Hello to you all,
I was diagnosed with PSC in 2012. At that time I had a dominant stricture in the main bile duct which was treated with balloon dilation. No IBD until now. No itching.
I am very interested in possible therapeutical options for PSC. My theory is that PSC is not a homogenous disease but much more a common bile ducts' syndrom/reaction to several undiscovered diseases with different causes, some of them immunological and some not.
Can some of you share your experience of participation in clinical trials? I found the following ones on clinicaltrials.gov: NorUDCA (phase 2), LUM001 (phase 2), Simtuzumab (phase 2), UDCA + ATRA (phase 3), Vancomycin (phase 3).
Wow, I don’t know enough about PSC to comment on this. I think we (I) need more laymen terms. I know I have PSC… elevated liver enzymes. Bile duct deterioration. No medication or treatment to aliviate my symptoms. Not a candidate for transplant. Many other symptoms that go along with PSC. I’ve gone from very active to virtually not able to do even The smallest tasks with out being fatigued and gum drop perspiration. So, in a nut shell, I playing a waiting game.
It sounds like you are not getting medical support. Are you thinking clinical triale may be a way to get some help?
For people who want to understand phases of clinical trials.
There may be some phase 3 clinical trials starting up if the phase 2’s look good. As PSC is rare, and you have not had a lot of medical intervention, maybe keep track of these to apply?
Hoping that you can get a proper diagnosis and some treatment!
Ruby, I suffered ulcerative colitis for mant years resulting in a total colectomy. Two years ago after 36 years posting op, I’m diagnosed with PSC. I do believe this is a proper diagnosis as per my liver biopsies and MRI’s. it can’t be determined how long I have been developing this disease,but my GI believes probably a long time. I just go by what I’m told and what I shared previously is what my GI shared with me. I do have a large non malignant mass on my liver which is being monitored on a regular basis. I have blood work done every three months and MRI every six. I have suffered three bad falls since my diagnosis and wondered if anyone has a mobility problem with their PSC? I am thankful for the information I have received from these sites. I have learned a lot.
I’ve had PSC for thirteen years and have participated in research and clinical studies. Of the drugs you mentioned, many informed PSC patients think NorUrso is the most promising. Oral Vanco has shown a lot of success with children and some success w adults. Trials for NorUrso are ongoing in Europe. Good luck finding a study.
I was in a open label trial for LUM100. I had to quit after 8 weeks of a twelve week trial because I experienced extreme fatigue and weakness. The doctor said I was the only subject with that problem. Everyone in the study had diarrhea, including me. I still have less energy than I did before the trial. We have to wait for the published results but I would not recommend the LUM100 trial.
Thanks Jennifer. I am not surprised that Lum001 caused diarrhea as its action is to prevent the uptake of bile acids in the intestines (normally, about 90% of bile acids are back to the liver through the blood circulation). So, on one side it is beneficial for the liver as there are less (toxic) bile acid circulating, on the other side however there are more bile acids in the darm, causing the diarrhea.
Personally, I am also convinced NorUrso will be the best solution.
Did anybody experienced it already?
You might be able to find out quicker than I can because the Nor-Urso trials are only in Europe. The lead researcher is Dr. Trauner in Vienna, Austria. He is a respected PSC researcher. There is probably a trial in Germany. You are correct about the cause of the diarrhea in LUM100 trial.
My hepatologist is associated with Vanderbilt University in Nashville, TN. I was going to do a clinical trial that is going on right now, but my numbers increased and it was determined that I needed to take prednisone and something else (can't remember the name offhand) to try to get the numbers back down. Therefore, I was not approved for the trial. I was really disappointed at not getting to be in the trial, but I have to believe that things work out the way they're supposed to. At least I got a free endoscopy, colonoscopy, liver biopsy, and several MRIs out if it!! :)
You might have your physician call Vanderbilt and see what other trials they might be planning or they might be able to put you in contact with other places conducting trials. I got the impression from my doctor that there are others going on around the country.
I tried to get into one psc trial but was rejected (my liver values were too close to normal - it would have been difficult to know if treatment helped…)
Other psc trials not listed above are e.g. Xifaxan (phase1 ended recently) and btt1023 by biotie (phase 2 starts next month) and oca by Intercept (phase 2 started two months ago).
So probably not much experiences to share yet on those.
Hi. I have been involved in the Simtuzumab (phase 2) trials for a year now. You have to have an MRCP, MRE & Liver biopsy done at the beginning of the trial, at the end of your first and your second year participating. You will give yourself a weekly injection of either one of two study medications or a placebo. The best part of the whole process for me is that I get very detailed labs done every month for free which I then pass on to my hepatologist. Has saved me quite a deal of money since they do these in the study. They also do an EKG each month. Hope this helps.
OCA phase 2 trial for psc starts soon, though it is depressing to see that it lasts until 2019.
One earlier oca trial was halted early because of clear success, so maybe psc has similar results…
Here is summary of oca trial for Nash liver disease:
I just wanted to give couple of high-level updates on clinical trials and the search for PSC cure or treatment.
First the bad news.
Two recently concluded phase2 trials were somewhat disappointing.
Simtuzumab and LUM001: These two were phase2 trials, but unfortunately, both seem to have failed (or more precisely, they showed some improvement, and analysis on the results still on-going, but sounds unlikely that these will be continued further as such - still some potential but seems low-priority).
Then the better news.
This is still being studied in several clinical trials in different places. Stanford University phase3 study results have been delayed (e.g. one key investigator left Stanford), but the trial is expected to end in June 2017. Since there are no corporate sponsors and no patents in force, this will not go for FDA approval (unless somebody has extra $100M million laying around). However, it is already available off-label, if doctor is familiar enough to prescribe it. Many PSC patients are already using this successfully i.e. have stopped the PSC progress.
Nor-udca (also known as Nor-urso, or oral 24-norursodeoxycholic acid):
Phase2 trial results in Europe were very promising. Preliminary results were announced at The International Liver Congress in April 2016. It seems phase3 clinical trial is being prepared now (with the intention to fully comply with all regulations and requirements for new drug approval in Europe/USA). So if things go fine, I expect that phase3 trial will be announced this year (hopefully also started this year). Planning the phase3 trial is not easy so I understand the delay. Anyway, I’m hopeful that the discussions with FDA and its EU counterpart go smoothly and well-designed phase3 trial (that can lead to approved New Drug Application) takes place. Maybe 4-5 years from now in pharmacy near you?
Several other promising phase2 trials are also on-going. Here are few of them:
OCA (Obeticholic acid): This has already been approved (breakthrough designation) for PBC as a first new drug in the last 20 years or so.
Fecal Microbiota Transplantation
ORBCEL-C (selected Mesenchymal stromal cells derived from human umbilical cord, EU Merlin project, phase1 and 2 trials announced)
For more details, here is link to most of the PSC clinical trials since 2012:
Great info. I hope that more work than don’t.
Great information Ted. I try to keep on top of the studies/trials for my 6 year old daughter who has the disease, but you found several more trials than I knew existed. Thanks for brightening me and my wife’s day with some good news concerning this battle. Best wishes, David
You can also find all clinical trials worldwide at TrialApp (free download)
Here is another link to the ongoing trials for norUrso. Published in May 2017 so fairly current as of this post. It is quite technical and lengthy; however, it contains some very good information and, more importantly, gives us PSCers hope that there is work being performed to solve our challenging problem!!