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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT05422222
Ethics application status
Date submitted
13/06/2022
Date registered
16/06/2022

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
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
Trial website
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
Medical Information
Address 0 0
Country 0 0
Phone 0 0
617-341-6777
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05422222