This population includes pre-symptomatic individuals at genetic risk for ALS or a related neurodegenerative disorder (i.e., FTD). AT-1501 (Developed by Anelixis) is an investigational antibody that targets the CD40 ligand (CD40LG), a protein present at the surface of some white blood cells that is involved in inflammation. Our greatest limitation to treating Amyotrophic Lateral Sclerosis (ALS) is the lack of understanding what causes or enables sporadic ALS. The mutation of the SOD1 gene leads to the production of an abnormal SOD1 protein that is likely to be toxic to cells and could possibly lead to the nerve cell death seen in patients with ALS. ALS Clinical Trials. These therapies can take the form of traditional medications taken in pill form or by injection, but have also included gene therapy studies and the study of medical devices. Credit: Unsplash/CC0 Public Domain In 2020, the Sean M. Healey & AMG Center at Massachusetts General Hospital launched the first platform trial for amyotrophic lateral sclerosis (ALS), in … Status: Currently EnrollingPrincipal Investigator: Nicholas J. Maragakis, M.D.Contact: Betsy Mosmiller | 410-502-0495Length of Study: ~8 months. Medical institutions that are equipped to perform clinical trials in ALS and motor neuron diseases OR researchers who are dedicated to ALS research may apply for membership. Participants must undergo surgery to implant electrodes onto the surface of the brain and a wireless transmitter under the skin of the chest. This binding may reduce the amount of both normal and toxic SOD1 protein. The goal of the study is to assess this particular BCI’s ability to safely assist with communication. Subjects will be assigned to one of 7 cohorts, depending on their ALS genetic status. This is an observational study in which participants will be followed from prior to starting Radicava through 6 months of treatment. These discoveries start in research laboratories across the institution as well as collaborations with investigators across the globe. (1st cycle 14 days of drug and 14 days of placebo, the 10 days of drug and 18 days of placebo for cycles 2-12). The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. It also modulates release of migration inhibitory factor (MIF). It also modulates survival and activation of immune cells in the CNS by preventing the production of pro-inflammatory agents from microglia. Patients taking the approved ALS drug Edaravone/Radicava® are not eligible for this study. This study has a 12 month double-blind phase, followed by a 6 month open label extension period. Please remove one or more studies before adding more. Participants must provide written informed consent prior to screening. Research goals include elucidation of the complex relationship between ALS and related neurodegenerative disorders, development of both wet and dry biomarkers of disease, and delineation of the full course of the natural history of disease from the early pre-symptomatic stages through established and even late-stage disease. Active: currently recruiting for the specified clinical trial or study Under normal conditions, the complement system will help remove pathogens and damaged cells without hurting your body. Email notifications have already been sent out to PALS meeting the specific study criteria. that might give them hope for … The intravenous form of edaravone has been approved as a treatment for ALS in 2017 by the FDA. Participants will train and test (for up to 4 hours each day, two days per week for a full year) with the BCI to control assistive typing technology and trigger a care-giver calling alert at their own home. At screening, eligible patient must be at least 18 years old with a decision made to prescribe Edaravone prior to consenting. The sponsor of the clinical trial, Neuralstem Inc., has found a way to grow neural stem cells and to freeze them until they are ready for use. Jonathan Glass from the Emory ALS Center explains two specific issues in ALS: diagnostic delay and insensitive outcome measures. Ann Romney Center for Neurologic Diseases Be sure to check back to ALS News Today for updates on ALS clinical trials in your area. The main focus of this study is to show that BIIB100 will cause no harm to humans. Its effects will be compared in 2 different ways. Published in Global News April 21, 2021 , written by Shelby Thom Two ALS patients – both Kelowna residents– are speaking out, advocating for access to clinical trials in B.C. There will be 5 cohorts with 8 subjects in each cohort across 10 sites in the US. ALS is a disease that causes motor nerve cells to gradually break down and die. This study requires