Contact SCGE
Your email
Message
Send
SCGE Consortium Home
About SCGE TCDC
Contact Us
License
Home
Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
A Study of AAV2-GDNF in Adults With Moderate Parkinson's Disease (REGENERATE-PD)
NCTID
NCT06285643
(View at clinicaltrials.gov)
Description
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.
(Show More)
Development Status
Active
Indication
Parkinson Disease
Disease Ontology Term
DOID:14330
Compound Name
AB-1005
Compound Description
AAV2-GDNF
Sponsor
AskBio Inc
Funder Type
Industry
Recruitment Status
Recruiting
Enrollment Count
87
Results Posted
Not Available
Therapy Information
Target Gene/Variant
GDNF
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Overexpression of protective allele/gene
Route of Administration
Convection enhanced delivery into the putamen
Drug Product Type
Viral vector
Target Tissue/Cell
Delivery System
Viral transduction
Vector Type
AAV2
Editor Type
none
Dose 1
Up to 1.8mL (3.3E12 vg/ml/gadoteridol 2mM co-infusion)
Dose 2
Dose 3
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase2
Submit Date
2024-02-14
Completion Date
2027-11-30
Last Update
2025-04-18
Participation Criteria
Eligible Age
45 Years - 75 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
32
Locations
United States,Poland,United Kingdom
Regulatory Information
Has US IND
True
FDA Designations
Fast Track
Recent Updates
Received FDA Fast Track designation
Resources/Links
Clinical Publications
Long-term safety of MRI-guided administration of AAV2-GDNF and gadoteridol in the putamen of individuals with Parkinson's disease
Persistent GDNF Expression 45 Months after Putaminal Infusion of AAV2-GDNF in a Patient with Parkinson's Disease
(Abstract) Phase 1b Safety and Preliminary Efficacy of Bilateral Intraputaminal Delivery of AAV2 GDNF (AB-1005) in Participants With Mild or Moderate Parkinson's Disease - AAN Meeting, April 2024
Trial of magnetic resonance-guided putaminal gene therapy for advanced Parkinson's disease
News and Press Releases
AskBio presents 18-month Phase Ib trial results of AB-1005 gene therapy for patients with Parkinson's disease
AskBio receives FDA Fast Track and MHRA Innovation Passport designations for AB-1005 investigational GDNF gene therapy for Parkinson's disease
AskBio Welcomes Brain Neurotherapy Bio to Expand Clinical Pipeline for Neurodegenerative Diseases
Preclinical Publications
Safety Assessment of AAV2-hGDNF Administered Via Intracerebral Injection in Rats for Treatment of Parkinson's Disease
Evaluation of an AAV2-based rapamycin-regulated glial cell line-derived neurotrophic factor (GDNF) expression vector system
Functional effects of AAV2-GDNF on the dopaminergic nigrostriatal pathway in parkinsonian rhesus monkeys
Anterograde axonal transport of AAV2-GDNF in rat basal ganglia
Interventional MRI-guided putaminal delivery of AAV2-GDNF for a planned clinical trial in Parkinson's disease
Intermittent convection-enhanced delivery of GDNF into rhesus monkey putamen: absence of local or cerebellar toxicity
Regeneration of the MPTP-lesioned dopaminergic system after convection-enhanced delivery of AAV2-GDNF
Clinically relevant effects of convection-enhanced delivery of AAV2-GDNF on the dopaminergic nigrostriatal pathway in aged rhesus monkeys
Safety evaluation of AAV2-GDNF gene transfer into the dopaminergic nigrostriatal pathway in aged and parkinsonian rhesus monkeys
Protocol
Clinical Trial Protocol
Related NCTID
Phase 1: NCT04167540
Phase 1: NCT01621581