Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor

Vertex Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Cystic Fibrosis

Treatments: Drug: LUM/IVA

Study type: Interventional

Funder types: Industry

Identifiers

NCT02544451

VX15-809-110

Details and patient eligibility

About

Study 110 is a Phase 3, multicenter study in subjects aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation and who participated in Study 109 (NCT02514473) or Study 011B (NCT01897233). Study 110 is designed to evaluate the safety and efficacy of long term treatment of lumacaftor in combination with ivacaftor.

Enrollment: 246 patients

Sex: All

Ages: 6+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria:

Subjects entering the Treatment Cohort must meet both of the following criteria:

• Completed study visits up to Week 24 of Study 109 or Week 26 of Study 011B and did not permanently discontinue treatment
• Willing to remain on a stable CF medication through the Safety Follow-up Visit.

Subjects entering the Observational Cohort must meet 1 of the following criteria:

• Completed 24 weeks of study drug treatment in Study 109 or completed 24 weeks of study drug treatment and the Week 26 Safety Follow up in Study 011B.
• Received at least 4 weeks of study drug and completed visits up to Week 24 of Study 109 or Week 26 of Study 011B.

Exclusion Criteria (Treatment Cohort Only):

• History of any comorbidity or laboratory abnormality 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 (e.g., cirrhosis with portal hypertension).
• Pregnant and nursing females.
• Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements.
• History of drug intolerance in the prior study that would pose an additional risk to the subject in the opinion of investigator
• History of poor compliance with study drug and/or procedure in the previous study as deemed by the investigator.
• Participation in an investigational drug trial (including studies investigating lumacaftor and/or ivacaftor).

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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246 participants in 4 patient groups

Treatment Period 1: LUM/IVA to LUM/IVA

Experimental group

Treatment:

Drug: LUM/IVA

Drug: LUM/IVA

Treatment Period 1: Placebo (PBO) to LUM/IVA

Experimental group

Treatment:

Drug: LUM/IVA

Drug: LUM/IVA

Treatment Period 1: Observational Cohort

No Intervention group

Treatment Period 2: LUM/IVA

Experimental group

Treatment:

Drug: LUM/IVA

Drug: LUM/IVA