Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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EUCTR |
Last refreshed on:
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4 September 2023 |
Main ID: |
EUCTR2018-000185-11-CZ |
Date of registration:
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09/07/2018 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy
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Scientific title:
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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 |
Date of first enrolment:
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15/10/2018 |
Target sample size:
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460 |
Recruitment status: |
Not Recruiting |
URL:
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https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2018-000185-11 |
Study type:
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Interventional clinical trial of medicinal product |
Study design:
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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
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Phase:
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Human pharmacology (Phase I): no
Therapeutic exploratory (Phase II): no
Therapeutic confirmatory - (Phase III): yes
Therapeutic use (Phase IV): no
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Countries of recruitment
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Australia
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Austria
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Belgium
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Canada
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Czech Republic
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France
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Germany
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Greece
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Italy
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Netherlands
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Sweden
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United Kingdom
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United States
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Contacts
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Name:
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Clinical Trials and Medical Info
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Address:
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50 Northern Avenue
MA 02210-1862
Boston
United States |
Telephone:
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001877 634 8789 |
Email:
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medicalinfo@vrtx.com |
Affiliation:
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Vertex Pharmaceuticals Incorporated |
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Name:
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Clinical Trials and Medical Info
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Address:
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50 Northern Avenue
MA 02210-1862
Boston
United States |
Telephone:
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001877 634 8789 |
Email:
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medicalinfo@vrtx.com |
Affiliation:
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Vertex Pharmaceuticals Incorporated |
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Key inclusion & exclusion criteria
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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
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Health Condition(s) or Problem(s) studied
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Cystic Fibrosis MedDRA version: 20.0
Level: PT
Classification code 10011762
Term: Cystic fibrosis
System Organ Class: 10010331 - Congenital, familial and genetic disorders
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Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
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Intervention(s)
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Trade Name: Trikafta Product Name: 100 mg ELX/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: 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-
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Primary Outcome(s)
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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
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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.
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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
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Timepoint(s) of evaluation of this end point: from baseline through safety follow-up (up to 100 weeks)
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Secondary Outcome(s)
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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
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Timepoint(s) of evaluation of this end point: from baseline through last dose of study drug (up to 96 weeks)
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Secondary ID(s)
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VX17-445-105
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2018-000185-11-SE
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Source(s) of Monetary Support
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Vertex Pharmaceuticals Incorporated
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Ethics review
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Status: Approved
Approval date: 10/10/2018
Contact:
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