Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials
42 studies found for:    Open Studies | "beta-Thalassemia"
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Rank Status Study
1 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
2 Not yet recruiting An Algorithm to Start Iron Chelation in Minimally Transfused Young Beta-thalassemia Major Patients
Condition: Beta Thalassemia Major
Intervention: Drug: Deferiprone
3 Unknown  Genetic Factors Affecting the Severity of Beta Thalassemia
Condition: Beta Thalassemia
Intervention:
4 Recruiting ACE-536 Extension Study - Beta Thalassemia
Condition: Beta-Thalassemia
Intervention: Drug: ACE-536
5 Recruiting Study of ACE-536 to Evaluate the Effects of ACE-536 in Patients With 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 Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia.
Conditions: Beta Thalassemia Major;   Beta Thalassemia Intermedia
Interventions: Drug: Dose level 1a;   Drug: Dose level 1b;   Drug: Dose level 2;   Drug: dose level 3;   Drug: dose level 4;   Drug: dose level 5
9 Recruiting Long Term Outcomes in β Thalassemia Major
Condition: Thalassemia Major
Intervention:
10 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
11 Recruiting Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia
Conditions: Iron Overload;   Beta-Thalassemia
Intervention: Drug: SP-420
12 Recruiting Post Hematopoietic Stem Cell Transplantation
Condition: Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major
Intervention: Drug: ICL670
13 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
14 Recruiting ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene
Condition: Confirmed Diagnosis of ß-thalassemia Major
Intervention: Genetic: Autologous CD34+ cells transduced with TNS9.3.55
15 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
16 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: CliniMacs (PLUS) Reagent System
17 Recruiting Reproductive Capacity and Iron Burden in Thalassemia
Condition: THALASSEMIA MAJOR
Interventions: Other: Blood Draw/Semen Exam;   Other: Retrospective data/Chart Review/Relevant Clinical Results;   Other: Pituitary MRI
18 Recruiting Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Severe Congenital Anemias Including Sickle Cell Disease and Beta-Thalassemia
Conditions: Peripheral Blood Stem Cell Transplantation;   Anemia, Sickle Cell;   Graft-Versus-Host Disease;   Sirolimus (Rapamune );   Alemtuzumab (Campath )
Interventions: Procedure: PBMC Transplant;   Drug: Alemtuzumab (Campath  );   Drug: Sirolimus (Rapamune  );   Drug: Cyclophosphamide (Cytoxan  );   Procedure: Low Dose Irradiation
19 Not yet recruiting Role of Vitamin C to Augment Iron Chelation With DFP or DFX
Conditions: Vitamin c;   Thalassemia Major;   Iron Chelation
Interventions: Drug: Vitamin C, Defriprone, deferisarox;   Drug: deferiprone , deferesarox
20 Unknown  Therapeutic Effect and Safety of Combined Hydroxyurea With Recombinant Human Erythropoietin.
Condition: Thalassemia Intermedia
Interventions: Drug: Hydroxyurea ,Epiao;   Drug: hydroxyurea, blood transfusion

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Indicates status has not been verified in more than two years