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News and Announcements
The Phase II Selection Trial of High Dosage Creatine and Two Dosages of Tamoxifen Preliminary Results Presented at PALS Webinar Sixty participants were enrolled in this trial and received one of three different experimental treatments (creatine 30 g, tamoxifen 40 mg, or tamoxifen 80mg) for 38 weeks. The trial's objectives were to rank the tolerability, safety and efficacy of the tested treatments and select the superior treatment for testing against placebo in a future larger trial. Creatine 30 g was less tolerable compared to tamoxifen 40 and 80 mg. A future placebo-controlled clinical trial is needed to test the efficacy of tamoxifen in ALS. Watch PI Dr. Nazem Atassi's webinar here.
NEALS Phase II Trial of Mexiletine to Begin Enrolling Soon
"A Safety and Tolerability of Mexiletine in Patients with Sporadic Amyotrophic Lateral Sclerosis" is a phase II randomized, double blind, placebo controlled trial investigating the safety and tolerability of two oral dosage levels of mexiletine, an anti-arrhythmic medication, versus placebo. The study will enroll 60 participants with sporadic ALS.
We anticipate enrollment for the Mexiletine Study to begin in late June/early July 2013.
Diaphragm Pacing System (DPS) Trial
In collaboration with Synapse Biomedical, the Muscular Dystrophy Association (MDA) , and the ALS Association, NEALS will lead a study to assess the ability of the NeuRx Diaphragm Pacing System (DPS) to improve respiratory function and quality of life in people with ALS. Survival and shortness of breath will also be determined. A group of people with ALS will receive the DPS and another group of people with ALS will receive the current standard treatment. The trial will enroll at approximately 20 NEALS sites in the US. Enrollment is anticipated to begin in July 2013.Clinical Research Learning Institute this OctoberThe Third Annual CRLI will be held on October 4-5, 2013 in Clearwater, FL. The CRLI is intensive program dedicated to educating attendees on clinical research and therapy development and empowering this group to be advocates for ALS clinical research. Be on the lookout soon for more news and announcements about applying to attend!
Brainstorm Statement
Brainstorm is currently conducting a Phase IIa dose-escalating trial at the Hadassah Medical Center in Israel. We are working with Brainstorm on a planned Phase II study in the US. We hope to begin the trial in the second half of 2013. We will continue to follow the progress of the study in Israel and will keep the NEALS website current on all new trials and start dates. Thank you for your interest.
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Talking Trials - Dr. Merit Cudkowicz Answers Your Clinical Trial Questions
NEALS Co-Chair Merit Cudkowicz, MD, MSc, was featured in an MDA article on the state of ALS clinical trials. She discusses promising drugs, challenges, and why we should be excited about clinical research in ALS. Read the Q&A.
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Enrolling ALS Clinical Trials
Phase II Trials
The Experimental Treatment of Bulbar Dysfunction in ALS
Nuedexta is a drug approved by the FDA for improving emotional lability in ALS and other disorders. Based on patient experience, a NEALS trial is seeking to evaluate whether this drug improves bulbar functions such as swallowing and speaking. The trial will enroll 60 participants.
A Phase IIb, Multi-National, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Safety, Tolerability and Efficacy of CK-2017357 in Patients With ALS (BENEFIT-ALS)
In the BENEFIT-ALS Phase IIB trial, researchers are examining tirasemtiv (formerly CK-2017257) for safety, tolerability and potential efficacy in patients with ALS. Participants will receive open-label tirasemtiv at 125 mg twice daily. They will then be randomized to receive 12 weeks of either placebo or tirasemtiv, again twice daily, beginning at 125 mg and increasing each week up to 250 mg twice daily.
Research sites are added to the NEALS website as they are activated. There will be 56 sites in the US and Canada, and between 14-20 sites in Western Europe.
Other Interventional Trials
Resistance and Endurance Exercise in ALS Trial Researchers at Johns Hopkins University, Massachusetts General Hospital, Carolinas Medical Center, and Washington University in St. Louis, Missouri are currently investigating 3 forms of exercise in people with ALS: weightlifting (resistance exercise), stationary bicycling (endurance exercise), and range of motion exercise (the current "standard of care" for ALS patients). The researchers will determine whether one type of exercise is better tolerated and/or safer than another form of exercise. They will also collect information about how the bodies of ALS participants respond to exercise. The trial will enroll 60 participants across the country. Study participants will take part in at-home regimen with the guidance of a study physical therapist during visits to the study center.Familial ALSArimoclomol in SOD1 Positive Familial ALS Arimoclomol is under investigation for safety and efficacy in patients with SOD1 positive familial ALS as part of a seamless, adaptive, randomized, double-blind, placebo-controlled phase II/III trial. Participants will be randomized 1:1 to receive either placebo or arimoclomol at a dose of 200 mg t.i.d. The trial is currently enrolling 80 participants at two sites: the University of Miami and Massachusetts General Hospital. The trial consists of both visits at the study site and visits in the participant's home.
Observational Studies
A Multicenter Study for the Discovery and Validation of Biomarkers in ALS
Biomarkers are markers of disease or responses to therapy that might be used to diagnose ALS, monitor disease progression, or monitor treatment effectiveness. To learn more about ALS, researchers are collecting blood and cerebrospinal fluid (CSF) from patients with ALS. Samples will be collected approximately every 4 months. This clinical study is enrolling at 6 NEALS sites: Boston MA, Worcester MA, Pittsburgh PA, Atlanta GA, Jacksonville FL, and Phoenix AZ.
The Biomarkers Webinar discusses the importance of biomarkers research into finding a cure for ALS. Through comparison of these specimens, the researchers hope to learn more about the underlying cause of ALS, as well as find unique biological markers, which could be used to develop new therapies.
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Enrollment Closures
Phase II High Fat/High Calorie Trial
On November 2nd 2012, enrollment closed for a phase II nutrition trial that investigated safety, tolerability, and preliminary efficacy of high fat versus high calorie versus normal diet in people with ALS. Weight loss is a common and severe symptom of ALS, caused both from inadequate calorie intake and an increased metabolic rate. People with ALS are generally instructed to increase their calorie intake; however, the ideal amount and type of calories has not been studied until now. The last participant visit took place in April 2013. Results are expected in August 2013. For more information on the Nutrition Trial, click here. |
PLS Research 
NEALS and the Spastic Paraplegia Foundation (SPF) recognize the critical need for improved clinical research in Primary Lateral Sclerosis (PLS), including epidemiologic studies, clinical trials, studies of disease mechanisms, and the development of new PLS outcome measures. The Virginia Freer-Sweeney Clinical Research Training Fellowship in PLS was devised in response to this critical need. We are delighted to share that Dr. Christina Fournier, MD, (Emory University) has been selected as the inaugural Virginia Freer-Sweeney Fellow for 2013.
Dr. Fournier will travel to the NEALS coordination center for a two-week training in being a primary investigator for multicenter clinical trials. She will also develop and conduct a research project in PLS and present her research at NEALS and SPF annual meetings. Of the training fellowship opportunity, she states, "(The fellowship) will allow me to explore the identification of biomarkers for disease progression in PLS. Ultimately, biomarkers can serve as a valuable tool in clinical trials to monitor response to treatment, guide new innovative therapies, and allow for more efficient research. This award will provide me the support needed to develop into an independent clinical researcher, and I am hopeful that through this grant and future efforts, I will ultimately be able to contribute to the advancement of PLS research."
Welcome Dr. Fournier!
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ALS Therapy Alliance and CVS/pharmacy Fundraising Campaign
 
CVS/pharmacy has kicked off its annual in-store fundraising campaign to support medical research and help improve quality of life for those living with ALS.
Customers visiting CVS/pharmacy during the month of June can donate $1 or $3 to the Advancing Medical Research campaign at checkout or online at www.cvs.com/als. The nationwide fundraising campaign will benefit the ALS Therapy Alliance, a national non-profit organization dedicated to advancing ALS research, now through June 29th.
Thank you for all that you do to support our mission to find a cure for ALS.
The ALS Therapy Alliance supports NEALS projects.
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Mark Your Calendars for the ALS Association/NEALS PALS Webinar Series! 
NEALS offers live, online seminars (or "webinars") for people with ALS and other motor neuron diseases. Experts explain the scientific rational and design of current ALS clinical research. Our webinars also offer participants the opportunity to ask questions directly to the experts running trials. Below is the information for our next webinar:
Genetic Research in ALS
Tuesday June 18th 2pm ET
Presented by Robert Brown, MD
(University of Massachusetts Medical Center)
View the rest of the summer schedule here!
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PALS Advocate Holly D. Ladd, JD
Holly Ladd, JD is a standout ALS advocate, with a strong voice in the ALS community. A lawyer by training, Holly has worked... |
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ALS Association/ NEALS Trial Expert
Devon Punch is the "ALS Association/NEALS Clinical Trial Expert." She is available to help people with motor neuron disease and their caregivers navigate the NEALS website and answer questions about clinical research during normal business hours eastern standard time.
ALS Trial Expert
(877) 458-0631
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