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

ArmDescriptionAssigned Intervention
Experimental
AAV2-GDNF
  • Drug: AAV2-GDNF Gene therapy
    Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose
    Other names:
    • Biological
Sham Comparator
Control Surgery
  • Procedure: Control Surgery
    Bilateral partial burr/twist holes without dural penetration

Recruiting Locations

University of Kansas Medical Center
Kansas City, Kansas 66103
Contact:
Sarina Fay
sfay2@kumc.edu

More Details

Status
Recruiting
Sponsor
Asklepios Biopharmaceutical, Inc.

Study Contact

Nisha Chhabria, MD
919-388-1040
askfirst@askbio.com