Thoughts on Clinical Trials

[Michael]

I had a thymectomy 26 years ago.  It didn’t put me in remission, but it may have made the MG more manageable.  That said, I think that’s a bazaar clinical trial.  They would have to make an incision in your body to make you think you had a thymectomy…right?  No way would I do that.  I’m with you and absolutely 100% would want the thymectomy and there would be zero guilt about not participating in that study.

I understand the need for clinical trials, but honestly, give me the medicine!  My MG definitely will not get better without treatment and will get 100% worse.  Lucky dog if you’re the one that gets a placebo and you get better.  Unfortunately, I don’t have the kind of luck.

I don’t feel that I could ever participate in a clinical trial because of the risk of getting a placebo or the therapy being tested doesn’t work.  I don’t want to have to deal with a flare…I have a miserable time dealing with double vision.   Anytime I had flare, it would take almost a year for all my symptoms to be almost back to normal.

[Jerry Trapp]

I was scheduled to start Vyvgart infusions this week and was then advised that I was accepted in a new clinical trial (Cartesian-08) that was starting next week. I had applied and had been turned down (after going through all interviews, exams and tests) on two pevious clinical trials. I am 82 year old male and have had generalized MG for 2 1/2- 3 years) and was starting to believe it was my age that was getting in the way.

The decision to cancel the Vyvgart infusions (they had been approved and I had secured 100% funding for the rest of the year) was difficult, but after much thought and research I have decided to go with the trial instead. Yes, I am having second thoughts about the decision, but am hopeful, that in the log run, it is best for me, my age and current condition. I did discuss the situation with my Neurologist as well as my GP. Neither, of course would say yes or no, but both agreed that I should do what I thought best for me and would support me in every they could. They really are great doctors and have provided me with excellent care and support getting me through these first few years.

I will keep the Forum as up-to-date as possible. There may be some restrictions of what and when I can report things, but will do the best I can.

[Nikki Meyers]

I was in the Castesion clinical trial. The first set for safety worked great but wore off after 4-5 months. The second clinical trial for the amount of the drug-I got the infusion monthly did not work at all. They could not get people to try clinical trial so it was extremely small-like 3 people.
Good luck

[Jonathan A]

So far, after 8 months of searching for a diagnosis for my disease….and now having a name for it  … ACUTE OCULAR and GENERALIZED MYASTHENIA GRAVIS,  I can now google and ZOOM with support groups to get educated.  My support groups have really helped me out, answered my questions, and directed me to websites and trial clinics  with information, to download, printout and give to my doctors, who don’t know what MG means in ENGLISH.

I have been in remission for 15 months now and am stable  … and an now in remission also from medically induced DIABETES II  … from all the STATINS, STEROIDS, and PREDNISONE that the hospitals were dumping in me.  So I am off LANTUS and am not forced to SHOOT UP any more.

The researchers for the UNIVERSITY of CALIFORNIA at Irvine, called me up and had me answer many MG questions, after submitting my medical papers and tests online.   Julia, the research contact, did not feel I had high enough BAD SCORES on that standard MG check sheet to qualify., after I answered all her questions, when she called.

I do so much want to help find a cure, and help others out, but at the same time, I do not want to risk falling out of REMISSION,  as I remember how sick I was undiagnosed  … and that I actually died (ACUTE RESPIRATORY FAILURE due to ANESTHESIA)  … CODE RED   ….   when stopped breathing for MRI/CAT SCAN tests  …. too much ROCURONIUM and PROPOFOL to an undiagnosed MG patient.

Why am I in remission, while my fellow ZOOMING MG support group members (belong to 4 groups) still are doing monthly IVIGs or trying desperately to taper off PREDNISONE?  Wouldn’t clinics, who know about MG, and are trying to figure it all out, want to figure out  .. how I can be stable  .. when others are not ??   I am on 2000 MGs of CELLCEPT daily and 180

MGs of MESTINON daily … with no more IVIG  .. beyond the original 5 hours for 5 days, after the nurse talked her supervisors into testing my ANTIBODIES  … all three tests were “thru the roof” high scores.  I took me over a year to get those test results with the great help of ALLSTRIPES and PICNIC HEALTH RARE DISEASE GROUPS.  They also finally found a THYMOMA X-RAY  … and I was clear with no masses.

Because I am on immune suppressants for transplant patients (have no transplant)  … my immune system is TOAST  …. and all doctors seem to be into BLOOD THINNERS also, so I am on 5, and bleed in arm or leg, whenever I am out now gardening and pruning.  It is wonderful to know that I can get on my knees again  … and not be undiagnosed weak with MG, that I have to crawl back into the house and lay down on the carpet to rest, and gain my legs again.

So I want to help out  …  but how can one sign up for a THYMUS removal  .. and then not have it removed ???  I understand pill placebos  … but an operation placebo  … with a very high risk of infection and bleeding excessively. NO WAY !!

