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RECRUITING Phase 1

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

NCT07287397 · View on ClinicalTrials.gov ↗

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.

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

FieldValue
Enrollment Target 12 participants
Start Date 2025-12-19
Est. Completion 2027-10-15
Phase Phase 1

Sponsor

Vector Y Therapeutics

1 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT07287397

The ClinicalTrials.gov registry entry for NCT07287397 describes a study currently listed as recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 12 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Vector Y Therapeutics, which has 1 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT07287397 reports 11 study locations spanning 7 distinct geographic areas — top geographies include Other, Florida, Arizona. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

What interventions are being tested in trial NCT07287397?

The interventions under investigation include: VTx-002 (GENETIC), Optional Rescue medication - Corticosteroids: Methylprednisolone (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

Who is sponsoring clinical trial NCT07287397?

This trial is sponsored by Vector Y Therapeutics, which has 1 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

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.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial