I am persisting with the min-102 trial and yesterday had my 10th visit. Nothing particularly different about the visit: the usual 6 minute walking and balance trials, the annual MRI scan and echocardiogram and the questionnaires and meeting with a specialist.
What I did learn is that Minoryx are likely going to expand the trial to include younger people with brain involvement (ALD). This is new because one of the prerequisites for the original trial (way back in 2018) was a clean MRI scan. Now that it appears that the drug may help prevent adult onset ALD, they want to study the impact it may have on people who already have ALD. I think they mentioned 16(18?)-25 age group, but it may be wider than that.
As for me, the main impact of 4 years of taking the drug is that I carry around 10-12kg of extra weight due to the (o)edema. Physically, I am definitely slower on the walking test but I am likely better at the balance test. However, neither of these effects may have much relation to the disease. I am almost certainly less fit than I was in 2018 and I am also significantly more confident wrt the balance test. However, of course, we don't know what the counterfactual is, and it may be that I'd now be in a much worse physical state if not for the drug.
I don't particularly mind continuing with the trial because it's only one day per year and I'd have to go in and have the annual MRI and see a consultant anyway. The oedema is a pain but aside from swollen feet/ankles, it's not costing me much. I suppose the next question is when does this trial end, and what happens after that. It seems clear to me that the drug will be licensed if they are happy it reduces the risk of ALD because for healthcare, me in a wheelchair isn't a big deal whereas me in a bone marrow transplant or dead is more of a problem. The original aim of physical improvement of AMN symptoms seems to have been largely abandoned.
Hope all is well
Angus
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wilburlois15
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Currently, there is a clinical trial for: The NEXUS Clinical Study in cALDThe NEXUS clinical trial is a phase 2, open-label clinical study that has been designed to assess the efficacy and safety of leriglitazone in male pediatric patients with early stage cerebral X-linked adrenoleukodystrophy (cALD). Also, there were 6 patients in Min-102 who've developed adult cerebral ALD. I am one of them. It's possible they reduce and prevent far worse advancing lesions. I am waiting for ALD Connect virtual conference in November for more information about the results. It may buy more time for pediatric patients before a bone marrow transplant. As for me, I decided not to due to the mortality risk and of my age. 59. I will be 60 in October. I'll report more on my situation after the November conference. Let's not forget that MIN-102 may prevent AMN for those pediatric patients who have had bone marrow transplants. However, that is my own understanding. Please bear that in mind.
The study raises lots of questions. Unfortunately it's going to take a few years to get more results with a new group. Fingers crossed there is a decent outcome
Thank you for taking the time to give us all a really useful first-hand experience of the trial. I am sorry that you have not experienced any benefit apart from perhaps improved balance.
The history of drug trials is, I suspect, fraught with these kinds of inconclusive outcomes. However, you are just one participant, and it is only when all the results are in can the scientists make a more definitive statement of usefulness. As you note, if it could help prevent the onset of adult ALD, (or as Songstream notes, for paediatric patients) that would indeed be something extremely valuable.
I've had one per year since the study started (four in total). It is to check if the water retention is causing problems with the heart. Their plan is to take people off the drug if the heart is compromised
I just had my annual visit at Stanford Medicine two days ago. Brain MRI with gadolinium contrast; balance and sway tests; 6-minute walk test; ECG; blood and urine samples; and full neurological exam: sensation, coordination, motor skills, vision, hearing, and cognition. My EDSS score is 6.5: I need constant bilateral assistance (canes, crutches, braces) required to walk about 20 meters without resting.
I agree the drug does not impact the initial endpoints (walking and balance) but it may have neuroprotective properties, which is huge. My weight gain has been "healthy weight", as in muscles, not fluid retention.
The good news is that it's all free (the sponsor pays for everything), and they put all the reports on the patient portal the very next day.
Lucky you without water retention! As with everything health over here, it's free, but we don't get much feedback on the results. I'd love to see how they interpret the results of the balance/sway test
I have a potty mouth with the Romberg stance (balance) with the research coordinator. It's not my favorite and I will admit, my balance is worse. The question I have for the researchers and experts is does cerebral ALD make balance worse with AMN?. My lesions are also in the cerebellum which is part of the brain that controls balance. I may be wrong but my balance is worse, I feel.
The results of the Min-102/leriglitazone study continue to trickle out.
I just saw this, which came out two months ago. They found that Min-102/leriglitazone helped maintain "myelin integrity" in the brain compared to the placebo. I don't really understand the methods, but myelin integrity seems like something worth maintaining.
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MIN-102 clinical trial, is it worth?
Bone marrow transplant possibility and leriglitazone update 
I saw Ken, Aaron, and Songstream at ALD connect annual meeting today.