a 4 day, 3 night stay in the hospital for drug administration and careful monitoring. Ibudilast is a capsule, taken orally with a 1:1 ratio of study drug to placebo. For the national central database of clinical trials for all conditions, including MS, Alzheimer’s disease, ALS, Parkinson’s disease, and brain tumors, visit: www.clinicaltrials.gov. Participants must be able to travel to the Johns Hopkins Hospital several times a week. Multiple different regimens may be active within the platform trial at the same time. Status: Currently enrollingPrincipal Investigator: Nicholas J. Maragakis, M.D.Contact: Betsy Mosmiller | 410-502-0495Route of Administration: OralLength of Study: ~6 weeks. Amyotrophic Lateral Sclerosis (ALS) Clinic Founded in 1998, the Amyotrophic Lateral Sclerosis (ALS) Clinic at Johns Hopkins is a world recognized leader in providing superior medical care and offering the latest in clinical trials and therapies to ALS patients. In a separate, small group of ALS patients (SOD1 ALS patients) the disease is caused by a genetic mutation in the SOD1 gene. The purpose of this study is to support and extend the results of the FORTITUDE-ALS clinical trial, a clinical trial of an investigational drug (reldesemtiv) for the treatment of amyotrophic lateral sclerosis (ALS). In general, ALS clinical trials are therapeutic or observational in nature. Clinical trials in people with ALS showed that riluzole prolongs survival by a few months, particularly in the bulbar form of the disease, but does not reverse the damage already done to motor neurons. Using emerging technologies, we hope to make clinical trials more efficient to reveal the most promising treatments faster. Ibudilast (developed by MediciNova) acts as a nonselective phosphodiesterase inhibitor and has been used as an anti-asthmatic medication as it reduces release of leukotrienes, cytokines and other molecules involved in bronchospasm. However, sometimes uncontrolled or improper activation of the complement system can damage your body. HEALEY ALS Platform Trial This is the first ALS platform trial, accelerating the path to new ALS therapies by testing multiple treatments at once, reducing the cost of research by 30%, decreasing the trial time by 50% and increasing patient participation by 67%. ALS research studies that fall under the interventional category are generally randomized clinical trials, which means that the subjects are split into two groups: one that receives a new treatment and another that gets the placebo.. Why Should I Register and Submit Results? As an open label study, all participants will receive the study drug. Edaravone has been shown to slow the decline in clinical assessment of daily functioning in persons with ALS. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. Its commercial name is Radicava. Amyotrophic lateral sclerosis (ALS) is a fatal motor neuron disease (MND) with no cure. Quick Approval Given COVID Vaccines Raise Concerns, Rare Disease Patients Say. Als Clinical Trials ‘ALS has taken so many things away’: Kelowna patients speak to need for clinical trials in B.C. This study will be conducted in participants who have sporadic or familial amyotrophic lateral sclerosis (ALS) as defined by revised El Escorial criteria. A Study to Assess The Amyotrophic Lateral Sclerosis (ALS) Patient Experience Jacksonville, FL. Information provided by (Responsible Party): CRiALS is an umbrella protocol through which people are recruited to participate in a range of research studies being conducted by the ALS Research Collaboration (ARC). ALS Research Notification for Clinical Trials and Studies The following are approved ALS studies and clinical trials that have used the National ALS Registry for patient recruitment purposes. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Biomarkers from blood and urine will be collected and analyzed to help better understand Edaravone’s effect on disease progression and its mechanism of action. This compound demonstrated preclinical results in a mouse model of ALS and in other preclinical studies and has a good safety profile. In the CENTAUR trial, 137 participants with ALS were randomized two-to-one to receive AMX0035 or placebo. Status: Currently EnrollingPrincipal Investigator: Jeffrey Rothstein, M.D., Ph.D.Contact: Betsy Mosmiller | 410-502-0495Route of Administration: Intrathecal (Lumbar Puncture)Length of Study: ~6 months. A secondary goal is to determine if the study drug can reduce total SOD1 protein. The research team can opt to manage the group using one of three approaches: See the Clinical Trials and Studies page on the Les Turner ALS Foundation website