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Clinical Trials Considering or participating in research studies

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Old Wed Jan 9, 2013, 12:39 AM
Neil Cuadra Neil Cuadra is offline
Join Date: Jul 2006
Location: Los Angeles, California
Posts: 2,520
Thank you to clinical trial participants!

Let's all say thank you to the many patients who participates in clinical trials.

Whether you chose a trial because you thought you had few other choices, because someone talked you into it, because you wanted access to a specific medication, or because you wanted to help the doctors or hospital with their research, you have provided a magnificent gift to future patients.

You've increasing scientific knowledge about the safety and efficacy of new drugs and drug combinations, treatment dosages and protocols, or other areas of research. You've contributed to the growing body of medical information that lets doctors save an ever-increasing number of lives.

Participating in a clinical trial is a wonderful example of "paying it forward." Being in a trial may have helped you with your own treatment and recovery but it has also helped countless other patients. Take a bow. You deserve it.
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Old Wed Jan 9, 2013, 12:56 AM
Heather8773 Heather8773 is offline
Join Date: Jan 2013
Location: Houston, TX
Posts: 254
Thumbs up

Like.. I was just reading about TT-30. It's all written to medically for me to understand any info?
Heather, wife of Ronald 36; dx PNH 2012; Dx VSAA 2013; eculizumab(Solaris) hATG 2/20/13 cyclosporine 400 mg daily. 37 units RBC and 15 units of platelets. Post BMT -pentam,vorconizole,valtrex, valcyte, actigall, Pepcid , prograf, magnesium.
10/10 MUD 10/10/13
Now no PNH or AA. Mixed Chimerisim
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Old Wed Jan 9, 2013, 01:37 AM
MDSPerth MDSPerth is offline
Join Date: Sep 2012
Location: Perth, Western Australia
Posts: 74
Hear! Hear! It is really suprising how much development has been made over the last couple of decades with bone marrow failure. It seems that its only in this timeframe that these diseases have gained a little forefront.
Sandi, partner of Paul 62, diagnosed MDS Intermediate 2; July 2012. Pancytopenia, Cytogenetics -7 +8 Chromosomes. Low Blast cell count. Currently on EPO & G-CSF and having great response. MUD found will be admitted to Royal Perth Hospital 27 March 2013 to start SCT process.
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Old Wed Jan 9, 2013, 01:43 PM
Neil Cuadra Neil Cuadra is offline
Join Date: Jul 2006
Location: Los Angeles, California
Posts: 2,520
A few tips about clinicaltrials.gov

The descriptions at clinicaltrials.gov use medical language that can't always be understood by us laypeople, but with a bit of practice you can learn to get enough information to know if a particular trial is of interest.

What Phase?

The first fact to notice is what "Phase" the trial is. Here's a good summary at Wikipedia:
Phase 1 trials: Researchers test an experimental drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.

Phase 2 trials: The experimental treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.

Phase 3 trials: The treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow it to be used safely.

Phase 4 trials: Postmarketing studies delineate additional information, including the treatment's risks, benefits, and optimal use.
There are slightly different definitions in the glossary at clinicaltrials.gov. They make the claim that Phase 1 trials are conducted with health volunteers, which I don't find to be the case for most trials related to bone marrow failure disease. They also mention that Phase 2 trials often involve placebos.

A Phase 1 trial may be the first time a drug is given to humans. Or it may instead be the first test of a drug' use for a given condition when its use is already well-established for other illnesses. The first test of a brand new drug may not be as likely to benefit the patients involved as a Phase 3 or Phase 4 trial of a drug that's already been through earlier trials to establish an effective dosage and compare its outcomes with other treatments. But a Phase 1 trial can be the first chance to get a drug that appears promising.

Phase 2 trials often answer the question "Does this drug or treatment protocol work?". Phase 3 trials often answer the question "Is this drug or treatment protocol better than other choices?" by comparing different treatments against each other head to head.

What else to look for

You can check who is running the trial, where it's taking place, and how long it will run. Ideally a trial would be at your own treatment center. If not, you'll want to know where you'd have to be, how often and for how long. Some patients are more able or willing than others to travel for treatment in a trial. Many trials are held at the NIH in Bethesda Maryland.

