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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02683629




Registration number
NCT02683629
Ethics application status
Date submitted
8/02/2016
Date registered
17/02/2016
Date last updated
14/05/2019

Titles & IDs
Public title
Investigation of the Safety and Efficacy of NTCELL® [Immunoprotected (Alginate-Encapsulated) Porcine Choroid Plexus Cells for Xenotransplantation] in Patients With Parkinson's Disease
Scientific title
A Phase IIb, Randomised, Double-blind, Placebo-controlled, Dose-range Investigation of the Safety and Efficacy of NTCELL® [Immunoprotected (Alginate-Encapsulated) Porcine Choroid Plexus Cells for Xenotransplantation] in Patients With Parkinson's Disease
Secondary ID [1] 0 0
LCT/PD-015
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
Treatment: Other - NTCELL Implantation
Other interventions - Sham Surgery

Experimental: NTCELL - NTCELL Implantation

Sham comparator: Sham Surgery - Sham Surgery


Treatment: Other: NTCELL Implantation
NTCELL Implantation

Other interventions: Sham Surgery
Sham Surgery
Intervention code [1] 0 0
Treatment: Other
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
The safety of xenotransplantation of NTCELL as measured by the incidence of adverse events related to treatment
Timepoint [1] 0 0
up to 26 weeks
Secondary outcome [1] 0 0
Change in total Unified Parkinson's Disease Rating Scale (UPDRS in the 'off' and 'on' state) over 26 weeks post-intervention compared with baseline
Timepoint [1] 0 0
Baseline and 26 weeks
Secondary outcome [2] 0 0
Change in Unified Parkinson's Disease Rating Scale (UPDRS Part III in the 'on' state) over 26 weeks post-intervention compared with baseline
Timepoint [2] 0 0
Baseline and 26 weeks
Secondary outcome [3] 0 0
Change in Quality of life as assessed by Parkinson's Disease Questionnaire (PDQ-39) over 26 weeks post-intervention compared with baseline
Timepoint [3] 0 0
Baseline and 26 weeks
Secondary outcome [4] 0 0
Change in L-dopa dosage over 26 weeks post-intervention compared with baseline
Timepoint [4] 0 0
Baseline and 26 weeks
Secondary outcome [5] 0 0
Change in 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-intervention compared with baseline
Timepoint [5] 0 0
Baseline and 26 weeks
Secondary outcome [6] 0 0
Change in scores measured by the modified walking test in accordance with the CAPSIT-PD protocol (Defer et al. 1999) over 26 weeks post-intervention compared with baseline
Timepoint [6] 0 0
Baseline and 26 weeks
Secondary outcome [7] 0 0
Change in Modified Hoehn and Yahr stage over 26 weeks post-intervention compared with baseline
Timepoint [7] 0 0
Baseline and 26 weeks

Eligibility
Key inclusion criteria
1. Adults (males or females) in the age range 40 to 65 years
2. Diagnosis of Parkinson's disease (minimum duration of 5 years) in accordance with the London Brain Bank criteria
3. Patients diagnosed with idiopathic Parkinson's disease
4. Optimum medication for Parkinson's disease
5. Expected to meet the criteria for DBS in the future, in the opinion of the Investigator
6. If female, no childbearing capability (those who are more than 2 years post-menopausal or have undergone voluntary sterilisation can be considered for enrolment)
7. 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
65 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Any history of central nervous system infection
2. Significant dementia as determined by neuropsychiatric assessment
3. Focal neurological defects
4. Evidence of significant ongoing medical or psychiatric disorders
5. Secondary parkinsonism
6. Severe autonomic symptoms
7. Atypical Parkinson's disease
8. History of substance abuse
9. Body mass index (BMI) = 30 kg/m2 or = 20 kg/m2
10. Serious comorbid conditions that, in the opinion of the Investigator, are likely to affect participation in the study, including:

1. 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
2. Previous cerebrovascular disease manifesting as transient ischaemic attacks (TIAs) or stroke
3. Peripheral vascular disease with foot ulcer and/or previous amputation
4. History of New York Heart Association (NYHA) class II, III or IV congestive heart failure (CHF) and/or chronic atrial fibrillation
5. 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
6. 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
7. Haematological disorders, including haemoglobin = 110 g/L or platelet count < 80 x 109/L
8. 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
9. Peptic ulcer disease and/or history of previous gastrointestinal bleeding
10. Malignancy other than basal cell carcinoma
11. History of epilepsy
12. Untreated hypothyroidism
13. Known adrenal insufficiency
11. Previous brain surgery for Parkinson's disease
12. Poor candidate for any surgery
13. HIV antibody and/or risk factors for HIV infection
14. Positive hepatitis C antibody, positive hepatitis B surface antigen, and hepatitis B core antibody
15. Current administration of immunosuppressive medications (e.g. cyclosporin, tacrolimus, sirolimus, mycophenolate mofetil, muromonab-CD3, daclizumab, basiliximab, antithymocyte globulin, interferons) for other disease conditions
16. 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
Randomised controlled trial
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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
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
1/02/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
2/05/2019
Sample size
Target
Accrual to date
Final
18
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
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Statistecol Consultants Limited
Address [1] 0 0
Country [1] 0 0

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 efficacy of xenotransplantation 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/NCT02683629
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Barry Snow
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/NCT02683629