Volunteering for clinical trials

Posted - June 2014 in HSPRF News

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Lot of interest being expressed

With the progress being made in the Australian research on HSP and potential drugs to cure it, we are foreshadowing the possibility of human clinical trials perhaps as early as 2016.

As a result, there is considerable interest being expressed by HSPers, and not just in Australia, in volunteering for clinical trials.

As part of the Foundation mission is “facilitating research towards a cure”, we are currently exploring how we can be most helpful and effective in matching HSP volunteers for clinical trials with the requirements of the researchers.

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There is a lot to be worked on in ensuring the multiple and complex considerations are factored in to coming up with the best answer. For example, the number of participants required for phase III of a clinical trial is somewhere between several hundred and several thousand… and it depends on how much variability there is in the effects of the drugs.

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It may well be that there are not enough qualified participants in Australia for this. Perhaps a multi-site clinical trial will be needed, including other countries. Any patient registry that gets developed will need to allow for such eventualities when it is designed, apart from issues such as compatibility with other databases, data ownership and access, ethics and of course, funding.

Rest assured that we are using the considerable lead time to work on this now. We will keep everyone updated as we always do. When the time comes, everyone will be informed and have equal opportunity in volunteering and signing up for participation in clinical trials. Not everyone will be medically suitable, so signing up is just the 1st step and does not mean you will be participating in the trials.

It is great that there is such a level of interest. The best thing that anyone can do now is to provide funding support for the 2 new projects that will hopefully lead to approval for a clinical trial. If it is to be, it’s up to us!

Comments on this story

andrew posted at 6:38 pm on 7 July 2014Reply

I have already emailed you previously regarding this matter. I have got HSP and also I have an 11 year old daughter in a wheelchair with cerbal palsey which makes life very difficult. I was wondering if I could apply to be on your list of candadates for the drug trials that you have been publishing. I live in England and I am prepared to travel to Sheffield if necessary. I have read your articles with great interest and i am very impresed with your results. I would be very greatful if you would give me the chance to be part of your trial in order to help give me a better quality of life.
Michelle posted at 8:15 pm on 12 July 2014Reply

I am so excited about these trials and am hoping to get my 14 yr old daughter involved,we are from WA and our specialist has told me he has never seen HSP as rare and as complex as my daughters,it affects her in ever possible way it can she has been wheelchair bound for 5 yrs and now over the last 2 yrs has affected her upper limbs,speech, eyesight and she has also had slight shrinkage of the brain,so fingers crossed for the trials because we will try anything at this stage.
grant posted at 2:49 pm on 14 July 2014Reply

I am signing up for any test that you may have
coming up when they need someone.
Claire posted at 3:01 am on 29 July 2014Reply

I have been diagnosed with HSP also my 7 year old son has this. I am most interested in signing for the drug trials that you have. We are based in the UK and this wonderful news is something that I have been waiting for. My dad and brothers all have this condition and I have seen how this progresses.. Anything that could help give my son a chance and improve/cure this condition would be amazing. This is the most positive research update that I have heard in a long long time… Well done and thank you for researching such a complex condition.
Alastair posted at 10:36 pm on 2 August 2014Reply

I have been diagnosed with HSP for 2 and a half years. I am interested in signing up for the drugs trials. I am the only one in my family who has this condition.
Donald (USA) posted at 5:57 am on 17 March 2015Reply

Where are the clinical trials going to be in Australia and in the United States and when would this happen in 2016? All my brothers have this disease except my oldest brother, now 65 years old. It started with all my brothers in our early 40 years of age. Each year it progresses, my legs are so stiff, and I trip and fall too much now. I have had many knee surgeries too, in my early teens, I played a lot of sports. My knees feel like rocks, they don`t bend at all, I get to certain point when I reach for something, and my legs want to collapse. I thought in the beginning when I was getting this disease it was all in my knees. I have been reading so much about this disease, I hate to see young people get this. It is very frustrating to me, I have been falling so much now, my feet make circles when I walk and they trip one another. I keep my upper body real strong so when I do fall, I am able to pull myself up from on my knees crawling to somewhere so I can pull myself up. This disease is getting to me, but I won`t give up, I played hockey since I was 7 years old, and I`m not a quitter, I want to lace up my skates again, but I`m 54 years of age now, trying to be active and it`s very hard to be.
Thank You,
Donald
- Editor posted at 8:08 am on 17 March 2015Reply
  
  Editor’s Note: Too early to say yet Donald. Here is the path forward as best we can determine at this point:
  1. There needs to be success with the 2 drug validation studies currently in progress (2015)
  2. Application to the TGA (Australia) to hold human clinical trials (early 2016)
  3. If approved, Phase I/II trials for safety and efficacy will be held in Australia with a relatively small number of HSPers, probably in Sydney, but possibly in another location as well depending on numbers of suitably qualified HSPers available (2nd half 2016)
  4. Again, if successful and approved, Phase III clinical trials, with much larger numbers participating, will be held in a location or locations, the decisions on which will depend on multiple factors including numbers and locations of suitably qualified participants; quality and adequacy of patient databases/patient registries; funding; logistics; and strategic partners. (2017?)
Roberta posted at 5:37 am on 3 December 2015Reply

I was diagnosed with HSP when I was about 40, but was able to continue working with no trouble until age 65. I am now almost 74, and have to use a walker to get around. I have a son who also started showing symptoms at age 40. He is now 48 and can barely walk. He is trying to get on disability, but is having difficulty getting a diagnosis from anyone, including the folks at Duke University, where he was sent for tests. He has the exact same problems that I started out with, but his progression has been much quicker. We are still trying to find a neurologist who will give him a diagnosis. We would BOTH be interested in the drug trials. Keep us posted! Thanks. I live in South Carolina, USA, and he lives in North Carolina, USA.
- Editor posted at 1:55 pm on 3 December 2015Reply
  
  Hi Roberta, we work closely with the SP Foundation in the USA http://sp-foundation.org/. Together we jointly fund the research currently underway, so they will have all the information about clinical trials for people in North America when the time comes. Make sure you’re signed up as a member with them.
emma posted at 5:24 pm on 12 February 2016Reply

I have a 6yo boy with HSP. I am looking for anything that will ease his condition as no-one serms to be able to help. Westmead children hospital and GPs just say its a monitoring game and to keep comfortable. But the aches in joints, gut pain and bowel pain, cuddles and love can not ease.
Rolland posted at 4:12 pm on 3 June 2018Reply

I live in Texas, I have been diagnosed with HSP since the age of 49. I am now 56. I am still working full time, I cannot walk a straight line or walk without loosing my balance. I walk like I am drunk is how I feel. I have begun using a walker at home now. I have no pain. I would to be part of any clinical trial you have for HSP. To improve my balance and quality of life and health. I have the SPG7, if this helps.
Josep posted at 11:33 am on 6 June 2018Reply

Hi, my name is Josep and I am from Barceñoma (Catalonia). My grandfather and my father suffered HSP (now they rest in peace). Of the five children of my father, I and a younger brother have HSP.

I am 54 years old and my brother 50; they diagnosed me at age 40. I worked until 2016, and since 2000 with crutches. I have a diagnosis of my neurologist at the Vall d’Ebron Hospital in Barcelona, which, if necessary, could be sent to see if we could be included in phase III clinical trials.
Thanks.