You should also read the Eligibility/Recruitment information to see if you qualify for the trial. For example, a study may be limited to patients who are newly diagnosed and haven't had other treatments, while other studies might be designed for patients who have failed to improve after other treatments. Even if you want to get into a trial, they may not want you if you don't match their research agenda.

Finally, to get a complete understanding of a trial and get past the medical terminology, you can talk to your own doctor and/or contact the person listed as the "Contact" (or the office of the Principal Investigator) to learn about what the trial involves and whether it might suit you, armed with your list of questions.

The bottom line questions to ask yourself are: What are the possible benefits? What are the risks? How do they compare with what I'll do if I'm not in the trial?

More help

Read How to Read a Study Record for tips on reading clinical trial descriptions.

You can also get help understanding clinical trials from the AA&MDSIF.
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Old Wed Jan 9, 2013, 08:58 PM
mausmish mausmish is offline
Join Date: Mar 2010
Location: Maryland
Posts: 453
Bravo for mentioning this, Neil! I always reply with a resounding, "Yes!" when asked if it's okay to take extra blood, marrow, tissue, etc. for research. My donor was also delighted that I provided some of "our" marrow to science. I also try to participate in other medical studies that require nothing more than a little of my time whenever I can.
Karen, age 62, dx MDS RAEB-2 1/8/10: pancytopenia WBC 2.7k/Hgb 7.4/Hct 22.1/Plt 19k; complex cytogenetics -3,del(5)(q14q33),-6,+8,+mar,17% blasts. MUD BMT Johns Hopkins 11/30/10. Dx tongue cancer 8/31/12. ok now. blog mausmarrow.com
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Old Thu Jan 10, 2013, 01:04 AM
Neil Cuadra Neil Cuadra is offline
Join Date: Jul 2006
Location: Los Angeles, California
Posts: 2,520
Originally Posted by Heather8773 View Post
Like.. I was just reading about TT-30. It's all written to medically for me to understand any info?
I'm guessing you spotted the Safety and Pharmacokinetics of TT30 in Subjects With PNH trial at clinicaltrials.gov.

Let's see if we can make sense of it from the "English" parts of the descriptions.

It's a Phase 1 trial, sponsored by Alexion Pharmaceuticals, to study varying dosages of TT-30. (To know what TT-30 is you have to look elsewhere, such as in PubMed. TT-30 was actually developed by another company, Taligen Therapeutics, which Alexion then acquired. But you may not care how the drug works or who developed it, just that it may help PNH patients!)

The trial has multiple locations: California, the Czech Republic, Italy, Poland, and the U.K. There's contact information (name, phone number, email address) for a nurse at the Norris Comprehensive Cancer Center in Los Angeles.

It's a "single-dose" study, which presumably means that they'll give you the drug only once, but it also says that "subjects will return for the required study evaluations over the next 59 days", which doesn't leave clear to me how often you have to be there over those 2 months.

The trial is available to people "18 years of age with a diagnosis of PNH & vaccination against meningococcus" as long as they don't have any of a list of medical problems, such as "abnormal renal or liver function". They are still recruiting patients even though they expect to conclude the study in February 2013. I guess if you start when the trial is already underway then you finish later than that.

Bottom line: I may not have understood the entire trial description, but now I would be able to tell whether to cross it off my list or ask my doctor about it.
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Old Fri Jan 11, 2013, 02:24 PM
Snuuze Snuuze is offline
Join Date: Sep 2010
Location: Rio Rancho, NM
Posts: 50
I agree with the accolades for those who participate in clinical trials, either for their own benefit or for others now and in the future. And let's not forget to thank Neil and Birgitta for their continuing participation and willingness to share their experience and knowledge of what's current in bone marrow disease treatment.
Sue, age 72; Dx 6/2010 MDS Int-2. Revlimid unsuccessful, began Aranesp 10/2010; additionally Dx PNH 2/2011, Soliris added 3/2011. ATG 5/2011, Cyclosporine 5/2011. Nplate 10/2011 to 10/2012 . Exjade began 12/2013 due to high ferritin level, discontinued 3/2014 because of increase in creatinine.
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Old Sat Jan 12, 2013, 03:49 PM
Birgitta-A Birgitta-A is offline
Join Date: Oct 2007
Location: Stockholm, Sweden
Posts: 1,918

Hi Sue,
Thank you! You know I have been following treatment of MDS since my dx 2006 and got much important info from trials and from support groups that I think have helped me to fight the disease.
Kind regards
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