Clinical Trial – NASH Patients Clinical Trial Exchange

Galmed

The most important part of my Galmed story is what transpired after they removed me from the ARMOR double blind Phase 3 Aramchol trial. Shortly thereafter the Phase 3 trial was suspended and Galmed CEO Baharaff stated that participants would be unblinded (notified whether they were receiving Aramchol or placebo). But having been already removed they would not unblind me in spite of my repeated pleas. This alone captures the essence of Galmed. Galmed is rotten to the core. If you are considering participation in such a trial understand that you are nothing more than a guinea pig and collateral damage. That said here is my Galmed story.

Trial starts with participant signing Informed Consent. FDA guidelines establish important but non-binding guidelines. Section 1.28 states “informing of all aspects relevant to the participant decision to participate in trial”. Informed Consent created by Sponsor (Galmed) approved by Institutional Review Board (IRB) omits several key points of information while misrepresenting others

Aramchol drug that was approved for Phase 3 study had patent expiring in 2023 and so would never be marketed. Instead Galmed’s intent was always to petition FDA to substitute with another similar drug called Meglumine with patent protection to 2035 without reverting back to Phase 2.
Informed Consent states “Clinical trial policy insurance to cover the Sponsor’s legal liability … for injuries that directly result from the study drug or procedures required by the study protocol will be maintained by the Sponsor”. In fact such insurance is not required in U.S. as it is in other countries. I asked for the name of the insurance provider and no name was provided. Informed Consent further states “If you have a research related injury reasonable and necessary medical care will be available to you. The cost of care … will be paid by the Sponsor of the study”. Since Sponsor is apparently not maintaining liability insurance in U.S. and Sponsor is located in Israel the chances of recovering such cost of care probably slim.

Had I been made aware of these things I would not have signed up for the trial.

In July 2020 I started on the trial and made all required trial protocol appointments. March 2020 FDA published document describing how to conduct clinical trials during COVID. Sponsors and investigators were given option to conduct video visits, to collect blood using local labs, and mailing study drugs. November 2020 earnings call Galmed gave investigators discretion in conducting the trial. January 25, 2021 scheduled visit I asked investigator to allow me such discretion. I expected that I would be receiving COVID vaccine shortly due to my age and co-morbidities. Investigator contacted Galmed who refused such. Interesting to note that investigator who is sitting on Galmed advisory board wrote this article. On January 20, 2021, one day before deadline to inform investigator whether or not I would show up, I was informed that my participation was being terminated. I was the only participant at this particular location (Jefferson Hospital in Philadelphia PA). Other locations throughout the U.S. were unable to recruit any participants due to strict Phase 3 requirements. Or possibly prospective participants were not so naive and wanted to have nothing to do with foreign corporation with no track record of having marketable drug or knew FDA had nothing to do with approving Phase 3 of double blind trial. Galmed expected 2000 participants worldwide and was only able to recruit about 50 (i was told about such small enrollment by investigator in another location). In fact the most difficult part of NASH trials is participant recruitment.

Having had to endure 6 months of pre-trial qualifications, painful and dangerous biopsy and other tests, another 6 months in trial taking investigational drug, multiple blood draws, only getting paid to cover parking and gas expenses. While I have no way of knowing if I was receiving the drug or just placebo, my results showed no improvement. While Galmed shared my test results with Halegoua who in turn shared them with me, I was told Sponsors test for significantly more things than they share with investigators. Such tests may include biomarkers such as proc-3, elf, and other sophisticated tests only research community knows about. Is it possible Galmed had tests that showed significant deterioration in my condition?

