Anyone participating in any of the 3 trials?

[Vik95]

If you watched the webinar you know that there are 3 trials going on for new meds to treat C3g. Anyone in any of them?

[dyates Ambassador]

I am participating in the Achillion ( ACH-5548) trial through the University of Iowa. Presently I am in the testing phase to ensure I meet all the qualification requirements. I am waiting on the final results from Europe on my biopsy and, should I meet the requirements, I expect to begin the trial within the next 7-10 days.

[Vik95 in reply to dyates]

I hope you get good results from this new medication. Will you please keep me updated on how you are doing while taking it? Am very hopeful for you and all of those who are fighting C3g! 🙏🏻

[DavidF_NKF Partner in reply to dyates]

Great! Thanks so much for sharing that. For people who are thinking of joining this, or any trial, but who may be reluctant to do so, perhaps you could share more of your experience on what this has been like for you so far- what have you needed to do? With what medical professionals have you interacted? Any paper work yet?

[dyates Ambassador in reply to DavidF_NKF]

Happy to!

The experience with the trial, thus far, has been good. The waiting game is the hardest part. Waiting for the trial site to get approved, waiting for test results, etc can get a bit nerve wracking. Otherwise the process has been good. I am working through the University of Iowa which is just awesome. The people and facilities are just incredible! My primary nephrologist is through the children's hospital there (Dr. Nester), so that has made it even easier.

So far I have had to do a full lab workup (blood and urine tests, ekg, and biopsy). The first part of the evaluation they are checking your blood and urine labs to make sure you meet the criteria for the trial, which I did. Once that was cleared they scheduled the biopsy, which was last Monday. The biopsy is now under evaluation in Europe to determine if I qualify (need to be no more than 50% sclerosed). That is where I am at currently, waiting on the final results. Based upon Iowa's review of the biopsy I meet, but the ultimate determination needs to come from Achillions review in Europe which I hope to know by end of this week or early next. If approved I will start the trial as early as next week.

Paperwork has been minimal. I had to sign a consent and a few other documents but other than that it was pretty easy. The biggest part was the decision making process as a family. Trying to determine if this was the right path to take. Thankfully Dr. Nester was very helpful in guiding me and my wife to ensure we were well prepared to take the leap. I am eager to begin and hopeful this will be the start towards a cure.

[dyates Ambassador in reply to dyates]

Update: I have been accepted into the trial (BTW, it is ACH-4471 not 5548) and will begin on Wednesday (7/18).

[Vik95 in reply to dyates]

Congratulations! Am hopeful for you and for our community of friends who battle this rare disease. Please communicate on how it is going once you have begun. It’s an oral medication right?

[dyates Ambassador in reply to Vik95]

Yes, oral is correct.

[Ghoskins in reply to dyates]

Great news on getting in. I’ve been with Dr, Nester the past few years, too. Hope it works for you and all involved .

[Vik95 in reply to dyates]

How are you doing? Just wanted to check in and see how things are going for you since you started trial.

[dyates Ambassador in reply to Vik95]

All is going well. Feel good but no changes in symptoms, positive or negative. They don’t tell you any results of the tests, so I am truly blinded of anything. All in all things are going well though. Appreciate you checking in and hope you are doing good as well.

[JMaharry]

I am in the screening process to begin the trial for Achillion ACH-4471. I expect to have the biopsy soon and hopefully begin the trial soon after that. I am working with doctors at the Ohio State University as well as Dr Nestor in Iowa

[scottiowa]

I am getting ready to start the blood work and stuff for the Achillion study. I tried for it 1 other time but my SSMACK level apparently wasn't high enough. They have now gotten rid of that requirement. But when I do my biopsy my kidney has to b 50% scarred. Seeing how I am down to 36% kidney function I think I unfortunately have that covered. My concern is,is it gonna do any good or do anything at all?

[scottiowa in reply to scottiowa]

I to am going through the University of Iowa

[Vik95 in reply to scottiowa]

Hi Scott. I’m hoping it does good for you! What does SSMACK mean?

[scottiowa in reply to Vik95]

I'm not entirely sure. The only way I even remotely understand it is the level of failure ur in,I think

[linfuller Ambassador in reply to Vik95]

I think Scott is referring to the s-mac level, soluble membrane attack complex, which is also known as soluble C5b-9. It has to do with activation of the terminal complement pathway, and can be used as a measure of disease activity. Many people with C3G have elevated C5b-9 due to alternative pathway dysfunction. It is also a clue as to where the problem in the complement system lies, as it is not the same for every patient. That info can help determine which medications might be most effective.

[linfuller Ambassador]

How is everyone getting along? Has anyone else been accepted? I'm so anxious to hear how you are feeling, though I know you don't get to see lab results during the trial.

[Vik95]

How is everyone doing in their trials? It’s been awhile since I’ve checked in. My daughter who is now 23 is still stable with fully functioning kidneys and continues to take 50mg losartan daily. She continues to see her doctor yearly who I am proud to say she has an amazing doctor, Dr Bomback. Our last visit was favorable. Less protein in urine. She’s has had to become gluten and dairy free and low sugar due to a year of suffering severe constipation. After every imaginable test with many different specialists with no diagnosis she went the holistic route and has to adhere to a strict diet and supplements. I still pray for a cure as the future and course of this disease is unknown patient to patient. I pray for everyone is has this disease and am thankful for the brilliant doctors who are passionate about finding a cure that targets the root of the disease. Thinking of everyone and hope to hear from you.

Vikki, Mom of daughter with C3g in New Jersey