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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01734733




Registration number
NCT01734733
Ethics application status
Date submitted
18/11/2012
Date registered
28/11/2012
Date last updated
9/06/2020

Titles & IDs
Public title
Open-label Investigation of the Safety and Clinical Effects of NTCELL in Patients With Parkinson's Disease
Scientific title
A Phase I/IIa, Open-label Investigation of the Safety and Clinical Effects of NTCELL [Immunoprotected (Alginate-Encapsulated) Porcine Choroid Plexus Cells for Xenotransplantation] in Patients With Parkinson's Disease
Secondary ID [1] 0 0
LCT/PD-012
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Parkinson's 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: NTCELL - NTCELL 40 microcapsules (+/- 20%)

The NTCELL microcapsules are drawn up into a catheter system and introduced intracranially by stereotactic insertion into the brain under guidance by neuroimaging.
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
the safety of xenotransplantation of NTCELL
Timepoint [1] 0 0
26 weeks
Secondary outcome [1] 0 0
Brain metabolism as demonstrated on PET with [18F]-fluorodopa measured at 26 weeks post-implant 1 compared with baseline
Timepoint [1] 0 0
26 weeks
Secondary outcome [2] 0 0
Brain metabolism as demonstrated on PET with [11C]-tetrabenazine measured at 26 weeks post-implant 1 compared with baseline
Timepoint [2] 0 0
26 weeks
Secondary outcome [3] 0 0
Scores measured by the Unified Parkinson's Disease Rating Scale (UPDRS Parts I, II, III, IV - Parts II and III will be performed in the 'off' and 'on' state) over 26 weeks post-implant 1 compared with the baseline scores
Timepoint [3] 0 0
26 weeks
Secondary outcome [4] 0 0
Modified Hoehn and Yahr stages over 26 weeks post-implant 1 compared with the baseline stages
Timepoint [4] 0 0
26 weeks
Secondary outcome [5] 0 0
Scores measured by the Unified Dyskinesia Rating Scale (UDysRS Parts I, II, III, IV - Parts III and IV will be performed in the 'off' and 'on' state) over 26 weeks post-implant 1 compared with the baseline scores
Timepoint [5] 0 0
26 weeks
Secondary outcome [6] 0 0
Times for hand-arm movement between 2 points (tapping test) in accordance with the CAPSIT-PD protocol (Defer et al. 1999) over 26 weeks post-implant 1 compared with the baseline times
Timepoint [6] 0 0
26 weeks
Secondary outcome [7] 0 0
Scores measured by the modified walking test in accordance with the CAPSIT-PD protocol (Defer et al. 1999) (walking 4.5m back and forth instead of 7m back and forth) over 26 weeks post-implant 1 compared with the baseline scores
Timepoint [7] 0 0
26 weeks
Secondary outcome [8] 0 0
Scores measured by the Parkinson's Disease Questionnaire (PDQ-39) over 26 weeks post-implant 1 compared with the baseline scores
Timepoint [8] 0 0
26 weeks
Secondary outcome [9] 0 0
Scores measured by neuropsychological tests at 26 weeks post-implant 1 compared with the baseline scores
Timepoint [9] 0 0
26 weeks
Secondary outcome [10] 0 0
Psychiatric assessment at 26 weeks post-implant 1 compared with the baseline psychiatric assessment
Timepoint [10] 0 0
26 weeks

Eligibility
Key inclusion criteria
To be assessed at the Week -10 to -4 Visit

* Adults (males or females) in the age range 40 to 70 years
* Diagnosis of Parkinson's disease (minimum duration of 5 years) in accordance with the London Brain Bank criteria
* Patients diagnosed with idiopathic Parkinson's disease
* Stable medication for Parkinson's for at least 1 month
* Patients with advanced and fluctuating Parkinson's disease who have met the criteria for DBS and who have been accepted for DBS at Auckland City Hospital. These criteria include exhaustion of available medication treatments for Parkinson's disease, normal brain MRI, intact cognitive, psychological and psychiatric function, appropriate carer support, and competence and willingness to consent to the placement of deep brain probes
* If female, no childbearing capability (those who are more than 2 years postmenopausal or have undergone voluntary sterilisation can be considered for enrolment)
* Provision of written informed consent. Patients will be required to agree to comply with all tests and visits specified in the protocol, and they (and their partners/close contacts) will also be required to consent to long-term microbiological monitoring, which is an integral part of the study.
Minimum age
40 Years
Maximum age
70 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
To be assessed at the Week -10 to -4 Visit