In December 2020 Galmed announced an open-label trial (NCT04104321) to run concurrently with the double-blind trial that I was participating in. All participants in the open-label trial would receive the actual drug. Galmed stated that all participants in the double-blind trial would be given the option of transferring to the open-label trial. I informed Investigator that I would like to exercise that option. Investigator informed Galmed and then I was removed from the trial. While I will never know reasons Galmed wanted me out of Phase 3 trial (expected to last 5 years), my suspicion is that they knew I am on the real drug and not placebo, and that the drug is ineffective in treating neither my NASH or fibrosis conditions. I pleaded with Galmed CEO Baharaff not to throw me out from trial I gave year of my life and did not even get a response. On 1/26/2021 Galmed presentation to investors, CEO Baharaff stated all Phase 3 participants will be unblinded. While I was kicked out of the trial on 1/20/2021 it seems like a decent thing to do for Galmed to inform me if I was taking the real drug. But they did not.

While I can only speculate that I was on real drug and Galmed wanted me out, at the same time I was kicked out from Phase 3 trial, Galmed prepared informed consent for open label trial participants. All accommodations I asked for now offered to new participants

“In case of circumstances directly related to the COVID-19 pandemic preventing holding the visits at the site, the study staff will make every effort for you to remain in the study. They may communicate with you to coordinate an alternative location for holding the visit. Dr. xxxxxx may also choose, for safety reasons related to the pandemic, to replace the face-to-face conversation during the visit, with a phone or video call. In such case you will be notified in advance and the call will be coordinated with you. Do not hesitate to contact the site for any related questions you may have”

This is part of email from Dr. xxxxxx (I am not releasing name because this doctor did not do anything bad to me unlike Dr.Halegoua-Demarzio who in my opinion is a monster for what she did to me). Answer to my question why conduct open label study “Regarding Galmed, I understand that the decision to lend open label access to drug is the incentive that if folks opt to enroll into clinical research during this time of COVID, then we need to minimize risk for receiving a placebo. However, the study will open back up to randomization to placebo in the future, but render access to open label drug to those who elect to participate now. This was strictly an effort to optimize recruitment during a very difficult and challenging time” I am not buying this answer because I don’t think it is possible to get NDA (new drug application) approved with trial that is not randomized and double blind. I am using this quote to show that Galmed was accommodating patients in Covid just not me.

I also noticed in open label informed consent something quite interesting. When I signed mine side effects were itching, now Galmed stating they had one person with lung blood clots and one with gallbladder inflammation. And this is all from handful of people they signed up for double blind Phase 3 last year?

As far as my results? My blood sugar went a lot higher, my liver enzymes did not change, my bilirubin became dangerously high. And I developed shortness of breath and chest pains. Plus I am worried now about liver damage cause by investigational drug toxicity. And I am still unable to see doctors in person or have any tests. And when I finally get that COVID vaccine which Jefferson had ability to provide to me but did not, I expect my medical expenses from tests will cost me thousands. And who is supposed to pay for all this? Obviously me. So much for Galmed responsibility outlined in Informed Consent. Pretty good way to deal with such “responsibility” to throw participant out of the trial and pretend we never heard about you. Here is safe investigational drug such as Aramchol that Dr.Halegoua-Demarzio repeatedly told me as main reason she “selected” Galmed for her trials. And I told her about what happened to me, her response was “I am not your doctor”, go where you came from to get treated. Not even decency to ask Galmed what exactly they fed me. This is Dr.Halegoua-Demarzio with her angelic looking face and 5 star reviews (that cant be possibly be real if you read content) in action. And when I complained to WIRB about my symptoms I was called a liar. Here is organization that is supposed to protect trial participants and who pays for this “protection”? Galmed. And where do some WIRB executives work before they joined IRB? FDA. And this is full circle how sponsors, investigators, IRB and FDA all in it together to allow abuse of trial participants. (Circle actually completes with FDA executives move to become drug companies executives. Probably unlikely considering Galmed is foreign company. But there is a very good example how statins were approved in US https://www.fda.gov/media/110452/download. It is interesting that FDA sees this as success to approve such widely used medication that can do may horrible things to person with liver disease)

And as far as advising open label participants that the drug they will sign up taking may be substituted if FDA gives Galmed approval, not in new informed consent. I am guessing if Galmed wanted to keep them on original drug they would have said so. But why do anything beyond FDA requirements? Interesting wording in new consent: “Clinically relevant information pirating to NASH or of this research will be communicated with you when we Dr. xxxxxxx get it” Is it a joke? Insult to trial participants? Any wording of mysterious liability insurance is gone from new Open Label Informed Consent.

