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A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs) for Transplantation in Pediatric and Adult Patients With Hematologic Malignancies and Other Indications
This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic maligna…
Deep Phenotyping of Hearing Instability Disorders: Cohort Establishment, Biomarker Identification, Development of Novel Phenotyping Measures, and Discovery of Therapeutic Targets
Background: Disorders of hearing instability (HI) are poorly characterized and ineffectively treated. HI can cause fluctuations in hearing thresholds and spee…
A Phase 2 Open-Label Study to Evaluate the Efficacy and Safety of Ruxolitinib on Hair Regrowth in Patients With Autoimmune Polyendocrinopathy Candidiasis Ectodermal Dystrophy (APECED)-Associated Alopecia Areata
Background: Autoimmune polyendocrinopathy candidiasis ectodermal dystrophy (APECED) is a problem of the immune system. In people with APECED, the immune syste…
A Phase II Prospective Study Evaluating Selective Depletion of CD45RA+ (Naïve) T Cells (TND) From Peripheral Blood Stem Cell (PBSC) Grafts for Prevention of Graft Versus Host Disease (GVHD) in Non-Malignant Diseases (NMDs)
This phase II trial investigates how well a naive T cell depleted graft work for the reduction of graft versus host disease in patients with non-malignant dise…
Longitudinal Investigation of Cortical Demyelination and Meningeal Inflammation in Multiple Sclerosis and Related Disorders
In this protocol, a combination of MRI, blood, and cerebrospinal fluid (CSF) analysis will be used to understand the natural history, underlying immunologic me…
Testing of an Educational Tool for Patients With Melanoma and Concomitant Autoimmune Disease Who Are Candidates for Adjuvant Therapy With Immune Checkpoint Inhibitors: Acceptability and Usability With Patients and Providers
This study learn how easily patients can use an educational tool that will be created for patients with melanoma and pre-existing autoimmune diseases who recei…
Primary Care Pragmatic, Real World Experience for Automated Insulin Delivery
The goal of this randomized controlled trial is to compare the efficacy and safety of the iLet Bionic Pancreas (BP) System in adults with insulin-treated diabe…
A Phase 3, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study With an Open-label Period and Long-term Extension to Assess the Efficacy and Safety of Rilzabrutinib in Participants With Warm Autoimmune Hemolytic Anemia (wAIHA)
This is a parallel-group, Phase 3, double-blind, 2-arm study to investigate the efficacy, safety, PK and PD of oral rilzabrutinib in achieving durable Hb respo…
Phase 2 Study Bridging Pre-Transplant Inflammatory Dampening for Primary Immune Regulatory Disorders (BRIDGE Trial)
The researchers are doing this study to find out whether emapalumab or a combination of fludarabine and dexamethasone are effective in preparing people with a …
Coagulopathy of Immunodermatologic Diseases The Molecular, Prognostic, and Therapeutic Implications of Crosstalk Between Coagulation, Fibrinolysis, and Inflammation in Immune-Mediated Skin Diseases
This study will examine the coagulation and fibrinolysis profiles of those with autoimmune skin diseases. Blood samples will be collected from participants wit…
A Phase 2b, Double-Blind, Placebo-Controlled, Multicenter Proof of Concept Clinical Study to Evaluate the Efficacy and Safety of ATH-063 Induction Therapy in Patients With Biologic Relapsed/Refractory Moderately to Severely Active Ulcerative Colitis (UC)
The primary objective of this trial is to evaluate the clinical efficacy of ATH-063 in participants with biologic/advanced therapy relapsed/refractory moderate…
Spermatogonial Stem Cell (SSC) Transplant and Testicular Tissue Grafting
Spermatogonial stem cell transplantation through ultrasound guided rete testis injection and testicular tissue grafting will be performed for participants who …