Trial or not to Trial

[ourlife]

I am now trialling SAR302503 - and am giving extra bloods, marrow & DNA samples to help future research. I would be interested in your experiences if you are on or have been on a trial - please share any views about clinical trials that you might have.

[UPBEAT]

HI Ourlife.

I am very interested in your experiences trialing SAR302503. I recently attended a consultation at Guys as a possible candidate to trial either the SAR or INC424. I have elected to trial the INC and hope to start in September.

My condition is high red blood count and low platelet count. The symptoms I suffer are extreme tiredness, headaches, nausea, weight loss, night sweats and cramps.

I applaud you taking the SAR trial; committing to the frequent bone marrow sampling.

My own experience with bone marrow sampling has been unpleasant. Three very painful attempts before a successful sample and both times followed by a large painful swelling over the puncture site, which lasted weeks. So, I am fearful of entering a programme that requires frequent bone marrow sampling. Of course I shouldn’t be as SAR302503 it is hoped will improve the condition of the bone marrow and so perhaps be something of a cure whereas INC424 is a relief of symptoms rather than the causes.

I do wish you well and thank you for trailing this drug which may benefit us all.

[ourlife in reply to UPBEAT]

Hi UPBEAT, I am also at Guys - my first BMB was at my local hospital in Hertford and was one of the worst experiences in my life so far ! The team at Guys are superb and well practised and the BMB at the start of this trial was a much better experience. Although unpleasant it is vital for the research and hopefully I will benefit in the long run or at least help someone else - they also let my wife in with me to stop me making a fuss !

I had to stop all meds for 3 weeks before commencing this trial - that was a more frightening prospect than taking a new pill or having a BMB.

So far the drug has not yet reduced my platelet count 798 after week 2 Monday is my next blood test week 4,thankfully all other counts are OK.

The fatigue at present is worse but all other side affects I've had seem to have disappeared,(Flaky skin on feet,nausea,upset stomach,) My night sweats were few and far between it's early days and I'm hoping they have gone too.

I wish you well if you take the plunge and would be very interested in your progress both mentally and physically.

[UPBEAT]

Hi Ourlife,

Thank you for your response. Its good to hear you are making some progress and may it continue.

I have just today heard from Guy's that I am called forward for the first batch of tests.

I think that I will stay with the INC trial. I will keep in touch and let you know how I get along.

[ourlife]

Day 1 of cycle 2. Had blood taken on hours 1,2,3,4,6 and 8, so lots of waiting around at the hospital,not much fun but was glad of the rest! platelet counts down to 531 so heading in the right direction but as a result of the drug my creatinine is up to 123 which means my kidneys are not too happy ! There is no cause for concern yet as in the grand scheme of things that's not too bad.Advice is not to drink alcohol (odd glass of wine OK) and to consume plenty of water, though if I drink much more per day I fear that I will turn into a fish !

[ourlife]

day 15 cycle 2 platelet count 529 so not much of a change wbc dropped to 3.0 and rbc dropped to 4.17, creatinine down to 106 all out side of normal range.

have had a strange rash develop on my arms and neck and a biopsy was taken as it is not yet clear if the trial drug had anything to do with it !!

[StreetPastor in reply to ourlife]

Ourlife: Just want to say a BIG thank you for trialing

[hackett]

Thank you Ourlfe for you description of progress on the SAR trial. I need to read it a few more times to absorb it. I had hoped to get on the trial last June but had to "withdraw" at the last moment because in the withdrawal period my platelet count was going too high.Regardless it went higher still and as you wrote the 2-3 weeks were frightening. All for nought at that stage. I may have another opportunity to participate in another arm of the trial in the next few months. I will remain watchful of you progress if you are willing to continue to share it and wish you well regardless. That which you write and and this site as a whole is invaluable for me as suspect I am on the far side of the world to most of you and fellow suffers are few and far between. Take care and may your journey be a good one.

[ourlife]

Hi and thank you for your interest, I have had the results of the biopsy and in conclusion I had a reaction to sunlight it seems to have been a one off but each time the sun comes out I do worry it might happen again,so wear plenty of sun screen now! I have been led to believe that SAR 302503 is a Tyrosine Kinase inhibitor which seem to be causing photo-sensitivity in some patients. My last platelet count was 588, so have gone back up and my hemoglobin has dropped for the first time in 12 years to 12.4. I am still very tired and am beginning to wonder if this drug is really going to work,the fatigue is still my main problem. I intend to stick it out and hope that progress will be made in the future.

[ourlife]

I have now come off this trial following cramp in both my calf muscles and in my hand which has been quite painful,and other pains in various places. My last blood test also gave cause for concern as my RBC and HB dropped again and my platelets were up to 628 so this drug is clearly not working for my ET which Is very disappointing.

This experience has not put me off future trials and good luck to everyone else trialing now and in the future.

[hackett]

Ourlife If I may offer condolences .................. or I don"t know what really what to say. I have been away a while and your last post was unexpected. I had always assumed that this drug was pretty good.(not quite the magic panacea, but pretty good never the less). I wish you well

[hackett]

Hello Ourlife. If your still around and If you dont mind I wonder if you can say how you feel now you have been off the trial some time and how you went about resuming some alternate treatment regime. As always take care.