If you are considering getting on one of their trials think about what they can do to you. I urge every prospective participant to consider my experience. I hope others will write about their experience in clinical trials.

More About Galmed

why it is important for investors to know about patent but not for trial participant risking his life for drug that will never be marketable drug? Because Galmed is public company listed on NASDAQ stock exchange in USA. Securities Exchange Commission (SEC) requires Galmed to disclose all important information to investors. Food & Drug Administration (FDA) does not require any such disclosure to trial participants. Is it because trial participants second class citizens? I hope anyone reading this will post comments below. I think reason is rich people invest in stocks and participants of clinical trials according to statistics I read are mostly poor and uneducated. So why would anyone care about Guinea pigs? Is it even possible for Galmed CEO with annual compensation of $1,269,700, company that employs 19 people treat trial participants as human beings? In my opinion Allen Baharaff does not care.

I thought long and hard why Baharaff failed to show any humanity towards me, unpaid volunteer who trusted him for 66% chance I will get aramchol which was promised to me as safest NASH experimental drug. What would be Baharaff getting out of it if aramchol was approved by FDA? As CEO who owns 20% of Galmed, Baharaff may get few hundred million dollars. But Baharaff has no respect for people who endanger their lives to make him that obscene amount of money. He treated me like garbage and here are my thoughts why it is not possible for Baharaff to act like human being expected to act. As I read about him, Baharaff and his wife Roni Gilat-Baharaff part of Tel Aviv super rich elite. Articles written how he is fighting liver disease but in in my opinion the only thing he is fighting is his bottom line. I doubt Baharaff can understand that ordinary people like myself actually have feelings and care that I was possibly hurt by his investigational drug. She is managing director of famous Christie auction house, he is on board of art museum and member of Israel bar association. He is elite snob and you just Guinea pig and he does not give a damn about you.

Galmed filed annual report around the same time Informed Consent for Phase 3 trial was prepared.

Page 29

“The composition of matter patents directed to Aramchol expired on March 25, 2019 worldwide. We will not be able to submit an NDA seeking approval of Aramchol (free acid) prior to the composition of matter patents’ expiration date. However, because Aramchol is regarded as a new chemical entity, or NCE, following approval of an NDA, if we are the first applicant to obtain NDA approval, we may be entitled to up to five years of patent term extension in the United States with respect to such NCE, and provided that the use patent with respect to Aramchol in the treatment of fatty liver will still be in force when the approval of the NDA is received from the FDA. The non-extended patent term for such use patent, is due to expire on April 15, 2022 worldwide and on April 17, 2021 in Israel. The U.S. patent was extended by a patent term adjustment of 567 days, resulting in an effective expiration date in the U.S. of November 3, 2023. In addition, a term of data exclusivity of up to 5 years will be available for the first approved clinical use of this NCE in the U.S., if Aramchol receives regulatory approval. Although we believe that we may be able to protect our exclusivity in our field of activity through such use patent portfolio and such period of exclusivity, the lack of composition of matter patent protection may diminish our ability to maintain a proprietary position for its intended uses of Aramchol. Moreover, we cannot be certain that we will be the first applicant to obtain an FDA approval for any indication of Aramchol and we cannot be certain that we will be entitled to NCE exclusivity.”

Galmed talked about uncertainty of continuing with new compound Aramchol salts with investors but not with trial participants. Even if FDA grants galmed right to substitute aramchol acid with aramchol salt in phase 3 trial, would you as trial participant want to know if aramchol salt went thru the same phases as aramchol acid? Will it be in informed consent? I doubt it.

“Our potential development of Aramchol salts may not result in improved bioavailability compared to the existing form of Aramchol. Furthermore, although we have submitted patent applications for our Aramchol salts in development, there is no assurance that we will receive any patents for them, and even if we receive one or more patents for our Aramchol salts in development, they may be of little or no commercial value.

