Safety and Tolerability Study of Human Neural Stem Cells for Huntington's Disease (REGEN4HD)

April 6, 2026 updated by: Leslie Thompson

Phase 1B/2A Study of the Safety and Tolerability 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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

21

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • California
      • Irvine, California, United States, 92697
        • Recruiting
        • University of California, Irvine
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: Cohort A
Unilateral, low dose
Biological: hNSC-01
neural stem cell therapy
Active Comparator: Cohort B
Bilateral, low dose
Biological: hNSC-01
neural stem cell therapy
Active Comparator: Cohort C
Bilateral, mid-dose
Biological: hNSC-01
neural stem cell therapy
Active Comparator: Cohort D
Bilateral, high dose
Biological: hNSC-01
neural stem cell therapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
Safety is assessed 6 weeks following each surgery for Cohort A and 12 weeks following cohort B-D completion

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
12 months
Change in Total Motor Score (TMS)
Time Frame: 12 months
To evaluate change in TMS after 1-year post-implantation.
12 months
Change in Functional Imaging: FDG-PET
Time Frame: 12 months
To evaluate change in Functional Imaging: FDG-PET at 12 months compared to Baseline
12 months
Change in structural MRI
Time Frame: 12 months
To evaluate change in structural MRI at 12 months compared to Baseline
12 months
Change in NfL in CSF.
Time Frame: 12 months
To evaluate change in NfL levels in CSF.
12 months
Change in NfL in plasma.
Time Frame: 12 months
To evaluate change in NfL levels in plasma.
12 months
Change in PENK levels in CSF.
Time Frame: 12 months
To evaluate change in PENK levels in CSF.
12 months
Change in cognition using the MMSE.
Time Frame: 12 months
To evaluate change in cognition using the MMSE.
12 months
Change in cognition using Stroop Word Reading Test.
Time Frame: 12 months
To evaluate change in cognition using the Stroop Word Reading Test.
12 months
Change in Total Functional Capacity (TFC)
Time Frame: 12 months
To evaluate change in Total Functional Capacity (TFC) after 1-year post-implantation
12 months
Change in Problem Behaviors Assessment-short version
Time Frame: 12 months
Assess improvement in Problem Behaviors Assessment - short version (PBA-s) score
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Leslie Thompson

Collaborators

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

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 6, 2026

Primary Completion (Estimated)

June 1, 2028

Study Completion (Estimated)

January 1, 2031

Study Registration Dates

First Submitted

February 8, 2026

First Submitted That Met QC Criteria

February 28, 2026

First Posted (Actual)

March 5, 2026

Study Record Updates

Last Update Posted (Actual)

April 13, 2026

Last Update Submitted That Met QC Criteria

April 6, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ASCC-26-01 [STUDY00000867]

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • ICF

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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