Clinical trial NCT06285643: A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD)
Official title: A Phase 2, Randomized, Double-blind, Sham Surgery-controlled Study of the Efficacy and Safety of Intraputaminal AAV2-GDNF in the Treatment of Adults With Moderate Stage Parkinson's Disease
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Parkinson Disease
Keywords: Gene therapy
Lead sponsor: AskBio Inc
Sponsor class: INDUSTRY
Collaborators: name: Bayer
First posted: 2024-02-29
Enrollment: 87
Sex: ALL
Minimum age (years): 45
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


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

Primary outcomes:
measure: Change from baseline to Month 18 on PD Motor Diary
timeFrame: 18 months
Secondary outcomes:
Other outcomes:
Locations:
city: Irvine
country: United States
facility: UCI Alpha Clinic (Neurology)
state: California
city: Loma Linda
country: United States
facility: Loma Linda University (Neurology)
state: California
city: Loma Linda
country: United States
facility: Loma Linda University (Surgical)
state: California
city: San Francisco
country: United States
facility: University of California San Francisco (Neurology)
state: California
city: San Francisco
country: United States
facility: University of San Francisco (Surgical)
state: California
city: Englewood
country: United States
facility: CenExel Rocky Mountain Clinical Research (Neurology)
state: Colorado
city: Washington D.C.
country: United States
facility: Georgetown University (Neurology)
state: District of Columbia
city: Boca Raton
country: United States
facility: Parkinson's Disease and Movement Disorders Center of Boca Raton (Neurology)
state: Florida
city: Port Charlotte
country: United States
facility: Parkinson's Disease Treatment Center of Southwest Florida (Neurology)
state: Florida
city: Atlanta
country: United States
facility: Emory University (Surgical)
state: Georgia
city: Chicago
country: United States
facility: Rush University Medical Center (Neurology)
state: Illinois
city: Kansas City
country: United States
facility: University of Kansas Medical Center (Neurology)
state: Kansas
city: Boston
country: United States
facility: Massachusetts General Hospital (Neurology)
state: Massachusetts
city: Boston
country: United States
facility: Massachusetts General Hospital (Surgical)
state: Massachusetts
city: East Lansing
country: United States
facility: Michigan State University (Neurology)
state: Michigan
city: Farmington Hills
country: United States
facility: Quest Research Institute (Neurology)
state: Michigan
city: Las Vegas
country: United States
facility: Cleveland Clinic Lou Ruvo (Neurology)
state: Nevada
city: Chapel Hill
country: United States
facility: University of North Carolina (Neurology)
state: North Carolina
city: Durham
country: United States
facility: Duke Neurosurgery (Surgical)
state: North Carolina
city: Durham
country: United States
facility: Duke University (Neurology)
state: North Carolina
city: Columbus
country: United States
facility: Ohio State University (Surgical)
state: Ohio
city: Philadelphia
country: United States
facility: University of Pennsylvania (Neurology)
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt University (Neurology)
state: Tennessee
city: Houston
country: United States
facility: Houston Methodist (Neurology)
state: Texas
city: Houston
country: United States
facility: Houston Methodist (Surgical)
state: Texas
city: Kirkland
country: United States
facility: EvergreenHealth Research Department (Neurology)
state: Washington
city: Madison
country: United States
facility: University of Wisconsin (Neurology)
state: Wisconsin
city: Berlin
country: Germany
facility: Charité - Universitätsmedizin Berlin (Surgical)
city: Marburg
country: Germany
facility: Philipps-Universität Marburg (Neurology)
city: Tübingen
country: Germany
facility: Universitätsklinikum Tübingen (Neurology)
city: Tübingen
country: Germany
facility: Universitätsklinikum Tübingen (Surgical)
city: Würzburg
country: Germany
facility: Universitätsklinikum Würzburg (Neurology)
city: Gdańsk
country: Poland
facility: Copernicus Podmiot Leczniczy, Szpital św. Wojciecha (Neurology)
city: Katowice
country: Poland
facility: Specjalistyczna Praktyka Lekarska Prof. Grzegorz Opala (Neurology)
city: Warszawa
country: Poland
facility: Mazowiecki Szpital Bródnowski (Neurology)
city: Warszawa
country: Poland
facility: Mazowiecki Szpital Bródnowski (Surgical)
city: Warszawa
country: Poland
facility: Samodzielny Publiczny Szpital Kliniczny im. Prof. W. Orłowskiego CMKP (Neurology)
city: Bristol
country: United Kingdom
facility: North Bristol NHS Trust - Southmead Hospital (Neurology)
city: Cardiff
country: United Kingdom
facility: University Hospital of Wales (Surgical)
Study officials:
References: