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
- Boston, Jacksonville, New York, Philadelphia, Phoenix, and San Diego, United StatesSee the 6 sites
- Phase
- Phase 1/2
Where in United States
- Sean M. Healey & AMG Center for ALS at Massachusetts General HospitalBoston, United StatesTaking people
- Mayo Clinic in FloridaJacksonville, United StatesTaking people
- The Eleanor & Lou Gehrig ALS CenterNew York, United StatesNot yet recruiting
- University of PennsylvaniaPhiladelphia, United StatesTaking people
- St. Joseph's Hospital and Medical Center/Barrow Neurological InstitutePhoenix, United StatesNot yet recruiting
- University of California San Diego Medical CenterSan Diego, United StatesTaking 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?
Trial details
- Who runs it
- Vector Y Therapeutics
- Trial code
- NCT07287397EU 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; -.