Posted November 3, 2009 by LaVonne Goodman

We need 60 more good Huntington's people to get the ACR16 trial done in North America. Do you know that 90% of the more than 400 Huntington's participants from the European trial have petitioned centers so that they may continue ACR16 compassionate use after the trial? This remarkably high number suggests that Huntington families in Europe believe that ACR-16 may be doing something very good.

Why are we in the U.S. and Canada risking our chance to get this drug? Do you know that slow enrollment is the reason 2 out of every 3 clinical trials in the U.S must close before enough information is gathered to bring a new drug forward to the FDA -- because the drug company runs out of money. Each day that we don't enroll is adding to the cost of this multimillion dollar trial.

If we don't fire up 60 more people to join this trial, we may be risking our chance to get this drug.

How hard can enrolling 60 more people be? There are 28 geographic locations in the U.S. and Canada still recruiting for this trial, and if each of these sites worked hard to enroll just 2 or 3 more participants, we'd be over the top.

Every part of the community shares the responsibility for slow recruiting. And it includes every person working in a Center of Excellence and everyone working for HDSA at the national and local level. But it would be a mistake to count on these organizations to move quickly.

Who can make the most difference right now? We Can!

We at the grass roots level may be at the bottom, but we are the important ones who can to get this job done quickly. Every HD family who is aware of this trial must join if we can, and if we can't we must seek out, educate, encourage and provide support to those who can participate. Part of education is to stress the critical importance of participation in the ACR16 trial right now.

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Start today:

* Find the locations where this trial is offered
* Get your local HDSA chapter on board
* Inform and encourage participation in your local support groups and web communities
* If a testing site seems too far, know that transportation costs -- even air fare -- may be covered. To get this trial -- and others to follow -- done we need families outside of centers
* Let your extended families from other areas of the North America know about the importance of this trial
* Make these contacts today. Don't put it off
* Let your local physicians who take care of Huntington families know about the trial.

We need only 60 more good people to get the ACR16 job done in North America. Only we have the power to make a difference - either by joining this trial ourselves or by helping others participate. We must energize our communities to get this job done now -- we otherwise are risking, and might lose the chance for this drug.

Hi Dr. Lavoone,

I have a family member I would love to participate in this trial! The closest site participating is in St. Louis. Since my family member is still working, it would be very difficult for us to complete the required 7 on site visits in St. Louis. Do you know if this trial is adding new sites? I live in the Kansas City area and would love to see KU Med participate. I'm sure there are other potential subjects in our area also. Perhaps adding a few additional investigator's to this trial could help close the enrollement gap. Thanks.

I would Love for my husband to participate in this trial, but he is already enrolled in the COQ10 study. He will be seeing his HD neurologist on Thursday and I will ask if he could switch.

My husband wanted to participate in the ACR16 clinical trial. But the current medication he is on excludes him from participating.

I have heard no more from UTSW Med Center in Dallas.
Perhaps Amit Gode or Dr O will schedule me this week.
Bob

Bob, that's terrible! Hasn't it been about 3 weeks since you phoned them, and said you'd like to take part in the trial?

Regardless of this trial, and it's outcome,(and as much as I hate this phrase), I think we have a "teaching moment" here.

The fact that this "call to arms" is necessary means we are not set up for trials, and have inadequate ways of providing for their success.

I agree the need for this trial is now. I do see this drug as a hopeful treatment as an excellent candidate for advancing treatment. But we have to be able walk and chew gum at the same time.

Personally I am not interested in laying blame for slow enrollment on anyone. Rather than criticizing, I think it's time for a critique of what we have and what we want and need and start moving in that direction. I think we have good people at HDSA, I think we have excellent hard working, attentive COE's. We have excellent researchers by way of CHDI, and HSG. And we have the rest of us. The finger pointing is going to keep everyone separate.

I am sorry but every one of these groups actually have their strengths... and we need to identify those and put them to better use. And we need more lines of communication open, and more enthusiasm, and completely different attitudes.

There is a big difference between identifying flaws and assigning blame, and identifying and carrying out a new vision using each other and the strengths of each to a unified end. There is no reason we can't accept it's time for some evolution to take place, without being bitter that it hasn't evolved already. There is no time machine to backwards. So lets not go there anymore.

