Clinical Trial Alert: Phase 2/3 Study of Tideglusib (AMO-02) in Children and Adolescents with Congenital Myotonic Dystrophy

Researchers at AMO Pharma Ltd are seeking children and adolescents living with congenital myotonic dystrophy (DM1), also known as Steinert’s disease, to participate in a phase 2/3 clinical trial (REACH CDM) to evaluate efficacy of the investigational drug tideglusib (AMO-02) to treat congenital DM1. Tideglusib is designed to disrupt the RNA repeat that causes congenital DM1, . . .

Read More

Clinical Trial Alert: Early Phase Study of SRP-9001 in Boys with DMD

Researchers at Stanford Neuroscience Health Center are seeking 3-year-old boys living with Duchenne muscular dystrophy (DMD), particularly in California, to participate in an early phase clinical trial (a new cohort of the ENDEAVOR trial) to evaluate efficacy of Sarepta’s investigational gene replacement therapy SRP-9001 to treat DMD. SRP-9001 uses an adeno-associated virus (AAVrh74) to introduce a . . .

Read More

Capricor Announces Positive Results from its Phase 2 Study of CAP-1002 to Treat DMD

On September 24, Capricor Therapeutics announced positive results at the World Muscle Society Virtual Congress from its phase 2 HOPE-2 trial of the investigational therapy CAP-1002 for treatment of people in advanced stages of Duchenne muscular dystrophy (DMD). The primary and secondary endpoints of the study, the improvement of upper limb and cardiac function, were met, . . .

Read More

Clinical Trial Alert: Phase 2 Study of Ifetroban in Individuals with DMD

Researchers at Cumberland Pharmaceuticals Inc. are seeking boys and men living with Duchenne muscular dystrophy (DMD) to participate in a phase 2 clinical trial to evaluate the safety, efficacy, and duration of effect of oral ifetroban to treat heart disease associated with DMD. Ifetroban is designed to reduce fibrosis and fat deposition in the heart and . . .

Read More

Clinical Trial Alert: Phase 2/3 and Open-Label Extension Study of ASP0367 in Individuals with Primary Mitochondrial Myopathy

Researchers at Astellas Pharma Inc. are seeking individuals living with primary mitochondrial myopathy (PMM) to participate in a phase 2/3 clinical trial and open-label extension to evaluate safety, tolerability and preliminary efficacy of the investigational drug ASP0367 (MA-0211) to treat PMM. ASP0367 is being investigated regarding whether it can increase the number and function of the . . .

Read More

Clinical Trial Alert: Phase 1b/2a Study of Descartes-08 in Adults with Generalized Myasthenia Gravis

Researchers at Cartesian Therapeutics are seeking adults living with generalized myasthenia gravis (gMG) to participate in a phase 1b/2a clinical trial to evaluate safety, tolerability and manufacturing feasibility of the investigational drug Descartes-08 to treat gMG. Descartes-08 is designed to eliminate the aberrant plasma cells that cause gMG, potentially reducing the clinical symptoms of the disease. . . .

Read More

Clinical Trial Alert: Phase 3 Study of Pamrevlumab (FG-3019) in Combination with Systemic Corticosteroids in Ambulatory Boys with DMD

Researchers at FibroGen are seeking boys living with Duchenne muscular dystrophy (DMD) to participate in a phase 3 clinical trial to evaluate efficacy of the investigational drug pamrevlumab (FG-3019) in combination with systemic corticosteroids to treat DMD. Pamrevlumab is designed to decrease fibrosis in the muscles, thereby protecting the heart and lungs. This has the potential . . .

Read More

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

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 . . .

Read More