Evaluation of VX-659/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age

Vertex Pharmaceuticals

Status and phase: Terminated; Phase 3

Conditions: Cystic Fibrosis

Treatments: Drug: IVA; Drug: VX-659/TEZ/IVA

Study type: Interventional

Funder types: Industry

Identifiers

NCT03633526

2018-001711-67 (EudraCT Number)

VX18-659-106

Details and patient eligibility

About

This study will evaluate the pharmacokinetics (PK), safety, tolerability, efficacy, and pharmacodynamic effect of VX-659, tezacaftor (TEZ), and ivacaftor (IVA) when dosed in triple combination (TC) in Cystic Fibrosis (CF) subjects with F/F or F/MF genotypes.

The study was discontinued after completion of Part A due to Sponsor's discretion.

Enrollment: 18 patients

Sex: All

Ages: 6 to 11 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Homozygous or heterozygous for F508del mutation (F/F or F/MF genotypes)
• Forced expiratory volume in 1 second (FEV1) value ≥40% of predicted mean for age, sex, and height.

Key Exclusion Criteria:

• Clinically significant cirrhosis with or without portal hypertension
• Lung infection with organisms associated with a more rapid decline in pulmonary status.
• Solid organ or hematological transplantation.

Other protocol defined Inclusion/Exclusion criteria may apply.

Trial design

18 participants in 1 patient group

VX-659/TEZ/IVA

Experimental group

Description:

Participants who received VX-659 120 milligram (mg)/TEZ 50 mg/ IVA 75 mg as fixed-dose combination (FDC) in the morning and IVA 75 mg as a mono tablet in the evening in the triple combination (TC) treatment period.

Treatment:

Drug: IVA

Drug: VX-659/TEZ/IVA