As part of our research and development studies, we have confirmed that several Aramchol salts have improved solubility as compared to the existing form of Aramchol acid. In 2014, we submitted new patent applications to protect such salts and we will need to conduct an appropriate bioequivalence study, or studies, of the biological equivalence of two proprietary preparations of a drug.

If we commence animal PK studies and formulation development in order to test the bioavailability of the Aramchol salt compounds, the results might not support the claims sought by us. Success in our earlier pre-formulation studies does not ensure that later studies will be successful, and the results of later studies may not replicate the results of our prior pre-formation studies. Furthermore, either or both of the animal PK and formulation development studies may fail to demonstrate that the Aramchol salts result in an improvement in solubility and bioavailability. Any such failure may cause us to abandon the Aramchol salt compounds and may delay development of other product candidates. If the animal PK studies do not support our claims, the completion of development of such potential product candidates may be significantly delayed or abandoned, which will significantly impair our ability to generate revenues and will materially adversely affect our results of operations.

There can be no assurance that the U.S. Patent and Trademark Office, or the USPTO, will issue any patents based on the patent applications that we submitted to protect our Aramchol salts, nor, should the USPTO issue any patents to us with respect to the Aramchol salts, that we will be provided with adequate protection against potentially competitive products. Furthermore, if the USPTO issues us one or more patents for the Aramchol salts, there can be no assurance that the issued patents will be of any commercial value, or that private parties or competitors will not successfully challenge these patents or circumvent these patents in the United States or their counterparts abroad. In the absence of adequate patent protection, our business may be adversely affected by competitors who develop “comparable technology or products”.

When a company in S.E.C. filing talks about materially adverse effect, what they say to investors we are unlikely to remain as a business entity and our stock will become worthless. What Informed Consent should be saying is that the trial will be stopped if such materially adverse effect takes place.

New patent was given to Galmed on 12/1/2020 long after I signed informed consent. Galmed initially filed for patent in 2014 and said above there is no guarantee US Patent office will grant such application. Why trial participants not entitled to know this? Because Galmed will do the absolute minimum that is required by law. I saw other biotech companies clearly showing patent expiration on their website. This kind of disclosure shows respect for patients and investors.

Even if patent granted it is still unknown if FDA will allow Glamed to substitute Aramchol acid with Aramchol salt. Aramchol Meglumine is salt version of Aramchol and considered to be NCE (New Chemical Entity) and was granted patent.

http://patft.uspto.gov/netacgi/nph-Parser?Sect1=PTO2&Sect2=HITOFF&p=1&u=%2Fnetahtml%2FPTO%2Fsearch-bool.html&r=1&f=G&l=50&co1=AND&d=PTXT&s1=10849911&OS=10849911&RS=10849911

And if you don’t want to read company reports provided to investors here is clinicaltrials.gov, primary source to find trials

https://www.clinicaltrials.gov/ct2/show/NCT04104321?term=galmed&cond=nash&draw=2&rank=1#contacts. Double blind study expected to complete in 2024 after Galmed lost or will lose patent protection. While Galmed does not refer here if participants will receive patent losing Aramchol acid or unapproved Armachol salt with patent to 2035, how you person considering trial will know what you are going to be on for duration of the trial expected to complete in 2027. If you go on assumption FDA will do right thing and do not allow substitution unless Aramchol salt is safe to substitute, I wish you luck. By definition there is no such thing as safe investigational drug. Right thing for patients for FDA to do is to require Galmed to go back to Phase 2 with Aramchol salts with the same number of people as initial Phase 2 people (247) and don’t allow Galmed jump line because it will cost Galmed lots of money. FDA has responsibility to give 2000 future Phase 3 double blind trial priority over Galmed money and schedule to get to NDA.

And lastly note Galmed added open label trial to Phase 3 double blind trial I was kicked out from with no explanation. If you considering it ask some independent party what is the purpose of doing this? How would FDA actually know at the end of the trial if participant got better by itself or on Aramchol? This is why double blind placebo controlled trials are done. The only sense I got out of one researcher about open label trials, they done for very sick group of patients. Expanding trial to participants with F1 fibrosis (less sick than Galmed double blind trial) makes no sense.

