ethics approval
Ethics application status

Summary
Brief 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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05422222
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries