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ALSNCT07287397

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

StatusRecruiting
PhasePhase 1/2
Primary completion (estimated)
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Record History

All 17 versions: 12 with changes below, 5 re-verified with no change. See every version on ClinicalTrials.gov ↗

  1. Enrollment increased: 12 → 36 participants
    • Eligibility criteria expanded, about 15 words
    • Site details revised at 2 of 11 sites
  2. Site details revised at 1 of 11 sitesv15
  3. Site details revised at 1 of 11 sitesv14
  4. Site details revised at 2 of 11 sitesv13
  5. Site details revised at 1 of 11 sitesv12
Show 6 earlier versionsHide 6 earlier versions
  1. Interventions: Optional Rescue medication - Corticosteroids: Methylprednisolone (Drug), VTx-002 (Genetic) to Preventative (Prophylactic) Medication - Corticosteroids: Methylprednisolone (Drug), VTx-002 (Genetic)
    • Study description expanded, about 5 words
    v11
  2. Site details revised at 1 of 11 sitesv10
  3. Site details revised at 1 of 11 sitesv9
  4. Site contacts updated at 1 of 11 sitesv8
  5. Site contacts updated at 1 of 11 sitesv5
  6. Trial status: Not Yet Recruiting to Recruiting
    • Study sites: 13 to 11
    • Start date changed from estimate of Dec 18, 2025 to Dec 19, 2025
    v4
  1. Study First Submitted
  • Notable: the plan changed
  • Minor: routine updates

Endpoints

Primary

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

Secondary (1)

To assess whether VTx-002 works to prevent the progression of ALS and what dose can be used for future clinical studies.

Arms and interventions

Gene Therapy Group 1: Dose 1 (Low Dose)Experimental

Gene Therapy: VTx-002 18 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

Gene Therapy Group 2: Dose 2 (High Dose)Experimental

Gene Therapy: VTx-002 18 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

Genetic: VTx-002

Drugs: Preventative (Prophylactic) Medication - Corticosteroids: Methylprednisolone

Sites

11 sites in 4 countries

United States 7, United Kingdom 2, Belgium 1, Netherlands 1