A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype

Vertex Pharmaceuticals

Status and phase: Active, not recruiting; Phase 2

Conditions: Alpha-1 Antitrypsin Deficiency

Treatments: Drug: VX-864

Study type: Interventional

Funder types: Industry

Identifiers

NCT05643495

VX22-864-108

2022-002746-40 (EudraCT Number)

Details and patient eligibility

About

The purpose of this study is to evaluate the efficacy and safety of VX-864 in participants with the PiZZ genotype over 48 weeks.

Enrollment: 20 estimated patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Participants 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 Ribonucleic acid interference (RNAi) therapy at any time previously
• Participants for whom discontinuation of augmentation therapy is not considered to be in their best interest, based on the clinical judgement of the treating physician

Other protocol defined Inclusion/Exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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20 participants in 2 patient groups

Group A

Experimental group

Description:

Participants will receive VX-864 every 12 hours (q12h) for 48 weeks.

Treatment:

Drug: VX-864

Group B

Experimental group

Description:

Participants will undergo a liver biopsy done before receiving VX-864 q12h for 48 weeks, and will undergo a second liver biopsy at either Week 24 or Week 48.

Treatment:

Drug: VX-864