NCT07287397

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

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

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 12 (estimated)
• Sponsor: Vector Y Therapeutics · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

All participants will receive a single injection of the study drug. During the first year of the study there will be 12 visits to the study center, including 1 overnight stay after dosing. There will be a further 4 remote visits (telephone or video call). From Year 2-5 there will be 8 further visits. These will be every 6 months and will be either in-person at the study site or remote (telephone or video call) if needed or preferred. Throughout the 5-year observation period, there will be up to 20 study visits to complete follow-up tests and assessments and monitor the ongoing effects of the study drug.

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
GENETIC	VTx-002	An investigational gene therapy targeting a specific protein.
DRUG	Optional Rescue medication - Corticosteroids: Methylprednisolone	This intervention will only be administered if the study doctor concludes this is necessary based on the review of clinical and laboratory findings.

Timeline

• Start date: 2025-12-19
• Primary completion: 2027-10-15
• Completion: 2027-10-15
• First posted: 2025-12-17
• Last updated: 2026-04-13

Locations

11 sites across 4 countries: United States, Belgium, Netherlands, United Kingdom

Regulatory

• FDA-regulated drug study