A Phase 1/2 interventional study of hNSC-01 in Huntington Disease, sponsored by Leslie Thompson. Recruiting at 1 site in United States. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2026-04-13.
Sponsored by Leslie Thompson · Phase 1/2, Interventional, and Treatment
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.
285 studies on the registry are indexed under Huntington Disease; 49 are open to participants now.
This study's planned enrollment of 21 is below the median of 40 across 203 interventional studies indexed under Huntington Disease.
Browse Huntington Disease studies →This is the only study on the registry with Leslie Thompson as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Please note this may not be a complete list of eligibility criteria.
Inclusion Criteria:
Exclusion Criteria:
Unilateral, low dose
Biological: hNSC-01
Bilateral, low dose
Biological: hNSC-01
Bilateral, mid-dose
Biological: hNSC-01
Bilateral, high dose
Biological: hNSC-01
neural stem cell therapy
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.
Time frame: Safety is assessed 6 weeks following each surgery for Cohort A and 12 weeks following cohort B-D completion
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.
Time frame: 12 months
Change in Total Motor Score (TMS)
To evaluate change in TMS after 1-year post-implantation.
Time frame: 12 months
Change in Functional Imaging: FDG-PET
To evaluate change in Functional Imaging: FDG-PET at 12 months compared to Baseline
Time frame: 12 months
Change in structural MRI
To evaluate change in structural MRI at 12 months compared to Baseline
Time frame: 12 months
Change in NfL in CSF.
To evaluate change in NfL levels in CSF.
Time frame: 12 months
Change in NfL in plasma.
To evaluate change in NfL levels in plasma.
Time frame: 12 months
Change in PENK levels in CSF.
To evaluate change in PENK levels in CSF.
Time frame: 12 months
Change in cognition using the MMSE.
To evaluate change in cognition using the MMSE.
Time frame: 12 months
Change in cognition using Stroop Word Reading Test.
To evaluate change in cognition using the Stroop Word Reading Test.
Time frame: 12 months
Change in Total Functional Capacity (TFC)
To evaluate change in Total Functional Capacity (TFC) after 1-year post-implantation
Time frame: 12 months
Change in Problem Behaviors Assessment-short version
Assess improvement in Problem Behaviors Assessment - short version (PBA-s) score
Time frame: 12 months
Plan to share: Yes
Supporting information: Study protocol, Sap, Icf
No publications or documents are linked to this record.
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