[ourlife in reply to hackett]

Hi hackett, I feel much better now thank you,the cramps have stopped and my counts are much better, I have gone back on to anagarlide and the fatigue is harder to manage and the stomach troubles have started again! I have been asked to try Interferon but am worried about lots of different meds in a short time period,my haemoglobin and creatin are heading in the right direction and I have been told these will take a while to recover,

I belive the SAR is working well for some people with MF but it dose not seem to be working for ET I know of other people who have ET and been unsuccessful on this trial. What are you taking and how are you?

[Jennie_Barnes in reply to ourlife]

Hi Ourlife, it might be worth pushing to get changed onto interferon, I have been on both anagralide and HU in the past for a number of years and was quite ill on both of them, with nausea and tiredness and generally feeling weak! I've been on interferon on the highest dose for the last 5 years and have found although I do get tired I can still work 50 hours a week and continue to live the life of a normal 23yr old. the only side effects I seem to have our slight pins and needles and a headache the morning after taking my medication. But as I take the slow releasing one I only have to put up with this one day a week.

[hackett]

Hello Ourlife It is good to hear that you are feeling better. Long may it last. The pain and discomfort must have been bad. Did you withdraw or were you exclude? (or was it a joint decision?) What do you mean by stomach troubles? I have as part of my suite of symptoms intermittent unbearable stomach pain (which now can happen anywhere on my torso), No one yet has been able to put a cause or label on it.

I have had diagnosed with PV for 7 years though the original diagnosis was of ET. I have been taking Anagrelide for a number of years. It struggles to keep my platelet counts down so I have the occasional pulse of busulfan. I am hopeful to get on the trial again later this year . I am fearful also that it will not work or I will have to withdraw again for some reason. If it does not work then I may consider interferon. My original haematologist (who is currently on extended leave at the moment) keeps on suggesting that irradiated phosphorus may be a good alternative?????? Again, as always take care.

[ourlife in reply to hackett]

Hello again. I withdrew from the trial on the advice from my hematologist following a scheduled blood test,she was not happy with my counts or the way I was feeling.The fact that my platelets were rising after 3 months on the highest dose of 400 mg led us to believe this drug was not working for me.

My stomach troubles are definitely a side affect of the Anagrelide,mainly diarrhea/loose bowels,sometimes immediately after eating with my food passing straight through me, it seems to be worse the more fatigued I am. I was the same on Hydrea,yet all these symptoms stopped on the SAR trial.

Are you aware of the web site mpdvoice.org.uk ? It contains more information on Anagrelide and it's side effects; I am convinced that your problems are related to the drug.

What is irradiated phosphorus ? it sounds quite serious!

Good luck and have fun,Ourlife

[hackett]

Hello and sorry again for a tardy reply.For some reason which is not yet clear some of my worst symptoms have subsided somewhat ( those that remain are tolerable at present) and I have attempted a return to work. Fatigue and pruritus remain as my constant companions. I will look closer at the web site mpdvoice.org.uk re anagrelide. I was trying to construct a considered response re irradiated phosphorus or P32 and found that mpdvoice also gives a sufficient description in "Melphalan, busulphan and P32" My dilemma has been that my original haematologist has/had been recommending the use of "treatments" such as P32 and busulfan should the anagrelide cease to be effective on one hand and on the other my current relief haematologist is suggesting drugs such as interferon (or one of the trail drugs) should the anagrelide fail. (sorry about the lengh of that sentence) These drugs seem to be at the opposite ends of the spectrum so to speak. Maybe we are a little behind the times here out in the colonies! May I ask how much anagrelide you take per day??? . I currently am on 2mg per day. Take care and good health.regards hackett

[ourlife in reply to hackett]

Hello hackett, I am on 3mg of AG, 3 pills when I get up & 3 at bedtime with aspirin,I have just read about P32/busulfan and am not sure if I would want to try them before trying interferon.I have been made aware of another trial called "magic" the treatment is Ruxolitinib another jak inhibitor. Is this trial available to you? I am hoping to get on this next year if the entry criteiria can be changed to allow patients who have been on other trials.I am glad I do not have to work at the moment but we are a foster family and our current placement (identical twins) keep us busy !! There is so much information nowadays and it can be hard to know what option to take! Have fun and don't work too hard.Regards ourlife

[hackett]

G'day Ourlife. I agree. My impression is that P32 is not the medication for me unless I am suicidal. I was surprised that it is still available here. Also I shudder every time I have to take a pulse of Busulfan. About 2 months ago I was offered the opportunity to go on the Ruxolitinib trial, but in the end decided against it because the again the washout period was too long. I will try wait for a second chance to go on the SAR 302 503 trial which I understand could start in late December. I think I will try put out a question to see if anyone else has been on a trial and what their experiences have been. Good luck with fostering, its not an easy undertaking, but you are probably more aware of the challenges than I am. regards Hackett