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Screening and Baseline Assessment of Patients With Abnormalities of Immune Function
This study will evaluate patients with abnormal immune function that results in recurrent or unusual infections or chronic inflammation. This may include inher…
Screening Protocol for Genetic Diseases of Lymphocyte Homeostasis and Programmed Cell Death
This study will determine the biochemical and genetic causes of inherited immune diseases affecting lymphocyte homeostasis. Lymphocytes are a type of white blo…
Single Center Phase I Study of Adoptive Immunotherapy of Refractory Viral Infection With ex Vivo Expanded Rapidly Generated Virus Specific T (R-MVST) Cells for Immunodeficient Children and Young Adults
The primary objective is to determine the safety and feasibility of administering R-MVST cells to patients with refractory viral reactivation and/or symptomati…
A Phase 3, Prospective, Open-label, Multicenter, Single-arm Study to Investigate the Efficacy, Safety, and Pharmacokinetics of IgPro20 in Subjects With Secondary Immune Deficiency Due to Hematologic Malignancies Treated With B-cell Targeting Chimeric Antigen Receptor T-cell and T-cell Redirecting Therapies
This is a prospective, multicenter, open-label, single-arm study to assess the efficacy, safety, and pharmacokinetics (PK) of IgPro20 in adults with hematologi…
Study of TCR Alpha Beta T-Cell and CD19 B-Cell Depletion for Hematopoietic Cell Transplantation From Haploidentical Donors in the Treatment of Primary Immunodeficiency and Inherited Metabolic Disorders in Children
This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is s…
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…
Let's Get REAL: A Pilot Trial of a Family Health Communication Tool in Pediatric Stem Cell Transplant and Cellular Therapy
The investigators will conduct a pilot feasibility and efficacy trial of a newly developed family health communication tool (called Let's Get REAL) in increasi…
Efficacy and Safety of Cryopreserved Autologous Mobilized Peripheral Blood CD34+ HSPCs Transduced Ex Vivo With the EFS-ADA Lentiviral Vector in Patients With Severe Combined Immune Deficiency Due To Adenosine Deaminase Deficiency
The aim of this study is to assess the safety and efficacy of autologous transplantation of hematopoietic stem cells (CD34+ cells) from mobilized peripheral bl…
Evaluating a Clinical Decision Support Tool for Antiretroviral Therapy Optimization
This study is testing software designed to help healthcare providers choose the best HIV treatment combinations for their patients. HIV medicines, known as ant…
A Pilot Study in the Treatment of Refractory Epstein-Barr Virus (EBV) Infection With Related Donor EBV Cytotoxic T-Lymphocytes in Children, Adolescents and Young Adult Recipients
Related donor Epstein-Barr Virus (EBV) specific cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be admin…
Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures
Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and po…
A Pilot Study in the Treatment of Refractory Adenovirus (ADV) Infection With Related Donor ADV Cytotoxic T-Lymphocytes (ADV-CTLs)in Children, Adolescents and Young Adult Recipients
Related donor Adenovirus (ADV) specific cytotoxic T cells (CTLs) manufactured with the Miltenyi CliniMACS Prodigy Cytokine Capture System will be administered …