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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT02452723
Registration number
NCT02452723
Ethics application status
Date submitted
18/05/2015
Date registered
25/05/2015
Date last updated
5/04/2019
Titles & IDs
Public title
A Study to Evaluate the Safety of Neural Stem Cells in Patients With Parkinson's Disease
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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
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Secondary ID [1]
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ISC-hpNSC
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Parkinson Disease
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Condition category
Condition code
Neurological
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Parkinson's disease
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Experimental: ISC-hpNSC -
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Incidence of treatment-emergent adverse events (TEAEs), serious TEAEs, related TEAEs, severe TEAEs
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Assessment method [1]
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Timepoint [1]
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12 month
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Secondary outcome [1]
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Change in UPDRS score from baseline
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Assessment method [1]
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Timepoint [1]
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Baseline and 12 months
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Secondary outcome [2]
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Proportion of patients with improvement defined as any reduction in UPDRS motor score
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Assessment method [2]
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Timepoint [2]
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12 months
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Eligibility
Key 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
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Minimum age
30
Years
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Maximum age
70
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key 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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
NA
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 1
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
UNKNOWN
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/07/2016
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/06/2020
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Actual
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Sample size
Target
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Accrual to date
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Final
12
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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Dept of Neurology, The Royal Melbourne Hospital - Melbourne
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Recruitment postcode(s) [1]
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3050 - Melbourne
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Cyto Therapeutics Pty Limited
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
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.
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Trial website
https://clinicaltrials.gov/study/NCT02452723
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Trial related presentations / publications
Garitaonandia I, Gonzalez R, Sherman G, Semechkin A, Evans A, Kern R. Novel Approach to Stem Cell Therapy in Parkinson's Disease. Stem Cells Dev. 2018 Jul 15;27(14):951-957. doi: 10.1089/scd.2018.0001. Garitaonandia I, Gonzalez R, Christiansen-Weber T, Abramihina T, Poustovoitov M, Noskov A, Sherman G, Semechkin A, Snyder E, Kern R. Neural Stem Cell Tumorigenicity and Biodistribution Assessment for Phase I Clinical Trial in Parkinson's Disease. Sci Rep. 2016 Sep 30;6:34478. doi: 10.1038/srep34478. Gonzalez R, Garitaonandia I, Poustovoitov M, Abramihina T, McEntire C, Culp B, Attwood J, Noskov A, Christiansen-Weber T, Khater M, Mora-Castilla S, To C, Crain A, Sherman G, Semechkin A, Laurent LC, Elsworth JD, Sladek J, Snyder EY, Redmond DE Jr, Kern RA. Neural Stem Cells Derived from Human Parthenogenetic Stem Cells Engraft and Promote Recovery in a Nonhuman Primate Model of Parkinson's Disease. Cell Transplant. 2016 Nov;25(11):1945-1966. doi: 10.3727/096368916X691682.
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Public notes
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Contacts
Principal investigator
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Address
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Fax
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT02452723
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