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

Summary

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.

Description

Eligibility

Age range

18+ years

Sex

All

Healthy volunteers

No

Interventions

• Genetic
  VTx-002

  An investigational gene therapy targeting a specific protein.

• Drug
  Optional Rescue medication - Corticosteroids: Methylprednisolone

  This intervention will only be administered if the study doctor concludes this is necessary based on the review of clinical and laboratory findings.

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