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 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.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04099966October 1, 20197 October 2019AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell DepletionAllogeneic Stem Cell Transplantation for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion - NYMC 588Acute Leukemia;Severe Aplastic Anemia;Non-hodgkin Lymphoma;Hodgkin Lymphoma;Kostmann;Diamond Blackfan Anemia;Amegakaryocytic Thrombocytopenia;Sickle Cell Disease;Beta-ThalassemiaDrug: alpha beta depletionMitchell CairoNot recruitingN/A30 YearsAll20Phase 2United States
2NCT03966053September 13, 201930 September 2019The Use of Trifluoperazine in Transfusion Dependent DBAPhase I/II, Open Label Study to Determine Safety of Trifluoperazine (TFP) in Adults With Red Blood Cell Transfusion-Dependent Diamond Blackfan AnemiaDiamond Blackfan Anemia;Pure Red Cell AplasiaDrug: TrifluoperazineAdrianna VlachosRecruiting18 Years65 YearsAll24Phase 1/Phase 2United States
3NCT03653338August 2, 201811 March 2019T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other AnemiasT-Cell Depleted, Alternative Donor Transplant in Pediatric and Adult Patients With Severe Sickle Cell Disease (SCD) and Other Transfusion-Dependent AnemiasSickle Cell Anemia;Beta-thalassemia Major;Diamond-blackfan AnemiaBiological: CD3/CD19 depleted leukocytes;Biological: CD45RA depleted leukocytes;Drug: Hydroxyurea;Drug: Rituximab;Drug: Alemtuzumab;Drug: Fludarabine;Drug: ThiotepaBeth Carella, DORecruiting5 Years40 YearsAll5Phase 1/Phase 2United States
4NCT03513328June 15, 20183 June 2019Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell TransplantationPEDS024, Phase I/II Feasibility Study of Busulfan Fludarabine and Thiotepa Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation (HSCT) for Children With Non-Malignant DisordersBone 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;AdrenoleukodystrophyDrug: Thiotepa--single daily dose;Drug: Thiotepa--escalated doseUniversity of FloridaLive Like Bella Pediatric Cancer ResearchRecruiting6 Months38 YearsAll40Phase 1/Phase 2United States
5NCT03733249January 201728 October 2019Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical StudyFollow-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 DisordersAcute 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 CellDrug: Rimiducid;Biological: rivogenlecleucelBellicum PharmaceuticalsRecruiting1 Month18 YearsAll193Phase 1/Phase 2Italy;Saudi Arabia;United Kingdom
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
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Target_
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PhaseCountries
6NCT02179359September 2, 20147 October 2019Hematopoietic Stem Cell Transplant for High Risk HemoglobinopathiesMT2014-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent DisordersSickle 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 DisordersDrug: Reduced Toxicity Ablative Regimen;Drug: Reduced Intensity Preparative Regimen;Drug: Myeloablative Preparative RegimenMasonic Cancer Center, University of MinnesotaRecruitingN/A55 YearsAll25N/AUnited States
7NCT02386267September 201416 March 2015L-leucine in Diamond Blackfan Anemia PatientsTherapeutic Use of the Amino Acid Leucine in the Treatment of Transfusion-Dependent Diamond Blackfan Anemia PatientsDiamond Blackfan AnemiaDrug: L-leucineFederal Scientific Clinical Centre of Pediatric Hematology, Oncology and Immunology named after Dmitry RogacheRecruiting1 Year20 YearsBoth30Phase 2Russian Federation
8NCT02065869April 20144 November 2019Safety Study of Gene Modified Donor T-cells Following TCR Alpha Beta Depleted Stem Cell TransplantPhase 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 DisordersAcute 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 CellBiological: BPX-501 T cells;Drug: rimiducidBellicum PharmaceuticalsNot recruiting1 Month18 YearsAll193Phase 1/Phase 2Italy;United Kingdom;Germany;Spain;United States
9NCT01917708January 201419 November 2018BMT Abatacept for Non-Malignant DiseasesAbatacept for Post-Transplant Immune Suppression in Children and Adolescents Receiving Allogeneic Hematopoietic Stem Cell Transplants for Non-Malignant DiseasesHurler 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 DiseaseDrug: AbataceptEmory UniversityNot recruitingN/A21 YearsAll10Phase 1United States
10NCT01362595June 201330 September 2019Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan AnemiaThe 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 AnemiaDiamond Blackfan Anemia;Blackfan Diamond Syndrome;DBA;Congenital Hypoplastic Anemia;Pure Red Cell AplasiaDrug: leucineNorthwell HealthNot recruiting2 YearsN/AAll50Phase 1/Phase 2United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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Recruitment_
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Inclusion_
agemin
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PhaseCountries
11NCT01586455April 20138 January 2018Human Placental-Derived Stem Cell TransplantationA 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 DisordersMucopolysaccharidosis 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 LeukemiaDrug: Human Placental Derived Stem CellNew York Medical CollegeNot recruitingN/A55 YearsAll43Phase 1United States
12NCT01966367March 201325 March 2019CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell TransplantationCD34+ Stem Cell Selection for Patients Receiving a Matched or Partially Matched Family or Unrelated Adult Donor Allogeneic Stem Cell Transplantation for Non-Malignant DiseaseBone 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-thalassemiaBiological: CD34 Stem Cell Selection TherapyDiane George, MDRecruitingN/A40 YearsAll25Early Phase 1United States
13EUCTR2011-006322-25-GB08/10/201217 October 2016Extending the treatment of Iron OverloadA 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 LLCNot RecruitingFemale: yes
Male: yes
1500Phase 2Egypt;United States;Canada;Thailand;Lebanon;Turkey;Italy;United Kingdom
14EUCTR2011-005675-16-IT15/05/201223 March 2015Treatment 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 overloadPatients 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 RecruitingFemale: yes
Male: yes
50Phase 2United States;Canada;Lebanon;Turkey;Italy
15NCT01529827February 28, 201230 September 2019Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic MalignanciesA Phase II Trial of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan and Low Dose Total Body IrradiationPrimary 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 LeukemiaDrug: 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 transplantationRoswell Park Cancer InstituteNot recruiting3 Years75 YearsAll94Phase 2United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT01464164January 20123 June 2019Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan AnemiaPhase I/II, Open-Label Study to Determine Safety and Efficacy of Sotatercept (ACE-011) in Adults With Red Blood Cell Transfusion- Dependent Diamond Blackfan AnemiaDiamond Blackfan AnemiaDrug: Sotatercept;Drug: Sotatercept with prednisone boostNorthwell HealthRecruiting18 YearsN/AAll20Phase 1/Phase 2United States
17NCT01419704May 201130 September 2019Phase I/II Pilot Study of Mixed Chimerism to Treat HemoglobinopathiesPhase I/II Pilot Study of Mixed Chimerism to Treat HemoglobinopathiesAnemia, Sickle Cell;Complex and Transfusion-dependent Hemoglobinopathies;Thalassemia;Diamond-Blackfan Anemia;Bone Marrow Failure Syndromes;Alpha-Thalassemia;Beta-ThalassemiaBiological: Enriched Hematopoetic Stem Cell InfusionUniversity of LouisvilleDuke UniversityNot recruitingN/A45 YearsAll30Phase 1/Phase 2United States
18NCT01319851September 201016 December 2017Alefacept and Allogeneic Hematopoietic Stem Cell TransplantationAlefacept and Allogeneic Hematopoietic Stem Cell Transplantation for Children With Non-Malignant Diseases Who Have Been Multiply Transfused: a Pilot StudyThalassemia;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 AnemiaDrug: AlefaceptEmory UniversityChildren's Healthcare of AtlantaNot recruitingN/A21 YearsAll3N/AUnited States
19EUCTR2010-019645-25-GB19/07/201017 October 2016A 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. - SPD602-201Patients 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 LLCNot RecruitingFemale: yes
Male: yes
50Phase 2United States;Thailand;Turkey;Italy;United Kingdom
20NCT01034592November 200916 December 2017Pilot Lenalidomide in Adult Diamond-Blackfan Anemia Patients w/ RBC Transfusion-Dependent AnemiaA Pilot Study of Lenalidomide in Adult Diamond-Blackfan Anemia Patients With Red Blood Cell Transfusion-Dependent AnemiaAnemia;Leukemia;Acute Myeloid Leukemia (AML);Myelodysplastic Syndromes (MDS)Drug: LenalidomideJason Robert GotlibCelgene CorporationNot recruiting18 YearsN/AAll2Phase 1United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21NCT00957931March 200919 February 2015Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCsPilot Study MUD HCT:Pts High Risk Sickle Cell,Other Non-Malignant RBC Disorders- Reduced Intensity Preparative Regimen, HAPLO-Identical Mesenchymal Stromal CellsSickle Cell Disease;Thalassemia;Diamond-Blackfan AnemiaProcedure: Bone marrow transplantation;Biological: Mesenchymal Stromal CellsStanford UniversityUniversity of Minnesota - Clinical and Translational Science Institute;University of Alabama at BirminghamNot recruiting1 Year25 YearsBoth6Phase 2United States
22EUCTR2007-000766-20-GB17/07/200820 March 2012A multicenter, randomized, open-label phase II trial evaluating deferasirox compared with deferoxamine in patients with cardiac iron overload due to chronic blood transfusions - CORDELIAA multicenter, randomized, open-label phase II trial evaluating deferasirox compared with deferoxamine in patients with cardiac iron overload due to chronic blood transfusions - CORDELIABeta-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 AGAuthorisedFemale: yes
Male: yes
192Phase 2United Kingdom;Italy
23NCT00673608November 200716 December 2017Magnetic Resonance Imaging (MRI) Assessments of the Heart and Liver Iron Load in Patients With Transfusion Induced Iron OverloadA 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 OverloadDrug: deferasiroxNovartisNot recruiting18 YearsN/AAll118Phase 4Australia
24NCT02512679February 200716 December 2017Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood CellsProtocol for Related Donor Hematopoietic Stem Cell Transplantation (HSCT) for Treatment of Symptomatic Genetic Lymphohematological DiseasesStem 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 DiseasesDrug: Cyclophosphamide Dose Level 1;Drug: Cyclophosphamide Dose Level 2;Drug: Cyclophosphamide Dose Level 3;Drug: Cyclophosphamide Dose Level 4Children's Hospital Los AngelesLucile Packard Children's HospitalNot recruiting3 MonthsN/AAll20Phase 2
25NCT00235391October 200519 October 2017Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron OverloadA 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 ChelatorsThalassemia;Sickle Cell Disease;Diamond Blackfan Anemia;MyelofibrosisDrug: DeferasiroxNovartis PharmaceuticalsNot recruiting2 YearsN/AAll1683Phase 3United States;Belgium;Canada;Germany;Greece;Italy;Netherlands;Spain;Taiwan;Thailand;Turkey;United Kingdom;Australia
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
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size
PhaseCountries
26NCT00229619September 200519 October 2017Rituximab to Treat Moderate Aplastic Anemia, Pure Red Cell Aplasia, or Diamond Blackfan AnemiaA 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 AnemiaAnemia, Aplastic;Red-Cell Aplasia, Pure;Anemia, Diamond-BlackfanDrug: RituximabNational Heart, Lung, and Blood Institute (NHLBI)Not recruiting2 YearsN/AAll11Phase 2United States
