Clinical Trial

Disease: Alpha-1 Antitrypsin Deficiency, AATD, (NCT06405633)

Disease info:

Alpha-1 antitrypsin deficiency (AATD) is an inherited genetic disorder that is most commonly caused by a G-to-A point mutation in the SERPINA1 gene. This mutation results in the production of insufficient levels of circulating M-AAT protein, which protects the lungs from proteolytic enzymes, and aggregation of misfolded Z-AAT protein in hepatocytes. This leads to lung and liver disease. Individuals with AATD carry Z mutations on both SERPINA1 alleles; this is known as the PiZZ genotype.

Augmentation therapy via delivery of functional AAT protein is currently the only treatment option for AATD-associated lung disease and requires weekly intravenous infusions. There are no treatments for AATD-associated liver disease, besides liver transplantation.

Frequency:
There are approximately 200,000 patients in the United States and Europe who have Z mutations on both alleles.
Official title:
A Phase 1b/2a Open-label Single Ascending Doses (SAD) and Multiple Ascending Doses (MAD) Research Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics in Participants With AATD Pi*ZZ on WVE-006 (RestorAATion-2)
Who:

Contact

Phone: 855-215-4687

Email: Clinicaltrials@wavelifesci.com 

Partners:
Locations:
Study start:
Jun. 1, 2024
Enrollment:
24 participants
Gene editing method:
RNA editing oligonucleotide
Type of edit:
mRNA correction of single G-to-A point mutation (Z allele) in the SERPINA1 gene
Gene:
SERPINA1
Delivery method:
GalNAc-mediated uptake/conjugation - In-vivo
IndicatorIndicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Not yet recruiting

Description

The purpose of this open-label study is to assess the safety, tolerability, pharmacodynamics, and pharmacokinetics of WVE-006 in participants with alpha-1 antitrypsin deficiency (AATD) following Period 1 single ascending dose (SAD) and Period 2 multiple ascending doses (MAD), respectively.

Last updated: May. 13, 2024
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