65. Primary immunodeficiency Clinical trials / Disease details
Clinical trials : 500 / Drugs : 614 - (DrugBank : 119) / Drug target genes : 92 - Drug target pathways : 217
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04965597 (ClinicalTrials.gov) | April 19, 2022 | 21/6/2021 | Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) | Hematopoietic Cell Transplantation Using Treosulfan-Based Conditioning for the Treatment of Bone Marrow Failure Diseases | Bone Marrow Failure Syndrome;Congenital Amegakaryocytic Thrombocytopenia;Congenital Pure Red Cell Aplasia;Hereditary Sideroblastic Anemia;Myeloid Neoplasms With Germline GATA2 Mutation;Paroxysmal Nocturnal Hemoglobinuria;Shwachman-Diamond Syndrome | Drug: Treosulfan;Drug: Fludarabine Phosphate;Drug: Tacrolimus;Drug: Methotrexate;Biological: Lapine T-Lymphocyte Immune Globulin;Procedure: Peripheral Blood Stem Cell Transplantation;Procedure: Allogeneic Bone Marrow Transplantation;Other: Quality-of-Life Assessment | Fred Hutchinson Cancer Center | Blood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor Program;National Heart, Lung, and Blood Institute (NHLBI) | Recruiting | 1 Year | 49 Years | All | 40 | Phase 2 | United States |
| 2 | EUCTR2018-000338-36-IT (EUCTR) | 12/05/2021 | 04/06/2021 | Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study. | Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study. - OLE-IEDAT | Neurological symptoms of Ataxia Telangiectasia MedDRA version: 21.0;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate (soluzione 25mg/ml) Product Code: [Dexamethasone sodium phosphate] INN or Proposed INN: DEXAMETHASONE SODIUM PHOSPHATE | ERYDEL S.P.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 155 | Phase 3 | United States;Spain;Poland;Belgium;Australia;Norway;Germany;Tunisia;United Kingdom;Italy;India | ||
| 3 | EUCTR2018-000338-36-NO (EUCTR) | 06/11/2020 | 04/02/2020 | Not Applicable | Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study - OLE-IEDAT | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 21.0;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate INN or Proposed INN: Dexamethasone sodium phosphate Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | EryDel S.p.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 155 | Phase 3 | United States;Spain;Poland;Belgium;Australia;Tunisia;Germany;Norway;United Kingdom;Italy;India | ||
| 4 | EUCTR2018-000338-36-GB (EUCTR) | 27/05/2020 | 20/01/2020 | Not Applicable | Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study - OLE-IEDAT, version 5.0 | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 21.0;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate INN or Proposed INN: Dexamethasone sodium phosphate Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | EryDel S.p.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 51 | Phase 3 | United States;Spain;Poland;Belgium;Australia;Israel;Norway;Germany;United Kingdom;India | ||
| 5 | EUCTR2018-004191-35-BE (EUCTR) | 12/04/2019 | 05/03/2019 | Use of an antidiabetic drug (Empagliflozin) to lower the blood level of 1,5-anhydroglucitol in patients deficient in the glucose-6-phosphate transporter (GSD1b) and the phosphatase G6PC3, both of the endoplasmic reticulum, to treat their recurrent infections by normalizing their blood neutrophil counts. Neutrophils are the most abundant white blood cells in our blood that are essential to help fighting infections. 1,5-anhydroglucitol is a sugar derivative with no known function. | Evaluation of the safety and efficacy of administration of Empagliflozin in a new treatment for neutropenia in patients with Glycogen Storage Disease type 1b (GSD1b) and G6PC3 deficiency. - GLYCO-1B | (1) Severe Congenital Neutropenia type 4 (SNC4) due to a deficiency in G6PC3, a phosphatase of the endoplasmic also known a Ubiquitous glucose-6-phosphatase and (2) the neutropenia in Glycogen Storage Disease type 1b due to a deficiency in the glucose-6-phosphate transporter (G6PT / SLC37A4) of the endoplasmic reticulum.;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Trade Name: Jardiance Product Name: Empagliflozin Product Code: A10BK03 | Cliniques universitaires Saint-Luc | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 4 | Belgium | ||
