GenePHIT: A study to learn more about how well a new gene therapy (AB-1002) works and its safety in participants with congestive heart failure

Study title: 
GenePHIT: A study to learn more about how well a new gene therapy (AB-1002) works and its safety in participants with congestive heart failure
Long title: 
A phase 2, adaptive, double-blinded, placebo controlled, randomized, multi-center trial to evaluate the efficacy, safety and tolerability of intracoronary infusion of AB-1002 in adult subjects with New York Heart Association (NYHA) Class III heart failure and non-ischemic cardiomyopathy
Date receipt dossier: 
11 juil 2025
EU record number: 
B/BE/25/BVW5
EudraCT number: 
2024-510581-17-00
Company / Sponsor: 
Asklepios BioPharmaceutical (AskBio)
Phase: 
II
Treated organism: 
Humans
Indication category: 
Congestive Heart failure
Disease: 
NYHA Class III heart failure and non-ischemic cardiomyopathy
Therapeutic approach: 
Gene therapy
Genetic modification: 
Non-replicating recombinant vector derived from adeno-associated virus AAV2i8 carrying the transgene encoding for the human activated Inhibitor-1c ( I-1c)
Method of transfer of nucleic acid of interest: 
Non-replicating recombinant vector derived from adeno-associated virus AAV2/AAV8
Administered biological material: 
Genetically modified virus
Route of administration: 
Intracoronary
Locations in Belgium: 
AZORG in Aalst UZ Antwerpen in Edegem UZ Gent in Gent AZ Delta in Roeselare
Nr of subjects: 
Up to 150 patients overall and 5 patients in Belgium
Foreseen duration: 
from Aug.2025 till 31.12.2030
Type of procedure: 
Contained use and Deliberate release
Current status: 
Under evaluation

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