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

NCT02683629 | Completed Phase 2 DMC

• 1 other identifier: LCT/PD-015
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

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).

Conditions

Parkinson's Disease

Keywords

Parkinson's Disease; Xenotransplantation; choroid plexus

Outcome Measures

Primary Outcomes (1)

• The safety of xenotransplantation of NTCELL as measured by the incidence of adverse events related to treatment

  Adverse events can result from, for example, abnormal clinical laboratory tests (including xenogeneic viral analysis), abnormal physical examination findings, any abnormal findings following review by an infectious disease physician. These multiple assessments result in the one outcome measure which is the incidence of treatment emergent adverse events

  up to 26 weeks

Secondary Outcomes (7)

• Change in total Unified Parkinson's Disease Rating Scale (UPDRS in the 'off' and 'on' state) over 26 weeks post-intervention compared with baseline

  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

  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

  Baseline and 26 weeks

• Change in L-dopa dosage over 26 weeks post-intervention compared with baseline

  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

  Baseline and 26 weeks

Study Arms (2)

NTCELL

EXPERIMENTAL

NTCELL Implantation

Biological: NTCELL Implantation

Sham Surgery

SHAM COMPARATOR

Sham Surgery

Other: Sham Surgery

Interventions

NTCELL Implantation BIOLOGICAL

NTCELL Implantation

NTCELL

Sham Surgery OTHER

Sham Surgery

Sham Surgery

Eligibility Criteria

• Age: 40 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adults (males or females) in the age range 40 to 65 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
• Optimum medication for Parkinson's disease
• Expected to meet the criteria for DBS in the future, in the opinion of the Investigator
• If female, no childbearing capability (those who are more than 2 years post-menopausal 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

You may not qualify if:

• Any history of central nervous system infection
• Significant dementia as determined by neuropsychiatric assessment
• Focal neurological defects
• Evidence of significant ongoing 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, in the opinion of the Investigator, 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

Sponsors & Collaborators

• Living Cell Technologies: lead
• Statistecol Consultants Limited: collaborator

Study Sites (1)

Auckland City Hospital

Auckland, New Zealand

Location

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Study Officials

• Barry Snow

  Auckland City Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 8, 2016
• First Posted: February 17, 2016
• Study Start: February 1, 2016
• Primary Completion: October 1, 2017
• Study Completion: May 2, 2019
• Last Updated: May 14, 2019

Record last verified: 2019-05

Data Sharing

• IPD Sharing: Will share

Locations

NZ (1)