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

Phase 2RecruitingNCT07451613

Leslie Thompson21 enrolled

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Huntington Disease

Interventions

hNSC-01BIOLOGICAL

neural stem cell therapy

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

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.

Locations (1)

University of California, Irvine

Irvine, California, United States

RECRUITING

Outcomes

Primary Outcomes

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.

Time frame: Safety is assessed 6 weeks following each surgery for Cohort A and 12 weeks following cohort B-D completion

Secondary Outcomes

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.

Time frame: 12 months

Change in Total Motor Score (TMS)

To evaluate change in TMS after 1-year post-implantation.

Time frame: 12 months

Change in Functional Imaging: FDG-PET

To evaluate change in Functional Imaging: FDG-PET at 12 months compared to Baseline

Time frame: 12 months

Change in structural MRI

To evaluate change in structural MRI at 12 months compared to Baseline

Time frame: 12 months

Change in NfL in CSF.

To evaluate change in NfL levels in CSF.

Time frame: 12 months

Change in NfL in plasma.

To evaluate change in NfL levels in plasma.

Time frame: 12 months

Central Contacts

UCI Alpha Clinic

CONTACT

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

Data from ClinicalTrials.gov

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