Shandra
Forum Replies Created
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I did Phase 2, Part 1 in August-October of 2015. That was a placebo-controlled, dose escalation study so I may or may not have been on the drug, and if I was I have no idea what dose I was on. It was also only for 3 months.
I just screened for the extension study this past Monday. If everything looks good I’ll start in a few weeks! I’m excited because it’ll be a much different process than the first time. We are guaranteed the active medication at the most effective dose (derived from the results of the part 1 study we were in!). I know you know that but i wrote it out for others reading the forum 🙂 . There is no exercise testing in this study, so that’ll definitely be different!
I’m excited to start hearing some results from the Phase 2 Part 2 study. I know Reata started recruiting people in 2017 and enrolled the last person in November 2018. So that means some have already finished the year-long trial and the very last person will be done this November. This is just a guess but they might be able to give us some very preliminary results at CHOP this fall? Data analysis takes awhile but I’m optimistic that in early-mid 2020 we will start hearing some full results. Remember this was a pivotal Phase 2 trial, meaning the results are intended to provide evidence for FDA approval (no phase 3 trial). We shouldn’t get ahead of ourselves but if the results trickling in now are good, we may start hearing about the potential for FDA approval next year! That’s pretty cool to me 😀
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@gunnhild Can you clarify the dose you’re taking? Is it 200mg twice a day with meals (so 400mg total) or 200 mg a day (100mg with one meal and 100mg with the other meal)?
I am just interested in keeping track of the people trying it while we wait for further research 🙂
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When I was first diagnosed, I was a child so I didn’t make my medical decisions lol. My parents had me take it for about 5 years. Then, I enrolled in a clinical trial and had to discontinue Idebenone for the trial. I never started back on it again afterwards, and I personally experienced no difference in progression rate or cardiac stability. It has been almost 4 years now that I have been off of it and any other supplements.
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Here is the full paper, if anyone is interested in reading: https://drive.google.com/file/d/1qaEvn_0GQAbyvI0nVcaXGpaD7beigj8R/view?usp=sharing
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I can’t wait!!!!!! It’s going to be awesome 🙂
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I thought this was interesting, because it takes a different route to rescuing Frataxin. I’ll explain.
Our cells make proteins like this: DNA -> RNA -> Protein
So normally Frataxin is made like this:
Frataxin Gene (DNA) -> Frataxin mRNA -> Frataxin Protein
Because of GAA repeats in the DNA, FA patients have trouble with the first step ( DNA -> RNA ), which is known as transcription. Since we don’t make sufficient Frataxin mRNA, we don’t have enough Frataxin protein. But, we do have some.
The cool thing about this study is it takes a post-transcriptional approach. In other words, instead of trying to get around the issues caused by the GAA repeats in the DNA, the protein in the study (GRP75) works with the small amount of Frataxin we already have, and basically helps keep it around longer and get to the mitochondria where it can then do its job.
It’s definitely preliminary work, but I think it’s pretty cool. Instead of trying to make our bodies produce more Frataxin, or simply mopping up the damage caused by not having much Frataxin, this research shows we might be able to just help the Frataxin we already have!
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Hmm I don’t know what happened, sorry lol:
https://drive.google.com/file/d/1Q4zZ8NIcMFszr310LC250ajPNJ1t9cOY/view?usp=sharing
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Matt you should sign up for the research in Gainesville!! The FA Biomarkers study is still recruiting: https://clinicaltrials.gov/ct2/show/NCT02497534
Also I live in Gainesville so it’s a good excuse to see my friends 🙂
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This will sound like an odd question but I have a good idea. Do you have a wii fit @gunnhild ?
Your ideas sound good also!
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Hi! I was wondering if the people taking it are documenting the effects they feel? Like photos/videos.
If the drug is doing good it would definitely help move things along in terms of having official human trials if the people taking it in Europe shared the proof of their experiences with researchers here. Also I’d love to follow their experiences. I am a science student but also a hopeful FAer 🙂