Taking people in BelgiumPhase 1/2

Tests the safety of a gene therapy, VTx-002, in adults with ALS

Tests the safety of VTx-002 given into the spine in adults with ALS.

Plain-language title generated from the registry record
Registry title: Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS

Key facts

Where: Leuven, BelgiumSee the site
Phase: Phase 1/2

AI summary: who the study is looking for

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for adults with ALS whose weakness began within the last 18 months and who do not have FUS or SOD1 gene mutations.

Age: 18 years or older
Sex: Women and men can join.
Disease stage: Probable, laboratory-supported probable, or definite ALS by the El Escorial criteria; weakness first appeared no more than 18 months ago; seated SVC % predicted of 80% or more; TRICALS risk score between -2 and -6.
Earlier treatment: Riluzole and/or edaravone are allowed if the dose was stable for at least 2 weeks (riluzole) or one treatment cycle (edaravone) before screening.
Biomarker or test result: Lab tests must confirm that ALS is not caused by FUS or SOD1 gene mutations.

Main reasons for not taking part

Another significant nervous system disease that could cause the ALS symptoms
Both a tracheostomy and a feeding tube at screening
Clinically significant abnormality on spinal, cervical or brain MRI/MRA
Unable to safely stop blood thinners before study procedures
Cannot have general anaesthesia or deep sedation, or cannot have MRI, MRA or CT scans

Read by machine on 9 November 2026.

Where in Belgium

UZ LeuvenLeuven, BelgiumTaking people

Questions to ask the study team

How often do I need to come in, and for how long each time?
Are travel costs paid?
Can I keep my own doctor?
What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Trial details

Who runs it: Vector Y Therapeutics
Trial code: NCT07287397 EU 2025-522697-37-00
Record updated: 6 November 2026
Original description: 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).Read full original descriptionShow less
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
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.
Inclusion: Capable of, and willing to, provide written informed consent and comply with study procedures, including visits to the study site and visit requirements; Male or female ≥ 18 years of age; Has a diagnosis of ALS according to the El Escorial criteria (Brooks, et al., 2000) (probable, laboratory results supported; clinically probable, clinically definite); Confirmed absence of ALS caused by FUS and SOD1 gene mutations confirmed by laboratory tests; A maximum of 18 months since first appearance of weakness (e.g., limb weakness, dysarthria, dysphagia, shortness of breath); Erect (seated) SVC % predicted ≥ 80% at Screening; Treatment Research Initiative to Cure ALS (TRICALS) risk score between -2 and -6 at Screening; Has a reliable caregiver/partner/legal representative willing and able to support the participant in participation in the study and to give informed consent on behalf of the participant in the case that disease progression prevents the participant of giving consent (local legal rules will apply); Treatment with riluzole and/or edaravone is allowed if treatment was started and has remained at a stable dose for at least 2 weeks (riluzole) or one treatment cycle (edaravone) before the Screening visit; Women of childbearing potential (WOCBP) and male participants with female partners who are WOCBP must agree to use highly effective contraception during and after the study. WOCBP cannot be pregnant or breastfeeding; Women of nonchildbearing potential must be post-menopausal or surgically sterile (e.g. hysterectomy, bilateral tubal ligation, ovaries removed).
Exclusion: Significant concomitant disease or condition within 6 months of Screening that could pose an unacceptable safety risk to the participant or interfere with the participant's ability to comply with study procedures, e.g. heart disease, uncontrolled diabetes, liver disease, autoimmune diseases needing strong immune-suppressing drugs, cancer, etc or a current psychiatric diagnosis; Contraindications to general anaesthesia (GA) or deep sedation; 10 Positive test for illegal drugs (except prescribed medications or permitted medicinal/recreational marijuana if used responsibly); Diagnosis of a significant CNS or peripheral nervous system disease other than ALS that may be a cause for the participant's ALS symptoms or may confound study objectives; Contraindications to corticosteroid use (e.g. due to osteoporosis, uncontrolled blood pressure, diabetes or cholesterol); Clinically significant abnormalities in laboratory test results at Screening for example poor liver or kidney function, abnormal clotting or infections such as Hepatitis or HIV; Spinal, cervical, or brain MRI/MRA indicating clinically significant abnormality; Presence of tracheostomy and feeding tube at Screening; Use of blood thinners (e.g., warfarin, heparin, and novel oral anticoagulants) and being unable to safely stop them before certain study procedures; Contraindications to imaging methods MRI, MRA, CT due to claustrophobia and/or intolerance to contrast agents; Generally frail or if the Investigator deems participation in the study would not be in the best interest of the participant or is likely to prohibit further participation during the study; Other protocol-defined inclusion/exclusion criteria may apply; -.
ClinicalTrials.gov NCT07287397 · EU CTIS 2025-522697-37-00