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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
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
Secondary ID [1] 0 0
ISC-hpNSC
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Parkinson Disease 0 0
Condition category
Condition code
Neurological 0 0 0 0
Parkinson's disease

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Experimental: ISC-hpNSC -

Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of treatment-emergent adverse events (TEAEs), serious TEAEs, related TEAEs, severe TEAEs
Timepoint [1] 0 0
12 month
Secondary outcome [1] 0 0
Change in UPDRS score from baseline
Timepoint [1] 0 0
Baseline and 12 months
Secondary outcome [2] 0 0
Proportion of patients with improvement defined as any reduction in UPDRS motor score
Timepoint [2] 0 0
12 months

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
Minimum age
30 Years
Maximum age
70 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
UNKNOWN
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/07/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/06/2020
Actual
Sample size
Target
Accrual to date
Final
12
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Dept of Neurology, The Royal Melbourne Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
3050 - Melbourne

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Cyto Therapeutics Pty Limited
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
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