A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy

Vertex Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Cystic Fibrosis

Treatments: Drug: ELX/TEZ/IVA; Drug: IVA

Study type: Interventional

Funder types: Industry

Identifiers

NCT03525574

2018-000185-11 (EudraCT Number)

VX17-445-105

Details and patient eligibility

About

The study evaluates the long-term safety and tolerability of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del mutation

Enrollment: 507 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Completed study drug treatment in a parent study i.e.VX17-445-102 (NCT03525444) and VX17-445-103 (NCT03525548); or had study drug interruption(s) in a parent study but completed study visits up to the last scheduled visit of the Treatment Period in the parent study.

Exclusion criteria

• History of drug intolerance in a parent study that would pose an additional risk to the subject in the opinion of the investigator.
• Current participation in an investigational drug trial (other than a parent study)

Other protocol defined Inclusion/Exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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507 participants in 1 patient group

ELX/TEZ/IVA

Experimental group

Description:

Treatment Period: Participants received ELX 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hrs (q12h) in the treatment period for 192 weeks. Extension Period: Participants from certain countries participated in extension period and continued to receive ELX 200 mg qd /TEZ 100 mg qd/IVA 150 mg q12h in the extension period for 48 weeks.

Treatment:

Drug: IVA

Drug: ELX/TEZ/IVA