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

NCT04474197 | Completed Phase 2 Results FDA Drug

• 2 other identifiers: VX19-864-1012019-004881-16
• Study Type: interventional
• Target: 44
• Locations: 6 countries
• Sites: 30

Brief Summary

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

Conditions

Alpha1-Antitrypsin Deficiency

Outcome Measures

Primary Outcomes (2)

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

  From Baseline at Day 28

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

  Day 1 up to Week 8

Secondary Outcomes (2)

• Change in Plasma Antigenic AAT Levels

  From Baseline at Day 28

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

  Pre-dose at Day 7, Day 14, Day 21 and Day 28

Study Arms (4)

Placebo

PLACEBO COMPARATOR

Participants received placebo matched to VX-864 in the treatment period for 28 days.

Drug: Placebo

VX-864 100 mg

EXPERIMENTAL

Participants received VX-864 100 milligrams (mg) every 12 hours (q12h) in the treatment period for 28 days.

Drug: VX-864

VX-864 300 mg

EXPERIMENTAL

Participants received VX-864 300 mg q12h in the treatment period for 28 days.

Drug: VX-864

VX-864 500 mg

EXPERIMENTAL

Participants received VX-864 500 mg q12h in the treatment period for 28 days.

Drug: VX-864

Interventions

VX-864 DRUG

Tablets for oral administration.

VX-864 100 mg; VX-864 300 mg; VX-864 500 mg

Placebo DRUG

Placebo matched to VX-864 for oral administration.

Placebo

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Vertex Pharmaceuticals Incorporated: lead

Study Sites (30)

University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

Location

David Geffen School of Medicine at UCLA

Los Angeles, California, 90095, United States

Location

University of California Davis Medical Center

Sacramento, California, 95817, United States

Location

National Jewish Health

Denver, Colorado, 80206, United States

Location

University of Florida

Gainesville, Florida, 11735, United States

Location

University of Miami Miller School of Medicine

Miami, Florida, 33136, United States

Location

Central Florida Pulmonary

Orlando, Florida, 32803, United States

Location

The University of Iowa Hospitals and Clinics

Iowa City, Iowa, 52242, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Blessing Corporate Services, Inc., dba Blessing Health System

Hannibal, Missouri, 63401, United States

Location

Cardinal Glennon Children's Hospital - St. Louis University

St Louis, Missouri, 63104, United States

Location

University of North Carolina Medical Center

Chapel Hill, North Carolina, 27514, United States

Location

Wake Forest University Baptist Medical Center

Winston-Salem, North Carolina, 27157, United States

Location

University of Cincinnati

Cincinnati, Ohio, 45229, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Temple University Hospital

Philadelphia, Pennsylvania, 19140, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Renovatio Clinical

Houston, Texas, 77380, United States

Location

The University of Texas Health Science Center at Tyler

Tyler, Texas, 75708, United States

Location

University of Utah Health

Salt Lake City, Utah, 84108, United States

Location

Inova Fairfax Medical Campus

Falls Church, Virginia, 22042, United States

Location

Queen Elizabeth II Health Sciences Center

Halifax, Canada

Location

Inspiration Research Ltd

Toronto, Canada

Location

University Hospital RWTH Aachen

Aachen, Germany

Location

Universitätsklinikum Essen

Essen, Germany

Location

Medizinische Hochschule Hannover

Hanover, Germany

Location

Royal College of Surgeons in Ireland Clinical Research Centre, Beaumont Hospital

Beaumont, Ireland

Location

Skanes Universitetssjukhus, Malmo

Malmo, Sweden

Location

Staploe Medical Centre

Soham Ely, United Kingdom

Location

MeSH Terms

Conditions

alpha 1-Antitrypsin Deficiency

Condition Hierarchy (Ancestors)

Liver Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Subcutaneous Emphysema; Emphysema; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Medical Monitor
• Organization: Vertex Pharmaceuticals Incorporated

medicalinfo@vrtx.com

617-341-6777

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 13, 2020
• First Posted: July 16, 2020
• Study Start: July 24, 2020
• Primary Completion: May 4, 2021
• Study Completion: May 4, 2021
• Last Updated: May 26, 2022
• Results First Posted: May 26, 2022

Record last verified: 2022-05

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1); IE (1); CA (2); US (22); DE (3); SE (1)