A Study Evaluating the Long-term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis

• Register: EUCTR
• Last refreshed on: 3 November 2021
• Main ID: EUCTR2017-004134-29-DE
• Date of registration: 05/03/2018
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: A Study Evaluating the Long-term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis
• Scientific title: A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation
• Date of first enrolment: 12/07/2019
• Target sample size: 460
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-004134-29
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Australia, Canada, Denmark, Germany, Ireland, Israel, Norway, Poland, Spain, Switzerland, United Kingdom, United States

Contacts

• Name: Clinical Trials and Medical Info
• Affiliation: Vertex Pharmaceuticals Incorporated

• Name: Clinical Trials and Medical Info
• Affiliation: Vertex Pharmaceuticals Incorporated

Key inclusion & exclusion criteria

Inclusion criteria:
1. Subject (or his or her legally appointed and authorized representative) will sign and date an informed consent form (ICF), 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. Did not withdraw consent from a parent study.

4. 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 completed study visits up to the last scheduled visit of the Treatment Period of a parent study.

5. Willing to remain on a stable CF treatment regimen through completion of study participation.
Are the trial subjects under 18? yes
Number of subjects for this age range: 153
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 307
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. History of any comorbidity that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject.

2. Pregnant and nursing females. Females of childbearing potential must have a negative pregnancy test at the Day 1 Visit before receiving the first dose of study drug.

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 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.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Cystic fibrosis
MedDRA version: 20.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

Intervention(s)

Product Name: VX-659/Tezacaftor/Ivacaftor
Product Code: VX-659/ TEZ/IVA
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: VX-659
Other descriptive name: VX-659
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 120-
INN or Proposed INN: TEZACAFTOR
CAS Number: 1152311-62-0
Current Sponsor code: VX-661
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 50-
INN or Proposed INN: IVACAFTOR
CAS Number: 873054-44-5
Current Sponsor code: VX-770
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 75-

Trade Name: Kalydeco 150 mg film-coated tablets
Product Name: Ivacaftor
Product Code: VX-770
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: IVACAFTOR
CAS Number: 873054-44-5
Current Sponsor code: VX-770
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-

Primary Outcome(s)

Primary end point(s): Safety and tolerability of long-term treatment with VX-659 in TC with TEZ and IVA based on adverse events (AEs), clinical laboratory values, ECGs, vital signs, and pulse oximetry.

Secondary Objective: 1) To evaluate the long-term efficacy of VX-659 in TC with TEZ and IVA
2) To evaluate the pharmacodynamics (PD) of VX-659 in TC with TEZ and IVA

Main Objective: To evaluate the long-term safety and tolerability of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del mutation.

Timepoint(s) of evaluation of this end point: Treatment period, ETT visit, Safety Follow-up after last dose.

Secondary Outcome(s)

Secondary end point(s): 1. Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1)
2. Absolute change in sweat chloride (SwCl)
3. Number of pulmonary exacerbations (PEx)
4. Time-to-first PEx
5. Absolute change in body mass index (BMI)
6. Absolute change in BMI z-score
7. Absolute change in body weight
8. Absolute change from baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score

Timepoint(s) of evaluation of this end point: 1. Treatment period, ETT Visit, Safety Follow-Up After Last Dose.
2. Day 1,15, Week 4,8,16,24, 96.
3. Treatment period, ETT Visit, Safety Follow-Up After Last Dose.
4. Treatment period, ETT Visit, Safety Follow-Up After Last Dose.
5. Treatment period, ETT Visit, Safety Follow-Up After Last Dose.
6. Treatment period, ETT Visit, Safety Follow-Up After Last Dose.
7. Treatment period, ETT Visit, Safety Follow-Up After Last Dose.
8. Day1, Weeks 4,8,24, 48, 72, 96, ETT Visit, Safety Follow-Up After Last Dose.

Secondary ID(s)

VX17-659-105

2017-004134-29-GB

Source(s) of Monetary Support

Vertex Pharmaceuticals Incorporated

Ethics review

Status: Approved
Approval date: 12/07/2019
Contact:

Results

• Results available: Yes
• Date Posted: 25/03/2021
• Date Completed: 09/09/2020
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2017-004134-29/results