A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD)
Purpose
The objective of this randomized, surgically controlled, double-blinded, Phase 2 study is to evaluate the safety and efficacy of AAV2-GDNF delivered to the putamen in subjects with moderate Parkinson's Disease.
Condition
- Parkinson Disease
Eligibility
- Eligible Ages
- Between 45 Years and 75 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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
Study Design
- Phase
- Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Arm Groups
Arm | Description | Assigned Intervention |
---|---|---|
Experimental AAV2-GDNF |
|
|
Sham Comparator Control Surgery |
|
Recruiting Locations
Kansas City, Kansas 66103
More Details
- Status
- Recruiting
- Sponsor
- Asklepios Biopharmaceutical, Inc.