Recruiting
Phase 1
Phase 2

hNSC-01

Sponsor:

Leslie Thompson

Code:

NCT07451613

Conditions

Huntington Disease

Eligibility Criteria

Sex: All

Age: 18 - 65

Healthy Volunteers: Not accepted

Interventions

hNSC-01

Study Details

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

Study ID

NCT07451613

Start date

Apr 6, 2026

Status verified date

Feb, 2026

Completion date

Jan, 2031

Anticipated

Primary completion date

Jun, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 65

Healthy Volunteers: Not accepted

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 Design

Enrollment

21 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

active comparator: Cohort A

Unilateral, low dose

active comparator: Cohort B

Bilateral, low dose

active comparator: Cohort C

Bilateral, mid-dose

active comparator: Cohort D

Bilateral, high dose

Interventions

hNSC-01

neural stem cell therapy

Primary outcome measure

  • 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 ]

Central Contacts and Locations

Central contacts

UCI Alpha Clinic

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

Locations

University of California, Irvine

Recruiting

Irvine, California, United States, 92697

Contacts

UCI Alpha Clinic

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

More Information

Sponsor

Leslie Thompson

Last update posted

Apr 13, 2026

Last verified

Feb, 2026

Keywords

  • Huntington's disease
  • stem cell
  • REGEN4HD

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Leslie Thompson on 2026-04-13.