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RecruitingPhase 1 / Phase 2interventionalRules rewrite

Phase 1 / Phase 2: testing Preventative (Prophylactic) Medication - Corticosteroids: Methylprednisolone for Amyotrophic Lateral Sclerosis

PIONEER-ALS is a Phase 1/2, multicenter, open-label, ascending dose, uncontrolled, first-in-human study that will measure 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.

Official registry: https://clinicaltrials.gov/study/NCT07287397 · Vector Y Therapeutics · enrollment listed as 36

What this phase means

Phase 2: researchers already know a roughly safe dose (from phase 1). This stage asks whether the treatment actually helps the condition, and watches side effects more closely.

Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.

What the registry says happens

If you join, the listed intervention(s) are VTx-002, or Preventative (Prophylactic) Medication - Corticosteroids: Methylprednisolone. The registry lists allocation as randomized. PIONEER-ALS is a Phase 1/2, multicenter, open-label, ascending dose, uncontrolled, first-in-human study that will measure the safety, tolerability and effects on clinical and biomarker endpoints of intracisternal administration of Vtx-002 in participants with Amyotrophic Lateral Sclerosis (ALS).

They plan to enroll about 36 people at 9 sites. Taking part typically means taking a study drug (or dummy pill), genetic testing or a gene-targeted therapy plus check-ins.

Official title (unrewritten)

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

Interventions: VTx-002, Preventative (Prophylactic) Medication - Corticosteroids: Methylprednisolone

Primary outcome (registry wording): The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs)

Who it is for (from inclusion criteria)

  • Ages 18 Years to any
  • 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
  • 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

Public study contact (from the registry)

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

patients@vectorytx.com

patients@vectorytx.com

Published on ClinicalTrials.gov. Contact them as a possible participant. They decide eligibility.

Sites listed on ClinicalTrials.gov

9 listed

+ 3 sites outside the US on the same record

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FAQ about this NCT record

Is NCT07287397 still recruiting?

The sponsor posted status “Recruiting” on ClinicalTrials.gov at last ingest. Sites can pause independently. Confirm with the official record and the listed contact.

What phase is NCT07287397?

Phase 1 / Phase 2. Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.

Can PlainTrials tell me if I qualify for NCT07287397?

No. The inclusion and exclusion lists on this page are readable extracts of the public eligibility block. Only the study team can screen you.

How to read a ClinicalTrials.gov record · Questions to ask the coordinator · Glossary