So this trial is enrolling slowly. Who needs what for the next trial? There will be a next one. This is a strength of this trial, to show us where we want to go from where we are. There is no shame that this one hasn't been picture perfect. It's a trial of a trial. We need to sneak some new ideas and attitudes across everyones blood/brain barrier.

I do want to remind every poster and every reader here that you are not just on the internet. You are a member of a real organization of people. And as a member your thoughts do count. And so do your attitudes. And we need to be willing to adjust our thinking to being relevant and willing to possibly contribute in different ways than we are used to doing. We have our strengths as well and can achieve positive things. If you post here, don't say you were just online .. refer to yourself as an active member of the HDAC. That's what you are..and I am. We are an organization of people concerned about HD and issues surrounding it. I use the name. I did with my rep. We have to be part of the whole if we want a whole. If you can.. as a member of the HDAC speak up and represent me. Help where I can't, and I will help you where you can't. And when you see a chance to cross a line and help the HDSA..HCF... HDA ..CHDI...HSG we need to do that. They can count on us as a group. As an organization. Maybe we need to invite them to us. All of those groups have one thing in common with our membership. We want the best for everyone with HD issues.

I am very thankful to the members here. I have watched people here be relevant.. do cool things in new ways. And a lot of current thinking and attitudes about HD and issues has started right here. The members here have helped chop HD down to size... faced the realistic, and we learned first to cope then to attack problems and issues. Go to any conference and the attitudes you hear there you heard first here. There are elements of us sprinkled throughout the HD community. We as a group have achievements. And we are a group, and we can help as one.

Steve and Marsha, I have watched you evolve. And everyone here darn well knows you are the spark that's made the engine move. You let anyone who wants to, tweak the motor in every direction.. I think it's time to see the horse power this baby of yours has. I think we have more than enough for ourselves now and can give the rest away to where it can be used. We all here can help more than just us. And I know you have seen it as possible to get things that way. There are no better attitudes than here. Many past possibilities are realities now. I think it's ok for people here to realize they are members, they are relevant, And as members here they can help not only themselves and the other individuals here, but everyone at the same time in small ways. That's how I feel now. I kept waiting for the "HD community" to happen from somewhere... and I missed it. It can start here. Not just our community, but the real one. We can be the catalyst of that community, We can be the example of good attitude and good will. Why not?

I have an idea that might help. I have just started receiving e-bulletins from the HSC...Huntington's Society of Canada...and i'm wondering if the HDSA has an email list too? What if i was to write the HSC and ask if they can send out a mail, telling everyone about the ACR16, and how more participants are needed, and how important this is? Could the HDSA send out something similar? Would this be helpful?



Edited 1 time(s). Last edit at 11/11/2009 01:55PM by Barb.

Barb Go for it.

Ok, it's a holiday here today, so tonight i will send out an email, to Don from HSC, and one to Louise of HDSA.

What does slow to enroll mean? Are the phones not ringing? If they aren't ringing are they not ringing because people are not interested or not qualified to participate?

I went on the HDSA website and looked at all the ongoing research, ACR-16 has a list of 21 exclusions. The longest list of exclusions of any study that is ongoing. CoQ10 has 9, Memantine has 10, Celexa has 9, CoHort has 1, Predict-HD has 7, Creatine has 13.

This is a tough study to fill because of the exclusions and because of the time a person has to commit, because a caregiver must be also able to commit to it, a PhD has to be stable on approved drugs.

My husband is disqualified because of his meds and that he is in another study. I am home and could drive him every day but I will not risk his stability and drop his meds. I don't think that this is a marketing problem. Here are the exclusions...

