Polycystic Kidney Disease (PKD) Support Group
Polycystic kidney disease (PKD) is a progressive, genetic disorder of the kidneys. It occurs in humans and other organisms. PKD is characterised by the presence of multiple cysts (polycystic) in both kidneys. The disease can also damage the liver, pancreas and rarely the heart and brain.
Hello everybody,
I posted in another discussion that I'm in the Tolvaptan clinical research and after I saw some interest in that I thought I'd start a discussion about what is like to be in a clinical study.
Currently I'm in phase III of the study, or am for one more week. Phase III is a double blind study, meaning that neither I nor my study team know whether I am on the actual drug or a placebo. I believe I am on the placebo as I have zero side effects.
I am participating in the student through Universities Hospital located on the Case-Western Reserve University campus in Cleveland, OH. There are many other locations throughout the country that participate in the study. I live north of Detroit, which means Cleveland is about a five hour drive for me. The reason I'm going through them is because I got in the study the last week before it closed and they were the only ones who would take me. I had to drop everything and rush down there in order to get in.
Getting into the study involved an interview over the phone, to see if I was even close to qualifying. Then I had to travel to the hospital for some blood work, EKG, and MRI. Then I had to repeat that again one week later, minus the MRI. Then I was given the "low dose" for one month. After confirming I could tolerate that I was given the "high dose" and a supply of the low dose that I could switch to if I couldn't tolerate the high dose. Again, I believe I'm on the placebo and therefore could tolerate it with no problems or side effects at all.
Should an emergency situation ever arise that is becomes critical to know if Im on the placebo or not the study team does have the ability to find out.
Every four months I have to return for more blood work, in addition to an annual MRI and EKG. I'm now at the end of my third year, which is the end of the study.
Since I am currently in the Phase III study I am automatically approved for the next phase where I am guaranteed the actual drug Tolvaptan, unless my safety becomes at risk.
I have the option to drop-out of either study at any time, but I did sign a contract indicating I would do final follow-up tests. I can't imagine why I would drop-out, but I thought I would mention it in case somebody is curious.
I have to travel to Cleveland the next three Mondays. The first Monday will involve the end of study tests: MRI, blood work, etc. I also stop taking the drug. The second Monday is the beginning of the new phase of the trial, which involves paperwork, an exam, and tests. The third Monday is where I'll receive the drug. They will give me both dosages and if I can tolerate the high dose I'll continue taking it, if not I'll switch out. Then I have to go back in one month, then two months after that, then every three months.
It is very rare that I see the doctor in the study team. He only did my initial evaluation and then he reviews all my tests before they go off to the pharmaceutical. Who I really deal with is a team of nurses that are involved in several clinical studies. They run an office in the hospital, but it is separate from the traditional offices. The nurses are a lot of fun as they joke around a ton.
The drug comes in this HORRIBLE package. It is hands-down my biggest complaint. The pills are in a blister pack, one pack for each week, that I literally have to use tools to open. As part of the study I have to return everything, including any unused pills and even the empty packaging. All the pills must be accounted for.
I do take blood pressure medications along with other medicines and will continue taking them on the next phase. I CANNOT take any diuretics (a.k.a. water pills).
I'm hoping that next week I'll be able to verify that I've been on the placebo or not.
I posted in another discussion that I'm in the Tolvaptan clinical research and after I saw some interest in that I thought I'd start a discussion about what is like to be in a clinical study.
Currently I'm in phase III of the study, or am for one more week. Phase III is a double blind study, meaning that neither I nor my study team know whether I am on the actual drug or a placebo. I believe I am on the placebo as I have zero side effects.
I am participating in the student through Universities Hospital located on the Case-Western Reserve University campus in Cleveland, OH. There are many other locations throughout the country that participate in the study. I live north of Detroit, which means Cleveland is about a five hour drive for me. The reason I'm going through them is because I got in the study the last week before it closed and they were the only ones who would take me. I had to drop everything and rush down there in order to get in.
Getting into the study involved an interview over the phone, to see if I was even close to qualifying. Then I had to travel to the hospital for some blood work, EKG, and MRI. Then I had to repeat that again one week later, minus the MRI. Then I was given the "low dose" for one month. After confirming I could tolerate that I was given the "high dose" and a supply of the low dose that I could switch to if I couldn't tolerate the high dose. Again, I believe I'm on the placebo and therefore could tolerate it with no problems or side effects at all.
Should an emergency situation ever arise that is becomes critical to know if Im on the placebo or not the study team does have the ability to find out.
Every four months I have to return for more blood work, in addition to an annual MRI and EKG. I'm now at the end of my third year, which is the end of the study.
