Version history

1 version on record. Newest first; the live version sits at the top with a live indicator.

  1. Live sha256:ab245
    4/30/2026, 2:41:24 PM
    Content snapshot 
    {
  "nct_id": "NCT04127578",
  "phase": "phase_2",
  "status": "active",
  "domain": "parkinsons",
  "indication": "GBA1-mutant Parkinson's Disease",
  "intervention": "PR001 (AAV9-GBA1 intracisternal)",
  "sponsor": "Prevail Therapeutics / Lilly",
  "n_enrolled": 78,
  "primary_endpoint": "Safety + GCase enzyme activity in CSF at 52 weeks",
  "summary": "First systemic AAV gene therapy for a PD subtype; targets the most common genetic risk factor (GBA1 LoF).",
  "metadata": {}
}