great post whytee.
just being a little better than the controls isn't going to cut it. obviously there has to be no long lasting serious side affects and i kind of doubt there are any since they went ahead with the largest dose some time after the 40 and 80 capsule treatments were done.
whytee, i'm just lazy and will ask you if the post 26 week data, i assume they will take post 26 week measurements on the first 2 groups but maybe i might be wrong, will be reported and considered even though in the trial protocol they just says report the 26week data?
https://clinicaltrials.gov/ct2/show/study/NCT02683629?show_desc=Y#desc
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.
Primary Outcome Measures:
- The safety of xenotransplantation of NTCELL as measured by the incidence of adverse events related to treatment [ Time Frame: up to 26 weeks ]
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
Secondary Outcome Measures:
- 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 ]
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just from a layman with pd perspective, short of not needing any meds (a cure), having to take less meds would be the most important factor to me because of the following:
1. meds have side affects - hypotension, headaches at times.
2. the newer ones are longer lasting but expensive brand names or you have to add older/cheaper drugs which help to increase the half-life of l-dopa which is about 90min, bottom line have to take meds 4-5 times during the day and still facing a lot of variability in symptom relief.
3. for most advanced pd'ers l-dopa benefit is reduced or entirely negated by eating, especially protein, so going out to eat socially is a challenge as is eating in general, if i have the will power i eat most of my protein at night. just being able to not have to think about what and when i eat would be wonderful even if i had to still take some meds.
4. no longer have OFFS where i freeze and/or really can be only a couch potato waiting for my meds to kick in. really messes up ones life.
5. bottom line i'm better in all aspects of my life if i need less meds. my ON's are pretty good, it's my OFF's that i want to lessen significantly. sorry if that doesn't make sense.
if i'm in the trial and i need less meds then all the other criteria should be better when OFF. i think that's why they selected so many tests, to better support their trial. best of luck again.
