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NCT07287397 · ClinicalTrials.gov registry record · Phase 1
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
A Phase 1 study of Amyotrophic Lateral Sclerosis, sponsored by Vector Y Therapeutics.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 12
- Enrollment target
- 11
- Study locations
NCT07287397: Recruiting Phase 1 study of Amyotrophic Lateral Sclerosis, sponsored by Vector Y Therapeutics.
NCT07287397 is a Phase 1 study of Amyotrophic Lateral Sclerosis that is actively recruiting participants, run by Vector Y Therapeutics. The registered enrollment target is 12 participants, below the 898-participant average among 102 other Amyotrophic Lateral Sclerosis trials with a reported enrollment target (99% lower). The trial reports 11 study locations across 7 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT07287397, a Phase 1 study of Amyotrophic Lateral Sclerosis, is actively recruiting participants, sponsored by Vector Y Therapeutics.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 12 participants
- Enrollment target
- 11
- Study locations
Study 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.
Primary Outcome
Assessed by reviewing the nature, incidence, severity, relatedness, seriousness and outcome of treatment emergent adverse events.
Conditions Studied
Interventions
- GENETIC VTx-002
- DRUG Optional Rescue medication - Corticosteroids: Methylprednisolone
Study Locations (11)
Other
- UZ Leuven - Leuven
- UMC Utrecht - Utrecht
- Kings College Hospital - London
- Royal Hallamshire Hospital - Sheffield
Florida
- Mayo Clinic in Florida - Jacksonville
- University of Miami School of Science - Miami
Arizona
- St Joseph's Hospital and medical Center - Barrow Neurological Institute - Phoenix
California
- University of California San Diego Medical Center - San Diego
Massachusetts
- Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital - Boston
New York
- Herbert Irving Comprehensive Cancer Center - New York
Pennsylvania
- University of Pennsylvania - Philadelphia
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2025-12-19 |
| Est. Completion | 2027-10-15 |
| Phase | Phase 1 |
What NCT07287397 shows while recruiting
NCT07287397 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 898-participant average among 102 other Amyotrophic Lateral Sclerosis trials with a reported enrollment target (99% lower).
The record links to 1 condition, with Amyotrophic Lateral Sclerosis appearing as the primary indexed condition, and to 2 interventions - of which VTx-002 is the first listed.
NCT07287397 lists 11 locations in 7 states (Other, Florida, Arizona).
Frequently Asked Questions
What is clinical trial NCT07287397 about?
NCT07287397 is a clinical study titled "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). T...
What is the current status of trial NCT07287397?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 12 participants. The study started on 2025-12-19. Estimated completion is 2027-10-15.
What conditions does trial NCT07287397 study?
This clinical trial studies the following conditions: Amyotrophic Lateral Sclerosis.
What interventions are being tested in trial NCT07287397?
The interventions under investigation include: VTx-002 (GENETIC), Optional Rescue medication - Corticosteroids: Methylprednisolone (DRUG).
Who is sponsoring clinical trial NCT07287397?
This trial is sponsored by Vector Y Therapeutics, which has 1 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT07287397 being conducted?
This trial has 11 study locations across Arizona, California, Florida, Massachusetts, New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT07287397's enrollment target sits among peer trials
12 88th of 102 higher than 14 of 102 other Amyotrophic Lateral Sclerosis trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Amyotrophic Lateral Sclerosis trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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