Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
Recruiting now · Phase 1/Phase 2
Conditions studied: Amyotrophic Lateral Sclerosis
In brief
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.
Key facts
- Study ID
- NCT07287397
- Run by
- Vector Y Therapeutics
- People needed
- 12
- Starts
- 2025-12-19
- Expected to finish
- 2027-10-15
- Last updated by the study team
- 2026-06-23
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- University of Pennsylvania — Philadelphia, Pennsylvania, United States (enrolling)
- University of California San Diego Medical Center — San Diego, California, United States (enrolling)
- UMC Utrecht — Utrecht, Netherlands (enrolling)
- Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital — Boston, Massachusetts, United States (enrolling)
- Mayo Clinic in Florida — Jacksonville, Florida, United States (enrolling)
- St Joseph's Hospital and medical Center - Barrow Neurological Institute — Phoenix, Arizona, United States
- Royal Hallamshire Hospital — Sheffield, United Kingdom
- University of Miami School of Science — Miami, Florida, United States
- Herbert Irving Comprehensive Cancer Center — New York, New York, United States
- UZ Leuven — Leuven, Belgium
- Kings College Hospital — London, United Kingdom
Full record on ClinicalTrials.gov
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