Brain stem cells implanted in first human trial for Huntington's
NCT ID NCT07451613
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial tests whether implanting human neural stem cells into the brain is safe for people with Huntington's disease. Twenty-one adults with confirmed Huntington's will receive the stem cell therapy and be monitored for side effects over 12 weeks and up to a year. It is the first time this treatment is being studied in humans.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- neural stem cells (hNSC-01)
- What this could lead to
- If safe and effective, this could point toward a treatment that slows or controls Huntington's disease progression.
- What could go wrong
- This is the first human test of this stem cell therapy, so safety and effectiveness are unknown. The trial is small (21 people) and early-stage, so results may not apply broadly.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 21 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Apr 2026
- Expected to finish
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Jan 2031
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 to 65 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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University of California, Irvine
RECRUITINGIrvine, California, 92697, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Can a genetic 'Patch' fix Huntington's disease? lab test aims to find out
- Could a brain disease change behavior years before diagnosis?
- Scientists decode gait signatures to spot brain diseases
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