Status: Recruiting
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.
Participants needed: 12
Trial details
Phase: Phase 1, Phase 2Age: 18+Biological sex: AllType: InterventionalSponsor: Vector Y TherapeuticsUpdated: Jun 23, 2026Locations: 11
Eligibility criteria
Capable of, and willing to, provide written informed consent and comply with stu... [+10]
Diagnosis of a significant CNS or peripheral nervous system disease other than A... [+3]