Huntington DiseasePhase 1/2REGEN4HDNCT07451613
Safety and Tolerability Study of Human Neural Stem Cells for Huntington's Disease
A Phase 1/2 study testing hNSC-01 in people with Huntington Disease, run by Leslie Thompson at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Huntington Disease
- What is being tested?
- hNSC-01
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Apr 2026to Jan 2031
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- Are pregnant
- Test positive for human immunodeficiency virus (HIV), hepatitis C or chronic hepatitis B at Screening
- Have an implanted deep brain stimulation device.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
University of California, Irvine
Irvine, California, 92697
UCI Alpha Clinic, 949-824-3990, alphaclinic@hs.uci.edu
About this study
Official title: 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.
- Conditions
- Huntington Disease
- Being tested
- hNSC-01
- Keywords
- Huntington's disease, stem cell, REGEN4HD
- Medical subject headings
- Huntington Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
hNSC-01Biological
neural stem cell therapy
| Study group | Role | What they receive |
|---|---|---|
| Cohort A | Active comparator | Unilateral, low dose Receives: Biological: hNSC-01 |
| Cohort B | Active comparator | Bilateral, low dose Receives: Biological: hNSC-01 |
| Cohort C | Active comparator | Bilateral, mid-dose Receives: Biological: hNSC-01 |
| Cohort D | Active comparator | Bilateral, high dose Receives: Biological: hNSC-01 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
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.
Measured: 12 months
Change in Total Motor Score (TMS)
To evaluate change in TMS after 1-year post-implantation.
Measured: 12 months
Change in Functional Imaging: FDG-PET
To evaluate change in Functional Imaging: FDG-PET at 12 months compared to Baseline
Measured: 12 months
Change in structural MRI
To evaluate change in structural MRI at 12 months compared to Baseline
Measured: 12 months
Change in NfL in CSF.
To evaluate change in NfL levels in CSF.
Measured: 12 months
Change in NfL in plasma.
To evaluate change in NfL levels in plasma.
Measured: 12 months
Change in PENK levels in CSF.
To evaluate change in PENK levels in CSF.
Measured: 12 months
Change in cognition using the MMSE.
To evaluate change in cognition using the MMSE.
Measured: 12 months
Change in cognition using Stroop Word Reading Test.
To evaluate change in cognition using the Stroop Word Reading Test.
Measured: 12 months
Change in Total Functional Capacity (TFC)
To evaluate change in Total Functional Capacity (TFC) after 1-year post-implantation
Measured: 12 months
Change in Problem Behaviors Assessment-short version
Assess improvement in Problem Behaviors Assessment - short version (PBA-s) score
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Mar 2026
- Study start6 Apr 2026
- Primary completionJun 2028 (estimated)
- Study completionJan 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Huntington DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 21 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Leslie Thompson
- Sponsor type
- University, hospital or other
- Collaborators
- California Institute for Regenerative Medicine (CIRM), University of California, Irvine
- Responsible party
- Sponsor-investigator, Leslie Thompson, University of California, Irvine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07451613
- Sponsor's protocol ID
- ASCC-26-01 [STUDY00000867]
- Acronym
- REGEN4HD
- Status last verified
- Feb 2026
- First submitted
- 8 Feb 2026
- Last updated
- 13 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 13 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1B/2A Study of the Safety and Tolerability of Human Neural Stem Cells for Huntington's Disease
- ClinicalTrials.gov ID
- NCT07451613
- Acronym / sponsor ID
- REGEN4HD / ASCC-26-01 [STUDY00000867]
- Conditions
- Huntington Disease
- Keywords
- Huntington's disease, stem cell, REGEN4HD
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 21 participants (estimated)
- Lead sponsor
- Leslie ThompsonUniversity, hospital or other
- Collaborators
- California Institute for Regenerative Medicine (CIRM), University of California, Irvine
- Study start
- 6 Apr 2026 (actual)
- Primary completion
- Jun 2028 (estimated)
- Study completion
- Jan 2031 (estimated)
- First posted
- 5 Mar 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort A | Active comparator | Biological: hNSC-01Unilateral, low dose |
| Cohort B | Active comparator | Biological: hNSC-01Bilateral, low dose |
| Cohort C | Active comparator | Biological: hNSC-01Bilateral, mid-dose |
| Cohort D | Active comparator | Biological: hNSC-01Bilateral, high dose |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (3 listed)
- Are pregnant
- Test positive for human immunodeficiency virus (HIV), hepatitis C or chronic hepatitis B at Screening
- Have an implanted deep brain stimulation device.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| hNSC-01neural stem cell therapy | Biological | Cohort A, Cohort B, Cohort C, Cohort D |
Primary outcome
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.
11 secondary outcomes
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 after 12 months after dosing.
Time frame: 12 months
Change in Total Motor Score (TMS)
Time frame: 12 months
Change in Functional Imaging: FDG-PET
Time frame: 12 months
Change in structural MRI
Time frame: 12 months
Change in NfL in CSF.
Time frame: 12 months
Change in NfL in plasma.
Time frame: 12 months
Change in PENK levels in CSF.
Time frame: 12 months
Change in cognition using the MMSE.
Time frame: 12 months
Change in cognition using Stroop Word Reading Test.
Time frame: 12 months
Change in Total Functional Capacity (TFC)
Time frame: 12 months
Change in Problem Behaviors Assessment-short version
Time frame: 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Apr 2026.
- Individual participant dataSharing planned: Yes
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing hNSC-01 in people with Huntington Disease, run by Leslie Thompson at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 13 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact UCI Alpha Clinic (949-824-3990, alphaclinic@hs.uci.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the 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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Apr 2026.