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Completed

NCT Number: NCT04474197

Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype

This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-864 in PiZZ subjects.

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Key information

Conditions

Alpha1-Antitrypsin Deficiency Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Emphysema Genetic Diseases, Inborn Liver Diseases Lung Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Respiratory Tract Diseases Subcutaneous Emphysema alpha 1-Antitrypsin Deficiency

Age range

18 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Queen Elizabeth II Health Sciences Center, Halifax, Canada

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

VX-864

Drug

Tablets for oral administration.

Placebo

Drug

Placebo matched to VX-864 for oral administration.

Primary outcomes

  1. Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels

    Time frame: From Baseline at Day 28

  2. Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Time frame: Day 1 up to Week 8

Secondary outcomes

  1. Change in Plasma Antigenic AAT Levels

    Time frame: From Baseline at Day 28

  2. Observed Pre-dose Plasma Concentration (Ctrough) of VX-864

    Time frame: Pre-dose at Day 7, Day 14, Day 21 and Day 28

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study title

A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 in PiZZ Subjects

Important dates

Study start
2020
Primary completion
2021
Study completion
2021
First posted
Jul 16, 2020
Registry last updated
May 26, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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