A Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects With CF, Homozygous for the F508del-CFTR Mutation

Vertex Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Cystic Fibrosis

Treatments: Drug: VX-809; Drug: VX-770; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02514473

VX14-809-109

Details and patient eligibility

About

To evaluate the efficacy and safety of lumacaftor in combination with ivacaftor in subjects aged 6 Through 11 years with cystic fibrosis (CF), homozygous for the F508del CF transmembrane conductance regulator (CFTR) mutation

Enrollment: 206 patients

Sex: All

Ages: 6 to 11 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Subjects who weigh ≥15 kg without shoes a the Screening Visit
• Subjects with confirmed diagnosis of CF at the Screening Visit.
• Subjects who are homozygous for the F508del CFTR mutation
• Subjects with ppFEV1 of ≥70 percentage points adjusted for age, sex, and height
• Subjects with a screening LCI2.5 result greater than or equal to 7.5

Exclusion criteria

• History of any comorbidity reviewed at the Screening Visit 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.
• Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject
• Clinically significant abnormalities in hemoglobin, liver function, or renal function at the Screening Visit.
• An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before Day 1
• History of solid organ or hematological transplantation at the Screening Visit

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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206 participants in 2 patient groups, including a placebo group

LUM/IVA

Experimental group

Description:

Fixed-dose combination with lumacaftor (LUM) 200 mg every 12 hours (q12h)/ ivacaftor (IVA) 250 mg q12h

Treatment:

Drug: VX-770

Drug: VX-809

Placebo

Placebo Comparator group

Description:

Matching placebo q12h

Treatment:

Drug: Placebo