A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy

• Register: EUCTR
• Last refreshed on: 13 December 2021
• Main ID: EUCTR2018-000185-11-DE
• Date of registration: 03/07/2018
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy
• Scientific title: A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation
• Target sample size: 460
• Recruitment status: NA
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2018-000185-11
• 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
• 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, Austria, Belgium, Canada, Czech Republic, Czechia, France, Germany, Greece, Italy, Netherlands, Sweden, 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 (as defined in Section 9.5) through completion of study participation.
Are the trial subjects under 18? yes
Number of subjects for this age range: 90
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 370
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 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.


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

Health Condition(s) or Problem(s) studied

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

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

Intervention(s)

Product Name: 100 mg VX-445/50 mg TEZ /75 mg IVA FDC
Product Code: VX-445/TEZ/IVA
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: VX-445
CAS Number: 2216712-66-0
Current Sponsor code: VX-445
Other descriptive name: VX-445
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
INN or Proposed INN: Tezacaftor
CAS Number: 1152311-62-0
Current Sponsor code: VX-661
Other descriptive name: 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-

Trade Name: Trikafta
Product Name: 100-mg ELX (VX-445) / 50-mg TEZ / 75-mg IVA FDC
Product Code: ELX/TEZ/IVA
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Elexacaftor
CAS Number: 2216712-66-0
Current Sponsor code: VX-445
Other descriptive name: ELX
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
INN or Proposed INN: Tezacaftor
CAS Number: 1152311-62-0
Current Sponsor code: VX-661
Other descriptive name: TEZ
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
Other descriptive name: IVA
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 75-

Trade Name: Kalydeco 150 mg film-c

Primary Outcome(s)

Main Objective: To evaluate the long-term safety and tolerability of VX-445 in TC with TEZ and IVA in subjects with CF who are homozygous or heterozygous for the F508del mutation

Primary end point(s): Safety and tolerability of long-term treatment with VX-445 in TC with TEZ and IVA based on adverse events (AEs), clinical laboratory values, ECGs, vital signs, and pulse oximetry.

Secondary Objective: To evaluate the long-term efficacy of VX-445 in TC with TEZ and IVA
To evaluate the pharmacodynamics (PD) of VX-445 in TC with TEZ and IVA

Timepoint(s) of evaluation of this end point: from baseline through safety follow-up (up to 196 weeks)

Secondary Outcome(s)

Secondary end point(s): Secondary Endpoints
Absolute change from baseline in ppFEV1
Absolute change in sweat chloride (SwCl)
Number of pulmonary exacerbations (PEx)
Time-to-first PEx
Absolute change in body mass index (BMI)
Absolute change in BMI z-score
Absolute change in body weight
Absolute change from baseline in CFQ-R respiratory domain score
Additional Endpoints
Absolute change in CFQ-R non-respiratory domain scores
Changes in inflammatory mediators
Changes in microbiology analysis

Timepoint(s) of evaluation of this end point: from baseline through last dose of study drug (up to 192 weeks)

Secondary ID(s)

VX17-445-105

2018-000185-11-SE

Source(s) of Monetary Support

Vertex Pharmaceuticals Incorporated