| 6 | EUCTR2018-000338-36-DE (EUCTR) | 20/09/2018 | 21/06/2018 | Not Applicable | Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study - OLE-IEDAT | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 21.0;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate INN or Proposed INN: Dexamethasone sodium phosphate Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | EryDel S.p.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 150 | Phase 3 | United States;Spain;Poland;Belgium;Australia;Israel;Norway;Germany;United Kingdom;India | ||
| 7 | EUCTR2018-000338-36-BE (EUCTR) | 23/08/2018 | 28/06/2018 | Not Applicable | Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study - OLE-IEDAT | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 20.1;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate INN or Proposed INN: Dexamethasone sodium phosphate Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | EryDel S.p.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 51 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;Poland;Spain;Belgium;Australia;Israel;Germany;India | ||
| 8 | EUCTR2018-000338-36-ES (EUCTR) | 31/05/2018 | 05/04/2018 | Not Applicable | Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study - OLE-IEDAT | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 20.1;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate INN or Proposed INN: Dexamethasone sodium phosphate Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | EryDel S.p.A. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 61 | Phase 3 | United States;Poland;Belgium;Spain;Australia;Israel;Norway;Germany;Italy;United Kingdom;India | ||
| 9 | NCT03333486 (ClinicalTrials.gov) | December 7, 2017 | 2/11/2017 | Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer | A Phase II Trial of Haploidentical Allogeneic Stem Cell Transplantation Utilizing Mobilized Peripheral Blood Stem Cells | Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Acute Leukemia in Remission;Acute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Acute Myeloid Leukemia With FLT3/ITD Mutation;Acute Myeloid Leukemia With Gene Mutations;Aplastic Anemia;B-Cell Non-Hodgkin Lymphoma;CD40 Ligand Deficiency;Chronic Granulomatous Disease;Chronic Leukemia in Remission;Chronic Lymphocytic Leukemia;Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Congenital Amegakaryocytic Thrombocytopenia;Congenital Neutropenia;Congenital Pure Red Cell Aplasia;Glanzmann Thrombasthenia;Immunodeficiency Syndrome;Myelodysplastic Syndrome;Myelofibrosis;Myeloproliferative Neoplasm;Paroxysmal Nocturnal Hemoglobinuria;Plasma Cell Myeloma;Polycythemia Vera;Recurrent Non-Hodgkin Lymphoma;Refractory Non-Hodgkin Lymphoma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndrome;Severe Aplastic Anemia;Shwachman-Diamond Syndrome;Sickle Cell Disease;T-Cell Non-Hodgkin Lymphoma;Thalassemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich Syndrome | Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Procedure: Peripheral Blood Stem Cell Transplantation;Radiation: Total-Body Irradiation | Roswell Park Cancer Institute | NULL | Active, not recruiting | 1 Year | 75 Years | All | 31 | Phase 2 | United States |
| 10 | NCT02770807 (ClinicalTrials.gov) | March 2, 2017 | 2/5/2016 | EDS in Ataxia Telangiectasia Patients | Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients With Ataxia Telangiectasia | Nervous System Disease;Genetic Syndrome | Drug: EDS-EP dose range of ~5-10 mg DSP/infusion;Drug: EDS-EP dose range of ~14-22 mg DSP/infusion;Drug: Placebo | Erydel | NULL | Completed | 6 Years | N/A | All | 175 | Phase 3 | United States;Australia;Belgium;Germany;India;Israel;Italy;Norway;Poland;Spain;Tunisia;United Kingdom |
| 11 | EUCTR2015-005241-31-NO (EUCTR) | 21/11/2016 | 02/06/2016 | n/a | A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients with Ataxia Telangiectasia - Ataxia Telangiectasia - Treatment with EryDex SysTem - ATTEST | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 21.0;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate INN or Proposed INN: Dexamethasone sodium phosphate Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | EryDel S.p.A. | NULL | Not Recruiting | Female: yes Male: yes | 175 | Phase 3 | United States;Spain;Poland;Belgium;Australia;Israel;Tunisia;Germany;Norway;United Kingdom;Italy;India | ||
