Clinical trialNCT07287397RecruitingPhase 1/PHASE2Interventional

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

SponsorVector Y TherapeuticsEnrollment12Amyotrophic Lateral Sclerosis

View the registry record on ClinicalTrials.gov

Summary

The lay summary the sponsor registered on ClinicalTrials.gov.

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.

clinicaltrials.gov ↗

Investigators

No overall officials are registered on this study record. Site-level investigators, where published, appear with their sites below. Why? →

A profile link is shown only when the registered name resolves to a person record with a confident, namesake-checked match; ambiguous names stay unlinked.

Sites

11 registered facilities, with the per-site recruitment status where the registry publishes one. ClinicalTrials.gov does not publish per-site enrollment counts.

Belgium

Uz Leuven
Leuven, Belgium
Dr Philip Van Damme, Doctor of Medicine
Not, yet, recruiting

Netherlands

Umc Utrecht
Utrecht, Netherlands
Prof Leonard Van Den Berg, Doctor of Medicine
Recruiting

United Kingdom

Kings College Hospital
London, United Kingdom
Not, yet, recruiting
Royal Hallamshire Hospital
Sheffield, United Kingdom
Not, yet, recruiting

United States

St Joseph'S Hospital and Medical Center - Barrow Neurological Institute
Phoenix, Arizona, United States
Dr Shafeeq Ladha, Doctor of Medicine
Not, yet, recruiting
University of California San Diego Medical Center
San Diego, California, United States
Dr John Ravits, Doctor of Medicine
Recruiting
Mayo Clinic in Florida
Jacksonville, Florida, United States
Dr Bjorn Oskarsson, Doctor of Medicine
Recruiting
University of Miami School of Science
Miami, Florida, United States
Dr Michael Benatar, Doctor of Medicine
Not, yet, recruiting
Sean M. Healey & Amg Center for Als at Massachusetts General Hospital
Boston, Massachusetts, United States
Dr Doreen Ho, Doctor of Medicine
Recruiting
Herbert Irving Comprehensive Cancer Center
New York, New York, United States
Dr Neil Schneider, Doctor of Medicine
Not, yet, recruiting
University of Pennsylvania
Philadelphia, Pennsylvania, United States
Dr Colin Quinn, Doctor of Medicine
Recruiting

Site investigator names are published by ClinicalTrials.gov only while a study recruits, so completed studies list sites without them.

Drugs studied

The drug and biological interventions registered on this study, normalized to their RxNorm ingredient where possible.

Preventative Medication - Corticosteroids: Methylprednisolone

Linked drugs open the directory filtered to KOLs working with them.clinicaltrials.gov ↗

Registration & key dates

Registry attributes as recorded on the ClinicalTrials.gov study record.

Start: 19 Dec 2025
Primary completion: 15 Oct 2027
Completion: 15 Oct 2027
Last update posted: 23 Jun 2026
Registered enrollment: 12
Registry id: NCT07287397

Cross-registrations

EU-CTR / CTIS (EudraCT)2025-522697-37-00

clinicaltrials.gov ↗

Source: ClinicalTrials.gov, via the CTTI AACT database, data as of 24 Aug 2026. Registry facts are shown as registered by the study sponsor; findmyKOL does not interpret them.