VTx-002-01-001 (PIONEER-ALS)
Research type
Research Study
Full title
Phase 1/2 Investigation Of Novel Experimental Regimen in Amyotrophic Lateral Sclerosis (PIONEER-ALS): An Open-Label, Uncontrolled, Multicenter Study to Assess the Safety and Tolerability of Two Doses of VTx-002
IRAS ID
1013480
Contact name
Olga Uspenskaya
Contact email
Sponsor organisation
VectorY Therapeutics B.V.
Eudract number
2025-522697-37
Clinicaltrials.gov Identifier
Research summary
PIONEER-ALS is the first study of a drug called VTx-002. Studies in the lab suggest that VTx-002 might help slow down, stop, or even reverse ALS by lowering harmful protein levels. This is the first time VTx-002 has been tested in people.
Participants in the study will receive VTx-002; there are two doses being tested: The first six participants will get the low dose, one at a time, with careful checks between each person. If the low dose is safe, another six participants will get the high dose, also one at a time. Each participant will be in the study for about 5 years and 5 weeks.
Adults who are 18 or older and have been recently diagnosed with ALS can take part.
Each participant will get a single injection of VTx-002, into the cerebrospinal fluid, in an area below the brain called the cisterna magna. Participants will also have several tests during the study, including MRI scans, spinal taps, blood, urine, saliva and faeces tests, and muscle strength checks. If someone has an inflammation reaction in the brain fluid or liver, they may get a medicine called methylprednisolone or prednisolone.
Ethical Considerations
People with certain health problems, children, pregnant or breastfeeding women cannot join. Women of childbearing potential must agree to use highly effective contraception. An expert committee will check MRI scans and may exclude anyone with extra risks.The injection will happen under general anaesthesia, by a trained doctor in a hospital with emergency care. Participants will stay in the hospital for 24 hours after the injection. If there is a reaction in the brain fluid or liver, they may get a steroid medicine.
The study has been planned to lower risks, such as giving the treatment to one person at a time. People with ALS and their caregivers helped design the study and educational materials for participants.
The study is funded by VectorY Therapeutics B.V.
REC name
South Central - Oxford A Research Ethics Committee
REC reference
26/SC/0018
Date of REC Opinion
25 Mar 2026
REC opinion
Further Information Favourable Opinion