Study of Lumacaftor in Combination With Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

Vertex Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Cystic Fibrosis

Treatments: Drug: Ivacaftor; Drug: Lumacaftor

Study type: Interventional

Funder types: Industry

Identifiers

NCT01897233

VX13-809-011

Details and patient eligibility

About

This is a Phase 3, 2-part (Part A and Part B), open-label, multicenter study to evaluate the pharmacokinetics, safety, and tolerability of lumacaftor in combination with ivacaftor in subjects with cystic fibrosis aged 6 to 11 years who have the F508del-mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Enrollment: 62 patients

Sex: All

Ages: 6 to 11 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Confirmed diagnosis of CF defined as: with 2 CF-causing mutations, chronic sinopulmonary disease or gastrointestinal/nutritional abnormalities
• Subjects who weigh ≥15 kg without shoes at Screening Visit
• Subjects who are homozygous for the F508del-CFTR mutation
• Subjects with percent predicted forced expiratory volume in 1 second (FEV1) of 70% to 105% (inclusive) (Part A) or ≥40% (Part B) at Screening Visit where the predicted values are adjusted for age, sex, and height using the Wang equation
• Subjects with stable CF disease and who are willing to remain on stable CF medication regimen
• Able to swallow tablets

Exclusion criteria

• History of any illness or condition that might confound the results of the study or pose an additional risk in administering study drug to the subject
• Acute respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before Day 1 of the study
• Abnormal liver function as defined in the protocol at Screening Visit
• Abnormal renal function as defined in the protocol at Screening Visit
• History of solid organ or hematological transplantation
• Ongoing participation in an investigational drug study or prior participation in an investigational drug study within 30 days prior of Screening Visit
• History or evidence of lens opacity or cataract at Screening Visit
• Colonization with organisms associated with a more rapid decline in pulmonary status at Screening Visit (Part A only)
• A standard 12-lead ECG demonstrating QTcF >450 msec at Screening Visit

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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62 participants in 1 patient group

Lumacaftor/Ivacaftor (LUM/IVA)

Experimental group

Description:

Part A Cohort 1: Participants aged 6 through 8 years will receive LUM 200 milligram (mg) in fixed-dose combination with IVA 250 mg orally every 12 hours (q12h) for 14 days. Part A Cohort 2: Participants aged 9 through 11 years will receive LUM 200 mg in fixed-dose combination with IVA 250 mg orally q12h for 14 days. Part B: Participants aged 6 through 11 years will receive LUM 200 mg in fixed-dose combination with IVA 250 mg orally q12h for 24 weeks.

Treatment:

Drug: Lumacaftor

Drug: Ivacaftor