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

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Parkinson Disease
• Intervention / Treatment: Drug: AAV2-GDNF Gene therapy; Procedure: Control Surgery

• Study Type: Interventional
• Enrollment (Estimated): 87
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Nisha Chhabria, MD; Phone Number: 919-388-1040; Email: askfirst@askbio.com
• Study Contact Backup: Name: Christian Urrea, MD; Phone Number: 919-388-1040; Email: askfirst@askbio.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Male and female adults 45-75 years of age inclusive, at the time of signing of informed consent
• Diagnosed with Parkinson's Disease in the past 4-10 years inclusive defined by the following:

Presence of bradykinesia PLUS any of the following:

Rigidity Rest Tremor Postural instability

• Modified Hoehn and Yahr stage II-III in the practically defined OFF medication state (>/= 12 hours from last dose of anti-parkinsonian medications)
• MDS-UPDRS Part III score in the practically defined OFF state of 33 to 60 (inclusive)
• Presence of motor fluctuations with >/= 2.5 hours of absolute time in the OFF state averaged over 3 days as measured by the PD Motor Diary, and assessed at screening, and baseline visit 1 and 2
• Stable anti-parkinsonian medication regimen for >/= 4 weeks prior to Screening and held stable throughout the baseline period o Responsiveness to levodopa as determined by change in modified H&Y stage or MDS-UPDRS Part III score from the practically defined OFF state to ON state after taking first daily dose of PD medication

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Motor symptoms-MDS-UPDRS parts 2 and 3	18 months
PD Motor Diary Non-Motor symptoms-MDS-UPDRS part1	18 months
PDQ39 Levodopa Equivalent Daily Dose	18 months

Secondary Outcome Measures

Outcome Measure	Time Frame
Change from baseline in the MDS-UPDRS parts 2 and 3, and the PD Motor Diary	18 months
Change from baseline in the MDS-UPDRS part 1 and PDQ-39	18 months
Change in Levodopa equivalent daily dose	18 months

Collaborators and Investigators

• Sponsor: Asklepios Biopharmaceutical, Inc.
• Collaborators: Bayer

Study record dates

Study Major Dates

• Study Start (Estimated): April 1, 2024
• Primary Completion (Estimated): November 30, 2027
• Study Completion (Estimated): November 30, 2027

Study Registration Dates

• First Submitted: February 14, 2024
• First Submitted That Met QC Criteria: February 23, 2024
• First Posted (Estimated): February 29, 2024

Study Record Updates

• Last Update Posted (Estimated): February 29, 2024
• Last Update Submitted That Met QC Criteria: February 23, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Keywords: Gene therapy
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Parkinsonian Disorders; Basal Ganglia Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases; Parkinson Disease
• Other Study ID Numbers: ASK-PD5-CS201

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No