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AutoInflammatory Disease Alliance Registry (AIDA)
Autoinflammatory diseases (AID) are clinical entities characterized by recurrent inflammatory attacks in absence of infection, neoplasm or deregulation of the adaptive immune system. Among them, hereditary periodic syndromes, also known as monogenic AID, represent the prototype of this disease group, caused by mutations in genes involved in the regulation of innate immunity, inflammation and cell death. Based on recent experimental acquisitions in the field of monogenic AID, several immunologic disorders have been reclassified as polygenic/multifactorial AID, sharing pathogenetic and clinical features with hereditary periodic fevers. This has paved the way to new treatment...
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Autologous Bone Marrow Aspirate Concentrate in Patients Undergoing Meniscectomy
The proposed research study aims to evaluate the effects of autologous bone marrow aspirate concentrate (BMAC) on the development and progression of osteoarthritis (OA) in patients undergoing meniscectomy. This prospective, randomized, double-blind clinical trial will compare patient-reported outcomes, specifically IKDC scores between patients who receive BMAC post-meniscectomy and those who receive a saline control injection. The study will also compare physical examination, MRI, radiographs, and synovial fluid analysis. Our hypothesis is that those who receive the autologous BMAC injection after the procedure will have better outcomes than those who do not.
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Autologous Stem Cell Transplant (ASCT) for Autoimmune Diseases
A subset of autoimmune diseases (ADs) in children and young adults are life-threatening and unresponsive to conventional treatments. In these patients, the delivery of high dose immunosuppressive therapy followed by autologous stem cell transplant (ASCT) offers a treatment strategy capable of purging the pathogenic, autoreactive immune system and an opportunity for "immune reset." This strategy has been used in adults across a myriad of indications with evidence for efficacy. This study proposes a pilot study to evaluate this therapeutic strategy in children and young adults with systemic sclerosis (SSc) and systemic lupus erythematosis (SLE), two potentially life threatening...
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Autologous Stem Cell Transplantation in Patients With Systemic Sclerosis
The purpose of this study is to determine whether a regimen of high-dose immunoablative therapy will demonstrate safety that is consistent or improved with other published regimens in SSc patients, while maintaining a treatment effect.
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BAFF CAR-T Cells (LMY-920) for Systemic Lupus Erythematosus
This phase 1 study seeks to examine the safety and recommended phase 2 dose (RP2D) of BAFF-ligand CAR-T cells (LMY-920) in adult patients with refractory systemic lupus erythematosus (SLE). It is hypothesized that BAFF CAR-T cells will be safe and will improve SLE disease activity scores.
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Baricitinib in the Treatment of Kohlmeier-Degos Disease in Patients With Neurological Involvement
Background: Kohlmeier-Degos (KD) is a rare disease that causes inflammation and blood clots, leading to blockages in small blood vessels. These blockages can result in K-D lesions throughout the body, affecting the skin, lungs, heart, spinal cord, and brain. KD can be fatal. No treatment exists for this disease. Objective: To test a study drug (baricitinib) in people with brain and spine lesions caused by KD disease. Baricitinib is FDA approved to treat other disorders but has not yet been tried in people with KD. Eligibility: People aged 18 years or older with KD-related lesions in the brain and spine. Design: Participants will be screened; they will have a...
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Bayer OA Knee Pain Pilot
The objective of this study is to investigate if MRI can be used to evaluated effect of knee artery embolization for knee osteoarthritis. Participants be evaluated in clinic, obtain a knee MRI, undergo embolization of the symptomatic knee, and follow up in clinic at 1, 6, and 12 months after embolization. A second MRI is obtained 6 months after embolization. Participants will keep a record of their pain level and treatment and answer questionnaires at each visit. In addition, this study aims to determine the effects of knee artery embolization on the amount of opioid (pain reliever drugs) needed to manage osteoarthritis-associated pain and change in quality of life.
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Belimumab and Rituximab Combination Therapy for the Treatment of Diffuse Cutaneous Systemic Sclerosis
This is a 52 week, single center, randomized, double-blind, placebo-controlled study. After patients maintain a stable dose of Mycophenolate Mofetil (MMF) for at least 1 month, they will be randomized to treatment with either Belimumab & Rituximab or placebo.Patients in both groups will be on background MMF for the entirety of the study. Belimumab will be administered subcutaneously and Rituximab intravenously. Placebo injections and infusions will be of normal saline. Randomization will be done in a 2:1 manner to favor the treatment group. It is hypothesized that that Rituximab and Belimumab combination therapy with Mycophenolate Mofetil background therapy will improve...
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BFRT for Subacromial Pain
The goal of this pilot clinical trial is to learn if blood flow restriction training is safe and feasible in patients with subacromial pain. The main questions the investigators aim to answer are: - What problems do participants with shoulder pain encounter when training with blood flow restriction? - Is it feasible to apply blood flow restriction training as part of a physical therapy intervention? - What are some preliminary effects of blood flow restriction training on the recovery of shoulder function and strength? The investigators will compare active blood flow restriction training to sham (an inactive procedure designed to mimic the active...
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Biofeedback Retention in Individuals With AKA
More than two million Americans are currently living with a full or partial limb loss, and an additional 185,000 amputations occur each year. The majority of amputations occur in the lower limbs. There are many potential causes for amputation, but the majority can be attributed to vascular diseases, such as diabetes, traumatic injury, and cancer. For these individuals, prosthetic devices play an important role in restoring mobility and enabling them to participate in everyday activities. However, when learning to use these devices, patients often alter their movement patterns to compensate for pain or discomfort, a decreased ability to feel what their prosthetic limb is doing,...
