Tag: Clinical Trials

News, Research

Clinical Trial Alert: Phase 2 Study of Pitolisant for Treatment of Excessive Daytime Sleepiness in Adults with Myotonic Dystrophy, Type 1 (DM1)

MDA Staff

Researchers at Harmony Biosciences are seeking adults living with myotonic dystrophy, type 1 (DM1) to participate in a phase 2 clinical trial and open-label extension to evaluate safety and efficacy of the investigational drug pitolisant (brand name Wakix) to treat excessive daytime sleepiness and other non-muscular symptoms of DM1. Pitolisant treatment increases the amount the chemical . . .

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News, Research

Clinical Trial Alert: Phase 1b and Open-Label Extension Study of ASP0367 in Boys with DMD

MDA Staff

Researchers at Astellas Pharma Inc. are seeking boys living with Duchenne muscular dystrophy (DMD) to participate in a phase 1b clinical trial and open-label extension to evaluate safety, tolerability and preliminary efficacy of the investigational drug ASP0367 (MA-0211) to treat DMD. ASP0367 is designed to increase the number and function of the mitochondria in cells, thereby . . .

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News, Research

Clinical Trial Alert: Phase 3 Study of Rozanolixizumab in Adults with Generalized Myasthenia Gravis

MDA Staff

Researchers at The Neurological Institute, PA, located in Charlotte, North Carolina, are seeking adults living with generalized myasthenia gravis (gMG) to participate in a phase 3 clinical trial to evaluate efficacy and safety of the investigational drug rozanolixizumab. Rozanolixizumab is designed to alter the immune response and reduce antibody production, potentially improving strength, fatigue, swallowing, and . . .

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News, Research

Clinical Trial Alert: Phase 3 Study of ION363 in Individuals with ALS with Fused in Sarcoma Mutations (FUS-ALS)

MDA Staff

Researchers at IONIS Pharmaceuticals are seeking individuals living with amyotrophic lateral sclerosis (ALS) with fused in sarcoma mutations (FUS-ALS) to participate in a phase 3 clinical trial to determine the efficacy of the investigational drug ION363. Drug efficacy will be determined by measuring clinical functioning and survival in trial participants.   The study consists of two parts, . . .

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News, Research

The Search for ALS Treatments Continues

MDA Staff

May is Amyotrophic Lateral Sclerosis (ALS) Awareness Month, and while this past year was different — given the global pandemic we all endured — the research and support for finding new treatments never stopped.  ALS is a neurodegenerative disease of the motor neurons that eventually causes muscular atrophy, paralysis, and death. The cause of ALS is understood . . .

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Clinical Trial Alert: Phase 2 Study of Pegcetacoplan in Adults with Sporadic ALS (MERIDIAN)

MDA Staff

Researchers at Apellis Pharmaceuticals are seeking individuals living with sporadic amyotrophic lateral sclerosis (ALS) to participate in a two-year phase 2 trial (MERIDIAN) to determine the efficacy of the investigational drug pegcetacoplan. Pegcetacoplan is designed to reduce the activity of the complement system (a component of the immune system), and potentially slow the progression of ALS. . . .

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News, Research

Clinical Trial Alert: Phase 1/2 Study of AT845 in Late-Onset Pompe Disease

MDA Staff

Researchers at Audentes Therapeutics, an Astellas company, are seeking individuals living with late-onset Pompe disease (LOPD) to participate in a phase 1/2 study to confirm safety and efficacy of the investigational drug AT845. This gene-replacement therapy may offer the benefit of long-term improvement of motor and respiratory function and quality of life in adults living with . . .

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News, Research

Clinical Trial Alert: Observational Study to Identify Biomarkers in Individuals Receiving Radicava for ALS

MDA Staff

Researchers at Mitsubishi Tanabe Pharmaceutical America (MTPA) are seeking individuals living with amyotrophic lateral sclerosis (ALS) to participate in a six-month observational study. This study is designed to identify biomarkers to serve as quantifiable, biological, non-clinical measures of Radicava (edaravone) effects on ALS. Observational measures might include assessment of vitals; blood and urine collection; safety assessments; use . . .

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Clinical Trial Alert: Phase 3 Study of Zilucoplan in Individuals With Generalized Myasthenia Gravis

MDA Staff

Researchers at Ra Pharmaceuticals (now part of UCB BioSciences) are seeking individuals living with generalized myasthenia gravis (gMG) to participate in a phase 3 study to confirm safety, tolerability, and efficacy of the investigational drug Zilucoplan. This therapy may improve muscle function by blocking the autoimmune reaction that destroys the junction between nerves and muscles in . . .

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News, Research

Clinical Trial Alert: Phase 3 Study of Rozanolixizumab in Individuals With Generalized Myasthenia Gravis

MDA Staff

Researchers at UCB BioSciences are seeking individuals living with generalized myasthenia gravis (gMG) to participate in a phase 3 study. The objective of the study is to confirm the clinical efficacy and to assess safety and tolerability of rozanolixizumab. The therapy may help reduce the typical signs and symptoms of gMG, which are caused by autoantibodies . . .

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