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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02452723
• Date of registration: 18/05/2015
• Prospective Registration: Yes
• Primary sponsor: Cyto Therapeutics Pty Limited
• Public title: A Study to Evaluate the Safety of Neural Stem Cells in Patients With Parkinson's Disease
• Scientific title: 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
• Date of first enrolment: July 2016
• Target sample size: 12
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT02452723
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

Australia

Key inclusion & exclusion criteria

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



Age minimum: 30 Years
Age maximum: 70 Years
Gender: All

Health Condition(s) or Problem(s) studied

Parkinson Disease

Intervention(s)

Biological: ISC-hpNSC

Primary Outcome(s)

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

Secondary Outcome(s)

Change in UPDRS score from baseline [Time Frame: Baseline and 12 months]

Proportion of patients with improvement defined as any reduction in UPDRS motor score [Time Frame: 12 months]

Secondary ID(s)

ISC-hpNSC

Source(s) of Monetary Support

Please refer to primary and secondary sponsors