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18-month Double-blind, Randomized, Placebo-controlled, Multicenter, Phase 3 Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease and in Late-infantile and Juvenile-onset Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in lat…
Centered Surgery - Preoperative Counseling and Patient Satisfaction
This study is a prospective cohort study which aims to assess patient satisfaction and preparedness for surgery through two methods of preoperative counseling:…
Using Behavioral Economics and Implementation Science to Advance the Use of Genomic Medicine Utilizing an EHR Infrastructure Across a Diverse Health System
Given the expansion of indications for genetic testing and our understanding of conditions for which the results change medical management, it is imperative to…
Intravaginal Injection of Poly-L-Lactic Acid for Treatment of Cystocele: A Randomized Trial
Pelvic organ prolapse (POP) is a condition characterized by descent of vaginal walls, uterus and cervix, or the perineum and causes a sensation of vaginal bulg…
Accelerometer Measured Early Recovery After Prolapse Surgery
The AccelERate study will evaluate the physical recovery of patients who have undergone Pelvic Organ Prolapse (POP) surgery. This will be evaluated via a self-…
Open Label Phase 1 and Target Validation Study of ONC206 in Children and Young Adults With Newly Diagnosed or Recurrent Diffuse Midline Glioma (DMG), and Other Recurrent Primary Malignant Central Nervous System (CNS) Tumors
This phase I trial studies the effects and best dose of ONC206 alone or in combination with radiation therapy in treating patients with diffuse midline gliomas…
Observational Study to Deeply Phenotype Major Organs in Sickle Cell Disease After Curative Therapies
Background: People with sickle cell disease (SCD) have problems with their heart, brain, kidneys, liver, and lungs as they age. These problems may improve aft…
Slow-SPEED: Slowing Parkinson's Early Through Exercise Dosage
The goal of this clinical trial is two-fold. First to investigate the feasibility of whether a remotely administered smartphone app can increase the volume and…
68Ga(Gallium)-DOTATATE Positron Emission Tomography (PET)/MRI in the Diagnosis and Management of Somatostatin Receptor Positive Central Nervous System CNS Tumors.
The study population consists of patients who undergo resection for somatostatin receptor-positive (SSTR-positive) CNS tumors, focusing on meningioma, and incl…
Phase I Trial of [212Pb]VMT-Alpha-NET in Metastatic or Inoperable Somatostatin-Receptor Positive Gastrointestinal Neuroendocrine Tumors, Pheochromocytoma/Paragangliomas, Small Cell Lung, Renal Cell, and Head and Neck Cancers
Background: Some cancers have high levels of proteins called somatostatin receptors (SSTRs) on the surface of the tumors. These tumors can be in the lung, hea…
The Treatment of Painful Diabetic Neuropathy With Diet, a Randomized-controlled Trial
The purpose of this clinical trial is to determine whether targeted nutritional changes can improve symptoms of painful diabetic neuropathy. Impaired blood flo…
Hematopoietic Stem Cell Transplantation (HSCT) for Common Variable Immunodeficiency (CVID) and Other Autoimmune Manifestations of Primary Immune Regulatory Disorders (PIRD)
This is a research protocol that will examine Hematopoietic Stem Cell Transplantation (HSCT) using a reduced conditioning regimen (RIC) with total body Irradia…