Phase 3 N=22 Stem Cell Transplant for Hemoglobinopathy
Sickle Cell Disease · Thalassemia · Severe Congenital Neutropenia
Primary: Number of Patients Who Experienced Grade 3-5 Treatment Related Toxicity — 0; 0; 2 Participants
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Phase 2 N=16 Allogeneic SCT of NiCord®, UCB-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies
Sickle Cell Disease & Thalassemia
Primary: Safety and Tolerability Will be Measured by Acute NiCord® Infusional Toxicity. — 0; 0; 3; 0 participants
Phase 2 N=100 Clinical Investigation to Evaluate the Hemanext® Oxygen Reduction System - Pivotal Trial
Whole Blood Donation and Leukoreduction
Primary: % of Red Blood Cells With Hemolysis — 0.29; 0.29 % of Red Blood Cells with Hemolysis
N/A N=22 Hypoxic Red Blood Cells for Burns and Hematological Malignancies at Haukeland University Hospital
Hematologic Neoplasms · Burns
Primary: Number of Participants Who Experienced an Adverse Event (All Types/Grades) Within a Time Frame up to 24 Hours Following the Transfusion. — 2; 3 Participants
Phase 1 N=15 Peripheral Blood Stem Cell Collection for Sickle Cell Disease (SCD) Patients
Sickle Cell Disease
Primary: Number of Participants With Sufficient Collection of Hemopoietic Stem Cells (HSCs) Without Serious Adverse Events — 14 Participants
N/A N=8 A Pilot Study of HSCT for Patients With High-risk Hemoglobinopathy Using a Nonmyeloablative Preparative Regimen
Sickle Cell Disease · Thalassemia · Hemoglobinopathies
Primary: Development of GVHD Within 1 Year of BMT — 2 participants
N/A N=32 Evaluation of the Spectra Optia® Mononuclear Cell Collection Procedure
Multiple Myeloma
Primary: Days Until Neutrophil Recovery Following Peripheral Blood Stem Cell Transplant Minus the Historical Median Day Until Recovery. — 0 Days — p=<0.025
Phase 2 N=2 Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies
Hemoglobinopathies · Sickle Cell Disease · Thalassemia
Primary: Stable Engraftment With Donor Stem Cells in Patients With Severe Hemoglobinopathy. — 2 participants
Phase 2 N=36 Transplantation of NiCord®, Umbilical Cord Blood-derived Ex Vivo Expanded Cells, in Patients With HM
Hematological Malignancies · Acute Lymphoblastic Leukemia (ALL) · Acute Myeloid Leukemia (AML)
Primary: Engraftment — 94 percentage of patients
N/A N=25 Cord Blood Transfusion In Preterm Neonates (CB-TrIP)
Premature Infant Disease · Transfusion Related Complication · Fetal Hemoglobin
Primary: Median Percentage of HbF at 32 Weeks of Post Menstrual Age — 93.2; 90.8; 58.0; 69.0 percentage of total Hb — p=<0.0001
Phase 3 N=135 Stem Cell Transplant for Inborn Errors of Metabolism
Adrenoleukodystrophy · Metachromatic Leukodystrophy · Globoid Cell Leukodystrophy
Primary: Overall Survival — 120; 92; 81 Participants
Phase 2 N=102 Darbepoetin Administration to Preterm Infants
Infant, Newborn
Primary: Number of Transfusions During Hospitalization — 1.2; 1.2; 2.4 number of transfusions
Phase 2 N=202 Specialized Blood Cell Transplants for Cancers of the Blood and Bone Marrow
Myeloproliferative Disorders · Acute Myelogenous Leukemia · Chronic Myelogenous Leukemia
Primary: Number of Participants Who Experienced Transplant Related Mortality — 5; 4 Participants
N/A N=5 Assess the Feasibility and Safety of Granulocyte Colony Stimulating Factor (GCSF) Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia Major
Beta Thalassemia Major · Congenital Anemias
Primary: Number of Participants With no Serious Adverse Events — 5 participants
