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";s:4:"text";s:26417:"The impact of an ALS clinical research & trial program in B.C – Mar 29, 2021. Data should come in the first half of the year, and will go a long way in telling whether zuranolone does indeed have a future. share to twitter. Inês holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Source Reference: Paganoni S, et al "Long-term survival of participants in the CENTAUR trial of AMX0035 for ALS" AAN 2021. share to facebook. Sabrina Paganoni, AAN 2021: CENTAUR Trial of AMX0035 for ALS Published Online: April 23rd 2021 It was a pleasure to meet with Sabrina Paganoni (Harvard Medical School, Boston, MA, USA) to discuss the CENTAUR study findings, and where AMX0035 is likely to be placed in the treatment paradigm for amyotrophic lateral sclerosis (ALS). Amylyx’s ALS drug met the goals of a mid-stage study, paving the way for regulatory submissions in Europe and Canada. Since skeletal muscle cells include those responsible for controlling breathing, Orion thought of evaluating whether oral levosimendan could help preserve lung health, delay the need for ventilation support, and maintain overall functionality in people with ALS. About 20% had bulbar onset ALS, when the disease first affects muscles involved in speaking, swallowing, and breathing. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. That's about the same number of Phase 2 or 3 ALS trials completed, terminated, or suspended from 2007 to 2018, Goyal calculated in a study in the journal Muscle & Nerve in January 2020. The impact of an ALS clinical research & trial program in B.C – Mar 29, 2021. Of note, supine slow vital capacity refers to the maximum volume of air patients could slowly inhale or exhale when in a supine position (lying horizontally face up). CReATe Biomarker Pilot Project Request for Applications 2021. Sabrina Paganoni, AAN 2021: CENTAUR Trial of AMX0035 for ALS Published Online: April 23rd 2021 It was a pleasure to meet with Sabrina Paganoni (Harvard Medical School, Boston, MA, USA) to discuss the CENTAUR study findings, and where AMX0035 is likely to be placed in the treatment paradigm for amyotrophic lateral sclerosis (ALS). No votes so far! But the FDA is requiring data from a larger Phase 3 clinical trial … They were joined by Kuldip Dave, PhD, of the ALS Association. Watch recording.October 8, 2020: Weekly Q&AMass General researchers Merit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD are joined by Sandy Morris of I AM ALS.Watch recording | Download slides (PDF)September 29, 2020, 2020: Monthly UpdateMerit Cudkowicz, MD, MSc, principal investigator, and Sabrina Paganoni, MD, co-principal investigator of the HEALEY ALS Platform Trial, provided an update on the progress of the HEALEY ALS Platform Trial. These data were presented in an oral session at the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference held March 15-18, 2021. Mar 9, 2021: Platform Trial Crosses 50% Enrollment Milestone The HEALEY ALS Platform Trial has passed the 50% participant enrollment milestone for the first 3 regimens. https://www.massgeneral.org/neurology/als/research/platform-trial-news Let us help you navigate your in-person or virtual visit to Mass General. That life-changing diagnosis came in May 2018 for Sean Healey, then the CEO of an asset management firm worth more than $8 billion. We are committed to an ongoing dialogue about the HEALEY ALS Platform Trial with the entire ALS patient community. The participant locating service, a novel approach to ALS research, could prove useful in other future ALS trials to collect survival data. The idea of weekly interactive sessions came from our Patient Advisory Committee and we absolutely love the opportunity to connect with all who might be interested on a frequent basis. KC man diagnosed with ALS will be part of highly competitive medical trial. Mar 11, 2021: Advances in Motion: Accelerating Treatments with the HEALEY Center Platform TrialIn this video from Advances in Motion, Merit E. Cudkowicz, MD, MSc, chief of the Department Neurology and director of the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, discusses the HEALEY ALS Platform Trial, which aims to accelerate the discovery of new drug therapies for people with amyotrophic lateral sclerosis (ALS). share to twitter. In particular, “short duration of disease, worse respiratory function, and faster progression of ALS suggested better response to levosimendan,” she said, adding that this type of subgroup analysis “may be of value in identifying patient groups with the stronger treatment effect for future trial designs.”. Patients’ mean age was 59.2 years, 61.9% were men, and they had been living with the disease for a median of 24.3 months (about two years). Notably, among the subgroups of patients with faster disease progression and worse lung function, significant therapeutic effects were only observed at 48 weeks, rather than throughout the study. collected, please refer to our Privacy Policy. Editor’s note: The ALS News Today team is providing in-depth coverage of the 2021 Virtual AAN Annual Meeting, April 17–22. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. The bottom line is this: we are committed to beginning the trial as soon and as safely as we can. CENTAUR was a 24-week placebo-controlled study of 137 participants with ALS that evaluated the safety and efficacy of AMX0035. This trial is designed as a perpetual platform trial. Three of those trials are expected to deliver results in 2021. ALS Drug Development Webinar Series Part 4: Site Considerations May 14, 2021 / 3:30 PM - 4:30 PM. Safe Care CommitmentGet the latest news on COVID-19, the vaccine and care at Mass General.Learn more. Explore fellowships, residencies, internships and other educational opportunities. email article. These findings highlighted that while levosimendan “did not significantly improve the respiratory function or overall functionality in the broad population of people with ALS, there was a trend to treatment effects in a subgroup of participants,” Cudkowicz said. NEALS welcomes researchers, principal investigators, and companies to share their work on the NEALS site in our clinical trial listings. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. We use cookies and other tools to enhance your experience on our website and Watch recording | Download slides (PDF)October 22, 2020: Weekly Q&AEnrollment updates, trial sites, and patient questions.Watch recording | Download slides (PDF)October 21, 2020: ALS/MND Alliance Clinical Trial Design WebinarMerit Cudkowicz, MD, MSc joined researchers from Europe and Scotland to discuss new research and clinical trial models, how they will change the landscape going forward and their practical implications for patients and researchers. The disease has a roughly three-year median survival. Background: AMX0035 is an orally administered, fixed-dose coformulation (sodium phenylbutyrate-taurursodiol) designed to reduce neuronal death by mitigating endoplasmic reticulum and mitochondrial dysfunction. Results showed that some subgroups of levosimendan-treated patients did significantly better in terms of maintaining their ALSFRS-R scores than those given a placebo. HEALEY ALS Platform Trial Investigational Products Tested in the Trial 1 Merit Cudkowicz, MD Principal Investigator Sabrina Paganoni, MD, PhD Co-Principal Investigator Verdiperstat February 18, 2021. For more information about these cookies and the data Nicholson and Babu will discuss some of the issues faced by sites in a clinical trial including the specific steps and resources a site needs to open and run a clinical trial and an expanded access program. These included patients with shorter disease duration (less than about two years), faster disease progression, worse lung function, and greater heart rate changes. September 18, 2019: Sean M. Healey & AMG Center for ALS at Mass General launches first ALS Platform Trial with 5 promising drugsThe Sean M. Healey & AMG Center at Massachusetts General Hospital will launch the first platform trial for amyotrophic lateral sclerosis (ALS) to accelerate the development of effective and breakthrough treatments for people with ALS. First Patient Enrolled in Phase 2a Trial ... - ALS News Today Read Announcement. to analyze our web traffic. Learn about the companies working with our team to produce new treatments. Marta Figueiredo holds a master’s in evolutionary and developmental biology and a PhD in biomedical sciences from the University of Lisbon, Portugal. We are committed to providing expert care—safely and effectively. Merit Cudkowicz understands speed. The General Hospital Corporation. Watch recording | Download slides (PDF)December 10, 2020: Weekly Q&ASabrina Paganoni, MD, PhD presented this week's updates on the HEALEY ALS Platform Trial, discussed the enrollment process and study visits, and answered questions from the audience.Watch recording | Download slides (PDF)December 3, 2020: Weekly Q&ASabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented the week's updates on the HEALEY ALS Platform Trial and the Expanded Access Program, discussed the relationship between clinical research trials for ALS and clinical care, and answered questions from the audience.Watch recording | Download slides (PDF)November 30, 2020: Monthly UpdateMerit Cudkowicz, MD, MSc, principal investigator, and Sabrina Paganoni, MD, co-principal investigator of the HEALEY ALS Platform Trial, provided an update on the progress of the HEALEY ALS Platform Trial. Read Announcement. Long-Term Survival of Participants in the CENTAUR Trial of AMX0035 for ALS. The REViVALS-1A study (NCT04632225) intends to recruit 18 patients across four clinical sites in the U.S. Enrollment is open to adult ALS patients displaying motor symptoms in their limbs for … This will improve access to experimental treatments for people with ALS. It works by increasing calcium sensitivity in both heart and skeletal muscle fibers, potentially improving their function. Effectiveness of Two Oral Appliances for Managing Oral Self-biting Injuries in Patients With ALS on March 22, 2021 at 4:00 pm The Phase I/II clinical trial will be initiated in late 2021 or early 2022 as a multi-center, three-part study to evaluate the safety, tolerability and efficacy of APB-102 in patients with SOD1 ALS … email article. An Open-label, Single-center, 6-month Trial of Theracurmin for Patients With Amyotrophic Lateral Sclerosis (ALS) Actual Study Start Date : August 28, 2020: Estimated Primary Completion Date : August 15, 2021: Estimated Study Completion Date : August 15, 2021 Paganoni S, et al. Researchers conducted subgroup analysis to evaluate whether patients’ characteristics at the study’s start (baseline) determined their response to treatment. AAN 2021 Virtual Congress, 17-22 April. Please join us as often as you would like! https://medicalxpress.com/news/2021-01-als-drug-pridopidine-trial.html Contact an ALS trial liaison. share to linkedin. But the FDA is requiring data from a larger Phase 3 clinical trial … Further studies are needed to confirm the predictive value of these biomarkers, the researchers