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The objective of this randomized, double-blinded, placebo-controlled Phase 1 investigation is to evaluate the safety and potential clinical effect of AAV2-GDNF delivered to the putamen in subjects with either a possible or probable diagnosis of Multiple System Atrophy.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Active Treatment | Experimental |
| |
| Placebo Surgery | Sham Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AAV2-GDNF gene therapy | Biological | Bilateral image-guided infusion of AAV2-GDNF into putamen, single dose |
|
| Measure | Description | Time Frame |
|---|---|---|
| The incidence of Treatment-Emergent Adverse Events (TEAE) and Serious Adverse Events (SAE) assessed clinically by physical and neurological examinations | Number of TEAE and SAE's reported post-treatment. | 3 years |
| Measure | Description | Time Frame |
|---|---|---|
| MSA symptoms/signs as assessed by the Unified Multiple System Atrophy Rating Scale (UMSARS) | Change from baseline in the Unified Multiple System Atrophy Rating Scale (UMSARS) and compared to placebo. UMSARS total scores range from 0-104 points with higher scores indicating greater severity of impairment. | 12 months |
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Inclusion Criteria:
Exclusion Criteria:
NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California Irvine | Irvine | California | 92697 | United States | ||
| Parkinson's Disease and Movement Disorders Center of Boca Raton |
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| Label | URL |
|---|---|
| Nurse Navigator Service for Potential Participants | View source |
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| ID | Term |
|---|---|
| D019578 | Multiple System Atrophy |
| ID | Term |
|---|---|
| D054969 | Primary Dysautonomias |
| D001342 | Autonomic Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D001480 | Basal Ganglia Diseases |
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| ID | Term |
|---|---|
| C005703 | salicylhydroxamic acid |
| D013514 | Surgical Procedures, Operative |
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Up to 9 study participants meeting eligibility criteria will be randomized in a 2:1 fashion to receive either the investigational medicinal product or sham surgery in this Phase 1 trial.
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| Sham (Placebo) Surgery | Procedure | Bilateral partial burr/twist holes without dural penetration |
|
| Change in striatal dopamine transporter binding as measured by [123-I] Ioflupane |
Percent and absolute change in ratio of specific to non-specific binding of 123I FP-CIT to DaT from baseline and compared to placebo by Single Photon Emission Computed Tomography (SPECT) dopamine transporter (DaT) imaging |
| 12 months |
| Change in the quality of life as measured by Multiple System Atrophy Quality of Life (MSA-QoL) | Change from baseline and compared to placebo in the Multiple System Atrophy Quality of Life (MSA-QoL) scale. MSA-QoL is a self-reported questionnaire that measures MSA impact in day to day activities. Scale consists of 40 items with a five response option format (0 - no problem to 4 extreme problem) and a "not applicable" response option. | 12 months |
| Boca Raton |
| Florida |
| 33486 |
| United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Quest Research Institute | Farmington Hills | Michigan | 48334 | United States |
| NYU Langone Health | New York | New York | 10016 | United States |
| The Ohio State University Medical Center | Columbus | Ohio | 43210 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | United States |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009069 | Movement Disorders |
| D000080874 | Synucleinopathies |
| D019636 | Neurodegenerative Diseases |