| 12 | EUCTR2015-005241-31-IT (EUCTR) | 02/11/2016 | 28/02/2018 | n/a | A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients with Ataxia Telangiectasia - EDS in Ataxia Telangiectasia Patients - IEDAT-02 | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 20.1;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexametasone Fosfato Sodico INN or Proposed INN: Dexametasone Fosfato Sodico Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | ERYDEL S.P.A. | NULL | Not Recruiting | Female: yes Male: yes | 180 | Phase 3 | Tunisia;United States;Costa Rica;Spain;Turkey;Israel;Italy;United Kingdom;Belgium;Poland;Australia;Germany;Norway | ||
| 13 | EUCTR2015-005241-31-BE (EUCTR) | 21/10/2016 | 01/06/2016 | n/a | A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients with Ataxia Telangiectasia - Ataxia Telangiectasia - Treatment with EryDex SysTem - ATTEST | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 20.1;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate INN or Proposed INN: Dexamethasone sodium phosphate Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | EryDel S.p.A. | NULL | Not Recruiting | Female: yes Male: yes | 180 | Phase 3 | United States;Spain;Poland;Belgium;Australia;Israel;Tunisia;Germany;United Kingdom;Italy;India;Norway | ||
| 14 | EUCTR2015-005241-31-DE (EUCTR) | 14/09/2016 | 27/05/2016 | n/a | A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients with Ataxia Telangiectasia - Ataxia Telangiectasia - Treatment with EryDex SysTem - ATTEST | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 21.0;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate INN or Proposed INN: Dexamethasone sodium phosphate Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | EryDel S.p.A. | NULL | Not Recruiting | Female: yes Male: yes | 175 | Phase 3 | United States;Spain;Poland;Belgium;Australia;Israel;Tunisia;Norway;Germany;United Kingdom;Italy;India | ||
| 15 | EUCTR2015-005241-31-ES (EUCTR) | 31/05/2016 | 09/06/2016 | n/a | A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients with Ataxia Telangiectasia - EDS in Ataxia Telangiectasia Patients - IEDAT-02 | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 19.0;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate INN or Proposed INN: Dexamethasone sodium phosphate Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | EryDel S.p.A. | NULL | Not Recruiting | Female: yes Male: yes | 180 | Phase 3 | United States;Spain;Costa Rica;Turkey;Israel;United Kingdom;Italy;Belgium;Poland;Australia;Tunisia;Germany;Norway | ||
| 16 | NCT01652092 (ClinicalTrials.gov) | September 4, 2012 | 25/7/2012 | Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies | Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies | SCID;Omenn's Syndrome;Reticular Dysgenesis;Wiskott-Aldrich Syndrome;Bare Lymphocyte Syndrome;Common Variable Immunodeficiency;Chronic Granulomatous Disease;CD40 Ligand Deficiency;Hyper IgM Syndrome;X-linked Lymphoproliferative Disease;Hemophagocytic Lymphohistiocytosis;Griscelli Syndrome;Chediak-Higashi Syndrome;Langerhan's Cell Histiocytosis | Drug: Alemtuzumab 0.3 mg;Drug: Cyclophosphamide;Drug: Busulfan;Biological: Stem Cell Transplantation;Drug: Fludarabine phosphate 40 mg;Drug: Melphalan;Drug: Alemtuzumab 0.2 mg;Drug: Fludarabine phosphate 30 mg;Drug: MESNA | Masonic Cancer Center, University of Minnesota | NULL | Recruiting | N/A | 50 Years | All | 30 | N/A | United States |
| 17 | NCT01529827 (ClinicalTrials.gov) | February 28, 2012 | 6/2/2012 | 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 | 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;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;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 | 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 | NULL | Completed | 3 Years | 75 Years | All | 94 | Phase 2 | United States |
