Neuromuscular Clinical Trials
Embassy Pompe Disease Study
The purpose of this study is to evaluate new ways of assessing treatment with alglucosidase alfa. Alglucosidase alfa is an enzyme replacement therapy that is approved by the FDA for patients Pompe disease (GAA deficiency). Learning more about how the study drug works may help researchers develop new treatment options for patients with Pompe disease. Enrollment now open
Rasagiline in ALS
Rasagiline has shown broad neuroprotective activity against a variety of neurotoxins.
The specific aim of this screen study is to determine whether rasagiline is safe in this patient population and if the drug has the potential to slow ALS disease progression. Should rasagiline prove effective in slowing motor function decline and prolong ALS patient survival, it would have an immediate impact on clinical management of this severe neurological disorder.
Methotrexate in Myasthenia Gravis
The specific aim of this study is to determine if oral methotrexate is a safe and effective therapy for myasthenia gravis (MG) patients who are on prednisone. The hypothesis is that adding methotrexate therapy in these patients will improve the MG manifestations so that prednisone dose can be reduced and clinical measures of MG severity will improve. Methotrexate (MTX) is an analogue of folic acid and has been used in the treatment of cancer and autoimmune diseases. Several pharmacological mechanisms have been reported to explain the drug’s effects as an autoimmune/anti-inflammatory agent.
AT2220 and Alglucosidase Alfa in Patients with Pompe Disease
To evaluate the safety of ascending oral doses of an investigational new compound called AT2220 administered 1 hour before administration of alglucosidase alfa in patients with Pompe disease This study is intended to provide proof-of-concept that pre-administration of AT2220 (duvoglustat hydrochloride) has the potential to safely enhance the action and properties of alglucosidase alfa.
Duchenne Muscular Dystrophy (DMD) Study (DELOS Trial)
The aim of this Phase III study is to assess the efficacy of idebenone on pulmonary function, motor function, muscle strength and quality of life in patients with DMD.
Contact Us
PLEASE NOTE:
To be involved in the neuromuscular drug trials, you must be evaluated and followed at the Methodist Neurological Institute Neuromuscular Clinical Center.
For more information about this study, please contact:
Sharon L. Halton, MSW, LCSW
Senior Research Coordinator
Phone: 713-441-3420
Fax: 713-793-7271
E-mail: SLHalton@tmhs.org
Luis F. Lay, Jr., MD
Senior Research Coordinator
Phone: 713-441-3057
Fax: 713-793-7271
E-mail: LFLayJr@tmhs.org