My non neurological doctors, still do not understand my MG  … so when they see my low red and white blood cell numbers, I need to remind them I am on 2000 MGS daily of CELLCEPT.  I also had to remind them that when you keep dumping STATINS and PREDNISONE in me  … that my blood sugar numbers HIT THE ROOF.  So one wants to help out doctors figure out and cure MG,   but we that actually have MG  … are still trying to educate the many doctors that we see  .. who still don’t even know what MG means in ENGLISH.

My unknowing doctors keep wanting to scope me out from top to bottom, and maybe “take Samples”  … and I keep having to remind them that I have MG, my immune system is toast, I am on 5 blood thinners/anticlots, and lost my hearing for 4 months, because TOBRAMYCIN is dangerous to undiagnosed MG patients.  Then there is the whole matter of MG ignorance, and other not understanding that improper use and quantity of SEDATIVES and ANESTHESIAS, can give me a second CODE RED OVERDOSE so I stop breathing again.  So I am all for participating in trials and learning  … but they need to be SAFE  … as well as “IN TOWN” … as travel out of one’s local city comfort zone  … is a NO GO.

[Amy Cessina]

Pardon but I think this post is misinformation. No one would ever cut you open and not remove the thymus. I believe the study group was split into two,  one got surgery the other didn’t. But you wouldn’t know at the start what group you would be assigned. And the surgery had to be crack the chest open style too not robotic.  There are stringent standards for clinical trials that have to be approved you can’t just do what you want to a human being and call it a clinical trial.

[Jools]

Hi. I had a thymectomy 42 years ago when they opened up your breastbone etc and it was a major operation with a long recovery period. I know that can do key hole surgery nowxwhich is a godsend. However, i am absolutely in shock to see a trial where you wouldnt know if youd had the thymectomy or not. It sound extremely weird. Its only a theory still that a thymectomy may have a significant effect. Im 42 years sown the line and had my 3 worst MG crisis’s in the last 18 months or so. My Neurologist cant say for certain if the Thymectomy played an important role with my MG (generalised) from the start. Where is this trial taking place…? Also, why would a company need to do such a trial when there are 100s of MG patients that could fill out a questionnaire so easily and explain what a theymectomy did or didnt do for them. Id forget this trial and push with your Neurologist for a real Thymectomy if you feel it may be of benifit. Even psychological benefit is better than not knowing if youd actually had the operation. 🤔😬😲 Good luck tjo and take care. X

[Jerry Trapp]

I said I would provide updates to my participation in the Cartesian-08 trial in my comments last May.  So, I now can say that the trial was a great success for me.  I had the “T” cell removal done, the infusions manufactured (modified in the drug company lab), and I received 6 infusions.  There was some technical issues with the first batch of “T” cells that was resolved and I actually received 1 infusion from the first batch and then after a few month delay received an additional 5 infusions (once per week) for 5 weeks from the second batch.  My last infusion was in mid October ’22. I had immediate reactions to the infusions in a very positive manner. Today, I can report that in  my opinion, I am in remission of most all MG issues.  I still have some high up double vision, but it is not an issue for daily living as it is usually completely out of my normal field of vision. My biggest issue with MG was muscle deteriation.  During the last 2+ years my strength and coordination had been reduced to something that was barely allowing me to function.  My biceps and calves had lost about 35% of original mass and much more in strenght. This month I will have my last 28 day follow-up and will go into the 3 month follow-up routine. I feel like all is mostly normal with my body functions.  As I age (I am now 83 years old) and experience other health issues, it is harder for me to tell what is the cause of some of my strenght issues.  I experienced some heart artery blockage last last October and had 6 stints placed to relieve the blockages.  I am not scheduled for any future infusions and personally believe that the infusions I received have had a permanent effect in relieving my MG symptoms. I think the trial went well and am very satisfied that I made the decision to participate and am looking forward to completing the follow-up visits I have remaining.  The details of the “T” cell removal and infusions are many – some trying and painful – but too much to attempt to cover in this report.  I will do another report when the trial officially ends later his year.

[Bambilin]

Thanks for the follow-up. I’m 81 and seem to be deteriorating in strength and stamina. Plus my lower back is very weak and painful after five or ten minutes of cooking, etc. I want to blame some of it on my age but I think three years of isolation from COVID-19 and inactivity has also taken a toll physically. I’m happy your treatment has worked.

[Pup]

I participated in an extensive Cartesian double blinded study which required the removal of my Tcells for the purpose of, if given the treatment would actively “kill off” Tcells once treated blood was reinfused. The re-infusions took place once a week over 6weeks. I knew I received the treated Tcells as by the end of week 2 I was already feeling stronger. Upon completion of 6 weeks I felt like wonder woman-and for a 68 yo woman with gMG that was amazing. Lasted 6 mos-then I crashed.

It was worth it and I encourage everyone to participate in any trials that come available.

It is the only way we can find solutions for this hideous disease. So please for us now and those diagnosed in future days Please, please participate when you can.