Busulfan (DrugBank: Busulfan)
13 diseases| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 19 | ライソゾーム病 | 8 |
| 20 | 副腎白質ジストロフィー | 9 |
| 28 | 全身性アミロイドーシス | 2 |
| 36 | 表皮水疱症 | 2 |
| 49 | 全身性エリテマトーデス | 1 |
| 51 | 全身性強皮症 | 1 |
| 60 | 再生不良性貧血 | 12 |
| 65 | 原発性免疫不全症候群 | 42 |
| 164 | 眼皮膚白皮症 | 3 |
| 274 | 骨形成不全症 | 1 |
| 284 | ダイアモンド・ブラックファン貧血 | 6 |
| 285 | ファンコニ貧血 | 10 |
| 326 | 大理石骨病 | 4 |
| 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 | NCT03513328 (ClinicalTrials.gov) | June 15, 2018 | 19/4/2018 | 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 | 3 Months | 39 Years | All | 40 | Phase 1;Phase 2 | United States |
| 2 | EUCTR2017-002430-23-IT (EUCTR) | 14/03/2018 | 09/03/2020 | Gene therapy study with autologous hemapoietic stem cells for patients affected by MPSIH | A phase I/II study evaluating safety and efficacy of autologous hematopoietic stem and progenitor cells genetically modified with IDUA lentiviral vector encoding for the human a-L-iduronidase gene for the treatment of patients affected by Mucopolysaccharidosis Type I, Hurler variant - TigetT10_MPS1H | Mucopolysaccharidosis type I Hurler MedDRA version: 20.1;Level: PT;Classification code 10056886;Term: Mucopolysaccharidosis I;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Other descriptive name: Cellule CD34+ autologhe trasdotte con IDUA LV codificante per il cDNA dell'alfa-L-iduronidasi Trade Name: BUSILVEX - 6 MG/ML - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO - 10 ML 8 FLACONCINI INN or Proposed INN: BUSULFANO Other descriptive name: BUSULFANO Trade Name: FLUDARABINA ACCORD - 25 MG/ML CONCENTRATO PER SOLUZIONE INIETTABILE O PER INFUSIONE 5 FLACONCINI IN VETRO DA 2 ML INN or Proposed INN: FLUDARABINA FOSFATO Other descriptive name: FLUDARABINA FOSFATO Trade Name: MABTHERA - 2 FIALE 100 MG 10 ML INN or Proposed INN: RITUXIMAB Other descriptive name: RITUXIMAB Trade Name: MYELOSTIM - 34 1 FLACONCINO LIOFILIZZATO 33.6 MIU + SIRINGA PRERIEMPITA SOLVENTE 1 ML INN or Proposed INN: LENOGRASTIM Other descriptive name: LENOGRASTIM Trade Name: MOZOBIL - 20 MG/ML - SOLUZIONE INIETTABILE - USO SOTTOCUTANEO - F | OSPEDALE SAN RAFFAELE | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 6 | Phase 1 | Italy | ||
| 3 | NCT02171104 (ClinicalTrials.gov) | July 10, 2014 | 20/6/2014 | MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis | MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG | Mucopolysaccharidosis Disorders;Hurler Syndrome;Hunter Syndrome;Maroteaux Lamy Syndrome;Sly Syndrome;Alpha-Mannosidosis;Fucosidosis;Aspartylglucosaminuria;Glycoprotein Metabolic Disorders;Sphingolipidoses;Recessive Leukodystrophies;Globoid Cell Leukodystrophy;Metachromatic Leukodystrophy;Niemann-Pick B;Niemann-Pick C Subtype 2;Sphingomyelin Deficiency;Peroxisomal Disorders;Adrenoleukodystrophy With Cerebral Involvement;Zellweger Syndrome;Neonatal Adrenoleukodystrophy;Infantile Refsum Disease;Acyl-CoA Oxidase Deficiency;D-Bifunctional Enzyme Deficiency;Multifunctional Enzyme Deficiency;Alpha-methylacyl-CoA Racmase Deficiency;Mitochondrial Neurogastrointestingal Encephalopathy;Severe Osteopetrosis;Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation);Inherited Metabolic Disorders | Biological: Stem Cell Transplantation;Drug: IMD Preparative Regimen;Drug: Osteopetrosis Only Preparative Regimen;Drug: Osteopetrosis Haploidentical Only Preparative Regimen;Drug: cALD SR-A (Standard-Risk, Regimen A);Drug: cALD SR-B (Standard-Risk, Regimen B);Drug: cALD HR-D (High-Risk, Regimen C);Drug: cALD HR-D (High-Risk, Regimen D) | Masonic Cancer Center, University of Minnesota | NULL | Recruiting | N/A | 55 Years | All | 100 | Phase 2 | United States |
| 4 | EUCTR2009-017349-77-IT (EUCTR) | 15/03/2010 | 26/04/2010 | A Phase I/II clinical trial of hematopoietic stem cell gene therapy for the treatment of Metachromatic Leukodystrophy - TIGET-MLD | A Phase I/II clinical trial of hematopoietic stem cell gene therapy for the treatment of Metachromatic Leukodystrophy - TIGET-MLD | metachromatic leukodystrophy MedDRA version: 9.1;Level: PT;Classification code 10024381 | Product Name: autologous CD34+ cells transduced with a lentiviral vector encoding the ARSA cDNA Trade Name: BUSILVEX INN or Proposed INN: Busulfan | FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Phase 1;Phase 2 | Italy | |||