Note: the criteria for acceptance for DBS would exclude patients with comorbidities that normally would exclude them from similar studies, including uncontrolled depression, dementia, focal neurological deficits, or secondary parkinsonism. Specific exclusion criteria in this category are:

* Any history of central nervous system infection
* Significant dementia as determined by neuropsychological assessment
* Focal neurological defects
* Evidence of significant medical or psychiatric disorders
* Secondary parkinsonism
* Severe autonomic symptoms
* Atypical Parkinson's disease
* History of substance abuse
* Body mass index (BMI) =30 kg/m2 or =20 kg/m2
* Serious comorbid conditions that are likely to affect participation in the study, including:

* Previous coronary heart disease manifesting as non-ST elevation myocardial infarction (NSTEMI), Q-wave infarction or unstable angina; coronary artery bypass graft (CABG); or percutaneous angioplasty
* Previous cerebrovascular disease manifesting as transient ischaemic attacks (TIAs) or stroke
* Peripheral vascular disease with foot ulcer and/or previous amputation
* History of New York Heart Association (NYHA) class II, III or IV congestive heart failure (CHF) and/or chronic atrial fibrillation
* Chronic obstructive pulmonary disease (COPD) or asthma with previous hospitalisation for decompensation; a requirement for mechanical ventilation at any stage; or long-term treatment with oral corticosteroids
* Liver disease with abnormal liver function tests defined as serum bilirubin =20 µmol/L, and/or ALT =100 U/L, and/or GGT =100 U/L, and/or albumin < 35 g/L
* Haematological disorders, including haemoglobin =110 g/L or platelet count < 80 x 109/L
* Kidney disease, defined as serum creatinine > 130 µmol/L in men and > 110 µmol/L in women and/or haematuria and/or active urinary sediment or casts
* Peptic ulcer disease and/or history of previous gastrointestinal bleeding
* Malignancy other than basal cell carcinoma
* History of epilepsy
* Untreated hypothyroidism
* Known adrenal insufficiency

Other exclusion criteria:

* Past history of brain surgery for Parkinson's disease
* Poor candidate for any surgery
* HIV antibody and/or risk factors for HIV infection
* Positive hepatitis C antibody, positive hepatitis B surface antigen, and hepatitis B core antibody
* Current administration of immunosuppressive medications (e.g. cyclosporin, tacrolimus, sirolimus, mycophenolate mofetil, muromonab-CD3, daclizumab, basiliximab, antithymocyte globulin, interferons) for other disease conditions
* Inability to travel on aeroplane to Vancouver (for PET scan)
* Any other condition that, in the opinion of the Investigator, may interfere with adherence to the study protocol.

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
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
12/07/2013
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
4/06/2020
Sample size
Target
Accrual to date
Final
4
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
New Zealand
State/province [1] 0 0
Auckland

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Living Cell Technologies
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
To assess the safety of xenotransplantation of NTCELL \[immunoprotected (alginate-encapsulated) choroid plexus cells\] in patients with Parkinson's disease, assessed over the duration of the study, by monitoring the occurrence of adverse events and serious adverse events, including clinical and laboratory evidence of xenogeneic infection in transplant recipients and their partners/close contacts. Subsequent safety follow-up will include lifelong monitoring for clinical and laboratory evidence of xenogeneic infection.

To assess the clinical effects of NTCELL \[immunoprotected (alginate-encapsulated) choroid plexus cells\] in patients with Parkinson's disease. This will be quantified by testing the secondary endpoints of the trial as described below (see Endpoints/Outcome Measures).
Trial website
https://clinicaltrials.gov/study/NCT01734733
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Barry Snow, MBChB
Address 0 0
Auckland City Hospital
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01734733