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

NCT07451613 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: ASCC-26-01 [STUDY00000867]
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

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.

Conditions

Huntington Disease

Keywords

Huntington's disease; stem cell; REGEN4HD

Outcome Measures

Primary Outcomes (1)

• 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.

  Safety is assessed 6 weeks following each surgery for Cohort A and 12 weeks following cohort B-D completion

Secondary Outcomes (11)

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

  12 months

• Change in Total Motor Score (TMS)

  12 months

• Change in Functional Imaging: FDG-PET

  12 months

• Change in structural MRI

  12 months

• Change in NfL in CSF.

  12 months

Study Arms (4)

Cohort A

ACTIVE COMPARATOR

Unilateral, low dose

Biological: hNSC-01

Cohort B

ACTIVE COMPARATOR

Bilateral, low dose

Biological: hNSC-01

Cohort C

ACTIVE COMPARATOR

Bilateral, mid-dose

Biological: hNSC-01

Cohort D

ACTIVE COMPARATOR

Bilateral, high dose

Biological: hNSC-01

Interventions

hNSC-01 BIOLOGICAL

neural stem cell therapy

Cohort A; Cohort B; Cohort C; Cohort D

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Leslie Thompson: lead
• California Institute for Regenerative Medicine (CIRM): collaborator
• University of California, Irvine: collaborator

Study Sites (1)

University of California, Irvine

Irvine, California, 92697, United States

RECRUITING

MeSH Terms

Conditions

Huntington Disease

Condition Hierarchy (Ancestors)

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

Central Study Contacts

UCI Alpha Clinic

CONTACT

949-824-3990; alphaclinic@hs.uci.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Bren Professor, Departments of Psychiatry and Human Behavior, Neurobiology and Behavior, and Biological Chemistry, Co-Director, UCI Sue & Bill Gross Research Center

Study Record Dates

• First Submitted: February 8, 2026
• First Posted: March 5, 2026
• Study Start: April 6, 2026
• Primary Completion (Estimated): June 1, 2028
• Study Completion (Estimated): January 1, 2031
• Last Updated: April 13, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF

Locations

US (1)