Praxis Releases Results from Essential1 Clinical Trial
Earlier this spring, Praxis Precision Medicines reported topline results from its Phase 2 Essential1 clinical study of ulixacaltamide (formerly PRAX-944) to treat essential tremor (ET). The IETF’s newest ET Virtual Education Event, “Dare for More in ET: Ulixacaltamide Study Findings,” focuses on results from this Phase 2 trial and what they mean for people living with ET.
Ulixacaltamide was developed with essential tremor in mind. It was designed to block T-type calcium channels in certain parts of the brain to help stop tremors and improve function. The study found participants taking ulixacaltamide scored better than placebo on nearly all the ADL measures (10 of 12, including dressing, using keys, pouring, writing, drinking, among others.) Additionally, participants and their doctors reported overall improvement with ulixacaltamide versus placebo, and it was generally well-tolerated.
As a next step, Praxis will be meeting with the Food and Drug Administration (FDA) in June to discuss the results and receive feedback on its plans for a Phase 3 trial to support a marketing application for ulixacaltamide.
Miss New Hampshire Contestant is Raising ET Awareness
Jillian Mars was 15 when she first noticed her essential tremor (ET). But it took her seven years to get a diagnosis. There’s still a perception that ET only impacts older adults, but she’s working to change all that.
Jillian has adopted essential tremor as her community service initiative as she participates in pageants through the Miss New Hampshire Scholarship Program. She talks about her work in our latest Talking Essential Tremor podcast, “Spreading ET Awareness through the Miss New Hampshire Scholarship Pageant.”
“It’s been incredible getting to speak to my communities about essential tremor,” she said.
Her pageant work has earned Jillian $5,000 in scholarships so far. She also received a college scholarship from the IETF in the spring of 2023.
What’s her advice for young people who have ET?
“It’s nothing to be afraid of or ashamed of,” she said. “There’s no reason why my body shaking should embarrass me.”
Remember a Loved One with a Memorial Donation
Memorial Day is a national day of mourning designed to honor military personnel who sacrificed their lives while serving in the armed forces. Over the years, it also has become a day to honor family and friends who have died.
As Memorial Day approaches, consider honoring a loved one with a memorial contribution to the IETF. There is no set minimum contribution and all memorials are posted on the IETF website and in Tremor Talk magazine.
We often see memorial contributions this time of year and also around birthdays and anniversaries of loved ones.
As with all donations to the IETF, memorial donations are tax-deductible.
Clinical Trials Need You!
If you want to make a difference in the search for better treatments for essential tremor, consider participating in a clinical trial. On our website, we keep an ongoing list of clinical trials recruiting participants. Here are some of the most recent ones:
· Researchers in the Voice Lab at The University of Texas at Austin invite individuals with essential tremor to participate in a study of voice. The goals of the study are to understand how speakers control their voice and to develop therapy that improves vocal control. For more information contact the UT Voice Lab, (512) 232-4428, or email voicelab@austin.utexas.edu.
· Jazz Pharmaceuticals’ EveresT Study is a research study testing the safety and effectiveness of an investigational medication that may help decrease tremor severity for people diagnosed with essential tremor (ET). To learn more and sign up for the study, please visit the study website here.
· The KINETIC 2 TRIAL is evaluating the safety and efficacy of three different doses of an investigational drug compared to a placebo on reducing tremors in adults diagnosed with essential tremor. Participation lasts approximately 19 weeks. Visit the study website at KINETIC2ETtrial.com to learn more.
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I have had a DBS system implanted in 2013. Yet still have certain side effects including speech problems and walking problems but overall my shaking has largely subsided I would be willing to be a subject in any study
Thank you for sharing your experience with DBS. It is more helpful to hear about first hand experiences after the procedure. There is a lot of information available about before DBS surgery and laser treatment, but the improved result is not touted as much.
I have head tremor, and I understand that little is being done to stop head tremor, other than an implant. Am I wrong?
One of our medical advisory board members, Dr. Albert Fenoy, wrote an article talking about head tremor. It includes symptoms and also discusses treatment options. You might find this helpful. https://essentialtremor.org/essential-tremor-is-the-most-common-cause-of-head-tremor/
Hello,
I have a head and voice tremor along with hand tremor. Hopefully one day I’ll get some relief from these problems. I’m not on medication at this time and my anxiety is high. My family history includes paternal family tremor with several members.
I had an MRI-FUS procedure 3 1/2 years ago for a right hand tremor. There has been a return of the tremor in the last several months, but not nearly to it’s pre-procedure level. I can live with the way it is now and it is, in my mind, a much better choice than DBS, which requires cutting your skull open and living the rest of your life with an implant/remote control. That’s not to mention possible dizziness and balance issues. I read recently that the FDA has now approved MRI-FUS for both hands. It’s tempting, but 2 factors are keeping me from doing it: My age (81) and the fact that I never use my left hand for writing, drinking, eating, etc..
Thank you for the feedback about the long term efficacy of the MRIFUS procedure. Would you do it again? Did you have side effects?
What about Ultra Sound treatments? Last news I heard was that people were being permanently cured of ET through MRI Guided Ultra Sound. Is that still happening?
Yes, it is still available and in fact, more research has been done on long-term effects. This story posted on our website shares some of those results. https://www.massdevice.com/fda-approval-essential-tremor-insightec/.
Thank GOD that my essential tremor got to point where it would not affect my profession before I retired many years ago as a Dental Prosthodontist, fine detailed work. I am 76 years old and enjoyed a very successful career. My mother had essential tremor in her later life but it never affected her as she had retired as I before symptoms became more of a problem. I have been waiting for a pharmaceutical house to provide the public with a medication that would get this menace under control. I realize that this type of research takes years of clinical trials-patience is a virtue! Thanks for all you do for those of us suffering from this disorder.