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

Parkinson Disease Clinical Trial

— 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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06285643
• Other study ID #: ASK-PD5-CS201
• Status: Not yet recruiting
• Phase: Phase 2
• Start date: April 1, 2024
• Est. completion date: November 30, 2027

Study information

• Verified date: February 2024
• Source: Asklepios Biopharmaceutical, Inc.
• Contact: Nisha Chhabria, MD
• Phone: 919-388-1040
• Email: askfirst[@]askbio.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 87
• Est. completion date: November 30, 2027
• Est. primary completion date: November 30, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 45 Years to 75 Years

Eligibility

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

Related Conditions & MeSH terms

• Parkinson Disease

Intervention

Drug:
• AAV2-GDNF Gene therapy
Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose

Procedure:
• Control Surgery
Bilateral partial burr/twist holes without dural penetration

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
Asklepios Biopharmaceutical, Inc.	Bayer

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Motor symptoms-MDS-UPDRS parts 2 and 3		18 months
Primary	PD Motor Diary Non-Motor symptoms-MDS-UPDRS part1		18 months
Primary	PDQ39 Levodopa Equivalent Daily Dose		18 months
Secondary	Change from baseline in the MDS-UPDRS parts 2 and 3, and the PD Motor Diary		18 months
Secondary	Change from baseline in the MDS-UPDRS part 1 and PDQ-39		18 months
Secondary	Change in Levodopa equivalent daily dose		18 months