Last week I went back to NHNN in London for the 12-week update. It was a long day starting with fasting bloods at 8am, breakfast and then the usual physical check up. Then the wait began. Even though I took back my bottles of medication, as instructed, I was to get a new set of bottles and only then take the medication for that day. This meant the nurse instructing the pharmacy and then waiting for the pharmacy to produce it. Eventually the new bottles turned up and I took the daily dose at about 10:50. The catch then is that on the 12 week visit they take bloods 2, 4 and 6 hours after taking the medication... lots of waiting. I'd finished by 5pm.
The nurse (just Ming this time as Erla had taken some time off) explained that the pharmacy can only produce the medicine on the day required and not before, hence the wait. Ming explained that they are trying to change this process but I'm not holding my breath.
Apparently the week before, the pharmacy had some sort of problem and the patient only managed to leave at 7:30pm!
Otherwise everything else is fine. Somewhat surprisingly, my weight had increased 4kg (from 76kg to 80kg) from the last visit which is likely a result of odema (water retention). My lungs were still clear though so for now everything is OK and I don't need to take a diuretic. I think I am seeing marginal improvements in my symptoms but it is at the margin and could easily just be part of a natural variability. School started this week and I've definitely lost some fitness over the summer as the school run is exhausting.
There are 9 people on the trial in London, of which I am number 5. Dr Murphy said they had room for a few others but they are not far from capacity.
Remember to take a good book to read!
Angus
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wilburlois15
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Thanks for the update. Very useful information. Have they given you any insight into how they plan to draw conclusions while only using 9 test subjects? I realize that there are more doing the study in other parts of the world, but it still seems a very small sample size.
Good question. My memory might be failing me but I'm sure they initially indicated that they wanted up to 20 people in London. From my conversations with them last week, they have found that with limited resources there are only so many people they can process... I think they only have space to do one person twice a week and with all the repeat appointments those few spaces fill up very rapidly. Maybe once most of the 9 have been through the baseline, week 4 and week 12 meetings then they will have time to add more.
I am 6 weeks behind you. Good to know what to expect for my 12 week update in hospital in Leipzig Germany. 16 patient in the trial so far in Leipzig. The same symptoms in my case too, water in legs, no walking improvements. They are going to perform an echo check, probably regarding the water in lungs.
I went last week to Boston it was 4 days trip. dr.kerim explained the process in detail I believed him what he said and then they took my blood and mri with contrast to check if I am mild condition.I thing I will get the medicine next visit or placebo.
I hope this medicine will help toeverybody in any situation.
Hi all, I am going to Mass General for my initial visit on October 17th. I'm very excited to be participating in this trial although not excited about travelling all the way from Dallas. Look forward to any medical breakthrough that could help us all!
The bright side is Boston has some great places to dine at which is covered by the trial (up to $58 a day). We definitely ate good! Haha I go back at the end of the month to start the drug.
Cheers for the update. I too was in London last week, on Thursday. It was my 3rd time in total. I'm 8 weeks behind you as I have my 12 week visit on November 2nd (I think I am patient 9). It's a Friday and as I knew it was a long day I've already booked a 2nd nights accommodation. We're talking about going to see a show on Friday night. Dr Murphy said she thought she could see a little water retention but I'm not sure? I'm still on 10ml at present, I hope to get an update / increase next week. I haven't noticed any difference in my condition at present.
I think you must have been there on the same day! Although I'd imagine your day was a lot shorter than mine. It took at least 3 weeks for them to tell me about the dose increase so it might take a little while to hear back.
Having said that, I took a call from Ming yesterday telling me to reduce dose to 12ml per day (from 17ml)! That was only about a week and a half after the appointment...
I'm now 4 weeks since my v1 appointment, and no word from anyone about adjusting doses. They did tell me that even people on the placebo might get told to adjust their dose to keep up the blindness of the test.
I haven't noticed anything to report from taking the medicine so far. If anything I feel like I'm getting worse, though in terms of progression of the condition I think I'm perhaps behind some other people on the test to start with as I'm fairly unaffected in my mobility for the time being (they told me I had just snuck in to the test on the scoring criteria).
I'm fairly convinced that I'm in the control camp, although I managed to put on 2kg between baseline and visit 1 somehow. There was no comment at visit 1 about any water retention, so maybe I was just piling it on for some other reason!
My pattern of urination through the day (and night) has changed significantly, my weight has increased by more than 5kg (from 76 to over 81kg) and my ankles are swollen so I'm pretty sure I am on the drug. Apart from that I am not seeing any consistent change. I think my balance and ability to walk around is better but any longer distance walking is just as hard.
As I have/had a variability in my symptoms day-to-day and week-to-week I am just not sure if any of this can be gauged as consistent improvement. Some days are relatively good and some are relatively bad. Today was a tough day getting the kids to school but yesterday was fine.
Also, I find I tire (muscular tiredness) more easily, either because I am focusing on balancing/walking much more or because I am dragging 5kg of water around with me. Or both.
Anyhow I'm still only quarter of a year into a 2 year study so maybe I shouldn't be expecting miracles...
After 7 weeks got a call to decrees the doses from 10 to 7ml daily. Ankles swalowing is getting bigger and bigger. Slowly. ALD symptoms within the margin
I had my V1 4 weeks visit on Friday I've gained 3kg and have a bit of swelling? I usually find it hard to gain weight and the doctor said its the side effects, I also have not had any improvement but I have noticed my nighttime pain has subsided which is great!
Thank you so much for sharing, guys. I appreciate the updates. I decided not to participate in the trial, due to the requirement of using birth control for 2.25 years. My wife and I are not sure whether we plan to have another child in that time, but we would like to keep the option open!
Did you say you were having back pain? I've noticed if I'm sat leaning forward my back hurts but not for long? Also I woke the other morning with pain in my hip I couldn't stand for a bit and was sore all day? Just wondering if you think it's the drug?
I am feeling more and more aches and pains. Not sure if it is the effect of the medication or the impact of dragging my rather heavier water-filled body around. Or both. Sitting here I can feel my stomach muscles, my glutes and my left ankle whenever I stand up, all of which I think are muscular strains. I banged my left elbow a few days ago and aside from the bruise, I could feel pain from my wrist to my shoulder. It's probably in my upper body where I feel it more because I am used to various aches and pains in my legs due to poor gait and falls.
The most extreme incident was a couple of months ago, when while sitting watching TV I got sudden pains in my groin. Felt like I'd pulled something and it was agony for a couple of days. I had a sudden feeling of foreboding that this was the way forward but fortunately it hasn't repeated since then.
I'm feeling pretty good at the moment walking-wise so hopefully that is a sign the drug is working. However, if the drug does work, I'd imagine that more aches and pains are part of the package.
Yes, hopefully. My point with the pain is that increased nerve activity and healthier nerves should mean we can feel all the damage we are doing / have done to our bodies from poor gait and falls.
However, on your point about VLCFA normalisation, apparently the drug doesn't do that. What it does do is prevent the VLCFAs causing damage to the nerve cells. Or that is how I understand it...
That is how I understand it as well Wilbur. It's not meant to fix much, but hopefully prevent things from happening in the future. Fingers definitely crossed!
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