A Phase 2 interventional study of NTCELL Implantation and Sham Surgery in Parkinson's Disease, sponsored by Living Cell Technologies. Completed at 1 site in New Zealand. Open to participants aged 40 Years to 65 Years. Per ClinicalTrials.gov, last updated 2019-05-14.
Sponsored by Living Cell Technologies · Phase 2, Interventional, and Treatment
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).
Parkinson's disease is characterized by widespread neural degeneration, particularly in the substantia nigra and its projections to the basal ganglia. Current therapy for Parkinson's disease is purely symptomatic. There is a pressing need for a treatment that reverses or slows the degeneration of the nigrostriatal pathway.
Numerous transplant-based therapies have attempted to support, repair or replace the degenerating nigrostriatal neurons. These have included the transplantation of foetal and other neuronal stem cells, gene transfers, and the implantation of devices releasing neural growth factors. All these have been shown to have some effectiveness in animal models, but have been generally disappointing in human studies.
Intracranial choroid plexus cell transplantation has the potential to deliver biological neural agents for the treatment of Parkinson's disease which cannot be achieved by conventional treatment. The overall aim of delivering neural proteins and compounds over many months to the basal ganglia of the brain is to enhance neural repair currently not possible with antiparkinsonian medication or deep brain stimulation (DBS).
As animal-derived tissues have to be protected from immune rejection when transplanted into humans, transplants are usually accompanied by immunosuppressive therapy. However, porcine choroid plexus cells are preferably implanted without the use of immunosuppressive drugs which cause significant morbidity. To protect them from immune rejection, the cells can be encapsulated in alginate microcapsules which permit the inward passage of nutrients and the outward passage of biologic neural proteins and compounds normally secreted by choroid plexus cells. Alginate-encapsulated porcine choroid plexus cells implanted into the brain without immunosuppressive drugs have survived rejection for many months in animal studies.
NTCELL comprises neonatal porcine choroid plexus cells encapsulated in alginate microcapsules.
The bilateral dose that will be administered to the 18 patients (initially 3 groups of 6 patients, randomised 4:2 NTCELL:sham surgery) enrolled in this trial will be up to a total of twice the human equivalent dose administered unilaterally in LCT's non-human primate study. Thus up to 240 NTCELL microcapsules (± 5%) administered on each side of the brain.
If there are no significant safety issues after implantation of the first group of patients, the second group of patients will then be scheduled to receive implants of NTCELL.
If there are no significant safety issues after implantation of the second group of patients, the third group of patients will then be scheduled to receive implants of NTCELL.
This study will adopt an adaptive design in respect to the choice of dose of NTCELL for the fourth group of patients (those patients who originally received sham surgery in Groups 1-3). Following unblinding of the study after Groups 1-3 have reached 26-week follow-up, an interim analysis, for safety and efficacy, will be undertaken.
If there are no significant safety issues, the last group of patients, Group 4, (who originally received sham surgery) will be scheduled to receive NTCELL implants. The dose of NTCELL given will be determined by the DSMB following a proposal from the Principal Investigator, based on the results of the interim analysis.
Parkinson's disease patients will be followed up for 26 weeks after receiving either an implantation of NTCELL or sham surgery.
4,487 studies on the registry are indexed under Parkinson Disease; 1,082 are open to participants now.
This study's enrollment of 18 is below the median of 40 across 3,294 interventional studies indexed under Parkinson Disease.
Browse Parkinson Disease studies →Living Cell Technologies is the lead sponsor of 3 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Serious comorbid conditions that, in the opinion of the Investigator, are likely to affect participation in the study, including:
NTCELL Implantation
Biological: NTCELL Implantation
Sham Surgery
Other: Sham Surgery
NTCELL Implantation
Sham Surgery
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
Time frame: up to 26 weeks
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 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 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 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
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 Modified Hoehn and Yahr stage over 26 weeks post-intervention compared with baseline
Time frame: Baseline and 26 weeks
Plan to share: Yes — International conference presentations, press releases, International journal publications
No publications or documents are linked to this record.
This study is completed, as verified in May 2019. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Living Cell Technologies