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Gene Therapy Trial Browser
Clinical Trial Report
Gene Therapy Trial Report
Summary
Study of OXB-102 (AXO-Lenti-PD) in Patients With Bilateral, Idiopathic Parkinson's Disease
NCTID
NCT03720418
(View at clinicaltrials.gov)
Description
This study consists of two parts. Part A will evaluate the safety and tolerability of multiple doses of OXB-102 (AXO-Lenti-PD) in participants with Parkinson's disease. Part B will assess the safety and efficacy of the selected dose of OXB-102 in participants with Parkinson's disease.
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Development Status
Inactive
Indication
Parkinson Disease
Disease Ontology Term
DOID:14330
Compound Name
OXB-102
Compound Alias
AXO-Lenti-PD, ProSavin
Compound Description
EIAV.CMV.TH-CH1.AADC
Sponsor
Sio Gene Therapies
Funder Type
Industry
Recruitment Status
Terminated
Enrollment Count
6
Results Posted
Not Available
Therapy Information
Target Gene/Variant
TH-GCH1.DDC (tricistronic)
Therapy Type
Gene transfer
Therapy Route
In-vivo
Mechanism of Action
Overexpression of protective allele/gene
Route of Administration
Intraparenchymal (putamen)
Drug Product Type
Viral vector
Target Tissue/Cell
Striatal neurons
Delivery System
Viral transduction
Vector Type
EIAV
Editor Type
none
Dose 1
1.9E7 TU
Dose 2
4.0E7 TU
Dose 3
1E8 TU
Dose 4
Dose 5
Study Record Dates
Current Stage
Phase1, Phase2
Submit Date
2018-10-16
Completion Date
2022-04-12
Last Update
2022-05-03
Participation Criteria
Eligible Age
30 Years - 70 Years
Standard Ages
Adult, Older adult
Sexes Eligible for Study
ALL
Locations
No.of Trial Sites
3
Locations
United Kingdom,France
Regulatory Information
Has US IND
False
FDA Designations
Recent Updates
Axovant rebranded as Sio Gene Therapies, the company dissolved in 2023, returned rights to Oxford Biomedica which has shelved the program
Resources/Links
Clinical Publications
Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial
Long-Term Follow-Up of a Phase I/II Study of ProSavin, a Lentiviral Vector Gene Therapy for Parkinson's Disease
News and Press Releases
Oxford Biomedica announces update to agreement with Sio Gene Therapies
Sio Gene Therapies to dissolve after years of setbacks
Preclinical Publications
Gene Therapy for Parkinson's Disease: Preclinical Evaluation of Optimally Configured TH:CH1 Fusion for Maximal Dopamine Synthesis
Multicistronic lentiviral vector-mediated striatal gene transfer of aromatic L-amino acid decarboxylase, tyrosine hydroxylase, and GTP cyclohydrolase I induces sustained transgene expression, dopamine production, and functional improvement in a rat model of Parkinson's disease
Dopamine gene therapy for Parkinson's disease in a nonhuman primate without associated dyskinesia
Optimizing Transgene Configuration and Protein Fusions to Maximize Dopamine Production for the Gene Therapy of Parkinson's Disease
Related NCTID
Phase 1/2: NCT00627588
Long Term Follow-Up: NCT01856439