Research Updates, Trials and More.
In this section of the Forum, I will try to explore and hunt down the latest research there is on dystonia, what it means to you, how you can participate in clinical trials and more.
If anyone comes across an interesting research item, articles, news, please post it in this section. So that everyone has access to the links and
information in one section of this Forum.
Contribute !
beka
If anyone comes across an interesting research item, articles, news, please post it in this section. So that everyone has access to the links and
information in one section of this Forum.
Contribute !
beka
Tags: clinical, disorders, dystonia, movement, neurology, news, research, trials, updates
Replies to This Discussion
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Permalink Reply by beka on -
Web Cast on Research Trials in the EU
A new 90-minute audio conference from BioWorld Today
The EU Clinical Trials Directive and Beyond: What You Must Know to Get Your Trial Approved
______________________________________________________________________
When: Tuesday, October 14, 2008
What Time: 11am to 12:30pm ET
Where: Your office or conference room
Cost: Just $349 per listening site no matter how many people are in the room! ( Really Just ..!!! )
Speaker: Francis P. Crawley
Duration: 90 Minutes
There are benefits to conducting a trial in the EU—but companies that don’t understand the complicated process and the many ramifications of the Clinical Trials Directive (2001/20/EC) risk wasting significant time and money, and may still not get their drug approved.
Each member state interprets the directive differently, requiring sponsors and sites to juggle 27 sets of rules. National Competent Authorities can impose additional, onerous requirements before approving a trial.
And once a phase is complete, sponsors have to go through the process again, since individual studies, not investigational drugs, are approved.
In this 90-minute audio conference, Francis P. Crawley will show attendees what they need to know about the directive to effectively navigate the European clinical trial environment.
Learning Points or Questions to Be Answered:
8 common "pain points" sponsors and sites feel in dealing with the EU Clinical Trial Directive
What 2001/20/EC says, and how it is applied in the various member states
The tangled web of ethics committees—local, regional, and national, and the roles they play
How trial authorization in the EU differs from the U.S., and the practical impact that will have on budgets, timelines, and go/no-go decisions
10 additional activities and types of information a National Competent Authority can demand before authorization, when they are likely to demand it, and how to prepare for such additional requests
How SUSARs (Suspected Unexpected Adverse Event Reports) vary from country to country, and what that means for your trial, especially multi-state trials
Plus — you'll get the chance to ask the speaker your own questions prior to the audio conference as well as during the live Q&A session following the presentation!
Target Audience:
Clinical trial coordinators
Clinical trial investigators
Regulatory/legislative professionals
Submissions professionals
Compliance officers
Executive management
General/corporate counsel
Anyone responsible for developing or executing clinical trial protocols
Level: All Levels
Speaker:
Francis P. Crawley is the Executive Director of the Good Clinical Practice Alliance, Europe (GCPA) and a WHO expert in ethics. He currently represents the GCPA partnership on the European Commission (EC) FP7 research projects nEUroped and RESPECT. He is also the co-founder of the Strategic Initiative for Developing Capacity in Ethical Review (SIDCER).
Since 1999, Francis has been a member of the Working Group on Ethics, Union of European Medical Specialists – European Academy of Paediatrics (UEMS-EAP). He was a member of the Steering Committee for the EC project "The Development of European Standards on Confidentiality and Privacy in Healthcare among Vulnerable Patient Populations" (EUROSOCAP).
Francis is an Honorary Member of the Faculty of Pharmaceutical Medicine, Royal College of Physicians, United Kingdom.
Registration includes:
Admittance to the 90-minute call for as many people as you can fit in a room with a speakerphone.
Access to the specially created presentation handouts (available 48 hours in advance).
Participation in the 30-minute, live Q&A with the speaker following the presentation.
Certificates of attendance for all audio conference attendees.
Click here to register.
If you prefer to register, or order the CD recording (MP3 format) by phone, then call us at 800-688-2421 or 1-404-262-5474. And when you do, please be sure to mention your priority code: T08516 - EM5169.
Please feel free to forward this announcement to others who might find it useful.
--------------------------------------------------------------------------------
Audio conferences are sponsored by BioWorld, 3525 Piedmont Road, Building 6, Suite 400, Atlanta, GA 30305 U.S.A. Phone: 1-800-688-2421, E-mail: customerservice@bioworld.com.
BioWorld reserves the right to substitute speakers and reschedule or cancel audio conferences due to unforeseen circumstances. BioWorld is not responsible for any problems stemming from registrants' organization's hardware or telecommunications services. Recording of audio conferences is prohibited.
beka
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Permalink Reply by beka on
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New Botulinum Toxin Study Recruiting :
The efficacy of botulinum toxin (btx) has now been demonstrated for a variety of diseases associated with involuntary muscle spasms or movement. The application of botulinum toxin therapy to movement disorders requires treatment tailored to the individual patient and specific techniques of injection. This protocol 1) provides for training of physicians in the use of botulinum toxin and 2) allows us to provide botulinum toxin injections for patients participating in other studies on the physiology of sensorimotor systems and physiological effects of botulinum toxin.
Eligibility
Genders Eligible for Study: Both
Accepts Healthy Volunteers: No
Criteria
INCLUSION CRITERIA:
Patients will be eligible for participation if they have a disorder that, in the judgment of the treating physician, might be amenable to treatment with BTX.
Applicable disorders include but are not limited to dystonia, hemifacial spasm, blepharospasm, tremor, spasmodic dysphonia, tics, vocal fold tremor, oral lingual dyskinesia, tardive dyskinesia, spasticity, and spasmodic dysphonia.
EXCLUSION CRITERIA:
Patients will be excluded form participation if
They are pregnant or breastfeeding, for the duration of the condition.
They require treatment with an aminoglycoside antibiotic, until treatment is complete.
For laryngeal injections, they have a paradoxical vocal fold movement with intermittent stridor due to either gastroesophageal reflux or emotional disorders.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00001208
Contacts
Contact: Patient Recruitment and Public Liaison Office (800) 411-1222 prpl@mail.cc.nih.gov
Contact: TTY 1-866-411-1010
Locations
United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike Recruiting
Bethesda, Maryland, United States, 20892
Sponsors and Collaborators
National Institute of Neurological Disorders and Stroke (NINDS)
beka
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Permalink Reply by beka on
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There ae close to 75 Clinical Trials being conducted on Dystonia :
Check them out at this site and see if you qualify.
http://clinicaltrials.gov/ct2/results?cond=%22Dystonia%22
beka
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Permalink Reply by Joe on -
Thanks, Sparky,
I'm not surprised that Dr. Montgomery is involved in this research. He was the first neurologist I saw upon the onset of symptoms, so consequently, I received the correct diagnosis after just one visit. By the grace of God I managed to escape the horror show of doc shopping to get correctly diagnosed. He also presided over my first DBS surgery in 2002.
He's a good man and a brilliant neurologist. I'm really glad he's in our corner.
I'm also thankful for you, Sparky. You obviously have the skills and patience necessary to find relevant and current information.
Many thanks,
Joe
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Permalink Reply by Joe on
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That's an interesting story, Sparky. When I first went under the knife in May of 2002, the plan was to do both sides. However, because my brain anatomy (as per my neurosurgeon) was "atypical', it took them about 10 hours to do just one side. The had already made the incision for the other side, so they just stitched me back up and sent me home to recuperate About 11 days after the unit was turned on, my symptoms began simply to disappear. It was truly miraculous. Unfortunately, it was not to last. What was occurring was something called the "micropallidotomy effect." Surgery to the brain causes the brain to swell, and, for as yet some totally unknown reason, relieves the dystonia. Of course, as my brain healed my symptoms gradually returned...not to my previous baseline...there was still significant residual improvement. So much so, in fact, that I sort of became a victim of my own success. Right after my first surgery, Dr. Montgomery resigned his post at the hospital I was being cared for (it will go unnamed...suffice it to say that it's in Cleveland...and it's a "Clinic") to take the post he has now at Madison. Unfortunately for me, that meant I was no longer under his care. He had always intended to do the other side. However, the new neurologist to whom I was assigned, thought he new better and suggested more Botox ("drill, baby, drill!"). To make a long story short, after 9 months of futility trying everything that had previously failed, he came to the brilliant conclusion that maybe we needed the other side done...and that's what happened early in 2003.
It seems like a long time ago, but those were the longest 9 months of my life. I knew the potential benefit for me from DBS surgery, but I wasn't being allowed the benefit simply because some chuckleheaded neurologist had an ego.
Well, that's my story and I'm stickin' to it.
All the best,
Joe
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Permalink Reply by beka on
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About this Post -
It is my belief that , even though I work in the medical field, many with dystonia and other movement disorders are true searchers online for more information. Thus, I applaud all of you for adding your knowledge base to this Forum and Post. There are not always things that I don't know, but I do try to find out. Each of you have helped in this process.
Applause, Applause.
I also believe that patients need encouragement, support, positive experiences and a rally cry along with a crying shoulder now and then...
I will search for more new trials about the toxins PurTox and Nt201. Thanks, Sparks for posting about a NEW potential treatment option...
beka
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Permalink Reply by Ellen Schnakenberg on -
A reminder sent to me by the group doing the webcast. I'm signed up and hoping to attend. Use the links below to sign up or get more information!
When Good Medicines Become Bad Drugs (SM)
Just a reminder about the upcoming free live video webcast:
When Good Medicines Become Bad Drugs(SM)
Tuesday, October 21, 2008 at 3:00 p.m. EDT
By learning practical tips on how to keep your medicines safe, you will be helping to protect yourself, your family and community from the risks associated with prescription medicine abuse.
http://video.webcasts.com/events/vxmd001/28151/
Featured Panelists:
Moderated by General Barry R. McCaffrey, former Director of the Office of National Drug Control Policy, the webcast will feature a panel of speakers, who are leaders in their respective fields:
-Jeff Gudin, M.D., Co-Director of the Pain Management Center at Englewood Hospital and Medical Center in New Jersey
-Steve Passik, Ph.D., Associate Attending Psychologist at Memorial Sloan-Kettering Cancer Center in New York City
-Jennifer Bolen, J.D., a patient living with chronic pain and founder of The Legal Side of Pain, an organization that provides legal guidance regarding use of pain medicines
To learn more about ways that you can protect yourself, your family and community from the risks associated with prescription medication abuse, please visit: www.goodmedicinesbaddrugs.com.
"When Good Medicines Become Bad Drugs" is a service mark (SM) of Cephalon, Inc. or its affiliates.
http://community.wegohealth.com/profiles/blog/show?id=2028394%3ABlo...
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Permalink Reply by Thorns on -
Joe; I assume by reading your surgery that you now have two pace makers on either side of your chest, my brother who had this surgery DBS in Feb 08 only has one and it is attached to two rods on either side of his brain. Each rod has four contacts which have been now ajusted 8 times to get the right settings, each time he has gone there has been gains in some area's and losses in other's but so far the surgery has been a success.The next time will be in Dec. they are always hoping for more inprovements each time and are very caring at each appointment. The swelling is normal for this type of surgery and no ajustments should be made until it has gone completely away, my brother is still on is meds because he will be slowly weened off them because of all the years he has taken them. My brother has general dystonia with every part of him affected with alot of pain in his torso area besides his feet and legs, since the DBS he only has a little pulling in his neck and alittle bit of shaking when his is stressed. Thorns
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Permalink Reply by beka on
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PurTox Update
Mentor Corporation (NYSE:MNT - News), a leading supplier of medical products for the global aesthetic market, today announced completion of the first of three Phase 3 clinical trials with its purified botulinum Type A neurotoxin (PurTox®) for the reduction of glabellar rhytides (frown lines). This newly completed study involved 400 subjects at ten investigational sites in the United States. Subjects received a single treatment with PurTox or placebo.
Using a validated 4-point photo scale as a reference, reduction in line severity was scored by subjects, investigators, and independent reviewers at time points through six months. Satisfaction with treatment outcome was measured using a standard global satisfaction assessment tool and safety was assessed by adverse event tracking. The results for the primary efficacy endpoint and for each of the eight secondary efficacy endpoints were all highly significant (p ≤0.0001 in each case). For each of these endpoints, the findings were indicative of a substantial treatment effect. In addition, a very large percentage of subjects expressed a high degree of satisfaction with the treatment outcome.
Data from the three Phase 3 studies will be used to support the filing of a BLA (Biologics License Application) with the U.S. Food and Drug Administration. The remaining two Phase 3 studies for this indication have completed enrollment and are currently in the follow-up stage.
About Mentor Corporation
Mentor is a leading supplier of medical products for the global aesthetic market. The Company develops, manufactures, and markets innovative, science-based products for surgical and non-surgical medical procedures that allow patients to retain a more youthful appearance and improve their quality of life. The Company's website is www.mentorcorp.com.
If interested in the use of PurTox, and Botox and MyoBloc neither work for you, INVESTIGATE. NEW Toxin coming !
beka
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Permalink Reply by beka on
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One more Toxin Coming... Another Treatment Option ...Reloxin
Reloxin Basics
Molecular Structure
Reloxin, like Botox, is a 900-KD molecule. This rather large molecule is one that includes the basic 150-KD subunit as well as the protein structure surrounding it to create the larger molecule known as botulinum toxin. The basic configuration and its subtle changes make each form of botulinum toxin slightly different, allowing for differentiations between time of onset, duration of action and diffusion characteristics.