I had many conversations with investigators at different locations in US.

When you hear person breathing heavily and says ” to be honest it is been very difficult trial ….”. it tells you there are some people where honesty comes before Galmed money

Investigator with many trials including Galmed told me in email “I am not fan of Galmed trial”

You may want to call investigators who participated in Phase 2 and not doing open label or Phase 3 double blind or open label and ask them why not. May be they tell you more than standard “difficult to recruit”.

Jefferson Hospital

Don’t think that not for profit is some kind of charity. It is just a scheme not to pay taxes and get all kind of benefits as outlined in this article.

It is stunning how much money Jefferson makes and how executives getting millions in compensation. Billions in revenue, at least 30 millions in executive compensation and yet treat patients like garbage. And speaking of garbage, what percent of that executive compensation Jefferson executives need to give up and pay city of Philadelphia for garbage pickup? Or lessen tax burden on Philadelphia taxpayers? This is not for profit for you. It is all about profit.

Clinical trials are source of income for Jefferson. And this is just from private companies. N.I.H (U.S. Taxpayer funding) is where money is. $151M according to Jefferson.

You probably will be in clinical trial in institution you have some experience with. And it is very important to consider your prior experience when deciding to participate.

My overall experience with Jefferson over 10 years with one exception is horrible. CEO Klasko could not care less about patients subsidizing his 5 million plus salary (double average non-profit hospital CEO salary). This culture comes from the top, so if your experience prior to trial is bad it will likely continue during trial.

I had one doctor telling me that his residents (medical doctors right out of med school) practically walk on water and if I ever complain again about them no matter what they do, I will be asked to leave practice.

I had experience of having complicated procedure and Jefferson did not disclose that resident will be doing it. I had very serious complications and had I known in advance would never have agreed to have it done.

I had one doctor in charge of entire specialty practice who admitted that he never reads research publications and he is only one who decides what is best for patient. That incompetence on his part ended up with me getting totally wrong prescription.

And lies with billing are non ending. They tell you test is covered in overall procedure, just to find out they lied when you get huge bill. Test that would cost $60 in Quest, you get bill for $500. And I was told that test is part of surgical fee. Ultrasound that takes few minutes at doctors office is $1200. Why? Because Jefferson owns that ultrasound machine and not doctor who just runs practice out of Jefferson building. And if you walk few feet to Jefferson outpatient imaging the same sonogram is $100. And who owns the same Sonogram machine there? Jefferson.

Pain pill after surgery I told them I don’t want, was $37, they still charged insurance and insurance did not want to pay.

Jefferson practice performed test they did not have contract with insurance that allowed them to do that test, Insurance denied. I can always receive bill for thousands because when practice is not in network like that test, Jefferson is allowed to charge just about anything. It is not that uncommon when insurance prints on their explanation of benefits that you are not responsible for non-network charges. Do not ignore it. Bills will come and collections will follow. So should I expect Dr. Halegoua-Demarzio not to be part of that culture?

But not all hospitals are the same. There are hospitals and doctors who actually lots better than Jefferson hospital and Jefferson’s doctors. It is all about culture from the top. Jefferson treated me like garbage over 10 years while getting thousands from my insurance why I should be surprised that Dr. Halegoua-Demarzio treated me like garbage as well? Email me, I will tell you how to find better ones. Don’t look at review sites, unless people describe something specific. But in general good doctors are very few and most I met retired. In general younger the doctor the more likely he/she going to think he/she is hot stuff. I guess when they spent ½ million dollars on medical school they feel they entitled to treat you like garbage.

