Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype

Vertex Pharmaceuticals

Status and phase: Terminated; Phase 2

Conditions: Alpha 1-Antitrypsin Deficiency

Treatments: Drug: Placebo; Drug: VX-814

Study type: Interventional

Funder types: Industry

Identifiers

NCT04167345

VX19-814-101

Details and patient eligibility

About

This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-814 in PiZZ subjects.

Enrollment: 48 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Subjects must have a PiZZ genotype confirmed at screening
• Plasma AAT levels indicating severe deficiency at screening

Key Exclusion Criteria:

• History of a medical condition that could negatively impact the ability to complete the study
• Solid organ, or hematological transplantation or is currently on a transplant list
• History of use of gene therapy or RNAi therapy at any time previously

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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48 participants in 5 patient groups, including a placebo group

Parts A1, A2 and B Combined: Placebo

Placebo Comparator group

Description:

Participants received placebo matched to VX-814 in the treatment period for 28 days.

Treatment:

Drug: Placebo

Part A1: VX-814 100 milligrams (mg)

Experimental group

Description:

Participants received VX-814 100 mg every 12 hours (q12h) in the treatment period for 28 days.

Treatment:

Drug: VX-814

Part A1: VX-814 200 mg

Experimental group

Description:

Participants received VX-814 200 mg q12h in the treatment period for 28 days.

Treatment:

Drug: VX-814

Parts A1 and A2 Combined: VX-814 400 mg

Experimental group

Description:

Participants received VX-814 400 mg q12h in the treatment period for 28 days.

Treatment:

Drug: VX-814

Part B: VX-814 600 mg

Experimental group

Description:

Participants received VX-814 600 mg q12h in the treatment period for 28 days.

Treatment:

Drug: VX-814