Safety Study of hNSC-01 for Huntington's Disease

This study is looking at a new treatment called hNSC-01, which are human neural stem cells, for Huntington's disease. This is the first time hNSC-01 is being tested in people. The main goal is to see if hNSC-01 is safe and tolerable (meaning it doesn't cause too many bad side effects). Researchers will be watching for any side effects for 6 to 12 weeks after you receive the treatment. You might be able to join if you are 18 to 65 years old, have a confirmed genetic diagnosis of Huntington's disease, and can undergo MRI scans, surgery, blood draws, and a lumbar puncture (spinal tap). The study plans to enroll 21 participants.

Study design
This is an interventional study, meaning participants will receive a treatment. It plans to enroll 21 participants.
What's involved
You would need to undergo MRI scans, a neurosurgical procedure, blood draws, and a lumbar puncture (spinal tap).
Compensation
Not stated in the trial record.
Follow-up
Safety will be assessed for 6 to 12 weeks after the treatment.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT07451613

Safety and Tolerability Study of Human Neural Stem Cells for Huntington's Disease

Recruiting
PHASE1Ages 18–65InterventionalTreatment
Leslie Thompson
~21 participants
Updated 2026-04-13 on ClinicalTrials.gov
What's tested:hNSC-01

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 after 6 and 12 weeks after dosing.
Measured over Safety is assessed 6 weeks following each surgery for Cohort A and 12 weeks following cohort B-D completion
Huntington Disease

NCT07451613

Where you'd take part

This study runs at 1 site. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • University of California, Irvine

    Irvine, Californiastudy coordinator listed

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

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Eligibility criteria

Inclusion

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

Are pregnant
Test positive for human immunodeficiency virus (HIV), hepatitis C or chronic hepatitis B at Screening
Have an implanted deep brain stimulation device.
  • 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.