A Study to Evaluate the Safety of Neural Stem Cells in Patients With Parkinson's Disease

January 16, 2026 updated by: Cyto Therapeutics Pty Limited

A Single Arm, Open-Label Phase 1 Study to Evaluate the Safety and Tolerability of ISC-hpNSC Injected Into the Striatum and Substantia Nigra of Patients With Parkinson's Disease

This study will evaluate the safety of an investigational cell transplantation therapy, ISC-hpNSC, in patients with Parkinson's disease. All patients will receive the therapy, which consists of human neural stem cells. Three dose levels will be examined in the study.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

ISC-hpNSC is a cellular therapeutic consisting of human parthenogenetic neural stem cells (hpNSC). ISC-hpNSC will be injected intracerebrally to the striatum and substantia nigra of patients with Parkinson's disease (PD).

The study will enroll 4 patients for cell injection at each of three different doses. A total of 12 patients with moderate to severe PD will be treated. Each patient receives a single dose. The main objective of the study is to evaluate the safety of the cell transplantation.

Study Type

Interventional

Enrollment (Actual)

12

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • Victoria
      • Melbourne, Victoria, Australia, 3050
        • Dept of Neurology, The Royal Melbourne Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

26 years to 66 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Signed informed consent form (ICF) indicating the patient has been informed of the procedures to be followed, the experimental nature of the therapy, alternatives, potential benefits, side-effects, risks, and discomforts
  • Patient diagnosed with idiopathic PD of ≤ 13 years duration, as defined by the United Kingdom (UK) Parkinson's Disease Society Brain Bank criteria
  • Outpatients (male and female) 30 - 70 years old. Females must be of non-child bearing potential, or with a negative pregnancy test and not breast-feeding
  • Patients receiving a stable dose of levodopa for at least 3 months with the expectation that the treatment will remain unchanged throughout the course of the patient's participation in the trial
  • Patients receiving an anti-parkinsonian treatment at a stable dose for at least 3 months with the expectation that the treatment will remain unchanged throughout the course of the patient's participation in the trial
  • Hoehn and Yahr stage II-IV during "ON" time
  • Unified Parkinson's Disease Rating Scale (UPDRS) motor score (Part III) in the "OFF" state ≤ 49
  • Positive dopaminergic response of ≥ 33% decrease in UPDRS motor scores between "OFF" and "ON" states at screening, and unequivocal clinical off periods
  • Patient is experiencing motor fluctuations with at least two cumulative hours of daily "OFF" -time during the waking period, which is measured on at least two consecutive days
  • History of anti-parkinsonian treatment with sufficient doses of levodopa
  • Stable, well-controlled concomitant disorders that would not contraindicate general anesthesia or stereotactic neurosurgery
  • No abnormalities on baseline brain MRI
  • Insufficient control of PD symptoms or intolerable side effects with optimized oral PD therapy
  • Montreal Cognitive Assessment (MOCA) score ≥ 26
  • Willing to fully comply with all study procedures and requirements of the trial
  • No surgery for PD or been treated with neuroleptics in the past 6 months except low-dose quetiapine fumarate or clozapine
  • No significant further improvement with physical therapy/rehabilitation

Exclusion Criteria:

  • Mild cognitive impairment of dementia (MOCA score < 26)
  • The extent or severity of the disease is not measurable
  • Severe dyskinesia in the "OFF" or "ON" states (violent dyskinesias, incompatible with any normal motor task)
  • Pre-existing medical conditions such as bleeding disorders, septicemia, major cardiovascular, cerebrovascular or psychiatric disease
  • Any current or relevant previous history of serious, severe or unstable physical or psychiatric illness or medical disorder that may make the participant unlikely to fully complete the study (depression, anxiety, cognitive impairment or impulse control disorder)
  • Clinically significant abnormal hematologic evaluation (blood count, partial thromboplastin time (PTT)), blood chemistry (glucose, blood urea nitrogen (BUN), creatinine, electrolytes), liver function tests (aspartate aminotransferase (AST), alanine aminotransferase (ALT), gamma glutamyl transpeptidase (GGTP), total protein, bilirubin)
  • Any active infectious disease of any nature, including clinically active viral infections (seropositive for Human Immunodeficiency Virus (HIV), Hepatitis C Virus (HCV), Hepatitis B Virus (HBV) and Syphilis
  • Severe obesity
  • Previous intracranial surgery, including deep-brain stimulation
  • History of seizures
  • Substance abuse (recent history of alcohol abuse or other drugs such as barbiturates, cannabinoids and amphetamines)
  • Use of anti-platelet agents or other anti-coagulants
  • Signs of any malignant disease
  • Any use of immunosuppressive drugs
  • Enrollment in other investigational drug trial or has completed any trial within the last 3 months
  • Patients that cannot undergo MRI or PET scanning (i.e. patients with implanted pacemakers)
  • Patients unable to travel to the PET scanning center
  • Any other condition which clinician regards as making patient unsuitable for trial

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: ISC-hpNSC
Biological: ISC-hpNSC

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence of treatment-emergent adverse events (TEAEs), serious TEAEs, related TEAEs, severe TEAEs
Time Frame: 12 month
12 month

Secondary Outcome Measures

Outcome Measure
Time Frame
Change in UPDRS score from baseline
Time Frame: Baseline and 12 months
Baseline and 12 months
Proportion of patients with improvement defined as any reduction in UPDRS motor score
Time Frame: 12 months
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Cyto Therapeutics Pty Limited

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 1, 2016

Primary Completion (Actual)

June 18, 2025

Study Completion (Actual)

September 1, 2025

Study Registration Dates

First Submitted

May 18, 2015

First Submitted That Met QC Criteria

May 20, 2015

First Posted (Estimated)

May 25, 2015

Study Record Updates

Last Update Posted (Actual)

January 20, 2026

Last Update Submitted That Met QC Criteria

January 16, 2026

Last Verified

January 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ISC-hpNSC

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Parkinson Disease

Clinical Trials on ISC-hpNSC

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Malta

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe