284. ダイアモンド・ブラックファン貧血 ［臨床試験数：34，薬物数：91（DrugBank：36），標的遺伝子数：21，標的パスウェイ数：106］
Searched query = "Diamond Blackfan anemia"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04099966 | October 1, 2019 | 7 October 2019 | AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion | Allogeneic Stem Cell Transplantation for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion - NYMC 588 | Acute Leukemia;Severe Aplastic Anemia;Non-hodgkin Lymphoma;Hodgkin Lymphoma;Kostmann;Diamond Blackfan Anemia;Amegakaryocytic Thrombocytopenia;Sickle Cell Disease;Beta-Thalassemia | Drug: alpha beta depletion | Mitchell Cairo | Not recruiting | N/A | 30 Years | All | 20 | Phase 2 | United States | |
| 2 | NCT03966053 | September 13, 2019 | 30 September 2019 | The Use of Trifluoperazine in Transfusion Dependent DBA | Phase I/II, Open Label Study to Determine Safety of Trifluoperazine (TFP) in Adults With Red Blood Cell Transfusion-Dependent Diamond Blackfan Anemia | Diamond Blackfan Anemia;Pure Red Cell Aplasia | Drug: Trifluoperazine | Adrianna Vlachos | Recruiting | 18 Years | 65 Years | All | 24 | Phase 1/Phase 2 | United States | |
| 3 | NCT03653338 | August 2, 2018 | 11 March 2019 | T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias | T-Cell Depleted, Alternative Donor Transplant in Pediatric and Adult Patients With Severe Sickle Cell Disease (SCD) and Other Transfusion-Dependent Anemias | Sickle Cell Anemia;Beta-thalassemia Major;Diamond-blackfan Anemia | Biological: CD3/CD19 depleted leukocytes;Biological: CD45RA depleted leukocytes;Drug: Hydroxyurea;Drug: Rituximab;Drug: Alemtuzumab;Drug: Fludarabine;Drug: Thiotepa | Beth Carella, DO | Recruiting | 5 Years | 40 Years | All | 5 | Phase 1/Phase 2 | United States | |
| 4 | NCT03513328 | June 15, 2018 | 3 June 2019 | Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation | PEDS024, Phase I/II Feasibility Study of Busulfan Fludarabine and Thiotepa Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation (HSCT) for Children With Non-Malignant Disorders | Bone Marrow Failure Syndrome;Thalassemia;Sickle Cell Disease;Diamond Blackfan Anemia;Acquired Neutropenia in Newborn;Acquired Anemia Hemolytic;Acquired Thrombocytopenia;Hemophagocytic Lymphohistiocytoses;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;Common Variable Immunodeficiency;X-linked Lymphoproliferative Disease;Severe Combined Immunodeficiency;Hurler Syndrome;Mannosidosis;Adrenoleukodystrophy | Drug: Thiotepa--single daily dose;Drug: Thiotepa--escalated dose | University of Florida | Live Like Bella Pediatric Cancer Research | Recruiting | 6 Months | 38 Years | All | 40 | Phase 1/Phase 2 | United States |
| 5 | NCT03733249 | January 2017 | 28 October 2019 | Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study | Follow-up of Phase 1/2 Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR aß+T Cells in Pediatric Patients Affected by Hematological Disorders | Acute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplastic Syndromes;Primary Immunodeficiency;Anemia, Aplastic;Hemoglobinopathies;Cytopenia;Fanconi Anemia;Diamond Blackfan Anemia;Thalassemia;Anemia, Sickle Cell | Drug: Rimiducid;Biological: rivogenlecleucel | Bellicum Pharmaceuticals | Recruiting | 1 Month | 18 Years | All | 193 | Phase 1/Phase 2 | Italy;Saudi Arabia;United Kingdom | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT02179359 | September 2, 2014 | 7 October 2019 | Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies | MT2014-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders | Sickle Cell Disease;Transfusion Dependent Alpha- or Beta- Thalassemia;Diamond Blackfan Anemia;Paroxysmal Nocturnal Hemoglobinuria;Glanzmann Thrombasthenia;Severe Congenital Neutropenia;Shwachman-Diamond Syndrome;Non-Malignant Hematologic Disorders | Drug: Reduced Toxicity Ablative Regimen;Drug: Reduced Intensity Preparative Regimen;Drug: Myeloablative Preparative Regimen | Masonic Cancer Center, University of Minnesota | Recruiting | N/A | 55 Years | All | 25 | N/A | United States | |
| 7 | NCT02386267 | September 2014 | 16 March 2015 | L-leucine in Diamond Blackfan Anemia Patients | Therapeutic Use of the Amino Acid Leucine in the Treatment of Transfusion-Dependent Diamond Blackfan Anemia Patients | Diamond Blackfan Anemia | Drug: L-leucine | Federal Scientific Clinical Centre of Pediatric Hematology, Oncology and Immunology named after Dmitry Rogache | Recruiting | 1 Year | 20 Years | Both | 30 | Phase 2 | Russian Federation | |
| 8 | NCT02065869 | April 2014 | 4 November 2019 | Safety Study of Gene Modified Donor T-cells Following TCR Alpha Beta Depleted Stem Cell Transplant | Phase II Extension Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR aß+T Cells in Pediatric Patients Affected by Hematological Disorders | Acute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplastic Syndrome;Primary Immunodeficiency;Anemia, Aplastic;Osteopetrosis;Hemoglobinopathies;Cytopenia;Fanconi Anemia;Diamond Blackfan Anemia;Thalassemia;Anemia, Sickle Cell | Biological: BPX-501 T cells;Drug: rimiducid | Bellicum Pharmaceuticals | Not recruiting | 1 Month | 18 Years | All | 193 | Phase 1/Phase 2 | Italy;United Kingdom;Germany;Spain;United States | |
| 9 | NCT01917708 | January 2014 | 19 November 2018 | BMT Abatacept for Non-Malignant Diseases | Abatacept for Post-Transplant Immune Suppression in Children and Adolescents Receiving Allogeneic Hematopoietic Stem Cell Transplants for Non-Malignant Diseases | Hurler Syndrome;Fanconi Anemia;Glanzmann Thrombasthenia;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;Severe Congenital Neutropenia;Leukocyte Adhesion Deficiency;Shwachman-Diamond Syndrome;Diamond-Blackfan Anemia;Dyskeratosis-congenita;Chediak-Higashi Syndrome;Severe Aplastic Anemia;Thalassemia Major;Hemophagocytic Lymphohistiocytosis;Sickle Cell Disease | Drug: Abatacept | Emory University | Not recruiting | N/A | 21 Years | All | 10 | Phase 1 | United States | |
| 10 | NCT01362595 | June 2013 | 30 September 2019 | Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia | The Use of Novel Therapies to Reconstitute Blood Cell Production and Promote Organ Performance Using Bone Marrow Failure as a Model: a Pilot, Phase I/II Study of the Amino Acid Leucine in the Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia | Diamond Blackfan Anemia;Blackfan Diamond Syndrome;DBA;Congenital Hypoplastic Anemia;Pure Red Cell Aplasia | Drug: leucine | Northwell Health | Not recruiting | 2 Years | N/A | All | 50 | Phase 1/Phase 2 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | NCT01586455 | April 2013 | 8 January 2018 | Human Placental-Derived Stem Cell Transplantation | A Single-Arm Study to Assess the Safety of Transplantation With Human Placental-Derived Stem-Cells Combined With Unrelated and Related Cord Blood in Subjects With Certain Malignant Hematologic Diseases and Non-Malignant Disorders | Mucopolysaccharidosis I;Mucopolysaccharidosis VI;Adrenoleukodystrophy;Niemann-Pick Disease;Metachromatic Leukodystrophy;Wolman Disease;Krabbe's Disease;Gaucher's Disease;Fucosidosis;Batten Disease;Severe Aplastic Anemia;Diamond-Blackfan Anemia;Amegakaryocytic Thrombocytopenia;Myelodysplastic Syndrome;Acute Myelogenous Leukemia;Acute Lymphocytic Leukemia | Drug: Human Placental Derived Stem Cell | New York Medical College | Not recruiting | N/A | 55 Years | All | 43 | Phase 1 | United States | |
| 12 | NCT01966367 | March 2013 | 25 March 2019 | CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation | CD34+ Stem Cell Selection for Patients Receiving a Matched or Partially Matched Family or Unrelated Adult Donor Allogeneic Stem Cell Transplantation for Non-Malignant Disease | 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 | Biological: CD34 Stem Cell Selection Therapy | Diane George, MD | Recruiting | N/A | 40 Years | All | 25 | Early Phase 1 | United States | |
| 13 | EUCTR2011-006322-25-GB | 08/10/2012 | 17 October 2016 | Extending the treatment of Iron Overload | A Phase 2, Open-label, Multicentre, Extension Safety and Tolerability Study for Transfusionally Iron Overloaded Children, Adolescents and Adults Using SSP-004184 (SPD602). | Patients with transfusional iron overload due to hereditary anemias such as sickle cell disease, beta-thalassemia and Diamond-Blackfan anemia; aquired anemias such as Myelodysplastic Syndrome and other forms of bone marrow failure. MedDRA version: 16.1 Level: LLT Classification code 10065974 Term: Chronic iron overload System Organ Class: 100000004861 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: SSP-004184AQ 50 mg Capsule Product Code: SSP-004184AQ 50 mg Pharmaceutical Form: Capsule, hard INN or Proposed INN: deferitazole CAS Number: 1173095-59-5 Current Sponsor code: SSP-004184AQ Other descriptive name: (S)-4,5-dihydro-2[hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 50- Product Name: SSP-004184AQ 100 mg Capsule Product Code: SSP-004184AQ 100 mg Pharmaceutical Form: Capsule, hard INN or Proposed INN: deferitazole CAS Number: 1173095-59-5 Current Sponsor code: SSP-004184AQ Other descriptive name: (S)-4,5-dihydro-2[hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 100- Product Name: SSP-004184AQ 200 mg Capsule Product Code: SSP-004184AQ 200 mg Pharmaceutical Form: Capsule, hard INN or Proposed INN: deferitazole CAS Number: 1173095-59-5 Current Sponsor code: SSP-004184AQ Other descriptive name: (S)-4,5-dihydro-2[hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 200- Product Name: SSP-004184AQ 250 mg Capsule Product Code: SSP-004184AQ 250 mg Pharmaceutical Form: Capsule, hard INN or Proposed INN: deferitazole CAS Number: 1173095-59-5 Current Sponsor code: SSP-004184AQ Other descriptive name: (S)-4,5-dihydro-2[hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 250- Product Name: SSP-004184AQ 375 mg Capsule Product Code: SSP-004184AQ 375 mg Pharmaceutical Form: Capsule, hard INN or Proposed INN: deferitazole CAS Number: 1173095-59-5 Current Sponsor code: SSP-004184AQ | Shire Development LLC | Not Recruiting | Female: yes Male: yes | 1500 | Phase 2 | Egypt;United States;Canada;Thailand;Lebanon;Turkey;Italy;United Kingdom | |||
| 14 | EUCTR2011-005675-16-IT | 15/05/2012 | 23 March 2015 | Treatment of chronic iron overload with a chelation therapy (FBS0701) | A Phase 2, 24 Week, Randomized, Open Label, Multi-Center Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Two FBS0701 Doses in the Treatment of Chronic Iron Overload Requiring Chelation Therapy - FBS0701 in the treatment of chronic iron overload | Patients with transfusional iron overload, with the following primary diagnosis:hereditary anemia (such as sickle cell disease),ß-thalassemia and Diamond Blackfan anemia;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: FBS0701 CAPSULE Product Code: FBS0701 Pharmaceutical Form: Capsule, hard INN or Proposed INN: iron chelating agents CAS Number: 1173092-59-5 Current Sponsor code: FBS0701 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 50- Product Name: FBS0701 CAPSULE Product Code: FBS0701 Pharmaceutical Form: Capsule, hard INN or Proposed INN: Iron chelating agents CAS Number: 1173092-59-5 Current Sponsor code: FBS0701 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 100- Product Name: FBS0701 CAPSULE Product Code: FBS0701 Pharmaceutical Form: Capsule, hard INN or Proposed INN: Iron chelating agents CAS Number: 1173092-59-5 Current Sponsor code: FBS0701 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 200- Product Name: FBS0701 CAPSULE Product Code: FBS0701 Pharmaceutical Form: Capsule, hard INN or Proposed INN: iron chelating agents CAS Number: 1173092-59-5 Current Sponsor code: FBS0701 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 375- Product Name: FBS0701 CAPSULE Product Code: FBS0701 Pharmaceutical Form: Capsule, hard INN or Proposed INN: iron chelating agents CAS Number: 1173092-59-5 Current Sponsor code: FBS0701 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 500- | FERROKIN BIOSCIENCES INC. | Not Recruiting | Female: yes Male: yes | 50 | Phase 2 | United States;Canada;Lebanon;Turkey;Italy | |||
| 15 | NCT01529827 | February 28, 2012 | 30 September 2019 | Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies | A Phase II Trial of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan and Low Dose Total Body Irradiation | Primary Myelofibrosis;Recurrent Adult Acute Lymphoblastic Leukemia;Recurrent Adult Acute Myeloid Leukemia;Recurrent Adult Burkitt Lymphoma;Recurrent Adult Diffuse Large Cell Lymphoma;Recurrent Adult Diffuse Mixed Cell Lymphoma;Recurrent Adult Diffuse Small Cleaved Cell Lymphoma;Recurrent Adult Grade III Lymphomatoid Granulomatosis;Recurrent Adult Hodgkin Lymphoma;Recurrent Adult Immunoblastic Large Cell Lymphoma;Recurrent Adult Lymphoblastic Lymphoma;Recurrent Adult T-cell Leukemia/Lymphoma;Recurrent Childhood Acute Lymphoblastic Leukemia;Recurrent Childhood Acute Myeloid Leukemia;Recurrent Childhood Anaplastic Large Cell Lymphoma;Recurrent Childhood Grade III Lymphomatoid Granulomatosis;Recurrent Childhood Large Cell Lymphoma;Recurrent Childhood Lymphoblastic Lymphoma;Recurrent Childhood Small Noncleaved Cell Lymphoma;Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma;Recurrent Grade 1 Follicular Lymphoma;Recurrent Grade 2 Follicular Lymphoma;Recurrent Grade 3 Follicular Lymphoma;Recurrent Mantle Cell Lymphoma;Recurrent Marginal Zone Lymphoma;Recurrent Mycosis Fungoides/Sezary Syndrome;Recurrent Small Lymphocytic Lymphoma;Recurrent/Refractory Childhood Hodgkin Lymphoma;Refractory Chronic Lymphocytic Leukemia;Refractory Hairy Cell Leukemia;Refractory Multiple Myeloma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndromes;Secondary Myelofibrosis;Severe Combined Immunodeficiency;Severe Congenital Neutropenia;Shwachman-Diamond Syndrome;Splenic Marginal Zone Lymphoma;T-cell Large Granular Lymphocyte Leukemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich Syndrome;Chronic Phase Chronic Myelogenous Leukemia;Congenital Amegakaryocytic Thrombocytopenia;Diamond-Blackfan Anemia;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Hepatosplenic T-cell Lymphoma;Juvenile Myelomonocytic Leukemia;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Nodal Marginal Zone B-cell Lymphoma;Paroxysmal Nocturnal Hemoglobinuria;Peripheral T-cell Lymphoma;Polycythemia Vera;Post-transplant Lymphoproliferative Disorder;Previously Treated Myelodysplastic Syndromes;Accelerated Phase Chronic Myelogenous Leukemia;Adult Acute Lymphoblastic Leukemia in Remission;Adult Acute Myeloid Leukemia in Remission;Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities;Adult Acute Myeloid Leukemia With Del(5q);Adult Acute Myeloid Leukemia With Inv(16)(p13;q22);Adult Acute Myeloid Leukemia With t(15;17)(q22;q12);Adult Acute Myeloid Leukemia With t(16;16)(p13;Adult Acute Myeloid Leukemia With t(8;21)(q22;Adult Grade III Lymphomatoid Granulomatosis;Adult Nasal Type Extranodal NK/T-cell Lymphoma;Anaplastic Large Cell Lymphoma;Angioimmunoblastic T-cell Lymphoma;Aplastic Anemia;Burkitt Lymphoma;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Chronic Myelogenous Leukemia;Childhood Diffuse Large Cell Lymphoma;Childhood Grade III Lymphomatoid Granulomatosis;Childhood Immunoblastic Large Cell Lymphoma;Childhood Myelodysplastic Syndromes;Childhood Nasal Type Extranodal NK/T-cell Lymphoma;Chronic Myelomonocytic Leukemia | Drug: fludarabine phosphate;Drug: melphalan;Radiation: total-body irradiation;Drug: tacrolimus;Drug: mycophenolate mofetil;Drug: methotrexate;Other: laboratory biomarker analysis;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantation | Roswell Park Cancer Institute | Not recruiting | 3 Years | 75 Years | All | 94 | Phase 2 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | NCT01464164 | January 2012 | 3 June 2019 | Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia | Phase I/II, Open-Label Study to Determine Safety and Efficacy of Sotatercept (ACE-011) in Adults With Red Blood Cell Transfusion- Dependent Diamond Blackfan Anemia | Diamond Blackfan Anemia | Drug: Sotatercept;Drug: Sotatercept with prednisone boost | Northwell Health | Recruiting | 18 Years | N/A | All | 20 | Phase 1/Phase 2 | United States | |
| 17 | NCT01419704 | May 2011 | 30 September 2019 | Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies | Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies | Anemia, Sickle Cell;Complex and Transfusion-dependent Hemoglobinopathies;Thalassemia;Diamond-Blackfan Anemia;Bone Marrow Failure Syndromes;Alpha-Thalassemia;Beta-Thalassemia | Biological: Enriched Hematopoetic Stem Cell Infusion | University of Louisville | Duke University | Not recruiting | N/A | 45 Years | All | 30 | Phase 1/Phase 2 | United States |
| 18 | NCT01319851 | September 2010 | 16 December 2017 | Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation | Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation for Children With Non-Malignant Diseases Who Have Been Multiply Transfused: a Pilot Study | Thalassemia;Sickle Cell Disease;Glanzmann Thrombasthenia;Wiskott-Aldrich Syndrome;Chronic-granulomatous Disease;Severe Congenital Neutropenia;Leukocyte Adhesion Deficiency;Schwachman-Diamond Syndrome;Diamond-Blackfan Anemia;Fanconi Anemia;Dyskeratosis-congenita;Chediak-Higashi Syndrome;Severe Aplastic Anemia | Drug: Alefacept | Emory University | Children's Healthcare of Atlanta | Not recruiting | N/A | 21 Years | All | 3 | N/A | United States |
| 19 | EUCTR2010-019645-25-GB | 19/07/2010 | 17 October 2016 | A 24 week trial to study the safety and tolerability of SSP-004184 in the treatment of patients with longterm iron overload who need iron chelation therapy, with the option of 72 weeks further dosing. | A Phase 2, 24 Week, Randomized, Open Label, Multi-Center Study to Assess the Safety, Tolerability, and Pharmacodynamics of SSP-004184 in the Treatment of Chronic Iron Overload Requiring Chelation Therapy, with a 72 Week Dosing Extension. - SPD602-201 | Patients with transfusional iron overload due to hereditary anemias such as sickle cell disease, ß-thalassemia and Diamond-Blackfan anemia; acquired anemias such as Myelodysplastic Syndrome and other forms of bone marrow failure. MedDRA version: 14.1 Level: LLT Classification code 10065974 Term: Chronic iron overload System Organ Class: 100000004861 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: SSP-004184 50 mg Capsule Product Code: SSP-004184 50 mg Pharmaceutical Form: Capsule, hard INN or Proposed INN: Not assigned CAS Number: 1173092-59-5 Current Sponsor code: SSP-004184 Other descriptive name: (S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 50- Product Name: SSP-004184 100 mg Capsule Product Code: SSP-004184 100 mg Pharmaceutical Form: Capsule, hard INN or Proposed INN: Not assigned CAS Number: 1173092-59-5 Current Sponsor code: SSP-004184 Other descriptive name: (S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 100- Product Name: SSP-004184 250 mg Capsule Product Code: SSP-004184 250 mg Pharmaceutical Form: Capsule, hard INN or Proposed INN: Not assigned CAS Number: 1173092-59-5 Current Sponsor code: SSP-004184 Other descriptive name: (S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 250- Product Name: SSP-004184 375 mg Capsule Product Code: SSP-004184 375 mg Pharmaceutical Form: Capsule, hard INN or Proposed INN: Not assigned CAS Number: 1173092-59-5 Current Sponsor code: SSP-004184 Other descriptive name: (S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 375- Product Name: SSP-004184 500 mg Capsule Product Code: SSP-004184 500 mg Pharmaceutical Form: Capsule, hard INN or Proposed INN: Not assigned CAS Number: 1173092-59-5 Current Sponsor code: SSP-004184 | Shire Development LLC | Not Recruiting | Female: yes Male: yes | 50 | Phase 2 | United States;Thailand;Turkey;Italy;United Kingdom | |||
| 20 | NCT01034592 | November 2009 | 16 December 2017 | Pilot Lenalidomide in Adult Diamond-Blackfan Anemia Patients w/ RBC Transfusion-Dependent Anemia | A Pilot Study of Lenalidomide in Adult Diamond-Blackfan Anemia Patients With Red Blood Cell Transfusion-Dependent Anemia | Anemia;Leukemia;Acute Myeloid Leukemia (AML);Myelodysplastic Syndromes (MDS) | Drug: Lenalidomide | Jason Robert Gotlib | Celgene Corporation | Not recruiting | 18 Years | N/A | All | 2 | Phase 1 | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 21 | NCT00957931 | March 2009 | 19 February 2015 | Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs | Pilot Study MUD HCT:Pts High Risk Sickle Cell,Other Non-Malignant RBC Disorders- Reduced Intensity Preparative Regimen, HAPLO-Identical Mesenchymal Stromal Cells | Sickle Cell Disease;Thalassemia;Diamond-Blackfan Anemia | Procedure: Bone marrow transplantation;Biological: Mesenchymal Stromal Cells | Stanford University | University of Minnesota - Clinical and Translational Science Institute;University of Alabama at Birmingham | Not recruiting | 1 Year | 25 Years | Both | 6 | Phase 2 | United States |
| 22 | EUCTR2007-000766-20-GB | 17/07/2008 | 20 March 2012 | A multicenter, randomized, open-label phase II trial evaluating deferasirox compared with deferoxamine in patients with cardiac iron overload due to chronic blood transfusions - CORDELIA | A multicenter, randomized, open-label phase II trial evaluating deferasirox compared with deferoxamine in patients with cardiac iron overload due to chronic blood transfusions - CORDELIA | Beta-thalassemia major (TM) or Diamond Blackfan anemia (DBA) or myelodysplastic syndromes (MDS) (low and INT-1 risk as per the IPSS for MDS) or sideroblastic anemia and myocardial iron overload as indicated by a T2* =6 but not greater than 20 ms, with no symptoms of cardiac dysfunction, and an MRI measured LVEF =56%. | Trade Name: Exjade Product Name: EXJADE Product Code: ICL670 Pharmaceutical Form: Dispersible tablet INN or Proposed INN: deferasirox CAS Number: 201530-41-8 Current Sponsor code: ICL670 Other descriptive name: EXJADE Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 125- Trade Name: Exjade Product Name: EXJADE Product Code: ICL670 Pharmaceutical Form: Dispersible tablet INN or Proposed INN: deferasirox CAS Number: 201530-41-8 Current Sponsor code: ICL670 Other descriptive name: EXJADE Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 250- Trade Name: Exjade Product Name: EXJADE Product Code: ICL670 Pharmaceutical Form: Dispersible tablet INN or Proposed INN: deferasirox CAS Number: 201530-41-8 Current Sponsor code: ICL670 Other descriptive name: EXJADE Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 500- Trade Name: Desferal Product Name: Desferal Product Code: DFO Pharmaceutical Form: Powder for solution for infusion INN or Proposed INN: deferoxamine CAS Number: 70-51-9 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 500- Trade Name: Desferal Product Name: Desferal Product Code: DFO Pharmaceutical Form: Powder for solution for infusion INN or Proposed INN: deferoxamine CAS Number: 70-51-9 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 2000- Trade Name: Exjade Product Name: Exjade Product Code: ICL670 Pharmaceutical Form: Dispersible tablet INN or Proposed INN: deferasirox CAS Number: 201530-41-8 Current Sponsor code: ICL670 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 125- INN or Proposed INN: deferasirox CAS Number: 201530-41-8 Current Sponsor code: ICL670 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 250- INN or Proposed INN: deferasirox CAS Number: 201530-41-8 Concentration unit: mg milligram(s) | Novartis Pharma Services AG | Authorised | Female: yes Male: yes | 192 | Phase 2 | United Kingdom;Italy | |||
