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

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

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.