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Recruiting NCT07451613

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

Phase I / Phase II Interventional Huntington Disease

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: hNSC-01.
Who it may be relevant to
Registry conditions: Huntington Disease. Basic parameters: 18 years — 65 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

Phase 1B/2A Study of the Safety and Tolerability of Human Neural Stem Cells for Huntington's Disease

Overview

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.

Interventions

  • Biological hNSC-01
    neural stem cell therapy

Primary outcome measures

  • 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]
Secondary outcome measures (11)
  • 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]

Eligibility 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Sequential
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 1 center
  • University of California, Irvine — Irvine

Identifiers

NCT: NCT07451613 · ASCC-26-01 [STUDY00000867]

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