Conclusion

You may want to ask how is it possible Dr. Halegoua-Demarzio has 5 star Google reviews and not a single person ever complained about her? Most reviews say she listens to patients. She is not a psychiatrist as far as I know. Most reviews are from people who never review anyone and some don’t even have real name. All reviews at Jefferson are controlled by Jefferson so I would completely ignore them. Answer lies to sample population she gets for her trials. In Pennsylvania 69% of adult population are not college educated. People have a tendency to trust doctors. Prospective participants should understand that she is not their doctor. People tend to believe government, specifically FDA, is there to protect them. People generally don’t have inclination to research and ask for second opinion. Most doctors you ask if you should be on clinical trial will say it more likely to hurt than benefit you. That what I was told. Most prospective participants will have no idea that only very small percent (maybe less than 58%) of clinical trials succeed. Failure rate in NASH trials is much higher. Few prospective participants will research best trial for them, read business analysts reports, research publications. It is almost impossible for most. And even then it may turn out that the best trial for you is thousands of miles away. Will you move? Obviously not. And then you have your friendly investigator peddling you her trial because company that is a leader in NASH trials with great results did not give her business. Is there a reason for getting mediocre trials?

And then investigator will try to scare you how bad your disease will get if you don’t get on a trial. Lets say you have stage 1 fibrosis. It takes 6-7 years progress to next stage.

By the time it gets really bad FDA may approve NASH drug. This is what I was told “The natural history of progression from F3 to F4 disease (based on results from prior Gilead simtuzimab studies) is approximately 20% by 30 months. The risk of F4 disease progressing to a clinically significant clinical event is also approximately 19% over 30 months.” This is why I decided to stay away from trials and concentrate on losing weight because likelihood of benefitting from study is very small and chance to get hurt by it a lot greater.

And lets say you did all homework and decided to participate:

