Investigation of the Safety and Efficacy of NTCELL® [Immunoprotected (Alginate-Encapsulated) Porcine Choroid Plexus Cells for Xenotransplantation] in Patients With Parkinson's Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02683629
• Date of registration: 08/02/2016
• Prospective Registration: No
• Primary sponsor: Living Cell Technologies
• 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
• Date of first enrolment: February 2016
• Target sample size: 18
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02683629
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

New Zealand

Contacts

• Name: Barry Snow
• Affiliation: Auckland City Hospital

Key inclusion & exclusion criteria

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

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



Age minimum: 40 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Parkinson's Disease

Intervention(s)

Other: Sham Surgery

Biological: NTCELL Implantation

Primary Outcome(s)

The safety of xenotransplantation of NTCELL as measured by the incidence of adverse events related to treatment [Time Frame: up to 26 weeks]

Secondary Outcome(s)

Change in total Unified Parkinson's Disease Rating Scale (UPDRS in the 'off' and 'on' state) over 26 weeks post-intervention compared with baseline [Time Frame: Baseline and 26 weeks]

Change in Quality of life as assessed by Parkinson's Disease Questionnaire (PDQ-39) over 26 weeks post-intervention compared with baseline [Time Frame: Baseline and 26 weeks]

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 [Time Frame: Baseline and 26 weeks]

Change in Unified Parkinson's Disease Rating Scale (UPDRS Part III in the 'on' state) over 26 weeks post-intervention compared with baseline [Time Frame: Baseline and 26 weeks]

Change in Modified Hoehn and Yahr stage over 26 weeks post-intervention compared with baseline [Time Frame: Baseline and 26 weeks]

Change in L-dopa dosage over 26 weeks post-intervention compared with baseline [Time Frame: Baseline and 26 weeks]

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 [Time Frame: Baseline and 26 weeks]

Secondary ID(s)

LCT/PD-015

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Statistecol Consultants Limited