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Phase 2 Completed N=14 Randomized Triple-blind Treatment

A Double-blind Study to Evaluate the Safety of Glutamic Acid Decarboxylase Gene Transfer in Parkinson's Participants

Source: ClinicalTrials.gov NCT05603312 ↗
Enrolled (actual)
14
Serious AEs
21.4%
Results posted
Sep 2025
Primary outcomePrimary: Incidence of Adverse Events Related to the Treatment — 0; 1; 0 Participants

Summary

The objective of this clinical trial was to evaluate the safety and tolerability of adeno-associated virus (AAV)-mediated delivery of glutamic acid decarboxylase (GAD) gene transfer into the subthalamic nuclei (STN) of participants with Parkinson's Disease.

Outcome Measures

OutcomeResultp-value
PRIMARY
Incidence of Adverse Events Related to the Treatment
0; 1; 0
PRIMARY
Incidence of Serious Adverse Events Related to the Treatment
0; 0; 0

Eligibility Criteria

Inclusion Criteria

  • Confirmed Parkinson's Disease
  • Levodopa responsiveness for at least 12 months
  • UPDRS Part 3 score of ≥25 points in the "off" state

Exclusion Criteria

  • History of brain surgery to treat Parkinson's Disease
  • Any history of cerebral insult or central nervous system infection
  • Atypical Parkinson's Disease
  • Focal or lateralized neurologic deficits
  • Evidence of significant medical or psychiatric disorders
  • Cognitive impairment as defined by the Montreal Cognitive Assessment (MoCA) ≤ 20
  • Beck Depression Inventory-II score of ≥ 20
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT05603312). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.

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