AAV2-hAADC for Parkinson's Disease (PDCS-01)
Trial statusNot yet recruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-75
SponsorKrzysztof Bankiewicz
About this trial
This study is looking at whether a gene therapy called AAV2-hAADC is safe and may help people with Parkinson's Disease.
AAV2-hAADC is intended to increase the levels of dopamine in your brain. It contains a virus called adeno-associated virus 2 (AAV2) that has been modified to carry the genetic code for an enzyme called Aromatic L-Amino Acid Decarboxylase, or AADC for short.
In this study, AAV2-hAADC is delivered to two parts of the brain called the putamen and the caudate. Increasing the amount of AADC gene in these parts of the brain converts more levodopa into dopamine, the chemical that is lacking in PD.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Male and female adults 18-75 years of age (inclusive), at the time of signing the informed consent.
Disease duration since diagnosis of >3 years.
Patient reported symptom onset prior to the age of 50 (inclusive).
Modified Hoehn & Yahr Staging ≥2.5 in the practically defined OFF medication state (≥ 12 hours from last dose of anti-parkinsonian medications)
Disqualifiers
Atypical or secondary parkinsonism, including but not limited to symptoms resulting from trauma, brain tumor, infection, cerebrovascular disease, other neurological disease, or to drugs, chemicals, or toxins, as determined by the Investigator.
Presence of clinically significant cognitive impairment as defined by a Montreal Cognitive Assessment (MoCA) score of less than 241 at screening and/or clinical diagnosis of dementia by Investigator based on MoCA diagnostic criteria.
Presence or history of psychosis, except for minor psychosis.
Presence of severe depression, as indicated by a BDI-II score >28 within 5 years of screening evaluation.
Trial design
Design model
Sequential
Treatments tested in this trial
AAV2-hAADC
DrugThe purpose of this study is to investigate AAV2-hAADC gene therapy (the study drug) for the treatment of moderately-advanced PD with motor fluctuations. AAV2-hAADC gene therapy works by using a modified virus called adeno-associated virus 2 (AAV2). This virus is naturally-occurring and not associated with any disease. The virus' DNA has been changed so that it can deliver the human enzyme called Aromatic L-Amino Acid Decarboxylase (AADC). This enzyme converts levodopa into dopamine. AAV2-hAADC can enter brain cells and transfer the copy of the AADC gene directly into these cells. Once the gene is inside the brain cells, those cells will make AADC. By increasing the levels of AADC, we aim to increase the levels of dopamine and improve the symptoms of PD. AAV2-hAADC will be delivered through a brain surgery to two parts of the brain called the putamen and the caudate. These parts of the brain are involved in PD. The goal of this study is to assess whether infusing AAV2-h
Treatment groups
9 Participants
are divided into 2 treatment groupsGroup A: Dose 1 (Low Dose)Experimental treatment 1 intervention
Group B: Dose 2 (High Dose)Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Adverse Events
Assessment of AEs or SAEs and their relationship to the study procedure and study drug (graded as definite, probable, possible, unlikely, or unrelated).
Time frame
Through 3 year Follow-up2
MRI findings
Intraoperative and post-operative MRI findings
Time frame
Through Month 6 Follow-up3
Physical Exam and labs
Changes from baseline in physical examination findings and routine clinical laboratory analysis (hematology, clinical chemistries, immunologic assessments).
Time frame
Through 3 year Follow-up4
C-SSRS
Changes in Columbia-Suicide Severity Rating Scale (C-SSRS) results
Time frame
Through 3 year Follow-upSponsors and contacts
Click on the lead sponsor to view all of their trials.
Krzysztof Bankiewicz
Lead sponsor
Ohio State University
Sponsor institution
Source ClinicalTrials.gov
This trial is not recruiting at the moment. You can still explore other options: