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An Open-label Phase 2a Study to Evaluate the Safety and Efficacy of AlloNK®, an Allogeneic Cord Blood-derived NK Cell Therapy, in Combination With Rituximab in Relapsing Forms of B-cell Dependent Rheumatologic Diseases
A Basket Trial of Refractory Rheumatoid Arthritis (RA), Sjögren's Disease (SjD), Idiopathic Inflammatory Myopathies (IIMs) and Systemic Sclerosis (SSc) subject…
Cannabidiol for the Treatment of Diabetic Peripheral Neuropathy: Pilot Study
The "Cannabidiol for the Treatment of Diabetic Peripheral Neuropathy: Pilot study (CBD-DPN1)" is a double-blinded, placebo-controlled, crossover pilot study ev…
A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial)
Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque- like sub…
Single Neuron Population Dynamics in Human Speech Motor Cortex for a Speech Prosthesis
The purpose of this study is to obtain preliminary device safety information and demonstrate proof of principle (feasibility) of the ability of people with tet…
Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC)
This is a treatment guideline for a second or greater allogeneic hematopoietic stem cell transplant (HSCT) using a reduced intensity conditioning (RIC) in pati…
The Myelin Disorders Biorepository Project and Global Leukodystrophy Initiative Clinical Trials Network
The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to s…
Photoplethysmography (PPG) to Predict Ejection Fraction and Other Echographic Data in the General Population
The investigators are aiming to investigate the association between ejection fraction (EF) determined by echocardiography and signals obtained from Photoplethy…
Center Without Walls for Imaging Proteinopathies With PET (CW2IP2): Imaging and Diagnostic Assessments
This current protocol will provide the key data to help determine the specificity of our to-be-developed radiotracers by implementing a multi-site diagnostic a…
Understanding and Restoring Speech Production Using an Intracortical Brain-computer Interface
The goal of this study is to improve our understanding of speech production, and to translate this into medical devices called intracortical brain-computer int…
Discarded Bone Marrow for Hematology Research
The primary objective of this study is to establish a mechanism to obtain discarded bone marrow-containing bone samples from hemoglobinopathy, as well as non-h…
Natural History Study of Synucleinopathies
Synucleinopathies are a group of rare diseases associated with worsening neurological deficits and the abnormal accumulation of the protein α-synuclein in the …
Effectiveness and Safety of Sotagliflozin in Slowing Kidney Function Decline in Persons With Type 1 Diabetes and Moderate to Severe Diabetic Kidney Disease
Powerful new drugs that can prevent or delay end stage kidney disease (ESKD) - so called sodium-glucose cotransporter-2 inhibitors (SGLT2i) - are now available…