| 5 | NCT01043640 (ClinicalTrials.gov) | December 2009 | 5/1/2010 | Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders | Allogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic Disorders | Mucopolysaccharidosis;Hurler Syndrome;Hunter Syndrome;Maroteaux-Lamy Syndrome;Sly Syndrome;Alpha Mannosidosis;Fucosidosis;Aspartylglucosaminuria;Adrenoleukodystrophy (ALD);Krabbe Disease;Metachromatic Leukodystrophy (MLD);Sphingolipidoses;Peroxisomal Disorders | Drug: Campath-1H;Drug: Cyclophosphamide;Drug: Busulfan;Procedure: Allogeneic stem cell transplantation;Drug: Cyclosporine A;Drug: Mycophenolate Mofetil | Masonic Cancer Center, University of Minnesota | NULL | Completed | N/A | 21 Years | All | 46 | Phase 2 | United States |
| 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 |
| 6 | NCT00668564 (ClinicalTrials.gov) | March 2008 | 25/4/2008 | Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism | Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Hematopoietic Cell Transplantation | Hurler's Syndrome;Maroteaux-Lamy Syndrome;Sly Syndrome;Alpha Mannosidosis;Fucosidosis;Aspartylglucosaminuria;Sphingolipidoses;Krabbe Disease;Wolman's Disease;Niemann-Pick Disease Type B;Niemann-Pick Disease, Type C | Procedure: Stem Cell Transplantation;Drug: Cyclophosphamide;Drug: Campath-1H;Drug: Busulfan | Masonic Cancer Center, University of Minnesota | NULL | Terminated | N/A | 21 Years | All | 18 | Phase 2 | United States |
| 7 | NCT00176917 (ClinicalTrials.gov) | May 1999 | 12/9/2005 | Stem Cell Transplantation for Hurler | Hematopoietic Stem Cell Transplantation for Hurler Syndrome, Maroteaux Lamy Syndrome (MPS VI), and Alpha Mannosidase Deficiency (Mannosidosis) | Mucopolysaccharidosis I;Mucopolysaccharidosis VI;Mannosidosis;Mucolipidosis Type II (I-cell Disease) | Procedure: Stem Cell Transplant;Drug: Busulfan, Cyclophosphamide, ATG | Masonic Cancer Center, University of Minnesota | NULL | Completed | N/A | N/A | All | 41 | Phase 2 | United States |
| 8 | NCT00176904 (ClinicalTrials.gov) | January 1995 | 12/9/2005 | Stem Cell Transplant for Inborn Errors of Metabolism | Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Bone Marrow Transplantation | Adrenoleukodystrophy;Metachromatic Leukodystrophy;Globoid Cell Leukodystrophy;Gaucher's Disease;Fucosidosis;Wolman Disease;Niemann-Pick Disease;Batten Disease;GM1 Gangliosidosis;Tay Sachs Disease;Sandhoff Disease | Procedure: Stem Cell Transplant;Drug: Busulfan, Cyclophosphamide, Antithymocyte Globulin | Masonic Cancer Center, University of Minnesota | NULL | Completed | N/A | N/A | All | 135 | Phase 2;Phase 3 | United States |
| 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 | EUCTR2018-001145-14-DE (EUCTR) | 23/01/2020 | 27/11/2018 | Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adrenoleukodystrophy (CALD) | A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects =17 Years of Age With Cerebral Adrenoleukodystrophy (CALD) | Cerebral Adrenoleukodystrophy (CALD) MedDRA version: 20.0;Level: PT;Classification code 10051260;Term: Adrenoleukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18] | Product Name: Lenti-D Drug Product INN or Proposed INN: elivaldogene autotemcel Other descriptive name: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA INN or Proposed INN: plerixafor Other descriptive name: PLERIXAFOR | bluebird bio, Inc. | NULL | Not Recruiting | Female: no Male: yes | 35 | Phase 3 | United States;France;Netherlands;Germany;United Kingdom;Italy | ||
