Status:
COMPLETED
Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype
Lead Sponsor:
Vertex Pharmaceuticals Incorporated
Conditions:
Alpha1-Antitrypsin Deficiency
Eligibility:
All Genders
18-80 years
Phase:
PHASE2
Brief Summary
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-864 in PiZZ subjects.
Eligibility Criteria
Inclusion
- Key
- Subjects must have a PiZZ genotype confirmed at screening
- Plasma AAT levels indicating severe deficiency at screening
- Key
Exclusion
- 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
Key Trial Info
Start Date :
July 24 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 4 2021
Estimated Enrollment :
44 Patients enrolled
Trial Details
Trial ID
NCT04474197
Start Date
July 24 2020
End Date
May 4 2021
Last Update
May 26 2022
Active Locations (30)
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1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
2
David Geffen School of Medicine at UCLA
Los Angeles, California, United States, 90095
3
University of California Davis Medical Center
Sacramento, California, United States, 95817
4
National Jewish Health
Denver, Colorado, United States, 80206