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

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

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2013-000604-41-IT

Enrollment

1000

Registered

2013-08-14

Start date

2014-01-21

Completion date

Unknown

Last updated

2016-06-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

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

Interventions

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 cod

Eligibility

Sex/Gender

All

Inclusion 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

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.

Design outcomes

Primary

Measure	Time frame
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	—

Measure

Time frame

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

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Secondary

Measure	Time frame
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).;Timepoint(s) of evaluation of this end point: Through week 100	—

Measure

Time frame

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

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Countries

Australia, Austria, Belgium, Canada, Czech Republic, Denmark, France, Germany, Ireland, Italy, Netherlands, Spain, Sweden, United Kingdom, United States

Contacts

Public ContactClinical Trials and Medical Info

Vertex Pharmaceuticals Incorporated

medicalinfo@vrtx.com+ 18776348789

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026