| 2 | EUCTR2018-001145-14-NL (EUCTR) | 24/06/2019 | 25/02/2019 | Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adrenoleukodystrophy (CALD) | A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects =17 Years of Age With Cerebral Adrenoleukodystrophy (CALD) | Cerebral Adrenoleukodystrophy (CALD) MedDRA version: 20.0;Level: PT;Classification code 10051260;Term: Adrenoleukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Lenti-D Drug Product Product Code: Not Applicable INN or Proposed INN: elivaldogene autotemcel Other descriptive name: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA | bluebird bio, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 35 | Phase 3 | France;United States;Germany;Netherlands;Italy;United Kingdom | ||
| 3 | EUCTR2018-001145-14-GB (EUCTR) | 17/04/2019 | 23/10/2018 | Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adrenoleukodystrophy (CALD). | A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects =17 Years of Age With Cerebral Adrenoleukodystrophy (CALD) | Cerebral Adrenoleukodystrophy (CALD) MedDRA version: 20.0;Level: PT;Classification code 10051260;Term: Adrenoleukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Lenti-D Drug Product INN or Proposed INN: elivaldogene autotemcel Other descriptive name: Autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA | bluebird bio, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 35 | Phase 3 | United States;France;Netherlands;Germany;Italy;United Kingdom | ||
| 4 | EUCTR2018-001145-14-FR (EUCTR) | 27/03/2019 | 23/10/2018 | Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adrenoleukodystrophy. | A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects =17 Years of Age With Cerebral Adrenoleukodystrophy (CALD) | Cerebral Adrenoleukodystrophy (CALD) MedDRA version: 20.0;Level: PT;Classification code 10051260;Term: Adrenoleukodystrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | bluebird bio, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 20 | Phase 3 | United States;France;Netherlands;Germany;Italy;United Kingdom | |||
| 5 | NCT03852498 (ClinicalTrials.gov) | January 24, 2019 | 13/2/2019 | Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adrenoleukodystrophy (CALD) | A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfan and Fludarabine in Subjects =17 Years of Age With Cerebral Adrenoleukodystrophy (CALD) | Cerebral Adrenoleukodystrophy (CALD) | Drug: Lenti-D; elivaldogene autotemcel | bluebird bio | NULL | Recruiting | N/A | 17 Years | Male | 35 | Phase 3 | United States;France;Germany;Italy;Netherlands;United Kingdom |
| 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 |
| 6 | NCT03513328 (ClinicalTrials.gov) | June 15, 2018 | 19/4/2018 | 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 | 3 Months | 39 Years | All | 40 | Phase 1;Phase 2 | United States |
| 7 | NCT02171104 (ClinicalTrials.gov) | July 10, 2014 | 20/6/2014 | MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis | MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG | Mucopolysaccharidosis Disorders;Hurler Syndrome;Hunter Syndrome;Maroteaux Lamy Syndrome;Sly Syndrome;Alpha-Mannosidosis;Fucosidosis;Aspartylglucosaminuria;Glycoprotein Metabolic Disorders;Sphingolipidoses;Recessive Leukodystrophies;Globoid Cell Leukodystrophy;Metachromatic Leukodystrophy;Niemann-Pick B;Niemann-Pick C Subtype 2;Sphingomyelin Deficiency;Peroxisomal Disorders;Adrenoleukodystrophy With Cerebral Involvement;Zellweger Syndrome;Neonatal Adrenoleukodystrophy;Infantile Refsum Disease;Acyl-CoA Oxidase Deficiency;D-Bifunctional Enzyme Deficiency;Multifunctional Enzyme Deficiency;Alpha-methylacyl-CoA Racmase Deficiency;Mitochondrial Neurogastrointestingal Encephalopathy;Severe Osteopetrosis;Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation);Inherited Metabolic Disorders | Biological: Stem Cell Transplantation;Drug: IMD Preparative Regimen;Drug: Osteopetrosis Only Preparative Regimen;Drug: Osteopetrosis Haploidentical Only Preparative Regimen;Drug: cALD SR-A (Standard-Risk, Regimen A);Drug: cALD SR-B (Standard-Risk, Regimen B);Drug: cALD HR-D (High-Risk, Regimen C);Drug: cALD HR-D (High-Risk, Regimen D) | Masonic Cancer Center, University of Minnesota | NULL | Recruiting | N/A | 55 Years | All | 100 | Phase 2 | United States |
