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News and Announcements
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 June 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.
In April 2013, the FDA approved a phase II Neuralstem trial. This phase II trial will increase the dose of cells into the cervical spinal cord and will collect preliminary data on potential efficacy of the treatment. Partial funding for the trial at Emory will be provided by a grant from the NIH. The ALS Association has also provided funds to extend the trial to a second site at the University of Michigan. The trial is expected to begin in June 2013. It will recruit and enroll established patients at the Emory and University of Michigan ALS clinics.
May is ALS Awareness Month
Hundreds of PALS, loved ones, and ALS advocates will be on Capitol Hill this month to urge Congress to step up the fight for a treatment and cure. Even those who cannot make it to Washington can make a difference in the ALS community.
The ALS Association campaign encourages you to Tell Your Story.
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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Clinical Research Learning Institute in Philadelphia - Application Due Date Extended
NEALS is seeking nominations for the first regional ALS Clinical Research Learning Institute (CRLI) to be held in Philadelphia, PA. The training educates attendees on clinical research and therapy development and empowers this group to be advocates for ALS clinical research.
Patients and patient-caregiver pairs who live in the Tri-state area are encouraged to apply.
Nominations will be accepted until May 14th.
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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 will evaluate whether this drug improves bulbar functions such as swallowing and speaking. The trial will enroll 60 participants. Neurology Associates in Lincoln, NE and Providence ALS Center in Portland, OR have begun enrolling.
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.
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Enrollment Closures
Phase II Selection Design
The Phase II Selection Trial of High Dosage Creatine and Two Dosages of Tamoxifen has been closed to enrollment and preliminary analyses have been completed. Sixty subjects were enrolled at 9 NEALS sites across the US. Each participant took one active study drug (creatine 30mg, tamoxifen 40mg, or tamoxifen 80mg) and one placebo for 38 weeks. This trial had a unique design which allowed selecting the most promising treatment to be tested in a future larger trial. Results will be shared in a NEALS webinar, with Dr. Nazem Atassi, on Monday May 20th. For more information on the Selection Trial, click here. Questions can be directed to Julie Berkley at jberkley@partners.org or 617-643-7429. Phase II High Fat/High Calorie Trial On November 2nd 2012, enrollment closed for a phase II nutrition trial, which 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. |
NEALS Coordination Center Mass. General NCRI and Non-Profit Prize4Life receive Bio-IT World Award for creation of ALS data platform
The Massachusetts General Hospital (MGH) Neurological Clinical Research Institute (NCRI) and Prize4Life, a nonprofit organization whose mission is to accelerate the discovery of treatments and a cure for ALS, received a Best Practices Award at the 2013 Bio-IT World Conference & Expo. Presented on April 10, the award acknowledged the creation of the Pooled Resource Open-access ALS Clinical Trials (PRO-ACT) platform.
Read the MGH Press Release.
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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 upcoming ALS Association/NEALS PALS Webinar Schedule:
Selection Trial of Creatine and Tamoxifen in ALS
Monday May 20th 3pm ET
Presented by Nazem Atassi, MD
(Massachusetts General Hospital)
Genetic Research in ALS
Tuesday June 18th 2pm ET
Presented by Robert Brown, MD
(University of Massachusetts Medical Center)
Registration information to come!
Clinical Trial Pipeline Series - July 2013
Monday July 8th 2pm ET
Presented by Merit Cudkowicz, MD
(Massachusetts General Hospital)
Registration information to come!
ALSUntangledWednesday July 31st 2pm ET
Presented by Richard Bedlack, MD (Duke University)
Registration information to come!
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Getting Involved in a Clinical Trial
How do I find out about clinical trials? How do I know which trial I would qualify for, and which is right for me?Get answers, and read about more commonly asked clinical trial questions here. |
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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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