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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05882357




Registration number
NCT05882357
Ethics application status
Date submitted
19/05/2023
Date registered
31/05/2023
Date last updated
15/08/2024

Titles & IDs
Public title
Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of Age
Scientific title
A Phase 3, Open-label Study Evaluating the Pharmacokinetics, Safety, and Tolerability of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 12 to Less Than 24 Months of Age
Secondary ID [1] 0 0
2023-503230-49-00
Secondary ID [2] 0 0
VX22-445-122
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 - ELX/TEZ/IVA
Treatment: Drugs - IVA

Experimental: Part A - Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.

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


Treatment: Drugs: ELX/TEZ/IVA
Fixed-dose combination granules for oral administration.

Treatment: Drugs: IVA
Granules 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 Concentration (Ctrough) of ELX, TEZ, IVA, and their Relevant Metabolites
Timepoint [1] 0 0
Day 1 up to Day 15
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
Day 1 up to Day 43
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
Day 1 up to Week 28
Secondary outcome [1] 0 0
Part B: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and their Relevant Metabolites
Timepoint [1] 0 0
Day 15 up to Week 16
Secondary outcome [2] 0 0
Part B: Absolute Change in Sweat Chloride (SwCl)
Timepoint [2] 0 0
From Baseline Through Week 24

Eligibility
Key inclusion criteria
Key

* Participants who have at least 1 F508del mutation in the CF transmembrane conductance regulator (CFTR) gene or another ELX/TEZ/IVA-responsive CFTR mutation

Key
Minimum age
12 Months
Maximum age
24 Months
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* History of any illness or any clinical condition that, in the opinion of the investigator, might either confound the results of the study or pose an additional risk in administering study drug(s) to the participant

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
27/06/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/10/2025
Actual
Sample size
Target
64
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Telethon Kids Institute - Nedlands
Recruitment hospital [2] 0 0
The Royal Children's Hospital - Parkville
Recruitment hospital [3] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment hospital [4] 0 0
The Children's Hospital at Westmead - Westmead
Recruitment postcode(s) [1] 0 0
- Nedlands
Recruitment postcode(s) [2] 0 0
- Parkville
Recruitment postcode(s) [3] 0 0
- South Brisbane
Recruitment postcode(s) [4] 0 0
- Westmead
Recruitment outside Australia
Country [1] 0 0
Canada
State/province [1] 0 0
Toronto
Country [2] 0 0
Canada
State/province [2] 0 0
Vancouver
Country [3] 0 0
Denmark
State/province [3] 0 0
Copenhagen
Country [4] 0 0
Germany
State/province [4] 0 0
Berlin
Country [5] 0 0
Germany
State/province [5] 0 0
Essen
Country [6] 0 0
Germany
State/province [6] 0 0
Hannover
Country [7] 0 0
Netherlands
State/province [7] 0 0
Rotterdam
Country [8] 0 0
Switzerland
State/province [8] 0 0
Bern
Country [9] 0 0
Switzerland
State/province [9] 0 0
Zurich
Country [10] 0 0
United Kingdom
State/province [10] 0 0
Cardiff
Country [11] 0 0
United Kingdom
State/province [11] 0 0
Leeds
Country [12] 0 0
United Kingdom
State/province [12] 0 0
Liverpool
Country [13] 0 0
United Kingdom
State/province [13] 0 0
London
Country [14] 0 0
United Kingdom
State/province [14] 0 0
Southampton

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

Ethics approval
Ethics application status

Summary
Brief summary
This study will evaluate the pharmacokinetics (PK), safety, tolerability, pharmacodynamics (PD), and efficacy of ELX/TEZ/IVA in CF subjects 12 to less than (\<) 24 months of age.
Trial website
https://clinicaltrials.gov/study/NCT05882357
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05882357