RecruitingPHASE1, PHASE2INTERVENTIONAL

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

This study, called PIONEER-ALS, is looking into a new medicine, VTx-002, for people with Motor Neurone Disease (ALS). It's the first time this medicine is being given to people, and it's in the early stages of testing (Phase 1/2). The main goal is to check how safe VTx-002 is and if people can tolerate it. Researchers will also be looking for any signs that the medicine is helping with the disease. Participants will receive a single injection of VTx-002, and some might also get a steroid medicine. The study will last up to five years for each person, with regular check-ups to monitor their health and the medicine's effects. It's for adults aged 18 and over.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

Vector Y Therapeutics

Enrolment target

Start

19 Dec 2025

Estimated completion

15 Oct 2027

Amyotrophic Lateral Sclerosis

What is this study about?

This study is called PIONEER-ALS, and it’s looking into a new potential treatment for Motor Neurone Disease (ALS) called VTx-002. ALS is a condition that affects how your nerves and muscles work, leading to weakness and other symptoms. The researchers want to find out if this new medicine is safe for people and if they can tolerate it without too many problems. This is an early-stage study, meaning it’s the first time VTx-002 is being given to people, so we’re still learning a lot about it.

Participants in the study will receive a single injection of VTx-002. Some people might also be given a steroid medicine called Methylprednisolone, which is sometimes used to help prevent unwanted reactions. The study doesn't say if this steroid is for everyone or specific groups. During the study, the medical team will also be looking for clues about whether VTx-002 has any helpful effects on ALS symptoms or markers in the body.

The study will last a long time for each person – up to five years. This extended period allows the researchers to carefully watch participants and understand how VTx-002 affects them over time. The main aim is to gather important information about the safety and tolerance of VTx-002, which is crucial for developing new treatments for ALS.

Key takeaways

PIONEER-ALS is a study for people with Motor Neurone Disease (ALS).
It's testing a new medicine called VTx-002 for safety and tolerance.
Participants will have one injection and be followed for up to five years.
The study involves regular visits to a clinic and some remote check-ups.
It's an early-stage study, meaning the medicine is still being explored.
Your diagnosis must be within 18 months of your first symptoms.

Who may be eligible?

This study is for adults aged 18 and over who have been diagnosed with Motor Neurone Disease (ALS). You would need to be able to understand the study and agree to take part, including attending all the necessary appointments.

To be eligible, you or your family would need to confirm that your ALS is not caused by specific gene problems (called FUS and SOD1 mutations). Also, your first symptoms of weakness should have appeared no more than 18 months ago. You would also need to have good lung function, measured by a test called SVC, and a certain risk score for your ALS progression.

There are also some reasons why you might not be able to join. For example, if you have another significant brain or nerve condition that could be mistaken for ALS and if you have certain findings on MRI scans of your brain or spine. If you already have a tracheostomy (a breathing tube) or a feeding tube, you cannot take part. Your doctor will carefully review all your medical information to see if this study is right for you.

Quick self-check

Are you 18 years old or older?
Do you have a confirmed diagnosis of Motor Neurone Disease (ALS)?
Did your first ALS symptoms appear within the last 18 months?
Do you have a reliable caregiver or partner who can support you if needed?
Are you able to travel to a study center for appointments?
Have you discussed your current medications with your doctor, especially riluzole or edaravone?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you join this study, it will be a long-term commitment, lasting up to five years and five weeks for each person. Before starting, there will be a screening period of up to five weeks to ensure you meet all the requirements. You will receive one injection of the study medicine, VTx-002.

During the first year, you will need to attend the study center 12 times. One of these visits will include staying overnight for at least one night after receiving the medicine. There will also be four additional check-ups via phone or video call. For the remaining four years (Years 2-5), you will have eight more visits, happening roughly every six months. These can be in person at the study site or by phone/video call, depending on what's best. All these visits are to monitor your health, check for any changes, and see how the study medicine is affecting your condition over time.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. For this study, potential benefits might include contributing to the understanding of ALS and helping to develop new treatments, as well as receiving close medical monitoring. However, there's no guarantee that VTx-002 will improve your condition or that you will experience any direct personal benefit. Potential risks are not fully known since this is an early-stage study, but they could include side effects from the medicine or the injection procedure. You will be told all known risks before you decide to join. Remember, you can choose to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (11)

St Joseph's Hospital and medical Center - Barrow Neurological Institute
Phoenix, United States· Not yet recruiting
University of California San Diego Medical Center
San Diego, United States· Recruiting
Mayo Clinic in Florida
Jacksonville, United States· Recruiting
University of Miami School of Science
Miami, United States· Not yet recruiting
Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital
Boston, United States· Recruiting
Herbert Irving Comprehensive Cancer Center
New York, United States· Not yet recruiting
University of Pennsylvania
Philadelphia, United States· Not yet recruiting
UZ Leuven
Leuven, Belgium· Not yet recruiting
UMC Utrecht
Utrecht, Netherlands· Not yet recruiting
Kings College Hospital
London, United Kingdom· Not yet recruiting
Royal Hallamshire Hospital
Sheffield, United Kingdom· Not yet recruiting

Common questions

What is VTx-002?

VTx-002 is a new investigational medicine being tested in this study to see if it's safe and tolerable for people with Motor Neurone Disease (ALS).

How long will the study last for me?

The study will last for up to five years and five weeks for each person who takes part.

Will I have to travel a lot for the study?

Yes, especially in the first year, you'll need to attend the study center regularly, including an overnight stay. Later visits may be more flexible, with some able to be done remotely.

Will this medicine cure my ALS?

As this is an early-stage study, its main goal is to check safety and tolerance. We don't know if VTx-002 will cure ALS or even improve it at this point.

Can I leave the study if I change my mind?

Yes, you can choose to stop participating in the study at any time, for any reason, without affecting your medical care.

How to find out more

Dr Olga Uspenskaya Chief medical Officer, VectorY Therapeutics, M.D; PhD

patients@vectorytx.com patients@vectorytx.com Official trial record

Always speak to your GP or specialist before deciding to take part in a study.