Phase 1/2 Investigation Of Novel Experimental Regimen in Amyotrophic Lateral Sclerosis (PIONEER-ALS): An Open-Label, Uncontrolled, Multicenter Study to Assess the Safety and Tolerability of Two Doses of VTx-002

Study title: 
Phase 1/2 Investigation Of Novel Experimental Regimen in Amyotrophic Lateral Sclerosis (PIONEER-ALS): An Open-Label, Uncontrolled, Multicenter Study to Assess the Safety and Tolerability of Two Doses of VTx-002
Long title: 
Phase 1/2 Investigation Of Novel Experimental Regimen in Amyotrophic Lateral Sclerosis (PIONEER-ALS): An Open-Label, Uncontrolled, Multicenter Study to Assess the Safety and Tolerability of Two Doses of VTx-002
Date receipt dossier: 
4 feb 2026
EU record number: 
B/BE/25/BVW9
EudraCT number: 
2025-522697-37-00
Company / Sponsor: 
VectorY Therapeutics B.V.
Phase: 
I/II
Treated organism: 
Humans
Indication category: 
Neurodegenerative disease
Disease: 
Amyotrophic Lateral Sclerosis
Therapeutic approach: 
Gene therapy
Genetic modification: 
Non-replicating recombinant AAV vector with a AAV5.2 capsid and carrying the transgene corresponding to the single-chain variable fragment that binds to human TDP-43
Method of transfer of nucleic acid of interest: 
Non-replicating recombinant vector derived from adeno-associated virus serotype 5
Administered biological material: 
Genetically modified virus
Route of administration: 
Intracisternal
Locations in Belgium: 
UZ Leuven
Nr of subjects: 
up to 12 patients overall and 3 patients in Belgium
Foreseen duration: 
From December 2025 till October 2031
Type of procedure: 
Contained use and Deliberate release
Current status: 
Under evaluation

Information for the public