Open-label Investigation of the Safety and Clinical Effects of NTCELL in Patients With Parkinson's Disease

NCT01734733 | Completed Phase 1 DMC

• 1 other identifier: LCT/PD-012
• Study Type: interventional
• Target: 4
• 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 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).

Conditions

Parkinson's Disease

Keywords

Parkinson's Disease; Xenotransplantation; choroid plexus

Outcome Measures

Primary Outcomes (1)

• the safety of xenotransplantation of NTCELL

  measured by the incidence of adverse events, clinical laboratory tests (including xenogeneic viral analysis), physical examination, review by infectious disease physician

  26 weeks

Secondary Outcomes (10)

• Brain metabolism as demonstrated on PET with [18F]-fluorodopa measured at 26 weeks post-implant 1 compared with baseline

  26 weeks

• Brain metabolism as demonstrated on PET with [11C]-tetrabenazine measured at 26 weeks post-implant 1 compared with baseline

  26 weeks

• 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

  26 weeks

• Modified Hoehn and Yahr stages over 26 weeks post-implant 1 compared with the baseline stages

  26 weeks

• 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

  26 weeks

Study Arms (1)

NTCELL

EXPERIMENTAL

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.

Other: NTCELL

Interventions

NTCELL OTHER

NTCELL

Eligibility Criteria

• Age: 40 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• To be assessed at the Week -10 to -4 Visit
• 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

Sponsors & Collaborators

• Living Cell Technologies: lead

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, MBChB

  Auckland City Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 18, 2012
• First Posted: November 28, 2012
• Study Start: July 12, 2013
• Primary Completion: June 4, 2015
• Study Completion: June 4, 2020
• Last Updated: June 9, 2020

Record last verified: 2020-06

Locations

NZ (1)