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

Conditions

• Amyotrophic Lateral Sclerosis

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• VTx-002 — GENETIC
  An investigational gene therapy targeting a specific protein.
• Preventative (Prophylactic) Medication - Corticosteroids: Methylprednisolone — DRUG
  To reduce the risk of reactions caused by the study treatment, steroid medicines will be given in advance.

Study Details

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.

Key Dates

Start date

Dec 19, 2025

Status verified

May 2026

Primary completion

Oct 15, 2027

Completion

Oct 15, 2027

Study Design

Enrollment

12 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Gene Therapy Group 1: Dose 1 (Low Dose)
  Gene Therapy: VTx-002 6 participants will receive dose 1 administered intra cisterna magna. Dosing of the first 3 participants will be staggered at specific timepoints apart and with a safety monitoring committee review of health-related information in between each participant being dosed. VTx-002 is a single dose therapy Drug: Preventative (Prophylactic) Medication: Methylprednisolone
• Experimental: Gene Therapy Group 2: Dose 2 (High Dose)
  Gene Therapy: VTx-002 6 participants will receive dose 2 administered intra cisterna magna. The participant dosing in group 2 will be staggered as it was in group 1. VTx-002 is a single dose therapy. Drug: Preventative (Prophylactic) Medication - Methylprednisolone

Primary Outcome Measure

The number of participants with treatment related adverse events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Over 5 years ]

Central Contacts

• Dr Olga Uspenskaya Chief medical Officer, VectorY Therapeutics, M.D; PhD
  [email protected]
  [email protected]

Locations (7)

Find similar trials in Phoenix, AZ

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