Since I am currently in the Phase III study I am automatically approved for the next phase where I am guaranteed the actual drug Tolvaptan, unless my safety becomes at risk.
I have the option to drop-out of either study at any time, but I did sign a contract indicating I would do final follow-up tests. I can't imagine why I would drop-out, but I thought I would mention it in case somebody is curious.
I have to travel to Cleveland the next three Mondays. The first Monday will involve the end of study tests: MRI, blood work, etc. I also stop taking the drug. The second Monday is the beginning of the new phase of the trial, which involves paperwork, an exam, and tests. The third Monday is where I'll receive the drug. They will give me both dosages and if I can tolerate the high dose I'll continue taking it, if not I'll switch out. Then I have to go back in one month, then two months after that, then every three months.
It is very rare that I see the doctor in the study team. He only did my initial evaluation and then he reviews all my tests before they go off to the pharmaceutical. Who I really deal with is a team of nurses that are involved in several clinical studies. They run an office in the hospital, but it is separate from the traditional offices. The nurses are a lot of fun as they joke around a ton.
The drug comes in this HORRIBLE package. It is hands-down my biggest complaint. The pills are in a blister pack, one pack for each week, that I literally have to use tools to open. As part of the study I have to return everything, including any unused pills and even the empty packaging. All the pills must be accounted for.
I do take blood pressure medications along with other medicines and will continue taking them on the next phase. I CANNOT take any diuretics (a.k.a. water pills).
I'm hoping that next week I'll be able to verify that I've been on the placebo or not.
1. Tolvaptan has already been approved for the treatment of hypervolemic and euvolemic hyponatremia. It is being marketed under the name SAMSCA (tolvaptan). You can find more information regarding SAMSCA on their website at: http://www.samsca.com/
Also, there is a very good video on that website. It is on the main page in a box labeled Featured Video
2. From what Ive been able to find SAMSCA costs as little as $1,000 per month, but what appears to be more accurate is closer to $300 per day!3
3. Tolvaptan for the treatment of PKD has been put on the Fast Track for approval by the FDA. You can learn more about what Fast Track means here: http://www.fda.gov/forconsumers/byaudience/forpatientadvocates/speedingaccesstoimportantnewtherapies/ucm128291.htm
Here is my perspective on the Fast Track approval: First, I started Phase III of the clinical study three years ago at the very end of the enrollment period. Im now one of the last people starting Phase IV, which is also expected to last three years. Once the pharmaceutical has started giving me a beneficial medicine they cannot stop giving it to me unless there is a safety issue. So, even if the study needs to continue, Ill be included.
Now, as I understand it the FDA review committee could, in theory, approve the drug at any time. Unless that happens I would suspect the drug would be approved in about three years. However, since it has already been approved for other treatments I would think it could move more quickly.
Thank you for that personal experience with the Tolvaptan trials. We applaud you for putting yourself out to be in the trials, all that driving and testing, etc. We thank you!
Here is the journal I mentioned in the other thread I would send you.
Water Prescription for PKD
Clin J Am Soc Nephrol. 2011 Jan;6(1):192-7. Epub 2010 Sep 28.
Design, setting, participants, & measurements In eight ADPKD patients eating typical diets, osmolality and volume were measured in 24-hour urine collections. The amount of additional ingested water required daily to achieve a mean urine osmolality of 285 45 mosm/kg was determined. Participants were instructed to distribute the prescribed water over waking hours for each of 5 days. Blood chemistries, 24-hour urine collections, BP, and weight were measured before and after the period of supplemental water intake. Results Five patients achieved the 285 mosm/kg urine target without difficulty. Mean urine osmolality decreased and mean urine volume increased; serum sodium, weight, and BP were unchanged. Daily osmolar excretion remained constant, indicating a stable ad lib dietary intake of solutes and protein over the 2-week study period. Conclusions The amount of additional water needed to achieve a urine osmolality target can be approximated from the urine osmolar excretion in ADPKD patients eating typical diets, providing a quantitative method to prescribe supplemental water for such individuals.
So, would it be fair to say, that if Tolvaptan does help us, then it would have the effect on us to make us so thirsty that we would drink that Gallon of water in one day whereas otherwise we would not be able to drink that much water thereby stopping the release of vasopressor, thereby slowing down cyst growth? I will be curious to see how much water you can drink or want to drink on the drug. I can't get more than 3-4 Liters in one day.
As far as the cost...will our insurance cover it as it has been covering my BP meds?
Be safe-with all that driving! Now for the 3 year wait for Phase IV to end.