Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS

PIONEER-ALS is a Phase 1/2, multicenter, open-label, ascending dose, uncontrolled, first-in-human study that will evaluate the safety, tolerability and effects on clinical and biomarker endpoints of intracisternal administration of Vtx-002 in participants with Amyotrophic Lateral Sclerosis (ALS). Two escalating dose (low dose and high dose) cohorts are planned. The duration of the study will be a maximum of 5 years and 5 weeks (265 weeks) for each participant. The screening period may last up to 5 weeks to complete screening procedures.

NCT Number	NCT07287397
Lead Sponsor	Vector Y Therapeutics
Conditions	Amyotrophic Lateral Sclerosis
Enrollment	12 participants
Start Date	2025-12-19
Primary Completion	2027-10-15 (estimated)
Study Completion	2027-10-15 (estimated)
Updated on ClinicalTrials.gov	2026-06-23