The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. Have you ever considered participating in a clinical trial (study) and thus contributing to … Some recent studies have focused on using different medications to enhance communication at the neuromuscular junction with the goal of improving muscle function as a result. For more information, please speak with your clinician (preferably at an ALS clinic) and visit ClinicalTrials.gov, where all legitimate, recognized ALS clinical trials are registered globally. Check boxes allow you to select more than one option in each field; Radio buttons allow for only "What antisense oligonucleotides can do is reduce the production of the mutant SOD1 protein by binding to and shutting down the genetic machinery that produces the mutant protein," he said. © 2020 Northeast Amytrophic Lateral Sclerosis Consortium. 7874 10 Street NE, Calgary, AB T2E 8W1info@alsab.ca | 403.228.3857 | Fax: 403.228.7752 | Toll Free: 1.888.309.1111Charitable Registration Number: 12063 0827 RR0001, World Health Organization International Clinical Trials Registry. Amyotrophic lateral sclerosis (ALS) is the disease that struck down baseball great Lou Gehrig and now bears his name. All rights reserved. BIIB078 is an antisense oligonucleotide (ASO) that is being studied to treat a familial form of ALS linked to mutations in the C9ORF72 gene. Observational trials aim to learn more about the disease and are essential to understanding, diagnosing and ultimately treating ALS. The goals of this study are: (1) to better understand the relationship between the phenotype and genotype of amyotrophic lateral sclerosis (ALS) and related diseases, including primary lateral sclerosis (PLS), hereditary spastic paraplegia (HSP), progressive muscular atrophy (PMA), and frontotemporal dementia (FTD); and (2) to develop biomarkers that might be useful in aiding therapy development for this group of disorders. In general, ALS clinical trials are therapeutic or observational in nature. Approximately 34% of all familial ALS cases are linked to C9ORF72, making it the most common genetic cause of ALS. With help from The ALS Association, NEALS provides up-to-date information for finding both federally and privately funded clinical studies focusing on ALS and motor neuron diseases. The treatment, called tofersen, was found to slow the decline of muscular function associated with a genetic form of ALS in a study to be presented next week, "ALS is a devastating and fatal disease that has no effective treatment options, so there is an opportunity to pioneer innovative treatments," said Dr. Timothy Miller, professor of neurology at. Apply Now for the 2020 Virtual NEALS Site Investigator Training Seminar July 06, 2020 Basic trial search; Browse recruiting trials Our mission is to discover treatments and a cure for ALS, and to serve, advocate for, and empower people affected by ALS to live their lives to the fullest. That's a huge leap forward. University of CalgaryActive (no longer recruiting). As the first ever platform trial for amyotrophic lateral sclerosis (ALS), the HEALEY ALS Platform Trial is testing three proposed drug regimens and will add two more over the next several months. for a search and may in fact limit your search results. All rights reserved. These substances are thought to contribute to the cellular toxicity that leads to disease. As the first ever platform trial for amyotrophic lateral sclerosis (ALS), the HEALEY ALS Platform Trial is testing three proposed drug regimens and will add two more over the next several months. You can also visit the EU Clinical Trials Register and the World Health Organization International Clinical Trials Registry for additional information. The ALS Team at the Healey Center at Mass General Hospital is committed to an ongoing dialogue about the HEALEY ALS Platform Trial with the entire ALS patient community. The patients were randomly assigned to receive either 20, 40, 60 or 100 milligrams of the tofersen treatment or a placebo for 12 weeks. Answer a few questions, and their search engine will get to work finding studies that are right for you. We partner with academia, industry, … This Phase 1 clinical trial will enroll 80 participants with a confirmed C9ORF72 mutation. "This potentially could be game-changing, at least for this subgroup of patients.". It's what's causing ALS in people with a genetic change in SOD1.". Clinical trials: between facts and fiction - Click here, What is new in the treatment of ALS? Look up everything on your opponents or yourself and see your game improve! It's a very modest effect, if any," Glass said. "Reducing the level of the SOD1 protein would be a good thing for people with SOD1 mutations causing ALS," Washington University's Miller said. After scientists test experimental therapies in the laboratory, those with promising results move to clinical trial to determine whether the therapy is safe and effective for use in humans. This trial is designed as a perpetual platform trial. That reduction of SOD1 led to reductions in how the ALS disease affected the body. Two drugs are approved by the US Food and Drug Administration for the. Answer a few questions, and their search engine will get to work finding studies that are right for you. In 2008, we conducted surveys that suggested a need for improved patient engagement in ALS research. See the ALS Ice Bucket Challenge progress! BIIB078 is designed to target repeat RNAs preventing them from being used to create the potentially toxic DPR proteins. The researchers also found that the majority of side effects were mild or moderate, such as pain at the injection site and headaches, Miller said. From harnessing innovative ideas, to translating concepts to therapies, to advancing treatments to people living with ALS – The ALS Association’s collaborative and global approach to funding research continues to lead to significant discoveries by top ALS scientists around the world. Among the participants who received 100 milligrams of the treatment, the researchers found a 37% reduction of the SOD1 protein in their spinal fluid compared with those who received the placebo. Trials with a NEALS emblem are those in which NEALS has a supporting role. Pimozide is a medication originally used in schizophrenia that has been shown to enhance communication at the neuromuscular junction in laboratory worms, fish and mice. Amyotrophic lateral sclerosis (a-my-o-TROE-fik LAT-ur-ul skluh-ROE-sis), or ALS, is a progressive nervous system disease that affects nerve cells in the brain and spinal cord, causing loss of muscle control. (JavaScript must be enabled to view this email address), Clinical Research Learning Institute (CRLI), NEALS Central Institutional Review Board (cIRB), HEALEY ALS Platform Trial Weekly & Monthly Webinar Series, HEALEY ALS Platform Trial Webinar with ALSA, First Participants Enrolled in the Groundbreaking Trial Led by the Sean M. Healey & AMG Center, Apply Now for the 2020 Virtual NEALS Site Investigator Training Seminar, NEALS 2020 Principal Investigator Training Seminar, Inaugural Mass General Hospital HEALEY Center International Prize for Innovation in ALS, ALS clinical research learning institutes (ALS-CRLI). Most patients also lose the ability to speak without assistance and die within two and five years of their diagnosis. You can locate both interventional trials, which examine if treatments are effective and safe under controlled environments, and observation trials, which examine people in more natural environments. As for the new treatment approach targeting a specific gene mutation, "we're in a new era of therapeutics for neurological disease," he said. Doctors usually don't know why ALS occurs. This incurable condition slowly breaks down the nerve links between the brain and body, eventually causing paralysis. Our scientists discover and develop potential treatments for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, a disease of the nerve cells in the brain and spinal cord that control voluntary muscle movement. //