Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Subjects 2 Years and Older

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Vertex Pharmaceuticals Inc.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Trial duration

1 Apr 2022 → 29 Jan 2026

Decision date (initial)

2024-08-29

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

Vertex Pharmaceuticals Inc.

External identifiers

EU CT number

2024-515606-90-00

EudraCT number

2020-002239-31

ClinicalTrials.gov

NCT05153317

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacodynamic, Others, Efficacy, Safety

To evaluate the long-term safety and tolerability of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) in cystic fibrosis (CF) subjects 2 years of age and older

Secondary objectives 1

1. To evaluate the long-term efficacy and pharmacodynamics (PD) of ELX/TEZ/IVA

Conditions and MedDRA coding

Cystic Fibrosis

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-002324-PIP01-17

Plan to share IPD

No

IPD plan description

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

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 10

1. Part A: Subject’s legal representative or guardian will sign and date an informed consent form (ICF).
2. Part A: As judged by the investigator, the legal representative or guardian must be able to understand protocol requirements, restrictions, and instructions and the legal representative or guardian should be able to ensure that the subject will comply with and is likely to complete the study as planned
3. Part A: Did not withdraw consent from the parent study.
4. Part A: Meets at least 1 of the following criteria: • Completed study drug treatment in the parent study, or • Had study drug interruption(s) in the parent study, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of the parent study.
5. Part A: Willing to remain on a stable CF treatment regimen (as defined in Section 9.5) through completion of study participation.
6. Part B: Subject’s legal representative or guardian will sign and date an ICF.
7. Part B: As judged by the investigator, the legal representative or guardian must be able to understand protocol requirements, restrictions, and instructions and the legal representative or guardian should be able to ensure that the subject will comply with and is likely to complete the study as planned.
8. Part B: Did not withdraw consent in Part A.
9. Part B: Meets at least 1 of the following criteria: • Completed study drug treatment in Part A, or • Had study drug interruption(s) in Part A, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of Part A.
10. Part B: Willing to remain on a stable CF treatment regimen (as defined in Section 9.5) through completion of study participation

Exclusion criteria 12

1. Part A: History of any illness or any clinical condition that might confound the results of the study or pose an additional risk in administering study drug(s) to the subject. This includes, but is not limited to, the following: • Clinically significant liver cirrhosis with or without portal hypertension • Solid organ or hematological transplantation • Cancer
2. Part A: History of drug intolerance in the parent study that would pose an additional risk to the subject (e.g., subjects with a history of allergy or hypersensitivity to the study drug).
3. Part A: Current participation in an investigational drug trial other than the parent study. Participation in a noninterventional study (including observational studies, registry studies, and studies requiring blood collections without administration of study drug) and screening for another Vertex study is permitted.
4. Part A: History of poor compliance with ELX/TEZ/IVA and/or procedures in the parent study as deemed by the investigator.
5. Part A: Use of restricted medication, as defined in Table 9-1, unless subject is on a study drug interruption at the time of rollover.
6. Part A: The subject or a close relative of the subject is the investigator or a sub-investigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study at that site.
7. Part B: History of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug(s) to the subject.
8. Part B: History of drug intolerance in the parent study or Part A that would pose an additional risk to the subject in the opinion of the investigator (e.g., subjects with a history of allergy or hypersensitivity to the study drug).
9. Part B: Current participation in an investigational drug trial other than the parent study or the current study. Participation in a noninterventional study (including observational studies, registry studies, and studies requiring blood collections without administration of study drug) and screening for another Vertex study is permitted
10. Part B: History of poor compliance with ELX/TEZ/IVA and/or procedures in the parent study or Part A, as deemed by the investigator.
11. Part B: Use of restricted medication, as defined in Table 9-1, unless subject is on a study drug interruption at the time of rollover.
12. Part B: The subject or a close relative of the subject is the investigator or a sub-investigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study at that site.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Safety and tolerability of ELX/TEZ/IVA based on adverse events (AEs), clinical laboratory values, standard 12-lead ECGs, vital signs, and pulse oximetry

Secondary endpoints 2

1. Absolute change in sweat chloride (SwCl)
2. Absolute change in lung clearance index (LCI)2.5

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 9

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Vertex Pharmaceuticals Inc.

Sponsor organisation

Vertex Pharmaceuticals Inc.

Address

50 Northern Avenue

City

Boston

Postcode

02210-1862

Country

United States

Scientific contact point

Organisation

Vertex Pharmaceuticals Inc.

Contact name

Clinical Trials and Medical Info

Public contact point

Organisation

Vertex Pharmaceuticals Inc.

Contact name

Clinical Trials and Medical Info

Locations

1 EU/EEA country · 2 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 11 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

4 submissions — initial application plus substantial / non-substantial modifications