Safety and tolerability of human neural stem cell implants for Huntington's disease
Phase 1B/2A Study of the Safety and Tolerability of Human Neural Stem Cells for Huntington's Disease
This research will test whether an implant of hNSC-01 human neural stem cells is safe and tolerable for adults with Huntington's disease.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 21 (estimated) |
| Ages | 18 Years to 65 Years |
| Sex | All |
| Sponsor | University of California, Irvine Academic / other |
| Locations | 1 site (Irvine, California) |
| Trial ID | NCT07451613 on ClinicalTrials.gov |
What this trial studies
This Phase 1/2, first-in-human interventional program implants hNSC-01 neural stem cells into the brains of eligible adults with genetically confirmed Huntington's disease to monitor safety and tolerability. Participants undergo neurosurgical implantation at a single center and are followed with MRI, clinical exams, blood tests and lumbar punctures to detect adverse events and biological signals. The study excludes people who are pregnant, have active HIV or hepatitis, or have implanted deep brain stimulation devices. Primary outcomes focus on safety, tolerability, and procedural feasibility with planned clinical and imaging follow-up.
Who should consider this trial
Good fit: Adults aged 18 to 65 with a genetically confirmed diagnosis of Huntington's disease who can give informed consent and tolerate MRI, neurosurgery, blood draws, and lumbar puncture are ideal candidates.
Not a fit: People who are outside the 18–65 age range, pregnant, test positive for HIV or chronic hepatitis, have an implanted deep brain stimulation device, or cannot undergo neurosurgery or required imaging/procedures are unlikely to be eligible or benefit.
Why it matters
Potential benefit: If successful, this approach could offer a new cell-based therapy that may slow or improve neurological decline in people with Huntington's disease.
How similar studies have performed: Preclinical studies and limited cell-therapy work in other neurodegenerative diseases show promise, but this is the first human trial of hNSC-01 so direct clinical evidence is lacking.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Irvine, California
- University of California, Irvine — Irvine, California, United States (Recruiting)
Study contacts
- Study coordinator: UCI Alpha Clinic
- Email: alphaclinic@hs.uci.edu
- Phone: 949-824-3990
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.