| 8 | NCT01043640 (ClinicalTrials.gov) | December 2009 | 5/1/2010 | Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders | Allogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic Disorders | Mucopolysaccharidosis;Hurler Syndrome;Hunter Syndrome;Maroteaux-Lamy Syndrome;Sly Syndrome;Alpha Mannosidosis;Fucosidosis;Aspartylglucosaminuria;Adrenoleukodystrophy (ALD);Krabbe Disease;Metachromatic Leukodystrophy (MLD);Sphingolipidoses;Peroxisomal Disorders | Drug: Campath-1H;Drug: Cyclophosphamide;Drug: Busulfan;Procedure: Allogeneic stem cell transplantation;Drug: Cyclosporine A;Drug: Mycophenolate Mofetil | Masonic Cancer Center, University of Minnesota | NULL | Completed | N/A | 21 Years | All | 46 | Phase 2 | United States |
| 9 | NCT00176904 (ClinicalTrials.gov) | January 1995 | 12/9/2005 | Stem Cell Transplant for Inborn Errors of Metabolism | Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Bone Marrow Transplantation | Adrenoleukodystrophy;Metachromatic Leukodystrophy;Globoid Cell Leukodystrophy;Gaucher's Disease;Fucosidosis;Wolman Disease;Niemann-Pick Disease;Batten Disease;GM1 Gangliosidosis;Tay Sachs Disease;Sandhoff Disease | Procedure: Stem Cell Transplant;Drug: Busulfan, Cyclophosphamide, Antithymocyte Globulin | Masonic Cancer Center, University of Minnesota | NULL | Completed | N/A | N/A | All | 135 | Phase 2;Phase 3 | United States |
| 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 | NCT00536601 (ClinicalTrials.gov) | June 29, 2006 | 27/9/2007 | High-Dose Chemotherapy With or Without Total-Body Irradiation Followed by Autologous Stem Cell Transplant in Treating Patients With Hematologic Cancer or Solid Tumors | Autologous Blood and Marrow Transplantation for Hematologic Malignancies and Selected Solid Tumors | 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 Nasal Type Extranodal NK/T-cell Lymphoma;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Burkitt Lymphoma;Childhood Diffuse Large Cell Lymphoma;Childhood Immunoblastic Large Cell Lymphoma;Childhood Nasal Type Extranodal NK/T-cell Lymphoma;Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor (PNET);Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Hepatosplenic T-cell Lymphoma;Intraocular Lymphoma;Nodal Marginal Zone B-cell Lymphoma;Peripheral T-cell Lymphoma;Plasma Cell Neoplasm;Primary Systemic Amyloidosis;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 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 Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor;Recurrent Grade 1 Follicular Lymphoma;Recurrent Grade 2 Follicular Lymphoma;Recurrent Grade 3 Follicular Lymphoma;Recurrent Malignant Testicular Germ Cell Tumor;Recurrent Mantle Cell Lymphoma;Recurrent Marginal Zone Lymphoma;Recurrent Neuroblastoma;Recurrent Small Lymphocytic Lymphoma;Recurrent/Refractory Childhood Hodgkin Lymphoma;Refractory Chronic Lymphocytic Leukemia;Refractory Multiple Myeloma;Regional Neuroblastoma;Splenic Marginal Zone Lymphoma;Testicular Lymphoma;Unspecified Adult Solid Tumor, Protocol Specific;Unspecified Childhood Solid Tumor, Protocol Specific;Waldenström Macroglobulinemia | Drug: etoposide;Drug: cyclophosphamide;Drug: carmustine;Drug: melphalan;Drug: busulfan;Drug: carboplatin;Drug: thiotepa;Radiation: total-body irradiation;Procedure: autologous hematopoietic stem cell transplantation;Procedure: autologous-autologous tandem hematopoietic stem cell transplantation | Roswell Park Cancer Institute | National Cancer Institute (NCI) | Completed | 4 Years | N/A | All | 174 | N/A | United States |
| 2 | NCT00007995 (ClinicalTrials.gov) | July 1999 | 6/1/2001 | Chemotherapy Plus Peripheral Stem Cell Transplant in Treating Patients Who Have Multiple Myeloma or Primary Systemic Amyloidosis | Phase 2 Study Of High Dose Chemotherapy Followed By Autologous Hematopoietic Stem Cell Support In Patients With Multiple Myeloma And Primary Light Chain Amyloidosis | Multiple Myeloma and Plasma Cell Neoplasm | Biological: filgrastim;Biological: recombinant interferon alfa;Biological: sargramostim;Drug: busulfan;Drug: cyclophosphamide;Drug: melphalan;Procedure: autologous bone marrow transplantation;Procedure: bone marrow ablation with stem cell support;Procedure: peripheral blood stem cell transplantation | Herbert Irving Comprehensive Cancer Center | National Cancer Institute (NCI) | Completed | 18 Years | N/A | Both | 75 | Phase 2 | United States |
