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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 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.

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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.

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

What the Registry Record Tells You About NCT07287397

The ClinicalTrials.gov registry entry for NCT07287397 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 12 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 898-participant average among 102 other Amyotrophic Lateral Sclerosis trials with a reported enrollment target (99% lower). The listed sponsor is Vector Y Therapeutics, which has 1 total studies on file at ClinicalTrials.gov.

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 reports 11 study locations spanning 7 distinct geographic areas - top geographies include 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.

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

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