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Gene therapy for adults with moderate Parkinson's Disease

A Phase 2, Randomized, Double-blind, Sham Surgery-controlled Study of the Efficacy and Safety of Intraputaminal AAV2-GDNF in the Treatment of Adults With Moderate Stage Parkinson's Disease

Phase 2 Interventional AskBio Inc · NCT06285643

This study is testing a new gene therapy for adults with moderate Parkinson's Disease to see if it can improve their movement and quality of life.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment127 (estimated)
Ages45 Years to 75 Years
SexAll
SponsorAskBio Inc Industry-sponsored
Locations39 sites (Irvine, California and 38 other locations)
Trial IDNCT06285643 on ClinicalTrials.gov

What this trial studies

This Phase 2 clinical trial evaluates the safety and efficacy of AAV2-GDNF gene therapy delivered to the putamen in adults diagnosed with moderate Parkinson's Disease. The study is randomized, surgically controlled, and double-blinded, ensuring that neither participants nor researchers know who receives the treatment versus the control. Participants must have been diagnosed with Parkinson's Disease within the last 4 to 10 years and show responsiveness to levodopa therapy. The trial aims to assess improvements in motor function and overall quality of life for those affected by this condition.

Who should consider this trial

Good fit: Ideal candidates are adults aged 45-75 with a diagnosis of moderate Parkinson's Disease who have been stable on anti-parkinsonian medications.

Not a fit: Patients with significant cognitive impairment, cardiovascular disease, or those with alternative diagnoses to idiopathic Parkinson's Disease may not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could significantly improve motor function and quality of life for patients with moderate Parkinson's Disease.

How similar studies have performed: Other studies have shown promise with gene therapy approaches for neurodegenerative diseases, indicating potential for success in this novel application.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

Age

1. Male and female adults 45-75 years of age inclusive, at the time of signing of informed consent Type of Subject and Disease Characteristics
2. Diagnosed with Parkinson's disease in the past 4-10 years (inclusive) as defined by the following:

   1. Presence of bradykinesia PLUS any of the following:

      * Rigidity
      * Rest tremor
      * Postural instability
   2. Presence of motor fluctuations as measured by the PD Motor Diary
   3. Stable anti-parkinsonian medication regimen for \>/= 4 weeks prior to screening
   4. Must demonstrate responsiveness to levodopa therapy

Exclusion Criteria:

* Known history or current evidence of medical, genetic, or neurological conditions that may provide an alternative to idiopathic PD diagnosis
* Presence or history of significant vascular and/or cardiovascular disease
* Presence of significant cognitive impairment, poorly controlled depression/anxiety
* Presence or history of psychosis or impulse control disorder
* History of malignancy other than treated cutaneous squamous or basal cell carcinomas
* Presence of clinically relevant conditions that could compromise surgical suitability and/or subject safety
* Contraindication to magnetic resonance imaging and/or use gadolinium-based contrast agents
* Prior history of brain surgery including, but no limited: DBS, pallidotomy focused ultrasound thalamotomy, or other experimental neurosurgical procedure
* Chronic immunosuppressive therapy

Where this trial is running

Irvine, California and 38 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Parkinson DiseaseGene therapy
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.