Like Botox, Reloxin is a type A botulinum, distinct from Myobloc, the other botulinum toxin previously tested, which was a type B. There are several other types of botulinum toxins currently in testing, with subtypes ranging from A through G. However, at this time, only A and B have been significantly developed.
Usage, Packaging, and Storage
Known as Dysport in Europe and other countries, where it is marketed by Ipsen, Reloxin has been in use since 1991.
It has both medical and cosmetic uses in various countries and, per the package insert, once reconstituted has an 8-hour length of usage, versus 4 hours for Botox. Additionally, it can be stored in the refrigerator rather than the freezer. Lastly, the vial contains lactose within it, while Botox has glucose.
When Reloxin comes to market, it appears it will have 300 units per vial as compared to Botox, which will be discussed later in this article.
Unit conversion rates are a very important concept when it comes to understanding this product, as this will determine its effectiveness and price comparison to Botox once it is on sale in the US.
Diffusion Characteristics
One of the potential differences between Botox and Reloxin relates to diffusion characteristics of the material once injected. Since the two products have different, although similar, protein structures, there are potential differences in diffusion rates operating before attachment to the presynaptic membrane, which may allow attachments to more areas or retention of an attachment prior to being degraded.
Diffusion characteristics may also allow Reloxin to potentially treat a larger area than an equivalent number of units of Botox. ( uhmm - larger muscles ??? )
While there are theoretical concerns about a higher incidence of adverse effects, none of the studies so far have shown any higher incidence of ocular adverse events compared to Botox and, in fact, have shown decreased adverse events when compared to the Botox approval studies. Whether this is a true correlation is difficult to say, as the studies were done at a different time and are not head-to-head in nature.
Diffusion characteristics can also have positive effects on outcome, especially in areas such as the off-label forehead areas, since Botox frequently has challenges reaching the entire forehead due to its lower diffusion rate. Additionally, a smoother look can be helpful in certain areas, and diffusion characteristics might aid in this goal.
Reconstitution and Comparison of Strengths to Botox
While there are no hard and fast rules regarding reconstitution and comparison of strengths, the prevailing wisdom based on studies performed in Europe by various investigators is that a 3:1 ratio of Reloxin to Botox is a good estimate.
Several studies showed that 2.5:1 was ineffective in certain situations, while 4:1 ratios led to adverse events. The studies performed were done with frontal muscles and showed less activity in the 2.5:1 ratio, but increased muscle weakness in the hand when used for hyperhidrosis in the higher ratio of 4:1.
It would seem then that the 300 unit vial that is proposed at this time for Reloxin will be more than likely an equivalent to the dose of Botox and can be reconstituted in the same way as with Botox for an equivalent effect. This should make the transition to the use of Reloxin smoother in those practices that wish to do so.
Side Effects
The side effect profile of Reloxin, in studies to date, shows a fairly similar profile to Botox. There are the expected ocular events (8% vs. 0% placebo), and other, non-statistically significant events, such as headache and injection site pain. No significant serious adverse events were noted in the one study that has been released so far.
Potential Competition in this Area
There has been a history of competition in the cosmetic marketplace and this area will be one more opportunity for competition. Allergan, the maker of Botox, has enjoyed a year in which they had the availability of both a filler, Juvéderm, and Botox. During this period, Medicis has been continuing to market the Restylane family of fillers (Restylane and Perlane), but hasn’t had a neurotoxin to complete the marketing package. The introduction of Reloxin will put these two competitors on even footing, but it should be noted that Allergan may have a challenge in marketing Botox if Reloxin is priced lower, because of certain Medicare-based price structures that can’t be changed easily.
Several countries in Western Europe, as well as Brazil, Iran, Russia and the Ukraine, have been exposed to the two products, Dysport and Botox, and appear to be using a fair amount of both products after several years of competition. While no exact numbers have been released, it appears that the two competitors are at parity in many of the countries where both are available. Many hope that the competition in the U.S. market will bring more options and benefits to the dermatologists and consumers.
Other Compounds in Development
As of press time, both PurTox and NT201 were in early Phase III trials. These botulinum toxin options are similar in some ways to both products in the market, but offer certain differences as well, including a different purification process for PurTox and a less bulky molecule (150KD) for NT201. As these products come closer to the marketplace, this publication will inform its dermatology readership of trial information and comparisons.
The Near Future
During the next year it is likely that Reloxin will enter the marketplace and fill a void as a competitor to Botox. It will be interesting to see what this will do to change and improve options for patients.
THERE IS TALK ABOUT A TOPICAL TOXIN... WHOO HOO !! NO Shots !!
beka
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