Clinical Trial

My daughter has been offered to participate in clinical study for pkd. Looking for any thought or cons to her doing this.

10 Replies

  • It would be an amazing thing for her to do, she would be helping people world wide with the results, I wanted to do the tolvaptan trail but was diagnosed with RA at the time so didn't do it, but I thank all those people brave enough to give it a go as I'm now taking it because it passed.

    Only she will know in her heart if it right for her, but a big thank you to her if she does it as we learn so much from people who are brave enough to do this for us. Jx

  • Thank you and she has decided to do this, its more a study than trials. We don't have the tolvaptan here in the US yet, so what she is doing is more a study to see if they can find a cure/slow it down someday. So more testing and monitoring for right now and in hopes that if there are trials they will let her participate.

    Good luck to you!

  • How old is your daughter? Is her PKD ex novo, or do you have some notion from familiy history about how quick it is expected to progress? I am aware that there is even considerable intra-familiar variability of PKD virulence.

    From what I understand Tolvaptan or any other treatment should be started early into the disease in order to slow down the progress, as once CKD shows up in eGFR, already a lot of damage will have be done.

    I have seen graphs regarding the progress of PKD over time and it looks that eGFR can remain constant over a long time, but once it starts falling, the decline will be relatively fast. The graphs look like a long horizontal line with a steep curve downward at the end, the x-axis being time and the y-axis being eGFR.

    I understand that the remaining nephrons (or whatsoever they are called) can compensate for quite some while for the damage done by the ever-growing cysts until a point of no return has been reached when the decline of GFR accelerates in direction towards kidney failure.

    Until they do not find some remedy on a molecular base, it is all about halting the disease. A cure there is not, for the moment, for the damaged kidneys and all the other systemic damage done.

    My nephro is considering to include me into a study as my kidney function is still relatively preserved (eGFR around 70, normoalbinuria and normal A/ Cr ratio). Tells me I might not be included because of my age, as I am 54, and ressources in my country of residence are limited while Tolvaptan is extremely expensive.

    Should I be offered treatment, I think I'd accept it. I am a bit wary because of the possible side effect on LFT's, as mine were completely deranged some years ago, and I am rather happy that they have normalized.

    As for the i creased thirst and urine output with consequent nycturia, I am not afraid of that. I do already have that, thank you. I drink water like a camel. Urine output >4l per day...

    Good luck for your daughter,


  • I have pkd and my stats are good. I have been refused tolvaptan as I got told my condition is deteriorating fast enough. I would gladly pay to start it now. Many thanks

  • Wishing you well Dean, and praying they find a cure soon!

  • I forget that I am from the US, we do not have the Tolvaptan trial here. What she is going to do is more a clinical study so they are just taking all her info, scans, blood, GFR tests etc and monitoring her, using her info for studying the progression. If trials become available they will let her know if they think would be good for her. They feel she is young and its at the beginning so they can monitor the progression.

    I worry because of our medical system and preexisting conditions and insurance. We do know what kind she has yet, as we never heard of PDK until her. She is 20 and still doing very well. As on now just small cysts on both kidneys, we just found out she had by an unrelated scan. They will start more testing with this study and let us know exactly where she is at as of now, and continue the study for a few years.

    She has decided to do the study in hopes of someday there will be a cure or they will find a way to slow this down for her and everyone.

    Good luck to you as well.

  • None of you has it, only her? I have read about a 10% being ex novo mutations.

    Must be terrible to have such a health care system. At least if you are not healthy and wealthy.

    In my country of residence not everything is well, either, but in general, I cannot complain about public health care. Not at all.

    Try not too worry too much. PKD is a very slow process. No good in having PKD in mind all the time. I basically forgot having the condition until it became symptomatic.

    Good luck to your daughter.


  • Thanks we are taking day by day, and trying not to worry. Was hard at first since was all new to us, but it has been helping just even with this site talking to people like you that say it can be a slow process, and not the horror stories I originally found on the internet. But as long as she is feeling good, we are just getting back to enjoying life.

    Right now we don't know of anyone else having it in the family so only time will tell.


  • Dean, you wrote 'fast enough'. Did you mean ' not fast enough'?

    Good luck,


  • Yeah my condition at the moment is not progressing fast enough for them to take action with tolvaptan apparently.