A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis

Vertex Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Cystic Fibrosis

Treatments: Drug: Placebo; Drug: VX-561; Drug: IVA

Study type: Interventional

Funder types: Industry

Identifiers

NCT03911713

2018-003970-28 (EudraCT Number)

VX18-561-101

Details and patient eligibility

About

The purpose of this study is to evaluate the efficacy, safety, pharmacodynamic (PD) and pharmacokinetic (PK) effect of VX-561.

Enrollment: 77 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D
• On ivacaftor therapy
• FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height

Key Exclusion Criteria:

• History of clinically significant cirrhosis with or without portal hypertension
• History of solid organ or hematological transplantation
• Lung infection with organisms associated with a more rapid decline in pulmonary status

Other protocol defined Inclusion/Exclusion criteria may apply

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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77 participants in 5 patient groups

Ivacaftor

Active Comparator group

Description:

Participants received IVA 150 milligrams (mg) orally every 12 hours (q12h) in the treatment period for 12 weeks.

Treatment:

Drug: IVA

Drug: Placebo

Drug: Placebo

VX-561: 25 mg

Experimental group

Description:

Participants received VX-561 25 mg orally daily (qd) in the treatment period for 12 weeks.

Treatment:

Drug: VX-561

Drug: Placebo

Drug: Placebo

VX-561: 50 mg

Experimental group

Description:

Participants received VX-561 50 mg orally qd in the treatment period for 12 weeks.

Treatment:

Drug: VX-561

Drug: Placebo

Drug: Placebo

VX-561: 150 mg

Experimental group

Description:

Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.

Treatment:

Drug: VX-561

Drug: Placebo

Drug: Placebo

VX-561: 250 mg

Experimental group

Description:

Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks.

Treatment:

Drug: VX-561

Drug: Placebo

Drug: Placebo