A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD)

Launched by ASKBIO INC · Feb 23, 2024

Keywords

ClinConnect Summary

This clinical trial, called REGENERATE-PD, is studying a new treatment for adults with moderate Parkinson's Disease, specifically looking at a gene therapy called AAV2-GDNF. The goal is to see if this treatment is safe and effective when delivered directly to a part of the brain involved in movement, known as the putamen. The trial is currently recruiting participants aged 45 to 75 who have been diagnosed with Parkinson's Disease within the last 4 to 10 years and are already on stable medication for their symptoms.

To be eligible for this study, participants must have specific symptoms of Parkinson's, such as slow movement and tremors, and they need to respond well to a common Parkinson's medication called levodopa. Those who join the study will receive either the gene therapy or a placebo (a treatment that looks like the real thing but has no active ingredients) and will be monitored closely to see how they respond. It's important to know that individuals with certain medical conditions, past brain surgeries, or severe mental health issues may not be able to participate for safety reasons.

Gender

ALL

Eligibility 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 regiment 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