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Natural History of Inflammatory Muscle Diseases
Objective: To collect information and biospecimens (such as blood, muscle, and skin samples) that will be used to research causes and treatments of inflammato…
Neurologic Bone Marrow Derived Stem Cell Treatment Study
This is a human clinical study involving the isolation of autologous bone marrow derived stem cells (BMSC) and transfer to the vascular system and inferior 1/3…
A Phase 1b Multicenter, Non-randomized, Open-label Study to Investigate the Safety, Tolerability, Pharmacokinetics, Immunogenicity, and Pharmacodynamics of Single Ascending Doses of a New Subcutaneous Formulation of Ocrelizumab in Patients With Multiple Sclerosis
The main purpose of this study is to evaluate the safety and tolerability of the ocrelizumab subcutaneous (SC) test formulation in participants with multiple s…
A Study to Evaluate the Biological Effect of Tofersen in Adults With Amyotrophic Lateral Sclerosis Without Mutations in SOD1
The goal of this clinical trial is to evaluate whether tofersen is safe and effective in adults with non-SOD1 ALS. Tofersen is currently approved by the U.S. F…
The Role of Anifrolumab in Improving Markers of Vascular Risk in Patients With Systemic Lupus Erythematosus
Background: People with systemic lupus erythematosus (SLE) are at risk of developing complications in their blood vessels. This can increase the risk of heart…
A Phase 1/2 Study of REGN5678 (Anti-PSMAxCD28) With or Without Cemiplimab (Anti-PD-1) in Patients With Metastatic Castration-Resistant Prostate Cancer and Other Tumors Associated With PSMA Expression
The main purpose of this study is to determine the safety, tolerability (how the body reacts to the drug\[s\]) and effectiveness (ability to treat the cancer) …
The Effect of Corticosteroids on Inflammation at the Edge of Acute Multiple Sclerosis Plaques: An Investigator-Blinded Study
Background: Multiple sclerosis (MS) affects the brain, spinal cord, and optic nerves. MS lesions can appear on the MRI (magnetic resonance imaging) scans in m…
Healing ALS Registry Observational Study (HAROS)
This is a prospective, observational, online study of people diagnosed with ALS, MND or PLS referred to as HAROS (Healing ALS Registry Observational Study). Pa…
Characterization of the Pathogenesis of Lymphangioleiomyomatosis (LAM)
Pulmonary lymphangioleiomyomatosis (LAM) is a destructive lung disease typically affecting women of childbearing age. Currently, there is no effective therapy …
TSC Alliance Tuberous Sclerosis Complex (TSC) Biosample Repository and Natural History Database
The TSC Biosample Repository collects and stores samples of blood, DNA, and tissues that scientists can request to use in their research. The samples we collec…
The Effects of Strength Training on Muscle Mass and Mental Health in Adults Taking Obesity Medication: A Pilot Study
The purpose of this study is to examine the effects of resistance exercise, such as weightlifting, on retention of muscle mass and mental health in individuals…
A Double-blind, Randomised, Placebo-controlled Trial Evaluating the Efficacy and Safety of Oral Nerandomilast Treatment in Patients With Systemic Sclerosis (SSc)
Nerandomilast is being developed to help people with systemic sclerosis by potentially improving symptoms and slowing disease progression. This study is open t…