27NCT00301834January 200516 December 2017Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic DisordersEvaluation 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)/LeukemiaCongenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Leukemia;Myelodysplastic Syndromes;Severe Congenital NeutropeniaBiological: 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 transplantationUniversity of California, San FranciscoNational Cancer Institute (NCI)Not recruitingN/A21 YearsAll35Phase 2United States
28NCT00176852June 20021 October 2018Stem Cell Transplant for HemoglobinopathyAllogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathy Using a Preparative Regimen to Achieve Stable Mixed ChimerismSickle Cell Disease;Thalassemia;Severe Congenital Neutropenia;Diamond-Blackfan Anemia;Shwachman-Diamond SyndromeDrug: Busulfan, Fludarabine, ATG, TLI;Drug: Busulfan, Cyclophosphamide, ATG, GCSF;Drug: Campath, Fludarabine, Cyclophosphamide;Radiation: Total Body Irradiation;Procedure: Stem cell infusionMasonic Cancer Center, University of MinnesotaNational Marrow Donor ProgramNot recruitingN/A50 YearsAll22Phase 2/Phase 3United States
29NCT00011505February 200119 February 2015Mobilization of Stem Cells With G-CSF for Collection From Patients With Diamond-Blackfan AnemiaInvestigation of G-CSF-Induced Stem Cell Mobilization Potential in Patients With Diamond-Blackfan AnemiaDiamond Blackfan AnemiaDrug: G-CSF;Procedure: LeukapheresisNational Heart, Lung, and Blood Institute (NHLBI)Not recruitingN/AN/ABoth15Phase 2United States
30NCT00305708August 200019 February 2015Busulfan, 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 RemissionBone Marrow Stem Cell Transplantation for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myeloid Leukemia in 1RemissionCongenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Fanconi Anemia;Leukemia;Severe Congenital Neutropenia;ThrombocytopeniaBiological: 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 therapyUniversity of California, San FranciscoNational Cancer Institute (NCI)Not recruitingN/A17 YearsBoth40Phase 1/Phase 2United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
31NCT00176878June 200019 October 2017Stem Cell Transplant for Bone Marrow Failure SyndromesBone Marrow Transplantation for Non-Malignant Congenital Bone Marrow Failure DisordersDiamond-Blackfan Anemia;Kostmann's Neutropenia;Shwachman-Diamond SyndromeProcedure: Stem cell transplant;Drug: Fludarabine monophosphate;Procedure: Total lymphoid irradiation;Drug: Busulfan;Biological: anti-thymocyte globulinMasonic Cancer Center, University of MinnesotaNot recruitingN/A35 YearsAll10Phase 2/Phase 3United States
32NCT00001962November 199919 October 2017A Study to Determine Whether Therapy With Daclizumab Will Benefit Patients With Bone Marrow FailureA 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 AnemiaAplastic Anemia;Pure Red Cell Aplasia;Diamond Blackfan AnemiaDrug: DaclizumabNeal Young, M.D.Not recruiting2 YearsN/AAll100Phase 2United States
33NCT00290628October 199916 December 2017Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic CancerTransplantation of Umbilical Cord Blood From Related and Unrelated DonorsChronic Myeloproliferative Disorders;Diamond-blackfan Anemia;Fanconi Anemia;Graft Versus Host Disease;Leukemia;Lymphoma;Multiple Myeloma and Plasma Cell Neoplasm;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative DiseasesDrug: 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: busulfanMasonic Cancer Center, University of MinnesotaNational Cancer Institute (NCI)Not recruitingN/A45 YearsAll43N/AUnited States
34NCT00001749July 199819 February 2015Medical Treatment for Diamond Blackfan AnemiaTreatment of Diamond Blackfan Anemia With Antithymocyte Globulin and Cyclosporine AFanconi's Anemia;Hematologic DiseaseDrug: Antithymocyte globulin;Drug: CyclosporineNational Heart, Lung, and Blood Institute (NHLBI)Not recruitingN/AN/ABoth25Phase 2United States

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