noted. March 25, 2020: COVID Updates and April 8th WebinarAs you all are, we too are closely monitoring the impact of COVID-19 on the HEALEY ALS Platform Trial timelines and operations. An Open-label, Single-center, 6-month Trial of Theracurmin for Patients With Amyotrophic Lateral Sclerosis (ALS) Actual Study Start Date : August 28, 2020: Estimated Primary Completion Date : August 15, 2021: Estimated Study Completion Date : August 15, 2021 We will continue to share enrollment updates as well as new ideas and opportunities. Determinants of clinical response to levosimendan in the REFALS phase 3 study in people with ALS, #AANAM – Trial Will Evaluate Tofersen in Presymptomatic SOD1 Patients, Quick Approval Given COVID Vaccines Raise Concerns, Rare Disease Patients Say, Distracting Myself From the Pain Helps Me Cope With It, An Ounce of Prevention is Worth a Pound of Cure (Benjamin Franklin, 1736), My Cuisine Adventures While Living with ALS. Inês currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner. Learn about the many ways you can get involved and support Mass General. Copyright © 2007-2021. Tofersen (BIIB067 – Previously IONIS-SOD1Rx). Watch recording | Download slides (PDF), April 8, 2021: Weekly Q&A Descriptions: Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial, discussed voice app biomarkers with guest speaker Jeremey Shefner, MD, PhD and answered questions from the audience. “We never want to miss a group of people that might be benefiting from the treatment,” Cudkowicz concluded. The Phase 2 trial … HEALEY ALS Platform Trial Investigational Products Tested in the Trial 1 Merit Cudkowicz, MD Principal Investigator Sabrina Paganoni, MD, PhD Co-Principal Investigator Verdiperstat February 18, 2021. Jan 12, 2021: HEALEY ALS Platform Trial Offers Study Drug Webinar Series A new webinar series gives members of the ALS community the opportunity to learn about the mechanism of action and science behind each of the 4 drugs currently being tested in the Platform Trial. A listing of Amyotrophic Lateral Sclerosis (ALS) medical research trials actively recruiting patient volunteers. We are sorry that this post was not useful for you! Long-Term Survival of Participants in the CENTAUR Trial of AMX0035 for ALS. Please contact Carly Allen, Communications Manager, Northeast ALS Consortium at (855) 437-4823 or [email protected] for more information about clinical trials. REFALS included a broad ALS patient population, reflecting what is typically seen in ALS trials, said Cudkowicz, who is also the chair of neurology at Massachusetts General Hospital in Boston. Amyotrophic Lateral Sclerosis (ALS) Clinical Trials. This analysis, along with REFALS data on potential ALS biomarkers, were presented at the 2021 virtual American Academy of Neurology annual meeting, running through April 22. An analysis of potential associations between the levels of several molecules and disease progression, as assessed with the ALSFRS-R and supine slow vital capacity, revealed that muscle-related biomarkers — creatinine, creatinine clearance, creatine kinase, and cardiac troponin T — were significantly associated with disease progression. We have remained at the forefront of medicine by fostering a culture of collaboration, pushing the boundaries of medical research, educating the brightest medical minds and maintaining an unwavering commitment to the diverse communities we serve. Watch recording | Download slides (PDF), April 15, 2021: Weekly Q&A Descriptions: Sabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented this week’s updates on the HEALEY ALS Platform Trial with guest speaker Adam Quick, MD, from The Ohio State University Wexner Medical Center and answered questions from the audience. Watch recording | Download slides (PDF), March 25, 2021: Weekly Q&A Sabrina Paganoni, MD, PhD presented the week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.Watch recording | Download slides (PDF), March 11, 2021: Weekly Q&ASabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented the week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.Watch recording | Download slides (PDF)March 4, 2021: Weekly Q&ASabrina Paganoni, MD, PhD presented the week’s updates on the HEALEY ALS Platform Trial and answered questions from the audienceWatch recording | Download slides (PDF)February 11, 2021: Weekly Q&ASabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented the week’s updates on the HEALEY ALS Platform Trial and answered questions from the audience.Watch recording | Download slides (PDF)January 28, 2021: Weekly Q&AMerit Cudkowicz, MD, MSc and Sabrina Paganoni, MD, PhD presented the week’s updates on the HEALEY ALS Platform Trial, discussed the trial design, and answered questions from the audience.Watch recording | Download slides (PDF)January 14, 2021: Weekly Q&ASabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented the week's updates on the HEALEY ALS Platform Trial, discussed the expanded access program, and answered questions from the audience.Watch recording | Download slides (PDF)January 7, 2021: Weekly Q&ASabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented the week's updates on the HEALEY ALS Platform Trial, discussed the expanded access program, and answered questions from the audience.Watch recording | Download slides (PDF)December 17, 2020: Weekly Q&ASabrina Paganoni, MD, PhD and Merit Cudkowicz, MD, MSc presented the week’s updates on the HEALEY ALS Platform Trial, discussed open label extension and COVID impact, and answered questions from the audience. 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