Min-102 ?’s

[Travzw]

Hi. Just wanted to ask the ones that are currently on the Min-102 trial if they’ve seen any improvements. I am in the US and talked to the coordinator today. I may be going for the first visit on October 19th. Please let me know your thoughts on your progress.

[wilburlois15]

Hi.

I've been on the trial in the UK since early June. I'm reasonably sure I'm on the drug because I've had some significant water retention and my weight has increased by as much as 10kg (6kg currently).

As far as I can tell I haven't seen any meaningful change. I'd like to think that my balance has improved and I am better at walking around for reasonably short distances, but my usual daily walk taking my kids to school in the mornings has not improved at all. Aside from getting about, I'd say that the one thing I have noticed is an increase in aches and pains in leg and arm muscles and some reduction in spasms in my leg muscles when I am sitting or lying in bed. All quite marginal and nothing unequivocal though.

Angus

[Travzw in reply to wilburlois15]

I’m trying to figure out if it’s worth driving 12 hours to the hospital that accepted me. Do you feel it’s worth the added side effects? I already have lymph edema so I have to wear compression socks a lot now. I’ve also found that marijuana helps with the evening nerve pain and spasms so I would have to quit using that to be on the trial.

[StevenSims in reply to Travzw]

Hi,

I'm in the Uk but I'm doing the trial in the Netherlands, I've been taking the drug for about 6 weeks now and the thing I've noticed is my night time pain has subsided and I think my spasms have eased! I've also gained 9lbs and have a bit of swelling in my calfs! It's early days yet but I think it's defo doing something?

All the best

Steve

[Travzw in reply to StevenSims]

I feel that since I already wear very high compression socks that it won’t be too bad if I get some swelling.

[wilburlois15 in reply to Travzw]

12 hours is a long drive and given it takes me 1-2 hours to get to my hospital, I can't really comment. However, on the treatment itself, it's still early days. I think one of the problems is that as far as I know there has never been a "cure" for any peripheral neuropathy (MS, AMN, anything) and as a result no-one really knows how the nervous system repairs itself, if at all. It's all very well curing an ALD-mouse or rat but we are an order of magnitude larger. Furthermore, after 15 years of steady decline I am pretty sure my expectations about the functioning of my peripheral nerves are totally different to what they would be in a normal person, so no idea what to expect.

I think more pain (muscular/joint pain as opposed to neuropathic pain) is a reasonable expectation after years of walking/standing badly. And fewer of the involutary problems like spasms, etc. Would guess that any significant improvement in gait probably requires a lot of physiotherapy and that is another thing that we're not supposed to do.

A bit of a leap into the unknown. If it works...

[KennyInPA in reply to Travzw]

Hi Travzw,

I am starting the trial next week in Boston and the trial has offered to pay for my airfare to go up. (Otherwise a 6-7 hour drive). That may make it more convenient for you. Something worth checking into.

Best!

Kenny

[Travzw in reply to KennyInPA]

Yes. They are paying for my travel either way I decide to go. I have to spend a weekend there so they are paying for a hotel as well.

[bsc78]

Thanks great Travzw! I just went for a screening at Stanford last week. I'm waiting to hear back. What site are you planning on visiting?

As an FYI to people in the US: There are still more spots available at all the sites: Stanford, Kennedy Krieger (Baltimore), and Mass General (Boston). If you're able, you should consider enrolling. It's important for rare disease patients to engage in clinical trials--it encourages more trials in this space.

[Travzw in reply to bsc78]

For some reason Boston is the one that responded. Baltimore is much closer to me and that’s the one that I signed up for but they never contacted me.

[bsc78 in reply to Travzw]

Call Connor Murray

, Clinical Services Coordinator II, Kennedy Krieger Institute, at 443-923-2750. The last I heard, they were still recruiting. Might be worth the call.

[Travzw in reply to bsc78]

I just found out that they are only accepting people that have been patients at the hospital. I guess it’s fine since I’ve never been to Boston. Gives me the chance to see a new place since I’ve been to Baltimore previously.

[bsc78 in reply to Travzw]

Just so you know, I'm on the board of ALD Connect and I contacted KKI. They said that they will see people that have not been seen there before. I think there might have been some miscommunication.

[Aaron98]

I ended up deciding not to participate in the trial, after having had extensive conversations with Connor Murray at KKI, and the rep at Stanford (which is the site that I would be going to). The reason for this is that my wife and I are not finished having children, and the trial necessitates using birth control for 2.25 years. This was a difficult decision, as I had been looking forward to participating in this trial.

My sincere thanks to all of you gentlemen who are participating. I am eager to see what the results will be.

[Travzw in reply to Aaron98]

I was hoping since most other countries has been doing it for 12 weeks there would be more info about how it’s working but I guess it’s different in every person so it probably won’t be much help for me.

[StevenSims in reply to Travzw]

Hi,

I think they started in December in Spain and the Netherlands and this link says they've started in the us?

ala.com/minoryx-therapeutic...

All the best

Steve

[Travzw in reply to StevenSims]

Yes. US has started. I go the 19th of October.

[KeithS]

I can't say I've seen any side effects or improvement. No weight gain or water retention and walking unchanged or a bit worse. I've been taking the meds for 7 and a half weeks. I either need a big increase in dose or I'm on the placebo :-|

[KeithS in reply to KeithS]

Up to 11ml for the past 5 days. Still no signs.

K

[florida59]

Hi I’m on the trial 2 weeks I travel from Ireland to Paris to take part I don’t see any great change yet I take 10ml every day

[KeithS]

I have a slight side effect! I am now taking 11.5 ml (I thought going from 10 to 11 was a waste of time!) and I have a slightly swollen left angle. My walking is definitely better than it was a couple of weeks ago but as previously mentioned it was worse than normal then. I'll keep you all informed and I'm also back down London next Friday.

[StevenSims]

Hi Keith,

Have you weighed yourself? I've been reduced to 9ml and I've dropped a kilo! I'm still 4kg up though! I'm back for my 12 week appointment on the 9th

All the best

[KeithS in reply to StevenSims]

Hi Steve, I'm probably a couple of pounds heavier than normal but nothing extreme. The swelling has gone down slightly but is still there. I have had some right hip pain the last couple of days, just an ache. Hopefully I'm pretty much on the right dose with limited side effects.

[KennyInPA]

I received by 1st dose and a month supply of MIN-102 today. Taste isn't bad. Will see where it takes me!

[KennyInPA]

I am definitely seeing some water weight on day #7, up almost 3lbs (1.3 kg). My appetite also seems to be heightened and I'm eating more, which is probably adding to it. Anyone else have any appetite changes?

[SongStream in reply to KennyInPA]

I was just reviewing this thread, and yes, my appetite seems to have increased which piqued my curiousity on whether this might be a side effect. It has now been 90 days into the study and my follow up is next Tuesday.

[KeithS]

Hi, I was at the neurological hospital from 9am until 6.30pm on Friday. They took bloods 4 times and I had to go to the University hospital for an echo cardio gram. Long day but I knew it would be. They booked me in for 2 nights at the hotel so we went to a show on the Friday night, brownie points I still think my walking is a tad better than normal but maybe it's just in my mind...

[KennyInPA]

I woke up last night with my left leg in some pain and definitely a feeling of aches in my joints. I believe this was mentioned above, I am feeling the pain. Pain is good though, something I haven't felt in awhile.

[KennyInPA]

3 days later I haven't had much pain and have actually felt slightly better with walking. Weight actually has gone down, which we are monitoring. This is definitely an interesting experience!

[KeithS]

Since my last hospital appointment I've put on 7lbs in 2 weeks! I thought my new meds tasted slightly different, more strawberry taste. My walking was poor yesterday, maybe due to lugging the weight around. The swelling looks no different, very slight. Haven't had any word back after bloods to say dose needs changing. Will keep you updated.

[SongStream in reply to KeithS]

Interesting comment on the taste of the drug. I've always associated the taste with "berry" and I lick the dose syringe like a kid.

[KennyInPA]

I am returning from a week long trip to Nevada where I can say I walked about 4-5 miles a day (as per my iPhone) and I never had one trip or fall the whole time. I did have one close call, but that was after a drink at the bar (I have limited myself to one a day). It definitely comes in spurts like it is working and then other times I feel like my normal self. Now that I'm in a few weeks I haven't had any leg swelling and my weight has been stable. I go to Boston next week for the usual testing.

While I was given a letter to transport all the medication for TSA to an from the site, I had my first TSA agent inspect one bottle of it today when coming through security. He gave it a good look and eventually said I was good to go.

[SongStream in reply to KennyInPA]

You can walk 4-5 miles in a day? Gulp! I'm pushing myself on the treadmill to walk just 1 mile in 30 minutes!

[KennyInPA in reply to SongStream]

Well that was definitely spread out throughout the day, plenty of time to rest!

[SongStream]

I just had to laugh at the thought. When I pick up my medication, I use a carry on with wheels.

[KennyInPA in reply to SongStream]

Haha I transport mine in the backpack, like a pack mule! It keeps me standing straight walking through the airport.

[SongStream in reply to KennyInPA]

I'd be more like the Hunchback of Notre Dame! Today I received my new supply and I forgot how much that thing weighed. : )

By the way, today was the 90 day mark and a full day event which also included almost 4 hours driving mostly stuck in Bay Area wretched traffic which really tested my relative AMN symptoms.. What was worth "the price of admission" was that they comped me lunch which meant that mostly anything in Stanford/Palo Alto was exorborant but satisfying after starting my day at 5 a.m.

[KennyInPA in reply to SongStream]

The food is the best part! We had some great fresh seafood in Boston last month.

[StevenSims in reply to KennyInPA]

Get yourself a cabin case with wheels! They wanted to give me 30 bottles on my last appointment but I took 20! The airport security staffs eyes light up when it went through! They checked 3 bottles and were OK!

All the best

[KennyInPA in reply to StevenSims]

I usually bring my roller case and last time I thought to put the box in there to lighten the load. (Duh!)! When going through security I just put it through the belt separate so they can check it out. One time every bottle was tested for explosives and I got full body search. I kid not when I say, full haha

I heard from my home nurse they may be upping my dose this week, which is good less to travel with!

[StevenSims in reply to KennyInPA]

Upping means more! 😂 You'll be ok

All the best

[KennyInPA in reply to StevenSims]

Darn it, I can't win..Haha The nurse said everything is kept very secretive and she can't confirm if I'll need an EKG or not, if they do indeed increase. Always an adventure. You as well!

[StevenSims in reply to KennyInPA]

The 12 week appointment will show what dose you need! I was told last time it won't be adjusted again! I didn't need a EKG she said its only needed if your weight gain is over 8kg since I've been on 9ml my weight is stabile! The 12 week appointment is a long day though!

All the best

[KennyInPA in reply to StevenSims]

I did do the 12 week appointment, it was definitely an interesting day. My weight has stabilized and I've been on 10.5ml for about a month or so. I'll definitely check in once I hear from them!

[SongStream in reply to KennyInPA]

On my 90 day appointment yesterday, I had an EKG performed and 4 blood draws, this along with neuro and weight monitored and BP (which didn't work). I had to turn right around after I left when I received a call to come back for my new drug supply. As I said, this day event started with me waking up at 5 a.m. and not getting home until 6 p.m. with 4 total hours in traffic. Today, was a day of recovery. It took a lot out of me. I guess I now wait to see if my dose will be changed. I already went from 10 ml to 8 ml. And I gained at least 5 pounds but I think this was just me going full whoopie and not sticking to my healthy diet but enjoying being bad! Of course I have to include that my gym time has diminished for 2 weeks. Got to get back on the program before I hit the point of no return!

[KennyInPA in reply to SongStream]

I did attribute some of the weight gain to the higher appetite. I did indulge for awhile and have since moved back to my sensible diet. All bets are off on the weekend though!

[SongStream in reply to KennyInPA]

Steve and Kenny, that is insane with airport security. It's already bad enough as it is but when you are carrying something suspicious, well, that's a whole new ball game.

[SongStream]

Is anyone here also participating at Stanford?