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| Name | Class |
|---|---|
| California Institute for Regenerative Medicine (CIRM) | OTHER |
| University of California, Irvine | OTHER |
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The purpose of this research study is to determine whether an implantation of hNSC-01 is a safe and tolerable study intervention for Huntington's disease. This study is the first time that hNSC-01 is being tested in people.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort A | Active Comparator | Unilateral, low dose |
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| Cohort B | Active Comparator | Bilateral, low dose |
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| Cohort C | Active Comparator | Bilateral, mid-dose |
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| Cohort D | Active Comparator | Bilateral, high dose |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| hNSC-01 | Biological | neural stem cell therapy |
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| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 after 6 and 12 weeks after dosing. | To determine the safety and tolerability of hNSC-01s implanted into the striatum of individuals affected with HD. | Safety is assessed 6 weeks following each surgery for Cohort A and 12 weeks following cohort B-D completion |
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 after 12 months after dosing. | Longer term (12 month) safety of implantation with absence of development of clinically significant inflammatory and/or immune reaction, and adverse events related to clinical safety laboratory tests, vital signs, ECGs, neurological and physical examinations. | 12 months |
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Please note this may not be a complete list of eligibility criteria.
Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| UCI Alpha Clinic | Contact | 949-824-3990 | alphaclinic@hs.uci.edu |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California, Irvine | Recruiting | Irvine | California | 92697 | United States |
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| ID | Term |
|---|---|
| D006816 | Huntington Disease |
| ID | Term |
|---|---|
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
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| Change in Total Motor Score (TMS) | To evaluate change in TMS after 1-year post-implantation. | 12 months |
| Change in Functional Imaging: FDG-PET | To evaluate change in Functional Imaging: FDG-PET at 12 months compared to Baseline | 12 months |
| Change in structural MRI | To evaluate change in structural MRI at 12 months compared to Baseline | 12 months |
| Change in NfL in CSF. | To evaluate change in NfL levels in CSF. | 12 months |
| Change in NfL in plasma. | To evaluate change in NfL levels in plasma. | 12 months |
| Change in PENK levels in CSF. | To evaluate change in PENK levels in CSF. | 12 months |
| Change in cognition using the MMSE. | To evaluate change in cognition using the MMSE. | 12 months |
| Change in cognition using Stroop Word Reading Test. | To evaluate change in cognition using the Stroop Word Reading Test. | 12 months |
| Change in Total Functional Capacity (TFC) | To evaluate change in Total Functional Capacity (TFC) after 1-year post-implantation | 12 months |
| Change in Problem Behaviors Assessment-short version | Assess improvement in Problem Behaviors Assessment - short version (PBA-s) score | 12 months |
| D003704 | Dementia |
| D002819 | Chorea |
| D020820 | Dyskinesias |
| D009069 | Movement Disorders |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D003072 | Cognition Disorders |
| D019965 | Neurocognitive Disorders |
| D001523 | Mental Disorders |