A study to assess the efficacy and safety of a combination of two experimental drugs in people with cystic fibrosis (a rare hereditary lung disease)

A Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long term Treatment With VX 661 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

EUCTR2014-004827-29-IE

Enrollment

1375

Registered

2015-07-09

Start date

2015-10-19

Completion date

Unknown

Last updated

2019-06-30

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

Conditions

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

Interventions

Product Name: VX-661/ivacaftor (VX-770) 100mg/150 mg Product Code: VX-661/VX-770 Pharmaceutical Form: Film-coated tablet INN or Proposed INN: IVACAFTOR

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Subjects entering the Treatment Cohort must meet all of the following criteria: •Elect to enroll in the Treatment Cohort. •Completed study drug treatment during the Treatment Period in a parent study (Studies 103, 106, 107, 108, or 109) or study drug treatment and the Safety Follow up Visit for subjects from Study 111. • Willing to remain on a stable CF medication (and supplement) regimen through the Safety Follow-up Visit. Subjects entering the Observational Cohort must meet the following criteria: • =65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: Treatment Cohort: • 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 urine pregnancy test at the Day 1 Visit and before receiving the first dose of study drug. • Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements. • History of drug intolerance in the parent study that would pose an additional risk to the subject. • History of poor compliance with study drug and/or procedures in the parent study as deemed by the investigator. • Participation in an investigational drug trial other than Studies 103, 106, 107, 108, 109, and 111 or use of a commercially available CFTR modulator (e.g., Kalydeco).

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the long-term safety and tolerability of VX-661 in combination with ivacaftor in subjects with CF, homozygous or heterozygous for the F508del-CFTR mutation who are in the Treatment Cohort.; Secondary Objective: Treatment Cohort To evaluate the long -term efficacy of VX 661 in combination with ivacaftor for subjects in the Treatment Cohort Observational Cohort To evaluate the post treatment safety of VX 661 in combination with ivacaftor for subjects in the Observational Cohort ; Primary end point(s): For the Treatment Cohort: Safety and tolerability of long-term treatment of VX-661 in combination with ivacaftor based on adverse events (AEs), ophthalmologic examinations (subjects <18 years of age, clinical laboratory values (serum chemistry, hematology, coagulation, lipids, vitamins, and urinalysis), standard digital electrocardiograms (ECGs), vital signs, and pulse oximetry ;Timepoint(s) of evaluation of this end point: 96 weeks	—

Measure

Time frame

Main Objective: To evaluate the long-term safety and tolerability of VX-661 in combination with ivacaftor in subjects with CF, homozygous or heterozygous for the F508del-CFTR mutation who are in the Treatment Cohort.; Secondary Objective: Treatment Cohort To evaluate the long -term efficacy of VX 661 in combination with ivacaftor for subjects in the Treatment Cohort Observational Cohort To evaluate the post treatment safety of VX 661 in combination with ivacaftor for subjects in the Observational Cohort ; Primary end point(s): For the Treatment Cohort: Safety and tolerability of long-term treatment of VX-661 in combination with ivacaftor based on adverse events (AEs), ophthalmologic examinations (subjects <18 years of age, clinical laboratory values (serum chemistry, hematology, coagulation, lipids, vitamins, and urinalysis), standard digital electrocardiograms (ECGs), vital signs, and pulse oximetry ;Timepoint(s) of evaluation of this end point: 96 weeks

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Secondary

Measure	Time frame
Secondary end point(s): For the Treatment Cohort: • Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1) • Relative change from baseline in ppFEV1 • Number of pulmonary exacerbations • Absolute change from baseline in body mass index (BMI) • Absolute change from baseline in BMI z score for subjects aged <20 years • Absolute change from baseline in Cystic Fibrosis Questionnaire–Revised (CFQ R) respiratory domain score • Absolute change from baseline in body weight • Absolute change from baseline in body weight z-score for subjects aged <20 years • Absolute change from baseline in height z-score for subjects aged <20 years • Time to first pulmonary exacerbation, • Pharmacokinetic (PK) parameters of VX 661, a VX-661 metabolite (M1 661), ivacaftor, and an ivacaftor metabolite (M1 ivacaftor) For the Observational Cohort: • Safety, as determined by related serious adverse events (SAEs) ;Timepoint(s) of evaluation of this end point: Through 100 weeks (Week 24 for PK endpoint)	—

Measure

Time frame

Secondary end point(s): For the Treatment Cohort: • Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1) • Relative change from baseline in ppFEV1 • Number of pulmonary exacerbations • Absolute change from baseline in body mass index (BMI) • Absolute change from baseline in BMI z score for subjects aged <20 years • Absolute change from baseline in Cystic Fibrosis Questionnaire–Revised (CFQ R) respiratory domain score • Absolute change from baseline in body weight • Absolute change from baseline in body weight z-score for subjects aged <20 years • Absolute change from baseline in height z-score for subjects aged <20 years • Time to first pulmonary exacerbation, • Pharmacokinetic (PK) parameters of VX 661, a VX-661 metabolite (M1 661), ivacaftor, and an ivacaftor metabolite (M1 ivacaftor) For the Observational Cohort: • Safety, as determined by related serious adverse events (SAEs) ;Timepoint(s) of evaluation of this end point: Through 100 weeks (Week 24 for PK endpoint)

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Countries

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

Contacts

Public ContactClinical Trials and Medical Info

Vertex Pharmaceuticals Incorporated

medicalinfo@vrtx.com001877634 8789

Outcome results

None listed

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