Then you should consider finances of the company. It may be less of concern with big pharma. But huge concern with small biotech company especially with limited pipeline (drugs in development). Take Galmed, they did IPO may be 5-6 years ago and raised 70 million on Nasdaq. They continue to burn money and continue to raise more money on Nasdaq by issuing more stock. Last offering 2/21 they were able to raise 9 million. This is nothing for company that needs to conduct phase 3 trial with expected enrollment of 2000. I read average company expected to spend 20k on each participant in NASH trial. More stock they issue more difficult is to raise money. It is real possibility company can run out of money and stop trial. If you Google “biotech cash burn reporting websites”, you can find website to get free trial and estimate if company you are considering has enough money to complete trial. That website just takes information from financial reports files with SEC. Bottom line if company can’t get money, trial will be stopped. Do not even think to rely on investigator opinion. They have no clue.
There is always wording in informed consent that allows investigator to remove you from trial for any reason. Or as they call it your best interest, which really means investigator or sponsor best interests. It is unlikely investigator or sponsor will agree to put specific reasons you can be removed from trial. But remember they can throw you out at any time without any reason. Just make sure you are ok with it. From my experience it hurts like hell to be treated like piece of garbage.
Look for wording promising you 5-7 years of free drug. This is just marketing ploy to sign you up.
Make sure you have guarantee in writing that all tests investigator receives will be shared with you.
Make sure you have health insurance. It is unlikely investigator will agree to cover your medical expenses if you get hurt from investigational drug. But process of going after sponsor should be outlined. Make sure you have everything including contact information. And verify it directly with sponsor. Obviously with small company like Galmed you should absolutely have health insurance.
You need to make decision if you want to be in trial where sponsor is breaking law and not reporting results on clinicaltrials.gov. If you decide to take risk with such company make sure you get all marketing materials from investigator and read business analysts reports to see what brokers told investors about success of specific drug. You should ignore all reports done by brokers that sell (underwrite) sponsor stock.
You need to have clear understanding how FDA approves drugs. https://www.fda.gov/drugs/information-consumers-and-patients-drugs/fdas-drug-review-process-continued To summarize there is very little FDA involvement after IND is granted (which is unlikely you will ever see) to multiyear process that leads to NDA application. Where is FDA role in this process that takes years with thousands of people taking investigational drugs for any given trial? To the best of my understanding is in tiny amounts of audits I already documented in my FDA post. Ask your investigator who approved whatever clinical trial phase you considering to be on. And see how she/he will answer. You may be told you have nothing to worry, DMC is in charge. While you will unlikely to know if DMC actually exists for your trial, this article shows how many things can go wrong https://www.nejm.org/doi/full/10.1056/NEJMra1510066 In 3/18/21 earnings conference call Galmed CEO announced “I’m happy to inform you today that the addition of the open-label part to our ongoing Phase 3 was approved in the US, Canada, Australia, UK, France, Belgium, and Spain and is expected to be approved in the coming weeks in Israel, Korea, Turkey, Mexico and Chile”. If you know which entity approved open label please post in comments below
You need to understand every word of informed consent which will be totality one sided in favor of sponsor and investigator. Fact that it was approved by IRB and ethics committee is useless. They are all in it together to take advantage of you
You should ask Investigator about purity of investigational drug. Sponsor suppose to disclose it in Investigator handbook. And you should ask question such as “what is half life of real drug I may be taking?” Knowing half life is important for many reason including participation in any other trials as long half life will preclude you from some trials. If answer is “proprietary” as pretty much what Cindi Miller was answer to almost every question I asked, tell investigator to take that drug and shove it in whatever part of his/her body you think is appropriate. And investigators will know thru epic system or social networking about your prior trial participation
You should look into using existing approved drugs off label to treat NASH. I started to look in Ozempic but side effects scared me.
You should pay close attention how you are qualified for trial. Currently standard set by FDA is liver biopsy. Most likely you will have fibroscan, MRI and then liver biopsy. I did not have MRI and I think it is mistake and probably was trial design by Galmed who wanted to save money on testing. I was told that liver MRI in clinical trial can cost $5000, so if sponsor of your trial wants you to go straight for biopsy and bypass MRI, knowing what I know now I would look for another trail. You want to have as much non evasive testing as possible before you agree to have liver biopsy. This may include PRO-c3, ELF tests, new devices that cleared by FDA based on handful of participants. You may hear that you don’t need biopsy and your disease diagnosis is clear enough for you to start on trial. But at the end you will be taking investigational drug for disease you may not have. Biopsy considered gold standard and don’t let anyone talk you of it. In my opinion possible danger of biopsy is magnitude of factors less than “safe” investigational drug you will be recruited to take. There are white papers circulating in Hepatology journal written by investigators probably with push from sponsors trying to convince FDA that liver biopsy is not reliable way to qualify for trial. I suspect this is done because sponsors can not recruit and investigators can not get paid due to such strict requirements as biopsy. I doubt it is so hard to run study presented in hepatology journal to create results someone want. I did not read any specific article on this subject as they are not free. I urge you to not fall for this tactic if FDA caves in and allows less strict testing to qualify for study. You are qualifying for study to take potentially dangerous investigational drug. You need to make sure you really have disease you are told you have. Biopsy is gold standard and if you told you don’t need it you may want to look for other study. Liver regenerates. You want to have best diagnosis possible. And you want to make sure your slides examined by more than one pathologist to make sure they arrive to the same conclusion. Insurance usually pays for second opinion, it is not that expensive to send slides some place else to make sure you have diagnosis you told you have.
If you still need answers before or when in a trial I would advise not to look for message boards that suppose to help patients. I found one called smartpatients. Run by person stating to be former Google executive. It has moderator, totally unqualified person that most experience is in virtual handholding and no substance. You would expect someone with resources previously working for Google will spend money to get someone qualified to help people to navigate thru clinical trials. When I brought it up I was promptly removed from the message board. Most people on smartpatients board with advanced cancer. But what struck me the most is one person asking if she should participate in some Phase 1 study because her potential investigator told her that investigational drug will kill her cancer. Not single person including myself told her that investigator has no way of knowing and Phase 1 is extremely dangerous, this is why it is phase 1. But this shows how no one should believe investigators on efficacy and safety of investigation drugs unless they can show something written to support that statement. And then you should still consider who wrote it and why.

About ½ century ago clinical trials were conducted on prisoners. Maybe best leave to that crowd.

