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| Name | Class |
|---|---|
| AskBio Inc | INDUSTRY |
| California Institute for Regenerative Medicine (CIRM) | OTHER |
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The objective of this Phase 1b investigation is to evaluate the safety and potential clinical effect of AAV2-GDNF delivered to the putamen in subjects with either a recent or a long-standing diagnosis of PD.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Earlier stage PD | Experimental |
| |
| Later stage PD | Experimental |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AAV2-GDNF | Biological | Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose |
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| Measure | Description | Time Frame |
|---|---|---|
| The incidence of Treatment-Emergent Adverse Events (TEAE) assessed clinically by physical and neurological examinations | Evaluation of the safety and tolerability through the assessment of incidence of TEAE, identified by MedDRA preferred term and grouped by MedDRA System Organ Class, as well as clinically meaningful changes in clinical exams or laboratory assays. | 5 years |
| Measure | Description | Time Frame |
|---|---|---|
| Motor symptoms as assessed by the Movement Disorder Society's Unified Parkinson's Disease Rating Scale (MDS-UPDRS) | Change from baseline in the MDS-UPDRS. The MDS-UPDRS contains 4 subscales: Part I, non-motor aspects of experiences of daily living (13 items); Part II, motor aspects of experiences of daily living (13 items); Part III, motor examination (33 scores based on 18 items); Part IV, motor complications (6 items). The rating for each item, or sub-item, is from 0 (normal) to 4 (severe). The total score for each Part is obtained from the sum of the corresponding item scores. |
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Key Inclusion Criteria:
Male and female adults 35-75 years of age (inclusive)
Diagnosed with Parkinson's disease
Modified Hoehn and Yahr stage I-III OFF medication
Time since receiving a clinical diagnosis of PD and disease severity consistent with one of the following:
Responsiveness to levodopa
Key Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California Irvine | Irvine | California | 92697 | United States | ||
| University of California San Francisco |
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| ID | Term |
|---|---|
| D010300 | Parkinson Disease |
| ID | Term |
|---|---|
| D020734 | Parkinsonian Disorders |
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
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A total of 12 study participants will be administered the investigational product in this Phase 1b trial. Participants will be enrolled into cohorts, based upon the duration and stage of their PD. Six (6) participants will be dosed in each cohort.
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| 18 months |
| Non-motor symptoms of Parkinson's disease as assessed by the Non-Motor Symptom Scale (NMSS) | Change from baseline in the NMSS. The NMSS evaluates 9 domains of non-motor of severity and frequency of PD symptoms associated with cardiovascular health, sleep and fatigue, mood and cognition, perceptual problems and hallucinations, attention and memory, gastrointestinal tract, urinary, sexual function, and a miscellaneous domain for other common non-motor conditions. Severity x frequency scores range 0-108, with 0 being less severe and less frequent. | 18 months |
| Brain dopaminergic cell integrity as measured by DaTscan | Percentage and absolute changes in Ioflupane retention as a marker for dopamine transporter protein expressed by dopamine producing cells within the brain. Measured by quantitative analysis of DaTscan SPECT imaging. | 18 months |
| San Francisco |
| California |
| 94103 |
| United States |
| The Ohio State University Medical Center | Columbus | Ohio | 43210 | United States |
| D009422 | Nervous System Diseases |
| D009069 | Movement Disorders |
| D000080874 | Synucleinopathies |
| D019636 | Neurodegenerative Diseases |