for information on current and past work. Bringing treatment to ALS patients is the main mission of this program’s efforts. The HEALEY ALS Platform Trial is a research trial that tests the safety and effectiveness of multiple treatments in ALS. Clinical Trials at the Healey Center for ALS If you're not sure which of these trials might be right for you, contact our ALS Research Access Nurse for guidance. Unaffected individuals from fALS pedigrees, including pre-symptomatic individuals at genetic risk for ALS or individuals from pedigrees of ALS related diseases. Current ALS Clinical Trials This is the full directory of ALS clinical trials from ClinicalTrials.gov. Gene therapy approaches, involving the delivery of antisense oligonucleotides into t … Our program offers patients two types of clinical trials: ones that explore new ways to treat ALS, and ones that aim to learn more about the disease. Following informed consent and screening, participants with ALS will take Theracurmin 1 capsule (90mg) twice daily for 6-months. Researchers and biopharmaceutical companies (“drug companies”) are currently exploring potential new treatments for ALS through clinical trials. Individual Participant Data (IPD) Sharing Statement: Studies a U.S. FDA-regulated Drug Product: Studies a U.S. FDA-regulated Device Product: Individuals affected with ALS or a related neurodegenerative disease, Unaffected individuals from pedigrees in which the genetic cause of ALS is known, Individuals affected with ALS or a related neurodegenerative disease (sporadic or familial). Recently, investigators demonstrated that AMX0035 slowed ALS disease progression over six months, with impacts on various activities of daily living such as a patient’s ability to walk, talk, use utensils or swallow food. The study drug BIIB067 is thought to bind to both the normal and mutated copies of the SOD1 gene. ALS clinical trials related to familial ALS may help sporadic ALS and vice versa. While sporadic ALS patients have two normal copies of the SOD1 gene, most SOD1 ALS patients have one mutated copy of the SOD1 gene and one normal copy of the SOD1 gene. You will have an equal chance of being assigned to any active regimen. However, Johns Hopkins researchers are uncovering powerful discoveries for this progressive disease. Status: Active, EnrollingPrincipal Investigator: Nicholas Maragakis, M.D.Contact: Betsy Mosmiller | 410-502-0495Route of Administration: Subcutaneous infusion + open label extension (OLE)Length of Study: ~52 Weeks + 52 Weeks OLE. 1 out of every 4 subjects will receive a placebo instead of BIIB100. Greg Gowe was diagnosed with the disease in 2019 and has since helped form an … The main goal of this study is to determine if the study drug is tolerated by ALS subjects. It is a twice daily, oral study drug that aims to be neuroprotective and to reduce inflammation in specialized brain cells called microglia and mast cells that are affected in people with ALS. In a study funded in part by The ALS Association’s TREAT ALS program. This study is being done to determine the safety and tolerability of multiple doses of AT-1501 in adults with ALS. However, if you complete a regimen and then choose to participate in another regimen, you will not be randomized to your first regimen again. This would include FTD, MSP, IBMPFD, etc. Patients who enroll in this study must also be taking the approved ALS drug Riluzole (Rilutek®). In ALS, the CD40LG protein is produced in excess and is thought to be involved in neurodegeneration. Activation of the complement system is believed to play a role in the development and progression of ALS based on research in people with ALS and ALS mouse models. Distracting Myself From the Pain Helps Me Cope With It. Ibudilast is being investigated as a potential new therapy to slow the progression of ALS. Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00136500. The Department of Neurology is a national leader in clinical trials for multiple sclerosis, Alzheimer's disease, and other neurological conditions. Please understand that our phone lines must be clear for urgent medical care needs. Browse our list of actively recruiting clinical trials to learn more and find out how you may be able to participate. This population be sporadic or familial ALS. CRiALS is an umbrella protocol through which people are recruited to participate in a range of research studies being conducted by the ALS Research Collaboration (ARC). A regimen is a specific course of treatment, each with a different study drug. Choosing to participate in a study is an important personal decision. 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