| 23 | NCT00673608 | November 2007 | 16 December 2017 | Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron Overload | A Study of Magnetic Resonance Imaging Assessment of Cardiac and Liver Iron Load in Patients With Haemoglobinopathies, Myelodysplastic Syndromes (MDS) or Other Anaemias Treated With Exjade® (Deferasirox) (The MILE Study) | Hemoglobinopathies;Myelodysplastic Syndromes;Other Inherited or Acquired Anaemia;MPD Syndrome;Diamond-Blackfan Anemia;Other Rare Anaemias;Transfusional Iron Overload | Drug: deferasirox | Novartis | Not recruiting | 18 Years | N/A | All | 118 | Phase 4 | Australia | |
| 24 | NCT02512679 | February 2007 | 16 December 2017 | Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells | Protocol for Related Donor Hematopoietic Stem Cell Transplantation (HSCT) for Treatment of Symptomatic Genetic Lymphohematological Diseases | Stem Cell Transplantation;Bone Marrow Transplantation;Peripheral Blood Stem Cell Transplantation;Allogeneic Transplantation;Genetic Diseases;Thalassemia;Pediatrics;Diamond-Blackfan Anemia;Combined Immune Deficiency;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;X-linked Lymphoproliferative Disease;Metabolic Diseases | Drug: Cyclophosphamide Dose Level 1;Drug: Cyclophosphamide Dose Level 2;Drug: Cyclophosphamide Dose Level 3;Drug: Cyclophosphamide Dose Level 4 | Children's Hospital Los Angeles | Lucile Packard Children's Hospital | Not recruiting | 3 Months | N/A | All | 20 | Phase 2 | |
| 25 | NCT00235391 | October 2005 | 19 October 2017 | Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload | A Study to Provide Expanded Access of (Exjade®) Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload From Blood Transfusions Who Cannot Adequately be Treated With Other Locally Approved Iron Chelators | Thalassemia;Sickle Cell Disease;Diamond Blackfan Anemia;Myelofibrosis | Drug: Deferasirox | Novartis Pharmaceuticals | Not recruiting | 2 Years | N/A | All | 1683 | Phase 3 | United States;Belgium;Canada;Germany;Greece;Italy;Netherlands;Spain;Taiwan;Thailand;Turkey;United Kingdom;Australia | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 26 | NCT00229619 | September 2005 | 19 October 2017 | Rituximab to Treat Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan Anemia | A Pilot Study of Recombinant Humanized Anti- Cluster of Differentiation Antigen 20 (Anti-CD20) Antibody (Rituximab) in Patients With Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan Anemia | Anemia, Aplastic;Red-Cell Aplasia, Pure;Anemia, Diamond-Blackfan | Drug: Rituximab | National Heart, Lung, and Blood Institute (NHLBI) | Not recruiting | 2 Years | N/A | All | 11 | Phase 2 | United States | |
| 27 | NCT00301834 | January 2005 | 16 December 2017 | Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders | Evaluation of Fludarabine, Busulfan and Alemtuzumab as a Reduced Toxicity Ablative Bone Marrow Stem Cell Transplant Regimen for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myelodysplastic Syndrome (MDS)/Leukemia | Congenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Leukemia;Myelodysplastic Syndromes;Severe Congenital Neutropenia | Biological: alemtuzumab;Drug: busulfan;Drug: cyclosporine;Drug: fludarabine phosphate;Drug: methotrexate;Drug: methylprednisolone;Procedure: allogeneic bone marrow transplantation;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantation | University of California, San Francisco | National Cancer Institute (NCI) | Not recruiting | N/A | 21 Years | All | 35 | Phase 2 | United States |