?Unable to give written informed consent.
?Treatment with any non-allowed antipsychotic medication within 12 weeks of randomisation. The non-allowed antipsychotic medication is any medication other than Amisulpride, Haloperidol, Olanzapine, Risperidone, Sulpiride, or Tiapride.
?Treatment with the antidepressants Fluoxetine or Paroxetine within 6 weeks of randomisation.
?Use of Tetrabenazine within 12 weeks of randomisation, or at any time during the study period.
?Treatment with any investigational product within 4 weeks of randomisation.
?Use of tricyclic antidepressants, class I antiarrhythmics, and strong CYP2D6 inhibitors such as Ajmalicine, Chinidin/Quinidine and Ritonavir, within 6 weeks of randomisation.
?Patients previously included into this study.
?A prolonged QTc interval at screen (defined as a QTc interval of > 450 msec for males or > 470 msec for females), or other clinically significant heart conditions.
?Creatinine clearance <40mL/min as measured at the screening visit.
?Any clinically significant, abnormal, baseline laboratory result which in the opinion of the Investigator, affects the patients' suitability for the study or puts the patient at risk if he/she enters the study.
?Clinically significant hepatic or renal impairment.
?Patients with a history of epilepsy or a history of seizure(s) of unknown cause.
?Severe intercurrent illness, which, in the opinion of the Investigator, may put the patient at risk when participating in the trial or may influence the results of the trial or affect the patients' ability to take part in the trial.
?Alcohol and/or drug abuse as defined by DSM IV-TR criteria for substance abuse - this includes the illicit use of cannabis within the last 12 months.
?Patients with suicidal ideation, defined as a positive score on criteria for major depressive episode, item A9 on the DSM-IV-TR criteria for a Major Depressive Episode.
?Females who are pregnant or lactating.
?Females who are of child bearing potential and not taking adequate contraceptive precautions are excluded from the trial. Females of child bearing potential taking acceptable contraceptive precautions can be included.
?Known allergy to any ingredients of the trial medication or placebo.
?Any previous participation in a clinical study with ACR16.
?Patients currently receiving deep brain stimulation.
?Patients with a history of surgical procedures aiming to improve the symptoms of Huntington's disease, such as neural transplantations, lesions of the central nervous system, infusions of neurotrophic agents or previous attempts of deep brain stimulation.

I didnt get an email out last night, i wasnt feeling well, so will try and do that tonight for sure

LOVE YA, ERIC!!!!! xxooxxooxxoo

Deborah

I would love it if my husband could participate, he has in another however his current med regiment excludes him....no so sure it's a great Idea to stop meds!

I was interested in this but the exclusions were just to overwhelming to
me.
Laura

Clinical trials are hard work, and people who join them do take risks when taking a new drug. But we'll never get better treatments if we don't do clinical trials. Eric is soooo right -- we all -- HDSA, HSC, HSG, CHDI, and we as the HD family community -- have responsibility. Eric is so right that we all have responsibility if we want to get new treatments faster.

No one should stop a drug to be in a clinical trial if it causes harm. Individual safety comes first. Having said that, I have two patients who stopped tetrabenazine (which was helpful for their chorea),so they could be in the ACR16 trial. Criteria limiting other drugs unfortunately is necessary, because investigators can't always sort out which drug might be adding benefit -- or risk. It is necessary both for FDA approval and your safety.

Institutions could make clinical trials easier for HD people: Saturday assessments, local assessments when possible (like for visits when only blood testing is required, etc.)

I personally think that we are running out of COE folks who can be in clinical trials -- that we must reach out into other communities, increase awareness and interest -- and find ways to make access easier. My opinion, these are all doable things -- but not if we stick to just the COE model.

I'm 99.99% sure my Mom has HD (same exact symptoms as her mom who had it), and even though relatives have brought it up over the past few years, she and my dad are in denial.

I'm tempted to mention the trial to them (and/or other promising treatments/trials I've read about here), but also don't want to spoil their denial. It's hard to say when the promise of these treatments begins to outweigh their right to denial. [Opinions welcome.]

I plan to discuss this further with my counsellor when I get the money saved up to get the genetic testing process. After counselling would be the EARLIEST I'd ever bring up HD to my parents again, if I do so at all. And that won't be for several months.

Also, nearest trial is 2hrs away; she won't even travel that far for Christmas anymore.



Edited 1 time(s). Last edit at 11/17/2009 09:38AM by smiling sara.

