Patients with Parkinson's Disease with motor fluctuations. MedDRA version: 20.0 Level: PT Classification code 10061536 Term: Parkinson's disease System Organ Class: 10029205 - Nervous system disorders
Conditions
Interventions
Product Name: VY-AADC02
Product Code: VY-AADC02
Pharmaceutical Form: Solution for injection/infusion
INN or Proposed INN: Pending
CAS Number: 2226647-27-2
Current Sponsor code: VY-AADC02
Other descrip
Sponsors
Voyager Therapeutics, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.- Males and females, 40 to 75 years of age (inclusive). 2.- Diagnosis of PD, consistent with United Kingdom Brain Bank Criteria. 3.- Unequivocal responsiveness to dopaminergic therapy, as judged by the Investigator. 4.- Disease duration from diagnosis of =4 years. 5.- In the judgment of the Investigator, a stable, optimal regimen of Parkinson's medications for at least 4 weeks prior to screening evaluation. 6.- Stable cognitive and psychological function based on screening evaluations. 7.- In the judgment of the Investigator, stable Parkinson's features and symptoms for at least 4 weeks prior to screening evaluation. 8.- Agrees to defer any neurological surgery, including deep brain stimulation, other invasive treatments for PD including duodopa, or the addition of new dopaminergic formulations until after completing the 12-month study visit. 9.- Ability to travel to study visits. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 38 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 4
Exclusion criteria
Exclusion criteria: 1.- Atypical or secondary parkinsonism, including but not limited to symptoms believed to be due to trauma, brain tumor, infection, cerebrovascular disease, other neurological disease, or to drugs, chemicals, or toxins, as determined by the Investigator. 2.- MoCA score <26. 3.- Use of tetrahydrocannabinol within 6 months of screening evaluation. 4.- Brain imaging abnormalities in the striatum or other regions that would substantially increase risk of surgery. 5.- Contraindication to MRI and/or gadolinium-based contrast agents. 6.- Prior brain surgery or infusion therapies that could complicate the study procedure or negatively impact study evaluations as determined from participant interview, screening MRI, or medical records. 7.- History of malignancy other than treated carcinoma in situ within 3 years of screening evaluation. 8.- Prior gene transfer, current treatment with any investigational agent (drug or device) within 2 months of screening evaluation, or participation or plans to participate in another research study. 9.- Ongoing treatments or planned treatments that might interfere with interpretation of the study outcome including deep brain stimulation.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the distribution, efficacy, and safety of VY-AADC02 in Patients with Parkinson's Disease with Motor Fluctuations.;Secondary Objective: Not applicable.;Primary end point(s): 1.Change in Patient Rated Motor Fluctuations in the VY-AADC02 group compared to placebo surgery group. 2.Percent coverage within the putamen at time of administration of VY-AADC02. 3.Change in AADC enzyme activity (Distribution). 4.Safety of VY-AADC02 as measured by number of treatment emergent adverse events (TEAEs) and Serious Adverse Events (SAEs). 5.Safety of VY-AADC02 as measured by changes in vital signs. 6.Safety of VY-AADC02 as measured by physical examinations and routine clinical laboratory analysis (hematology and clinical chemistry). 7.Safety of VY-AADC02 as measured by changes in findings on brain images. 8.Safety of VY-AADC02 as measured by the Columbia-Suicide Severity Rating Scale (C-SSRS). 9.Safety of VY-AADC02 based on change in impulse control disorders. ;Timepoint(s) of evaluation of this end point: 1.- Change in Patient Rated Motor Fluctuations in the VY-AADC02 group compared to placebo surgery group will be assessed from Baseline to 12 months post op. 2.- Percent coverage within the putamen at time of administration of VY-AADC02 will be determined on the Day of Surgery. 3.- Change in AADC enzyme activity (Distribution) will be evaluated on study 45 and at 12 months post op. 4.Safety assessments will be measured from time of Informed Consent to 12 months post op + 30 day Follow Up. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1.- Change in activities of daily living in the VY-AADC02 group compared to placebo surgery group. 2.- Change in PD related quality of life in the VY-AADC02 group compared to placebo surgery group. 3.- Change from baseline in time course response to levodopa in the VY-AADC02 group compared to placebo surgery group measured by the area under the curve (AUC) of repeated UPDRS III scores following a single dose of oral levodopa. 4.- Change in global function in the VY-AADC02 group compared to placebo surgery group. 5.- Change in overall non-motor symptoms in the VY-AADC02 group compared to placebo surgery group. ;Timepoint(s) of evaluation of this end point: Baseline to 12 months post-op. | — |
Countries
Poland, United States
Contacts
Public Contactclinicaltrials@vygr.com
Voyager Therapeutics, Inc.
Outcome results
None listed