N/A N=61 Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia
Dyskeratosis Congenita · Aplastic Anemia
Primary: Incidence of Neutrophil Engraftment — 100; 100; 96; 100 Percentage of participants
Phase 2 N=2 Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes
Sickle Cell Anemia · Severe Aplastic Anemia · Paroxysmal Nocturnal Hemoglobinuria (PNH)
Primary: Number of Patients With Neutrophil Engraftment — 2 participants
N/A N=30 Exercise Prehab in Older Adults With Hematologic Malignancies
Hematopoietic and Lymphoid Cell Neoplasm
Primary: Number of Patients With Adverse Events (AE), Graded According to Common Terminology Criteria for Adverse Events Version 5.0 — 0 Participants
Phase 2 N=29 Megadose CD34 Selected Progenitor Cells for Transplantation in Patients With Advanced Hematological Malignant Diseases
Leukemia · Lymphoma
Primary: Number of Participants With Absolute Neutrophil Count Engraftment — 21 participant
Phase 2 N=84 Cord Blood With T-Cell Depleted Haplo-identical Peripheral Blood Stem Cell Transplantation for Hematological Malignancies
Leukemia · Myelodysplastic Syndrome · Lymphoma
Primary: Percent of Engrafted Participants With Successful Neutrophil Recovery — 96 % w/incidence of neutrophil recovery
Phase 4 N=146 A Study of Anagrelide and Hydroxyurea in High-Risk Essential Thrombocythemia Patients
Thrombocythemia, Hemorrhagic
Primary: Change From Baseline in Left Ventricular Ejection Fraction (LVEF) Over Time — 66.4; 66.9; 0.5; -1.1 percentage of ejection fraction
Phase 2 N=22 Bone Marrow Transplantation in Young Adults With Severe Sickle Cell Disease
Sickle Cell Disease
Primary: Event -Free Survival Rate — 19 Participants
Phase 2 N=6 Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs
Sickle Cell Disease · Thalassemia · Diamond-Blackfan Anemia
Primary: Count of Participants With Stable Engraftment Post Hematopoietic Cell Transplantation (HCT) — 3 Participants
Phase 4 N=169 Study on Bone Marrow Morphology in Adults Receiving Romiplostim for Treatment of Thrombocytopenia Associated With Immune Thrombocytopenia Purpura (ITP)
Thrombocytopenia · Idiopathic Thrombocytopenic Purpura
Primary: Percentage of Participants With Collagen Fibrosis — 0.0; 0.0; 3.4 percentage of participants
Phase 3 N=118 Erythropoietin (EPO)+/- Filgrastim (G-CSF) vs. Supportive Therapy Alone for Patients With Myelodysplastic Syndromes
Anemia · Myelodysplastic Syndromes
Primary: Proportion of Patients Free of Transfusion at 4 Months — 0.459; 0.714 Proportion of patients
Phase 2 N=21 Reduced Intensity Transplant Conditioning Regimen for Severe Thalassemia
Severe Thalassemia
Primary: Primary Objective: Event-free Survival at 1 Year. — 17 Participants
Phase 2 N=104 Red Blood Cell (RBC) Survival Following Transfusion in Infants
Neonatal Anemia
Primary: Number of Discrete Biotinylated RBC Densities (up to 5 Discrete Densities) That Can be Accurately Measured by Flow Cytometry, i.e., Without Overlap. — 5; 5 flow…
Phase 2 N=41 Stem Cell Transplantation for Hurler
Mucopolysaccharidosis I · Mucopolysaccharidosis VI · Mannosidosis
Primary: Mean Percentage of Donor Cells in Study Population (Chimerism). — 85.8; 73.2; 84.6; 81.1 Percentage
Phase 3 N=30 Stem Cell Transplantation for Sickle Cell Anemia
Sickle Cell Disease
Primary: Number of Participants With Graft Failure — 3 Participants
Phase 2 N=29 HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity
Sickle Cell Disease · Beta Thalassemia-Major
Primary: Number of Participants With Sustained Cell Engraftment of Donor Cells — 29 Participants