The COE model is fine... the HD model is not. I think we have spent too much time worrying about people like me and my wife and basing everything that can and will be done on us. That's the problem. The whole HD community needs to be active upstream of the caregiver/patient outreach. By then all you have is tired worn out people who are active... but only have so much to give. We may have 30.000 gene positive people but the core of people we see who are probably 5000-10,000, once the symptoms have progressed to the point of needing treatment. And only a percentage of those get to the COE. There should be much more attention paid to the people at risk, not to their dilemma but to our need for them. They don't feel as stigmatized by HD as the older generation. And they do have examples of "activism" succeeding. I see no use in building from the ground up ... we have existing infrastructure in place. We just have to tap into the rest of the people in various ways. And that means realizing that an organization who is focused on trials, for example, may need to support the community in another way to achieve that goal. A person that comes into the community because they they want to pass a bill is a future trial participant. I am not, and my wife is not. My kids are. One kid is not a kid now. But they are not going to show up at a group meeting. If we wait for them to be ready for that..you will see them in 20 yrs. It's too late to hit me and my piers up and we are not the only people who have interests in HD. My son can get twenty people to a walk in a week.,.. my friends are dust in the wind. I am not saying deny or pull back from we caregivers, we are the ones in immediate need. But we are likely to take at any given time as much as we give. There are ways to interest a lot more people than just us. The "entrenched in HD crowd" has carried things as far as they are going to. It's going to be a slow crawl if this is the model we stick with.

The weirdest thing is for some reason I and several others can see things differently from the perspective of what we didn't have available, and would be happy to let that go and work ahead. But I keep hearing that there are obstacles and boundaries that are decades old and I don't care about them at all. I am just tired of hearing about what's gone wrong, what won't work, etc.. when it seems to work just fine for other groups. We hang on old stigma's that don't even apply in this day and age. Who the heck ever didn't get a job cause they were at risk for HD? Why should my son at college hide his status based on that? The dumb interviewer won't even know what HD is. So he may get HD in 20 years... they are lucky to keep a person for 5 yrs anymore before they move on. They don't care. I have never met at at risk person who was fired based on being at risk. There may be a incidental instance... but there is no systemic problem like this. There are not a bunch of at risk people sitting at home not working cause no one will hire them because they are at risk. There is no reason to not just say.."that's stupid to worry about that". But noooo... we let that fallacy perpetuate so HD is secretive for yet another reason. Over codling. Maybe these people would rather spill it at and actually have co-workers at the walk too. I am not saying be stupid and wave family death certificates in front an insurance agent. But come on... the guy in the next cubicle might have AIDS and going desk to desk for money and the HD person who may not even have a thing wrong with him/her is watching and feeling pent up and powerless... it's all wrong. Talk about overcoming stigmas... AIDS people came out and they went from modern day lepers to celebrity heroes.

We have to change thinking... not the institutions that could adapt to new thinking. If my kids doesn't get one job he wanted because of being at risk... too bad. He will get another one. He's more likely not to get it because he dressed wrong. In the mean time he could be working for his concern rather than hide from it. He might even take that circumstance to heart and work to change it. People have "callings" and talents that are being wasted this way. It's not right. And it's internal because we are not using what we have the best ways possible. And the fact that there are past achievements only points to the fact their could be more of them.

I like what NC has done with communicating doctors appointments with specialists via the internet. Why can't trials be done this way too? Using the existing COE model? And if HD can't afford the equipment alone.. lets team up with like diseases who we can share with.. or drug companies who see that having fewer sites but more participants available is cost effective and they chip in. Or both. We have good places already.. and good people available and good institutions. We have outdated thinking. We do have savvy people as a doctor once stated when the topic of "cure" came up. But savvy doesn't mean not excitable. It may not be over the word "cure"... but progress is something that does excite us. And that's what we need to focus on.. progressing from this point to the next. That means personal progress, social progress, medical progress, and organizational progress. I think if we just identify where progress can occur, there are usually simple steps to make it happen. People can be enthused with achievable goals. The people we have already can be enthused and people we don't have yet could be.

Thanks Eric - very thoughtful. Robi

Robi Blumenstein
CHDI Management/CHDI Foundation
robi.blumenstein@chdifoundation.org
www.chdifoundation.org

Oh l? l?, Eric! When the muses visit you, you are really something
I couldn't agree with you more. Specially when it comes to the secrecy part. Maybe the stigma nowadays is mostly in our heads.
And maybe the way we still think about this disease plays a role in the way things are developping with the ACR16 trial. I'm not saying it's not the medication factor in northamerica that's been preventing this trial to go faster. But maybe the fact that we are not coming out of the closet, that we are still choosing to suffer in isolation, that we are not connecting to each other is causing this, too. I don't know how things are in Europe, but maybe they've found a better way to reach out to those people.
Anyway, I think it's time to seriously think about every issue you mention, Eric, because more and more trials will be available soon and we cannot afford not to complete them if we want this disease treated or (warning: the "c" word will be used) ?cured?.