A study in people with Cystic Fibrosis (a rare hereditary pulmonary disease) to assess the long-term efficacy and safety of a combination of two experimental drugs

• Register: EUCTR
• Last refreshed on: 13 June 2016
• Main ID: EUCTR2013-000604-41-IT
• Date of registration: 14/08/2013
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: A study in people with Cystic Fibrosis (a rare hereditary pulmonary disease) to assess the long-term efficacy and safety of a combination of two experimental drugs
• Scientific title: A Phase 3, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation
• Date of first enrolment: 21/01/2014
• Target sample size: 1000
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2013-000604-41
• 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: yes Other trial design description: Open for the Treatment Cohort Part B If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: yes Other specify the comparator: Different dosage of the same medicial product Number of treatment arms in the trial: 3
• 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, Denmark, France, Germany, Ireland, Italy, Netherlands, Spain, 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:
? Subjects entering the Part A Treatment Cohort must have completed 24 weeks of study drug treatment in Study 103 or Study 104 and wish to enroll in the Part A Treatment Cohort.
? Subjects entering the Part A Observational Cohort must have completed at least 4 weeks of study drug treatment in Study 103 or in Study 104, or 24 weeks of study drug treatment in Study 103 or Study 104, but do not wish or do not qualify to enroll in the Part A Treatment Cohort.
? Treatment Cohort Subjects entering the Part B Treatment Cohort must have completed 56 days of study drug treatment in Cohort 4 of Study 102 and wish to enroll in the Part B Treatment Cohort.
? Willing to remain on a stable CF medication regimen through the end of study (Treatment Cohort only).
Are the trial subjects under 18? yes
Number of subjects for this age range: 389
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 611
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
? Any comorbidity or lab abnormality that might confound the results of the study or pose an additional risk in administering study drug to the subject.
? Pregnant and nursing females, sexually active subjects of reproductive potential who are not willing to follow the contraception requirements.
? History of drug intolerance in prior study that would pose an additional risk to the subject.
? History of poor compliance with study drug and/or procedures in prior study.
? Subjects may not participate in an investigational drug study until the completion of this study.


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 in patients homozygous or heterozygous for the F508del-CFTR Mutation
MedDRA version: 16.1 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders

Intervention(s)

Product Name: lumacaftor/ivacaftor 200mg/125mg tablets
Product Code: VX-809 / VX-770
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: lumacaftor
CAS Number: 936727-05-8
Current Sponsor code: VX-809
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 200-
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: 125-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use

Product Name: lumacaftor/ivacaftor 200mg/83mg tablet
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: lumacaftor
CAS Number: 936727-05-8
Current Sponsor code: VX-809
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 200-
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: 83-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use

Product Name: ivacaftor 125mg tablet
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: 125-
Pharmaceutical form of the placebo: Film-coated tablet
Route of administration of the placebo: Oral use

Primary Outcome(s)

Main Objective: To evaluate the long term safety and tolerability of lumacaftor in combination with ivacaftor in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the for the F508del-CFTR mutation

Secondary Objective: To evaluate the long term efficacy and durability of lumacaftor in combination with ivacaftor for subjects in the Treatment Cohort

To evaluate the post treatment safety and tolerability of lumacaftor in combination with ivacaftor for subjects in the Observational Cohort

Primary end point(s): Safety of long term treatment of lumacaftor in combination with ivacaftor based on adverse events (AEs), clinical laboratory values (serum chemistry, hematology, coagulation studies, and urinalysis), standard digital electrocardiograms (ECGs), vital signs, and pulse oximetry.

Timepoint(s) of evaluation of this end point: Through week 100

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: Through week 100

Secondary end point(s): For the Treatment Cohort: Relative change from baseline in percent predicted forced expiratory volume in 1 second (FEV1), Absolute change from baseline in percent predicted FEV1, Absolute change from baseline in body mass index (BMI), Number of pulmonary exacerbations starting from the previous study (Part A only), Absolute change from baseline in Cystic Fibrosis Questionnaire Revised (CFQ R) respiratory domain score, Absolute change in BMI z score (for Part A only), Absolute change from baseline in body weight, Rate of change in percent predicted FEV1, Time-to-first pulmonary exacerbation including pulmonary exacerbations in the previous study (Part A only), Event of having at least 1 pulmonary exacerbation including pulmonary exacerbations in the previous study (Part A only)
For the Observational Cohort: Safety, as determined by serious adverse events (SAEs).

Secondary ID(s)

VX12-809-105

Source(s) of Monetary Support

Vertex Pharmaceuticals Incorporated