MedTrace Logo

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

NCT07287397 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2026-05-14

No results posted yet for this 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.

Conditions

Interventions

GENETIC

VTx-002

An investigational gene therapy targeting a specific protein.

DRUG

Preventative (Prophylactic) Medication - Corticosteroids: Methylprednisolone

To reduce the risk of reactions caused by the study treatment, steroid medicines will be given in advance.

Sponsors & Collaborators

  • Vector Y Therapeutics

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-12-19
Primary Completion
2027-10-15
Completion
2027-10-15
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • Netherlands
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07287397 on ClinicalTrials.gov