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

ConditionHuntington Disease
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-65
SponsorLeslie Thompson

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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 design

Design model

Sequential

Treatments tested in this trial

  • hNSC-01

    Biological/Vaccine

    neural stem cell therapy

Treatment groups

21 Participants
are divided into 4 treatment groups
Group A: Cohort AActive comparator 1 intervention
Group B: Cohort BActive comparator 1 intervention
Group C: Cohort CActive comparator 1 intervention
Group D: Cohort DActive comparator 1 intervention

Trial outcomes

Primary outcomes

1

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

1

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
2

Change in Total Motor Score (TMS)

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

Time frame
12 months
3

Change in Functional Imaging: FDG-PET

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

Time frame
12 months
4

Change in structural MRI

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

Time frame
12 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Leslie Thompson

Lead sponsor

University of California, Irvine

Sponsor institution

California Institute for Regenerative Medicine (CIRM)

Collaborator

University of California, Irvine

Collaborator

Source ClinicalTrials.gov
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