World Health Organization site
Skip Navigation Links

Main
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: EUCTR
Last refreshed on: 19 February 2018
Main ID:  EUCTR2014-004838-25-IT
Date of registration: 30/03/2015
Prospective Registration: Yes
Primary sponsor: Vertex Pharmaceuticals Incorporated
Public title: A study in people with Cystic Fibrosis ( a rare hereditary lung disease) to assess the efficacy and safety of a combination of two experimental drugs
Scientific title: A Phase 3, Randomized, Double-Blind, Ivacaftor-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation and a Second CFTR Allele With a Gating Defect That Is Clinically Demonstrated to be Ivacaftor Responsive
Date of first enrolment: 20/05/2015
Target sample size: 200
Recruitment status: Not Recruiting
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2014-004838-25
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: yes
Randomised: yes
Open: no
Single blind: no
Double blind: yes
Parallel group: yes
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product: yes
Placebo: no
Other: no
Number of treatment arms in the trial: 2
 
Phase:  Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no
Countries of recruitment
Austria Belgium Canada France Germany Ireland Italy United Kingdom
United States
Contacts
Name: Clinical Trials and Medical Info   
Address:  50 Northern Avenue 02210 Boston, Massachusetts United States
Telephone: 1877634-8789
Email: medical_info@vrtx.com
Affiliation:  Vertex Pharmaceuticals Incorporated
Name: Clinical Trials and Medical Info   
Address:  50 Northern Avenue 02210 Boston, Massachusetts United States
Telephone: 1877634-8789
Email: medical_info@vrtx.com
Affiliation:  Vertex Pharmaceuticals Incorporated
Key inclusion & exclusion criteria
Inclusion criteria:
• Heterozygous for F508del-CFTR mutation and a second CFTR allele with a gating defect that is clinically demonstrated to be ivacaftor responsive
• FEV1 =40% and =90% of predicted normal for age, sex, and height during screening
• Stable CF disease as judged by the investigator.
Are the trial subjects under 18? yes
Number of subjects for this age range: 100
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 100
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
• 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.
• Pregnant and nursing females (females of childbearing potential must have a negative pregnancy test at Screening and Week -4 Visits).
• Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
Cystic Fibrosis
MedDRA version: 17.1 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-661/ivacaftor 100mg/150mg
Product Code: VX-661/VX-770
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Not yet assigned
CAS Number: 1152311-62-0
Current Sponsor code: VX-661
Other descriptive name: VRT-893661 VRT-0893661
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
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-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use

Trade Name: Kalydeco
Product Name: ivacaftor
Product Code: VX-770, VRT-813077
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-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use

Primary Outcome(s)
Main Objective: To evaluate the efficacy of VX-661 in combination with ivacaftor in subjects with CF who are heterozygous for the F508del mutation on the CFTR gene and a second CFTR allele with a gating defect that is clinically demonstrated to be ivacaftor responsive
Secondary Objective: To evaluate the safety of VX-661 in combination with ivacaftor

To investigate the pharmacokinetics (PK) of VX-661 and its metabolites, M1 and M2 (M1-661 and M2-661, respectively) and ivacaftor and its metabolite M1 (M1 ivacaftor)
Timepoint(s) of evaluation of this end point: Week 8
Primary end point(s): Absolute change in percent predicted forced expiratory volume in 1 second (FEV1) from baseline
Secondary Outcome(s)
Secondary end point(s): • Relative change in percent predicted FEV1 from baseline through Week 8
• Absolute change in sweat chloride from baseline through Week 8
• Absolute change in Cystic Fibrosis Questionnaire – Revised (CFQ-R) respiratory domain score from baseline through Week 8
• Safety and tolerability assessments based on adverse events (AEs), clinical laboratory values, standard 12-lead electrocardiograms (ECGs), vital signs, and pulse oximetry; from screening through 4 weeks after receiving last dose
• PK parameters of VX-661, M1-661, M2-661, ivacaftor, and M1 ivacaftor through Week 8
Timepoint(s) of evaluation of this end point: Weeks 8 and 12
Secondary ID(s)
VX14-661-109
Source(s) of Monetary Support
Vertex Pharmaceuticals Incorporated
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
Disclaimer: Trials posted on this search portal are not endorsed by WHO, but are provided as a service to our users. In no event shall the World Health Organization be liable for any damages arising from the use of the information linked to in this section. None of the information obtained through use of the search portal should in any way be used in clinical care without consulting a physician or licensed health professional. WHO is not responsible for the accuracy, completeness and/or use made of the content displayed for any trial record.
Copyright - World Health Organization - Version 3.6 - Version history