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

Sponsor
Vertex Pharmaceuticals Incorporated (Industry)
Overall Status
Recruiting
CT.gov ID
NCT05643495
Collaborator
(none)
20
Enrollment
2
Locations
2
Arms
24
Anticipated Duration (Months)
10
Patients Per Site
0.4
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease Intervention/Treatment Phase
Phase 2

Study Design

Study Type:
Interventional
Anticipated Enrollment :
20 participants
Allocation:
Non-Randomized
Intervention Model:
Parallel Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Phase 2, Open-label Study Evaluating Efficacy and Safety of VX-864 in Subjects With Alpha-1 Antitrypsin Deficiency Who Have the PiZZ Genotype, Over 48 Weeks
Anticipated Study Start Date :
Dec 1, 2022
Anticipated Primary Completion Date :
Nov 1, 2024
Anticipated Study Completion Date :
Dec 1, 2024

Arms and Interventions

Arm Intervention/Treatment
Experimental: Group A

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

Drug: VX-864
Tablets for oral administration.
Experimental: Group B

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.

Drug: VX-864
Tablets for oral administration.

Outcome Measures

Primary Outcome Measures

  1. Change in Blood Functional Alpha-1 Antitrypsin (AAT) Levels [From Baseline at Week 48]

Secondary Outcome Measures

  1. Change in Blood Functional AAT Levels [From Baseline up to Week 48]

  2. Change in Blood Antigenic AAT Levels [From Baseline up to Week 48]

  3. Change in Blood Z-polymer Levels [From Baseline up to Week 48]

  4. Part B: Change in Z-polymer Accumulation in the Liver [From Baseline up to Week 48]

  5. Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [Day 1 up to Week 52]

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years to 80 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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.

Contacts and Locations

Locations

Site City State Country Postal Code
1 Hannibal Regional Healthcare System Hannibal Missouri United States 63401
2 Renovatio Clinical Houston Texas United States 77380

Sponsors and Collaborators

  • Vertex Pharmaceuticals Incorporated

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT05643495
Other Study ID Numbers:
  • VX22-864-108
  • 2022-002746-40
First Posted:
Dec 8, 2022
Last Update Posted:
Dec 23, 2022
Last Verified:
Nov 1, 2022
Individual Participant Data (IPD) Sharing Statement:
No
Plan to Share IPD:
No
Studies a U.S. FDA-regulated Drug Product:
Yes
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Alpha 1-Antitrypsin Deficiency

Study Results

No Results Posted as of Dec 23, 2022