They can always advertise here for professional guinea pigs here

You need to think long and hard why you want to get involve in trial.

Global pandemic, life threatening disease such as cancer is one thing. NASH, I am not sure risks are justified. And fact that everyone is against you is very important consideration.

Please look at results only and take them to a doctor that doesn’t do trials. And assess your risk. You life is not to make investigator and sponsor rich. Sponsor will make hundreds of millions with successful NASH drug. Assuming you are not F4 which probably will disqualify you from most trials anyway you should try to lose considerable amount of weight and see if your liver enzymes got better. I know easier said that done, but I was able to do it once. You should never ever forget you can die from investigational drug. Phase 2 is done on few hundred people. What kind of indication of safety is that? And that is assuming best case scenario. No biases in reporting results, etc.

If you still decide to participate in trial you have to be ok with idea you are nothing more than Guinea pig, don’t expect anything out of investigator and sponsor. You can be thrown out at any time. You have nobody to complain to. You will hear “you were told everything in consent, you signed it“. You are unpaid volunteer. You are to be used and thrown out when we no longer need you for whatever reason.

I don’t think there is entity left that I did not complain, Jefferson CEO Klasko, Jefferson President Webster, WCGIRB, Jefferson ethics committee, FDA, PA AG, senators, congressman, state representatives. Not even a response. The most I got out of Jefferson. We are still going to treat you as a patient (meaning we want your insurance money), don’t think because we thrown you out from clinical trial it has any bearing on your continuing treatment (abuse in my experience) at Jefferson. Needless to say I left Jefferson entirely.

The only place I have left to complain about Dr.Halegoua-Demarzio is PA medical board. I am sure result will be no different from all my other complaints.

I decided to stay away from trials and let someone else to endanger their lives from taking investigational drug. There are 40 trials going on now, most phase 2. Which I would never do anyway. Most phase 3 failed so far.

NASH drug from Intercept probably will be approved this year. I don’t want this drug, NASH / fibrosis improvement is small, risks with increased LDL is huge. Madrigal drug looks promising but reducing fibrosis is not primary endpoint. Akero, NGM, Hanmi reporting impressive results but all are Phase 2. All of them injectable. Number of big pharma doing trial as well. So I will take my chances and wait for approved drug. And I will read everything about that drug to decide if I should take it.

I understand new drugs need to be developed and they only way to develop them is to conduct clinical trial. But unless clinical trials conducted in transparent matter, there is liability insurance that sponsor must pay for, Investigator employers are responsible for medical treatment of trial participants if there is even small connection to investigational drug, FDA audits all sponsors, all FDA guidance has legal enforcement and trial participants actually get paid money that have some resemblance of what investigators, IRBs, DMC get and possibly some loyalty payments if drug is approved and becomes huge financial success, I no longer want to be taken advantage of. But how this dream above can become reality? By Guinea pigs demanding to be treated like human beings. This is how change is made. You dont need them, you are a tool to make billions from approved drug. They cant do it without you. Clinical trials participants union anyone? There are 100,000 of us at any given time. Lets change US from green to red.

There is big debate going on about paying clinical trial participants to participate in trials. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5728432/

I think it is just pretense to save money for sponsors. Take Galmed, I got $50 for each blood draw visit and only if Jefferson actually able to get blood from me. I actually had to come back at my own expense because Jefferson does not train nurses to take blood from people with difficult veins. So lets say Galmed would pay me 100 for visit or 200 or more for each visit. Paid for my parking. Would that be enough to make me risk my life for investigational drug? There is no money that would make me to do that. I decided participate for 67% chance of getting real drug. Would it be incitement to some people? Probably. Did NIH/FDA/IRB ever do study what % of sick people do it for money? No and they never will because it is a lot easier to offer some idiotic logic in interests to save money to cheap skates like Galmed (that has no problem paying over million to ceo and ½ million to other executives). I saw informed consent of other nash sponsor and they paid 3x of money Galmed paying plus parking and hotel. And even with that 99% of intelligent people would not consider as enticement.