| 28 | NCT00176852 | June 2002 | 1 October 2018 | Stem Cell Transplant for Hemoglobinopathy | Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathy Using a Preparative Regimen to Achieve Stable Mixed Chimerism | Sickle Cell Disease;Thalassemia;Severe Congenital Neutropenia;Diamond-Blackfan Anemia;Shwachman-Diamond Syndrome | Drug: Busulfan, Fludarabine, ATG, TLI;Drug: Busulfan, Cyclophosphamide, ATG, GCSF;Drug: Campath, Fludarabine, Cyclophosphamide;Radiation: Total Body Irradiation;Procedure: Stem cell infusion | Masonic Cancer Center, University of Minnesota | National Marrow Donor Program | Not recruiting | N/A | 50 Years | All | 22 | Phase 2/Phase 3 | United States |
| 29 | NCT00011505 | February 2001 | 19 February 2015 | Mobilization of Stem Cells With G-CSF for Collection From Patients With Diamond-Blackfan Anemia | Investigation of G-CSF-Induced Stem Cell Mobilization Potential in Patients With Diamond-Blackfan Anemia | Diamond Blackfan Anemia | Drug: G-CSF;Procedure: Leukapheresis | National Heart, Lung, and Blood Institute (NHLBI) | Not recruiting | N/A | N/A | Both | 15 | Phase 2 | United States | |
| 30 | NCT00305708 | August 2000 | 19 February 2015 | Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First Remission | Bone Marrow Stem Cell Transplantation for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myeloid Leukemia in 1Remission | Congenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Fanconi Anemia;Leukemia;Severe Congenital Neutropenia;Thrombocytopenia | Biological: anti-thymocyte globulin;Drug: busulfan;Drug: fludarabine phosphate;Procedure: allogeneic bone marrow transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantation;Radiation: radiation therapy | University of California, San Francisco | National Cancer Institute (NCI) | Not recruiting | N/A | 17 Years | Both | 40 | Phase 1/Phase 2 | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 31 | NCT00176878 | June 2000 | 19 October 2017 | Stem Cell Transplant for Bone Marrow Failure Syndromes | Bone Marrow Transplantation for Non-Malignant Congenital Bone Marrow Failure Disorders | Diamond-Blackfan Anemia;Kostmann's Neutropenia;Shwachman-Diamond Syndrome | Procedure: Stem cell transplant;Drug: Fludarabine monophosphate;Procedure: Total lymphoid irradiation;Drug: Busulfan;Biological: anti-thymocyte globulin | Masonic Cancer Center, University of Minnesota | Not recruiting | N/A | 35 Years | All | 10 | Phase 2/Phase 3 | United States | |
| 32 | NCT00001962 | November 1999 | 19 October 2017 | A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow Failure | A Pilot Study of Recombinant Humanized Anti-Interleukin (IL-2) Receptor Antibody (Daclizumab) in Patients With Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan Anemia | Aplastic Anemia;Pure Red Cell Aplasia;Diamond Blackfan Anemia | Drug: Daclizumab | Neal Young, M.D. | Not recruiting | 2 Years | N/A | All | 100 | Phase 2 | United States | |
| 33 | NCT00290628 | October 1999 | 16 December 2017 | Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic Cancer | Transplantation of Umbilical Cord Blood From Related and Unrelated Donors | Chronic Myeloproliferative Disorders;Diamond-blackfan Anemia;Fanconi Anemia;Graft Versus Host Disease;Leukemia;Lymphoma;Multiple Myeloma and Plasma Cell Neoplasm;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative Diseases | Drug: cyclophosphamide;Drug: cyclosporine;Drug: filgrastim;Drug: melphalan;Drug: methylprednisolone;Drug: mycophenolate mofetil;Procedure: radiation therapy;Procedure: umbilical cord blood transplantation;Drug: anti-thymocyte globulin;Drug: busulfan | Masonic Cancer Center, University of Minnesota | National Cancer Institute (NCI) | Not recruiting | N/A | 45 Years | All | 43 | N/A | United States |
| 34 | NCT00001749 | July 1998 | 19 February 2015 | Medical Treatment for Diamond Blackfan Anemia | Treatment of Diamond Blackfan Anemia With Antithymocyte Globulin and Cyclosporine A | Fanconi's Anemia;Hematologic Disease | Drug: Antithymocyte globulin;Drug: Cyclosporine | National Heart, Lung, and Blood Institute (NHLBI) | Not recruiting | N/A | N/A | Both | 25 | Phase 2 | United States |