| 18 | EUCTR2005-003201-81-DE (EUCTR) | 20/12/2006 | 19/01/2007 | GACHE: German trial of Acyclovir and Corticosteroids in Herpes-simplex-virus-Encephalitis. A multicenter, multinational, randomized, double-blind, placebo-controlled German, Austrian and Dutch trial. - GACHE | GACHE: German trial of Acyclovir and Corticosteroids in Herpes-simplex-virus-Encephalitis. A multicenter, multinational, randomized, double-blind, placebo-controlled German, Austrian and Dutch trial. - GACHE | Herpes simplex virus type-1 (HSV-1) is the most frequent cause of fatal sporadic encephalitis in humans. Herpes simplex encephalitis (HSE) was one of the first viral infections to be successfully treated with antiviral drugs. Mortality has been significantly reduced since the introduction of acyclovir, a specific inhibitor of HSV replication. Despite appropriate and promptly initiated antiviral therapy the incidence of persistent neurological deficits remain unacceptably high. | Trade Name: Fortecortin Inject 40 mg Amp. Product Name: Fortecortin INN or Proposed INN: dexamethasone 21-dihydrogen phosphate | Universitätsklinikum Heidelberg | NULL | Not Recruiting | Female: yes Male: yes | 450 | Phase 3 | Germany | ||
| 19 | NCT00358657 (ClinicalTrials.gov) | May 24, 2006 | 28/7/2006 | Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders | HLA-Haploidentical Related Marrow Grafts for the Treatment of Primary Immunodeficiencies and Other Nonmalignant Disorders Using Conditioning With Low-Dose Cyclophosphamide, TBI and Fludarabine and Postgrafting Cyclophosphamide | Immunodeficiency Syndrome;Non-Cancer Diagnosis;Severe Aplastic Anemia;Donor | Procedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Drug: Sirolimus;Drug: Tacrolimus;Radiation: Total-Body Irradiation | Fred Hutchinson Cancer Research Center | National Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI) | Active, not recruiting | N/A | 55 Years | All | 14 | Phase 2 | United States |
| 20 | NCT00295971 (ClinicalTrials.gov) | April 2005 | 23/2/2006 | Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency Disease | Stem Cell Enriched, T Cell Depleted Haplocompatible Peripheral Blood Transplantation for Children With Myelodysplastic Disease, Leukemia, Marrow Failure Syndromes, or Severe Immunodeficiency Diseases | Congenital Amegakaryocytic Thrombocytopenia;Leukemia;Myelodysplastic Syndromes;Severe Congenital Neutropenia | Biological: anti-thymocyte globulin;Biological: therapeutic allogeneic lymphocytes;Drug: fludarabine phosphate;Drug: thiotepa;Procedure: allogeneic bone marrow transplantation;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: in vitro-treated peripheral blood stem cell transplantation;Radiation: total-body irradiation | University of California, San Francisco | National Cancer Institute (NCI) | Completed | 1 Year | 17 Years | Both | 21 | Phase 1 | United States |
| 21 | NCT00301834 (ClinicalTrials.gov) | January 2005 | 9/3/2006 | 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) | Completed | N/A | 21 Years | All | 35 | Phase 2 | United States |
| 22 | NCT00305708 (ClinicalTrials.gov) | August 2000 | 21/3/2006 | 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) | Completed | N/A | 17 Years | Both | 40 | Phase 1/Phase 2 | United States |
| 23 | EUCTR2015-005241-31-PL (EUCTR) | 01/07/2016 | n/a | A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Patients with Ataxia Telangiectasia - EDS in Ataxia Telangiectasia Patients - IEDAT-02 | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 21.0;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate INN or Proposed INN: Dexamethasone sodium phosphate Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | EryDel S.p.A. | NULL | Not Recruiting | Female: yes Male: yes | 180 | Phase 3 | United States;Costa Rica;Spain;Turkey;Israel;Italy;United Kingdom;India;Poland;Belgium;Australia;Germany;Norway;Tunisia | |||
| 24 | EUCTR2018-000338-36-PL (EUCTR) | 06/11/2018 | Not Applicable | Open-label, Long-term, Extension Treatment using Intra-Erythrocyte Dexamethasone Sodium Phosphate in Patients with Ataxia Telangiectasia Who Participated in the IEDAT-02-2015 Study - OLE-IEDAT | Patient with neurological symptoms of Ataxia Telangiectasia MedDRA version: 21.0;Level: PT;Classification code 10003594;Term: Ataxia telangiectasia;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Dexamethasone sodium phosphate INN or Proposed INN: Dexamethasone sodium phosphate Other descriptive name: DEXAMETHASONE SODIUM PHOSPHATE PH. EUR. | EryDel S.p.A. | NULL | NA | Female: yes Male: yes | 155 | Phase 3 | United States;Spain;Belgium;Poland;Australia;Tunisia;Norway;Germany;United Kingdom;Italy;India |