A Phase 3 Study of VX-121 Combination Therapy in Subjects With Cystic Fibrosis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Vertex Pharmaceuticals Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Trial duration

28 Feb 2023 → ongoing

Decision date (initial)

2024-07-01

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Vertex Pharmaceuticals Inc.

External identifiers

EU CT number

2024-514173-22-00

EudraCT number

2021-000713-17

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacodynamic, Safety, Efficacy

To evaluate the long-term safety and tolerability of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in subjects with cystic fibrosis

Secondary objectives 1

1. To evaluate the long-term efficacy of VX-121/TEZ/D-IVA

Conditions and MedDRA coding

Cystic Fibrosis

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-003052-PIP01-21

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. Subject (or his or her legally appointed and authorized representative) will sign and date an informed consent form, and, when appropriate, an assent form.
2. Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures.
3. Part A: Did not withdraw consent from a parent study. Part B: Did not withdraw consent from Part A. Part C: Did not withdraw consent from Part B.
4. Part A: Meets at least 1 of the following criteria: • Completed study drug treatment in a parent study. • Had study drug interruption(s) in a parent study, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of a parent study. Part B: Meets at least 1 of the following criteria: • Completed study drug treatment in Part A. • 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. Part C: Meets at least 1 of the following criteria: • Completed study drug treatment in Part B. • Had study drug interruption(s) in Part B, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of Part B.
5. Willing to remain on a stable Cystic Fibrosis treatment regimen through completion of study participation.

Exclusion criteria 5

1. New development of a comorbidity during the parent study, Part A, or Part B that might pose an additional risk in administering study drug to the subject. This includes, but is not limited to, the following: - Hepatic cirrhosis with portal hypertension, moderate hepatic impairment, or severe hepatic impairment, that might pose an additional risk in administering study drug to the subject. - Solid organ or hematological transplantation. - Any other comorbidity that, in the opinion of the investigator, might post an additional risk in administering study drug to the subject.
2. Pregnant or breast-feeding at the time of enrollment in Part A.
3. History of drug intolerance in a parent study 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.)
4. Current participation in an investigational drug trial (other than a parent study). Participation in a non-interventional study (including observational studies, registry studies, and studies requiring blood collections without administration of study drug) and screening for another Vertex study is permitted.
5. 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. However, an adult (aged 18 years or older) who is a relative of a study staff member may be enrolled in the study provided that: - the adult lives independently of and does not reside with the study staff member, and - the adult participates in the study at a site other than the site at which the family member is employed.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Safety and tolerability of long-term treatment with VX-121/TEZ/D-IVA based on adverse events, clinical laboratory values, ECGs, vital signs, and pulse oximetry

Secondary endpoints 3

1. Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1)
2. Absolute change from baseline in sweat chloride (SwCl)
3. Number of pulmonary exacerbations (PEx)

Investigational products

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

Test 2

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

Third parties 1

Locations

16 EU/EEA countries · 81 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 258 files

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

Application history

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