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

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

Status

Recruiting

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT06285643

Acronym

REGENERATE-PD

Enrollment

127

Registered

2024-02-29

Start date

2024-06-11

Completion date

2028-08-31

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Parkinson Disease

Keywords

Gene therapy

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.

Interventions

DRUGAAV2-GDNF gene therapy

Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose

PROCEDUREcontrol surgery

Bilateral partial burr/twist holes without dural penetration

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender

ALL

Age

45 Years to 75 Years

Healthy volunteers

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 regimen 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

Design outcomes

Primary

Measure	Time frame	Description
Change from baseline to Month 18 on PD Motor Diary	18 months	Normalized Good ON time per Hauser Diary

Countries

Germany, Poland, United Kingdom, United States

Contacts

CONTACTNisha Chhabria, MD

askfirst@askbio.com919-388-1040

CONTACTGayathri Palety, MD