Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of Age
Sponsor
Vertex Pharmaceuticals Incorporated (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT05882357
Collaborator
(none)
64
2
25
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 3 |
Study Design
Study Type:
Interventional
Anticipated Enrollment
:
64 participants
Allocation:
Non-Randomized
Intervention Model:
Sequential Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official 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
Anticipated Study Start Date
:
Sep 1, 2023
Anticipated Primary Completion Date
:
Oct 1, 2025
Anticipated Study Completion Date
:
Oct 1, 2025
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Part A Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening. |
Drug: ELX/TEZ/IVA
Fixed-dose combination granules for oral administration.
Other Names:
Drug: IVA
Granules for oral administration
Other Names:
|
| 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. |
Drug: ELX/TEZ/IVA
Fixed-dose combination granules for oral administration.
Other Names:
Drug: IVA
Granules for oral administration
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Part A: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and their Relevant Metabolites [Day 1 up to Day 15]
- Part A: Safety and Tolerability as Assessed by Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) [Day 1 up to Day 43]
- Part B: Safety and Tolerability as Assessed by Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) [Day 1 up to Week 28]
Secondary Outcome Measures
- Part B: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and their Relevant Metabolites [Day 15 up to Week 16]
- Part B: Absolute Change in Sweat Chloride (SwCl) [From Baseline Through Week 24]
Eligibility Criteria
Criteria
Ages Eligible for Study:
12 Months
to 24 Months
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Key Inclusion Criteria:
- 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 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.
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Vertex Pharmaceuticals Incorporated
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.Responsible Party:
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT05882357
Other Study ID Numbers:
- VX22-445-122
- 2023-503230-49-00
First Posted:
May 31, 2023
Last Update Posted:
May 31, 2023
Last Verified:
May 1, 2023
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Product Manufactured in and Exported from the U.S.:
No
Additional relevant MeSH terms: