New clinical trial.

[monkeybus]

I'd sign up for this if I had the time.

minoryx-advance.com/english

Enrolling now. Looks promising.

[bluejadedwho]

Never anything in Canada. Shame.

[wilburlois15]

Thanks for this. According to my consultant at NHNN in London, I think participants in the UK are being selected by the hospital/Minoryx rather than this platform. May be different elsewhere?

[deengo]

I signed up for it and got a response but it's not going on in my area yet.

[KeithS]

My sister has just pointed out your post (I usually get email alerts on Sunday evenings). Well spotted MB, signed up this morning. I'm not that local to London but willing to travel. Any glimmer of hope in treatment is better than none.

Emailed Robin Lachman too, he's in charge of the London trial but got an auto reply to say he's off until mid April. Will keep you all updated if I hear anything.

Regards Keith

[mvruggink]

I applied as well even though I am in the United States.

Mitch

[JasonHarnum in reply to mvruggink]

the US site is at Stanford. Where are you located Mitch?

[mvruggink in reply to JasonHarnum]

I am located in Grand Rapids, MI

[Aaron98 in reply to mvruggink]

I live in western Washington, just a short plane ride down to California. I signed up. I wonder if they will consider me.

[monkeybus]

I signed up. Looks like I'll have to find the time.

Let's stay in contact with this one, folks. We might eventually get a result.

[tbird]

I've been "diagnosed by a doctor with AMN" and genetic testing was "performed to confirm the diagnosis of X-ALD", however I'm 68 years old and they want males between 18 and 65. AGE DISCRIMINATION?!?!?! What's up with that? When you're over 65, you're not worth messing with?

I'm retired, I feel like I take good care of myself (diet and exercise), I can't walk without the aid of a walker or forearm crutches, I have urgent urination problems---I think I'd be a good candidate for a clinical trial!

I took part in the Kennedy-Krieger Adult Lorenzo's Oil clinical trial but, of course, I was "only" 60 years old then.

So does anybody have any ideas on the age limit?

[monkeybus in reply to tbird]

You are right. Ageism, pure and simple.

If it was me, I'd lie about my age.

[tbird]

Good idea, monkeybus. People are always telling me I don't look my age!

[JasonHarnum in reply to tbird]

ask for an exception...

[KeithS]

No news as such from London hospital, they just told me I needed a letter of referral from my neurologist to have any chance of being accepted on the trial. However, they already have plenty of people by the sound of it. After 3 emails I finally got a letter at the end of last week, a copy of the letter my neurologist had sent to London. Unfortunately things have dragged on so long I think I've probably missed the boat...

[homeworking in reply to KeithS]

If any consolation, I don’t think you’ve missed the boat. I was contacted last night by the research nurse who has pre-screened me for a pre-screening! Will be interesting to see what comes of it.

[KeithS in reply to homeworking]

I got an email on Friday afternoon. I tried to ring the nurse twice but I never managed to speak with her. I have left my number on a voicemail and replied to the email leaving my phone number too. I have printed off, read and filled in all of the forms. Hopefully I will speak to the hospital on Monday. Small steps.

[KeithS]

Hi, I finally spoke to the nurse on Friday. She told me to get a blood test from my own doctor and asked me if I was allergic to shell fish as they use as a dye in MRI scans. Thankfully I'm not allergic. She asked if I could attend the hospital on July 12th for a full day of assessments. Not a problem and so she told me she'd book a hotel for the 11th. When she confirms the booking I'll ask about travel expenses. I told work about my plans via email late on Friday. Probably find out tomorrow if they'll back me in terms of paid leave.

I'll update when I know more.

Keith

[homeworking in reply to KeithS]

This is the nurse in london, Erla? She called me on Friday as well - I’m now booked in for July 21st. Look forward to exchanging notes!

[KeithS in reply to homeworking]

Yes, Erla. I rang her 3 or 4 times during the week, she rang me once when I was in a meeting. I finally got through to her on Friday.

Good luck

[KeithS]

Had more email correspondence with Erla today. She's booking a hotel on 11th of July for me and my wife. I have to keep my receipts for the train to get reimbursement for my travel expenses.

I'll wait for her to confirm the hotel booking before reserving the train.

[monkeybus in reply to KeithS]

How long will you be in the hotel for, Keith?

[KeithS in reply to monkeybus]

A single night, the day before as I live about 4 hours from London by train / car.

[monkeybus in reply to KeithS]

Cheers Keith,

And what's the deal with the trial?

How many times will you have to visit the hospital? Do you yet know? Need to fit it round my job. I'll ring them later.

Nice hotel?

[homeworking in reply to monkeybus]

If you pm me your email address I’ll send you the info I have from Erla about the trial process.

[KeithS in reply to monkeybus]

As mentioned previously, 9 visits over 2 years. My work are giving me time off to attend.

[monkeybus]

Right, I am in.

Pre-screening in either Aug or Sept. Just rang them up, "we were just about to ring you."

Seems they still need people. I'd advise anyone to jump in. They seem to be in no rush at all.

Looking forward to this. Light at the end of the tunnel, maybe.

How many of us are signed up for this?

[homeworking in reply to monkeybus]

That’s good news.

I think you’re right that they’re not in a rush, and there seems no requirement for those who get enrolled on the program to start the trial at the same time.

I just got my hotel confirmation for my visit to london for prescreening, which is actually happening over two days the week after next because they couldn’t get lachman and the mri scanner available at the same time.

Initially I’d thought it was very decent of them to provide expenses, but in actual fact I think it’s the least minoryx can do to make sure we aren’t out of pocket taking part in a trial that could well be very beneficial to them as well as us, our children and those others in the future who inherit this faulty gene.

[Aaron98]

This trial is exciting, and I have signed up to take part once it begins in the United

States (whenever that may be). That being said, I'm not quite sure how Minoryx will track efficacy of this drug. My reason for stating that is this - our disease, "classic" AMN, is typically slowly degenerative. So how would they be able to determine results in a two or three year trial? For instance, I doubt that my walking is going to get significantly worse in two or three years, regardless of whether I take this medication or not.

I don't mean to sound negative, just inquisitive. If anyone has information on what would be considered a successful trial of this drug, I'd be interested to hear it. Cheers!

[Vanliz in reply to Aaron98]

Hi......justa perspective my husband has AMN and is 60. He has gone from walking to wheelchair bound and lifting equipment in the last 8 weeks. Prior to that he was independently mobile with a walking frame. We were looking into the trial but sadly too late for us.

[monkeybus in reply to Aaron98]

I was thinking the same thing.

Though when they tested the parent compound, pioglitazone on mice VLCFA levels were reduced and:

"Mitochondrial levels were increased and the locomotor defects found in the double Abcd1/2 mutants normalized by pioglitazone."

I can only guess "normalised" means made normal, as in improved.

academic.oup.com/brain/arti...

[nikolahn]

Thank you for sharing the link, i have joined today the MIN-102 clinical trial II