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NCT Number: NCT07451613

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

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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Key information

Conditions

Huntington Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Chorea Cognition Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dementia Dyskinesias Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Mental Disorders Movement Disorders Nervous System Diseases Neurocognitive Disorders Neurodegenerative Diseases

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

University of California, Irvine

Irvine, California, 92697, United States

Location status: Recruiting

Location contact

UCI Alpha Clinic

CONTACT

[email protected]

949-824-3990

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

hNSC-01

Biological

neural stem cell therapy

Primary outcomes

  1. Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 after 6 and 12 weeks after dosing.

    Time frame: 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 outcomes

  1. Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 after 12 months after dosing.

    Time frame: 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.

  2. Change in Total Motor Score (TMS)

    Time frame: 12 months

    To evaluate change in TMS after 1-year post-implantation.

  3. Change in Functional Imaging: FDG-PET

    Time frame: 12 months

    To evaluate change in Functional Imaging: FDG-PET at 12 months compared to Baseline

  4. Change in structural MRI

    Time frame: 12 months

    To evaluate change in structural MRI at 12 months compared to Baseline

  5. Change in NfL in CSF.

    Time frame: 12 months

    To evaluate change in NfL levels in CSF.

  6. Change in NfL in plasma.

    Time frame: 12 months

    To evaluate change in NfL levels in plasma.

  7. Change in PENK levels in CSF.

    Time frame: 12 months

    To evaluate change in PENK levels in CSF.

  8. Change in cognition using the MMSE.

    Time frame: 12 months

    To evaluate change in cognition using the MMSE.

  9. Change in cognition using Stroop Word Reading Test.

    Time frame: 12 months

    To evaluate change in cognition using the Stroop Word Reading Test.

  10. Change in Total Functional Capacity (TFC)

    Time frame: 12 months

    To evaluate change in Total Functional Capacity (TFC) after 1-year post-implantation

  11. Change in Problem Behaviors Assessment-short version

    Time frame: 12 months

    Assess improvement in Problem Behaviors Assessment - short version (PBA-s) score

Study contacts

Contact information is provided by the study sponsor or research team.

UCI Alpha Clinic

CONTACT

[email protected]

949-824-3990

Sponsors and collaborators

Lead sponsor

Leslie Thompson

Other

Collaborators

  • California Institute for Regenerative Medicine (CIRM)
  • University of California, Irvine

Registry information

Official study title

Phase 1B/2A Study of the Safety and Tolerability of Human Neural Stem Cells for Huntington's Disease

Acronym: REGEN4HD

Important dates

Study start
2026
Primary completion
2028
Study completion
2031
First posted
Mar 5, 2026
Registry last updated
Apr 13, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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