Clinical Trials

    Showing 1 - 10 of 12 Appel or shroff or greene or thonhoff

    A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1

    Status: Enrolling

    Investigator: Ericka Greene

    Study Coordinator: Aramide Balogun

    Phone: 713.441.6955

    A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1... Read more >

    A Randomized, Double-Blind, Placebo-Controlled Phase 3 Trial of Descartes-08 in Patients With Generalized Myasthenia Gravis (MG)

    Status: Enrolling

    Investigator: Sheetal Shroff

    Study Coordinator: Amelia Nounes

    Phone: 346.356.3639

    The AURORA Study is evaluating the safety, tolerability, and efficacy of an investigational mRNA CAR T-cell therapy known as Descartes-08 in adults with acetylcholine receptor autoantibody -positive generalized myasthenia gravis. Part 1 of the study ... Read more >

    A Study of Biological Markers for the Nervous and Immune Systems and Free Radical Mediated Processes in Amyotrophic Lateral Sclerosis (ALS)

    Status: Enrolling

    Investigator: Stanley Appel

    Study Coordinator: Aramide Balogun

    Phone: 713.441.6955

    The purpose of this protocol is to develop and maintain a biospecimen bank comprised of blood, urine, CSF collected during diagnostic evaluations, tissue from biopsies and autopsies collected from patients with neurological disease and non-neurolog... Read more >

    A Study to Investigate the Efficacy, Safety and Tolerability of Remibrutinib Versus Placebo in Adult Patients With Generalized Myasthenia Gravis (RELIEVE)

    Status: Open Not Enrolling

    Investigator: Ericka Greene

    Study Coordinator: Aramide Balogun

    Phone: 713.441.6955

    This study is a randomized, double-blind, placebo-controlled, multicenter, Phase III study, to evaluate the efficacy, safety and tolerability of remibrutinib in gMG patients who are on stable SOC treatment. Approximately 180 eligible participants wil... Read more >

    COMPILATIONS OF CLINICAL FINDINGS IN AMYOTROPHIC LATERAL SCLEROSIS

    Status: Open Not Enrolling

    Investigator: Stanley Appel

    Study Coordinator: Aramide Balogun

    Phone: 713.441.6955

    The purpose of this protocol is to develop and maintain a clinical findings data bank including diagnostic evaluations obtained from medical records sent from prior medical evaluations, diagnostic intake evaluations and follow-up care in the Methodis... Read more >

    ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis) (ConTTRibute)

    Status: Enrolling

    Investigator: Sheetal Shroff

    Study Coordinator: Samia Saad

    Phone: 832.915.6410

    The purpose of this study is to: Describe epidemiological and clinical characteristics, natural history and real-world clinical management of ATTR amyloidosis patients Characterize the safety and effectiveness of patisiran and vutrisiran as part ... Read more >

    Global Study of Del-desiran for the Treatment of DM1 (HARBOR)

    Status: Open Not Enrolling

    Investigator: Ericka Greene

    Study Coordinator: Aramide Balogun

    Phone: 713.441.6955

    The study consists of a Screening Period of up to 6 weeks and 54-week Treatment Period. The anticipated duration is approximately 60 weeks. Participants will be randomized to receive an intravenous infusion of either del-desiran or placebo at the ... Read more >

    HEALEY ALS Platform Trial

    Status: Enrolling

    Investigator: Sheetal Shroff

    Study Coordinator: Aramide Balogun

    Phone: 713.441.6955

    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.... Read more >

    HEALEY ALS Platform Trial - Regimen I NUZ-001

    Status: Enrolling

    Investigator: Sheetal Shroff

    Study Coordinator: Aramide Balogun

    Phone: 713.441.6955

    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. Regimen I will evaluate the safety and efficacy of a single study drug... Read more >

    Long-Term, Observational, Registry of Patients With Generalized Myasthenia Gravis Who Have Received Treatment With Complement C5 Inhibition Therapies

    Status: Open Not Enrolling

    Investigator: Ericka Greene

    Study Coordinator: Samia Saad

    Phone: 832.915.6410

    Complement component 5 (C5) inhibition therapies have been available for patients with generalized Myasthenia Gravis (gMG) in the United States since the introduction of Soliris® in late 2017. Alexion is developing potential new therapies that leve... Read more >

    RESET-MG: A Study to Evaluate the Safety and Efficacy of CABA-201 in Participants With Generalized Myasthenia Gravis

    Status: Enrolling

    Investigator: Sheetal Shroff

    Study Coordinator: Delrose Vernon

    Phone: 346.238.9068

    Myasthenia gravis (MG) is a rare autoimmune disorder characterized by autoantibody responses that cause defective transmission of signals at the neuromuscular junction, resulting in a distinctive pattern of weakness. Patients with generalized MG (gMG... Read more >

    TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (TRITON-PN)

    Status: Enrolling

    Investigator: Sheetal Shroff

    Study Coordinator: Delrose Vernon

    Phone: 346.238.9068

    The purpose of this study is to: Determine the efficacy of nucresiran in patients with hATTR-PN by evaluating the effect on neurologic impairment, quality of life, nutritional status, disability, and gait speed Demonstrate superiority of nucresir... Read more >