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VTX-002

Clinical trials are investigating VTX-002 in people with amyotrophic lateral sclerosis (ALS). This article explains what the study is trying to learn, who may take part, and which safety measures and outcomes are being checked. The main focus is safety and tolerability in a small Phase 1/2 study.

Table of Contents

Trial overview

The available study of VTX-002 is titled “A Phase 1/2 Study of the Safety and Tolerability of ICM VTx-002 in participants with ALS.”[1] It is an interventional trial, which means researchers give the study treatment and then watch what happens.[1] The trial is listed as Authorised and plans to enroll 12 participants.[1]

Who is being studied

This trial is for people with amyotrophic lateral sclerosis (ALS).[1] ALS is the condition named in the trial record, and no other target population is listed.[1] The source data do not provide more detailed inclusion or exclusion rules, so the exact entry criteria are not known from the trial summary alone.[1]

What the study measures

The main goal is to assess the safety and tolerability of increasing doses of a single administration of VTX-002.[1] Safety means whether unwanted medical problems happen, and tolerability means how well participants can handle the treatment.[1]

The primary outcome looks at the nature, number, severity, relatedness, seriousness, and outcome of treatment-emergent adverse events (new health problems that start after treatment begins).[1] The study also checks laboratory values, MRI findings, the Treatment-induced Peripheral Neuropathy Assessment Scale (TNAS), cellular responses to both the vector and the transgene encoded protein, and the Columbia Suicide Severity Rating Scale (C-SSRS).[1]

These measures help researchers see whether the treatment affects the body in ways that may matter for future research in ALS.[1] They also give a fuller safety picture than side effects alone because they include scans, blood or other test results, nerve symptom checks, and mental health screening.[1]

Trial design and phase

This study is in Phase 1/2, which is an early stage of clinical research.[1] Early-phase studies usually focus on safety first, and they may also begin to look for other signals that help plan later trials.[1]

The brief summary says the study is designed to assess increasing doses of a single administration of VTX-002.[1] The trial data also list VTx-002 Diluent as an intervention, but the main study focus remains VTX-002 in participants with ALS.[1]

Why this trial matters

ALS is a serious disease, so early studies like this are important for learning whether a new treatment can be studied safely in this group.[1] Because the trial is small and focused on safety, it is not designed to answer all questions about benefit.[1] Instead, it provides early information that can help decide whether larger studies should be done later.[1]

Trial IDPhaseCondition studiedStatusEnrollment
2025-522697-37-00Phase 1/2Amyotrophic Lateral SclerosisAuthorised12

FAQ

  • What is being studied in the VTX-002 trial? The trial is studying a single administration of VTX-002 in people with ALS. The main goal is to see whether increasing doses are safe and well tolerated.
  • Who can take part in this VTX-002 study? The study is for participants with amyotrophic lateral sclerosis (ALS). The trial data do not give more detailed entry rules.
  • What phase is the VTX-002 trial? This is a Phase 1/2 study. Phase 1/2 studies usually look at early safety and tolerability, and may also begin to explore other effects.
  • What outcomes are being measured? The study checks treatment-emergent adverse events, laboratory values, MRI findings, nerve symptom scores, cellular immune responses, and suicide severity ratings. These measures help show safety and how the body responds.
  • What is the status of the VTX-002 trial? The trial is listed as Authorised. It is an interventional study with 12 planned participants.

Ongoing Clinical Trials on VTX-002

  • A study to evaluate the safety of VTx-002 in patients with amyotrophic lateral sclerosis.

    Recruiting

    2 1 1
    Investigated drugs:
    Belgium The Netherlands Spain

Glossary

  • Amyotrophic Lateral Sclerosis (ALS): A serious disease that affects nerve cells and causes muscle weakness over time.
  • Phase 1/2: An early stage of clinical research that first checks safety and tolerability and may also look for early signs of benefit.
  • Safety: How well a treatment can be given without causing harmful problems.
  • Tolerability: How well people can handle a treatment and its effects.
  • Interventional study: A study where researchers give a treatment and then observe what happens.
  • Treatment-emergent adverse events (TEAEs): New health problems that start or get worse after treatment begins.
  • Laboratory values: Results from blood or other tests that help check body functions.
  • Magnetic resonance imaging (MRI): A scan that creates detailed pictures inside the body.
  • Treatment-induced Peripheral Neuropathy Assessment Scale (TNAS): A tool used to measure nerve-related symptoms, especially in the hands and feet.
  • Columbia Suicide Severity Rating Scale (C-SSRS): A questionnaire used to assess thoughts of self-harm or suicide.

References

  1. https://clinicaltrials.gov/study/2025-522697-37-00