Gene therapy for adults with moderate Parkinson's Disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• UCI Alpha Clinic (Neurology)
  Irvine, California, United States Recruiting
• Loma Linda University (Neurology)
  Loma Linda, California, United States Recruiting
• University of California San Francisco (Neurology)
  San Francisco, California, United States Recruiting
• CenExel Rocky Mountain Clinical Research (Neurology)
  Englewood, Colorado, United States Recruiting
• Georgetown University (Neurology)
  Washington D.C., District of Columbia, United States Recruiting
• Parkinson's Disease and Movement Disorders Center of Boca Raton (Neurology)
  Boca Raton, Florida, United States Recruiting

Study contacts

• Study coordinator: Nisha Chhabria, MD
• Email: askfirst@askbio.com
• Phone: 919-388-1040

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.