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A Phase I/II Study of Liposomal Cytarabine and Daunorubicin (CPX-351) in Combination With Quizartinib in Patients With Acute Myeloid Leukemia (AML) and High Risk Myelodysplastic Syndrome (MDS)
This phase I/II trial studies the side effects and best dose of CPX-351 in combination with quizartinib for the treatment of acute myeloid leukemia and high ri…
Metformin as an add-on or Monotherapy in Treatment of Aging People With Multiple Sclerosis (MS): Randomized, Placebo-controlled Pilot Study.
The goal of the study is to learn about treating older people with multiple sclerosis (MS) with metformin. Metformin may be used as a single therapy or as an a…
Spinal Cord Associative Plasticity for Amyotrophic Lateral Sclerosis
Veterans are at higher risk than non-Veterans of falling ill with amyotrophic lateral sclerosis (ALS). The investigators have shown that synchronized stimulat…
An Open-label Single Center, Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for Amyotrophic Lateral Sclerosis (ALS) Due to CHCHD10 Gene Mutation
This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with amyotrophic lateral sclero…
T Cell Profiling in Patients with Multiple Sclerosis
The goal of this research study is to to learn more about the body's immune response in patients with multiple sclerosis (MS). In MS, the body's immune cells m…
Increasing Physical Activity Via Provider Prescription and Engagement: Efficacy of ExerciseRx for Adults With Multiple Sclerosis (MS)
This study aims to advance the scientific understanding and potential future implementation of physical activity promotion by testing the efficacy of a phone-b…
A Phase III, Multicentre, Randomised, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Elafibranor in Adult Participants With Primary Sclerosing Cholangitis
The purpose of this study is to find out how well and safely elafibranor works compared to placebo in adult participants with Primary sclerosing cholangitis (P…
A Randomized, Double-blind, Parallel Group, Placebo-controlled Multicenter Study to Evaluate Efficacy, Safety and Tolerability of Ianalumab in Participants With Diffuse Cutaneous Systemic Sclerosis
The purpose of this study is to evaluate efficacy, safety and tolerability of s.c. ianalumab administered in participants with diffuse cutaneous systemic scler…
STICH3C Cardiac Magnetic Resonance (CMR) Observational Study (STICOS)
STICOS will test the hypothesis that residual jeopardized myocardium, late gadolinium enhancement, and non-ischemic substrate after revascularization is associ…
A Pilot Study of the Pharmacodynamics and Clinical Effects of Oral Extended Release (OER) Glibenclamide in Multiple Sclerosis Patients With Neuropathic Pain
This is an early phase safety evaluation of the use of oral extended release (OER) glibenclamide, which is otherwise known as glyburide, for use as a treatment…
Toward Ubiquitous Lower Limb Exoskeleton Use in Children and Young Adults: Exploratory Observational Analysis of Novel Robotic Control Strategies
People with cerebral palsy (CP), muscular dystrophy (MD), spina bifida, or spinal cord injury often have muscle weakness, and problems moving their arms and le…
The Pre-symptomatic Familial Amyotrophic Lateral Sclerosis (Pre-fALS) Study
Pre-fALS is a prospective natural history and biomarker study of people not yet affected with ALS, but who are at genetic risk for developing ALS. The investig…