36 studies found for:    Open Studies | "beta-Thalassemia"
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Open Studies | "beta-Thalassemia" (36 records)
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Rank Status Study
1 Recruiting Gene Therapy for Transfusion Dependent Beta-thalassemia
Condition: Beta-Thalassemia
Intervention: Genetic: Autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene
2 Recruiting A Study Evaluating the Safety and Efficacy of the LentiGlobin® BB305 Drug Product in Beta-Thalassemia Major Subjects
Condition: Beta-thalassemia Major
Intervention: Genetic: LentiGlobin® BB305 Drug Product
3 Not yet recruiting An Algorithm to Start Iron Chelation in Minimally Transfused Young Beta-thalassemia Major Patients
Condition: Beta Thalassemia Major
Intervention: Drug: Deferiprone
4 Unknown  Genetic Factors Affecting the Severity of Beta Thalassemia
Condition: Beta Thalassemia
5 Recruiting ACE-536 Extension Study - Beta Thalassemia
Condition: Beta-Thalassemia
Intervention: Drug: ACE-536
6 Recruiting A Study Evaluating the Efficacy and Safety of LentiGlobin BB305 Drug Product in Beta-Thalassemia Major and Sickle Cell Disease
Conditions: Beta-Thalassemia Major;   Sickle Cell Disease
Intervention: Genetic: LentiGlobin BB305 Drug Product
7 Recruiting Efficacy of Basiliximab in the Prevention of Acute Graft-versus-host Disease in Unrelated Allogeneic Hematopoietic Stem Cell Transplantation Therapy for Thalassemia Major
Condition: Beta-Thalassemia Major
Interventions: Drug: Basiliximab,;   Drug: cyclosporine A;   Drug: Methotrexate;   Drug: Mycophenolate mofetil
8 Recruiting Long Term Outcomes in β Thalassemia Major
Condition: Thalassemia Major
9 Unknown  Prevalence Of Microalbuminuria Among Children Suffering From Sickle Cell Nephropathy and Sickle Cell/Beta-Thalassemia
Conditions: Sickle Cell Anemia;   Beta-Thalassemia;   Microalbuminuria
Intervention: Other: No intervention
10 Recruiting Unrelated Umbilical Cord Blood Following HLA-haploidentical Hematopoietic Stem Cell Transplantation in Patients With β-thalassemia Major
Condition: Thalassemia Major
Intervention: Genetic: unrelated CB following haplo-identical hematopoietic stem cells transplantation
11 Recruiting Denosumab vs Placebo in Patients With Thalassemia Major and Osteoporosis
Conditions: Thalassemia Major;   Osteoporosis
Interventions: Drug: Denosumab;   Drug: Placebo
12 Not yet recruiting An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia
Conditions: Erythrocyte Transfusion;   Beta-Thalassemia
Interventions: Drug: Luspatercept;   Other: Placebo
13 Unknown  Study Of Efficacy,Safety of Combined Deferasirox and Deferiprone Versus Combined Deferiprone and Desferal In Conditions of Iron Overload
Conditions: Beta-thalassemia Major;   Sickle Cell Disease;   Iron Hemosiderosis
Interventions: Drug: DFP (ferriprox) and deferasirox (ICL 670);   Drug: DFP, DFO
14 Not yet recruiting Efficacy and Safety of Efficacy and Safety of Continued Iron Chelation Therapy In Poly-transfused Thalassemia Patients With Low Serum Ferritin (< 500 ng/ml)
Conditions: Beta-thalassemia;   Serum Ferritin;   Iron Chelation Therapy
Intervention: Drug: desferal, ferriprox, blood transfusion
15 Recruiting CD34+ Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation for Non-Malignant Disease
Conditions: Bone Marrow Failure Syndrome;   Severe Aplastic Anemia;   Severe Congenital Neutropenia;   Amegakaryocytic Thrombocytopenia;   Diamond-Blackfan Anemia;   Schwachman Diamond Syndrome;   Primary Immunodeficiency Syndromes;   Acquired Immunodeficiency Syndromes;   Histiocytic Syndrome;   Familial Hemophagocytic Lymphocytosis;   Lymphohistiocytosis;   Macrophage Activation Syndrome;   Langerhans Cell Histiocytosis (LCH);   Hemoglobinopathies;   Sickle Cell Disease;   Sickle Cell-beta-thalassemia
Intervention: Biological: CD34 Stem Cell Selection Therapy
16 Recruiting Reproductive Capacity and Iron Burden in Thalassemia
Interventions: Other: Blood Draw/Semen Exam;   Other: Retrospective data/Chart Review/Relevant Clinical Results;   Other: Pituitary MRI
17 Recruiting A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Conditions: Hematologic Malignancies;   Certain:;   Inherited Disorders of Metabolism;   Inherited Abnormalities of Erythrocyte Differentiation or Function;   Inherited Abnormalities of Platelets;   Disorders of the Immune System;   Histiocytic Disorders;   X-linked Lymphoproliferative Syndrome;   Primary Immunodeficiency Diseases;   Bone Marrow Failure;   Beta-thalassemia
Intervention: Drug: A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)
18 Recruiting CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant
Conditions: Leukemia;   Lymphoma;   Bone Marrow Failure;   Immunodeficiencies;   Histiocytosis;   Sickle Cell Disease;   Beta Thalassemia;   Inborn Errors of Metabolism
Interventions: Drug: Full Intensity with TBI;   Drug: Full Intensity;   Drug: Reduced Intensity;   Drug: Reduced Intensity (Fanconi)
19 Recruiting Stem Cell Transplant in Sickle Cell Disease and Thalassemia
Conditions: Sickle Cell Disease;   Beta Thalassemia
Interventions: Drug: Busulfan;   Drug: Fludarabine;   Drug: Alemtuzumab;   Procedure: Allogeneic stem cell transplant
20 Recruiting HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity
Conditions: Sickle Cell Disease;   Beta Thalassemia-Major
Interventions: Drug: alemtuzumab (Campath IH);   Drug: Fludarabine;   Drug: Melphalan;   Drug: Cyclosporine;   Drug: Mycophenolate mofetil;   Drug: Tacrolimus;   Biological: Hematopoietic Stem Cell Transplantation

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Indicates status has not been verified in more than two years