Phase 2
Completed N=44
Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype
Alpha1-Antitrypsin Deficiency
Source: ClinicalTrials.gov NCT04474197 ↗
Enrolled (actual)
44
Serious AEs
4.6%
Results posted
May 2022
Primary outcomePrimary: Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels — -0.1; 2.3; 2.3; 2.1 micromole per liter — p=<0.0001
Summary
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-864 in PiZZ subjects.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels |
-0.1; 2.3; 2.3; 2.1 | <0.0001 sig |
| PRIMARY Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) |
4; 7; 7; 17; 0; 0 | — |
| SECONDARY Change in Plasma Antigenic AAT Levels |
-0.1; 3.4; 2.9; 2.6 | <0.0001 sig |
| SECONDARY Observed Pre-dose Plasma Concentration (Ctrough) of VX-864 |
1.13; 1.62; 2.74; 0.852; 1.47; 4.35 | — |
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT04474197). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.