Huntington Disease
Conditions
Keywords
Huntington's disease, stem cell, REGEN4HD
Brief summary
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.
Interventions
BIOLOGICALhNSC-01
neural stem cell therapy
Sponsors
Leslie Thompson
California Institute for Regenerative Medicine (CIRM)
University of California, Irvine
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
Please note this may not be a complete list of eligibility criteria. Inclusion Criteria: * Have decision-making capacity and be able to provide written informed consent. * Are between 18 to 65 years of age * Have genetically confirmed diagnosis of Huntington's Disease * Able to undergo MRI scans, tolerate neurosurgical procedure, blood draws and lumbar puncture.
Exclusion criteria
* Are pregnant * Test positive for human immunodeficiency virus (HIV), hepatitis C or chronic hepatitis B at Screening * Have an implanted deep brain stimulation device.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 after 6 and 12 weeks after dosing. | Safety is assessed 6 weeks following each surgery for Cohort A and 12 weeks following cohort B-D completion | To determine the safety and tolerability of hNSC-01s implanted into the striatum of individuals affected with HD. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 after 12 months after dosing. | 12 months | 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. |
| Change in Total Motor Score (TMS) | 12 months | To evaluate change in TMS after 1-year post-implantation. |
| Change in Functional Imaging: FDG-PET | 12 months | To evaluate change in Functional Imaging: FDG-PET at 12 months compared to Baseline |
| Change in structural MRI | 12 months | To evaluate change in structural MRI at 12 months compared to Baseline |
| Change in NfL in CSF. | 12 months | To evaluate change in NfL levels in CSF. |
| Change in NfL in plasma. | 12 months | To evaluate change in NfL levels in plasma. |
| Change in PENK levels in CSF. | 12 months | To evaluate change in PENK levels in CSF. |
| Change in cognition using the MMSE. | 12 months | To evaluate change in cognition using the MMSE. |
| Change in cognition using Stroop Word Reading Test. | 12 months | To evaluate change in cognition using the Stroop Word Reading Test. |
| Change in Total Functional Capacity (TFC) | 12 months | To evaluate change in Total Functional Capacity (TFC) after 1-year post-implantation |
| Change in Problem Behaviors Assessment-short version | 12 months | Assess improvement in Problem Behaviors Assessment - short version (PBA-s) score |
Countries
United States
Contacts
CONTACTUCI Alpha Clinic
Outcome results
None listed