| 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 | NCT02582775 (ClinicalTrials.gov) | March 2016 | 16/10/2015 | MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs | MT2015-20: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Cell Transplantation and Serial Donor Mesenchymal Cell Infusions | Epidermolysis Bullosa | Drug: Thymoglobulin;Drug: Cyclophosphamide;Drug: Fludarabine;Radiation: Total Body Irradiation;Procedure: Bone marrow infusion;Drug: Tacrolimus;Drug: Mycophenolate Mofetil;Biological: Donor mesenchymal stem cell infusions;Drug: Busulfan | Masonic Cancer Center, University of Minnesota | NULL | Recruiting | N/A | 25 Years | All | 84 | Phase 2 | United States |
| 2 | NCT00478244 (ClinicalTrials.gov) | April 2007 | 23/5/2007 | Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa | Allogeneic Hematopoietic Cell Transplantation to Correct the Biochemical Defect and Create Tolerance to Donor Tissue in Subjects With Epidermolysis Bullosa | Epidermolysis Bullosa | Drug: busulfan;Drug: cyclophosphamide;Drug: fludarabine phosphate;Procedure: hematopoietic bone marrow transplantation | Masonic Cancer Center, University of Minnesota | NULL | Terminated | N/A | 25 Years | All | 7 | N/A | United States |
| 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 | NCT00684255 (ClinicalTrials.gov) | August 2007 | 22/5/2008 | Reduced Intensity Transplant in Medically Refractory Systemic Lupus Erythematosus (SLE) and Systemic Sclerosis (SSc) | Reduced Intensity Conditioning And Allogeneic Stem Cell Transplantation in Patients With Medically Refractory Systemic Lupus Erythematosus and Medically Refractory Systemic Sclerosis (SSc) | Systemic Lupus Erythematosus;Systemic Sclerosis | Procedure: Reduced Intensity Allogeneic Transplant;Drug: Fludarabine;Drug: Busulfan;Drug: Campath | New York Medical College | NULL | Terminated | 7 Years | 50 Years | All | 1 | Phase 1 | United States |
| 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 | NCT00684255 (ClinicalTrials.gov) | August 2007 | 22/5/2008 | Reduced Intensity Transplant in Medically Refractory Systemic Lupus Erythematosus (SLE) and Systemic Sclerosis (SSc) | Reduced Intensity Conditioning And Allogeneic Stem Cell Transplantation in Patients With Medically Refractory Systemic Lupus Erythematosus and Medically Refractory Systemic Sclerosis (SSc) | Systemic Lupus Erythematosus;Systemic Sclerosis | Procedure: Reduced Intensity Allogeneic Transplant;Drug: Fludarabine;Drug: Busulfan;Drug: Campath | New York Medical College | NULL | Terminated | 7 Years | 50 Years | All | 1 | Phase 1 | United States |
| 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 | NCT03579875 (ClinicalTrials.gov) | November 13, 2018 | 25/5/2018 | T Cell Receptor a/ß TCD HCT in Patients With Fanconi Anemia | T Cell Receptor Alpha/Beta T Cell Depleted (a/ß TCD) Hematopoietic Cell Transplantation in Patients With Fanconi Anemia (FA) | Fanconi Anemia;Severe Aplastic Anemia;Myelodysplastic Syndromes | Drug: Total Body Irradiation (TBI) (Plan 1);Drug: Cyclophosphamide (CY) (Plan 1);Drug: Fludarabine (FLU);Drug: Methylprednisolone (MP);Device: Donor mobilized PBSC infusion;Drug: G-CSF;Drug: Cyclophosphamide (CY) (Plan 2);Drug: Rituximab;Drug: Busulfan | Masonic Cancer Center, University of Minnesota | NULL | Recruiting | N/A | 65 Years | All | 48 | Phase 2 | United States |
| 2 | ChiCTR1900023509 | 2017-01-01 | 2019-05-31 | Allogeneic Hematopoietic Stem Cell Transplantation with Risk-adapted Conditioning Regimens for Children with Aplastic Anemia or Refractory Cytopenia of Childhood: a Single-Center Prospective Trial | Allogeneic Hematopoietic Stem Cell Transplantation with Risk-adapted Conditioning Regimens for Children with Aplastic Anemia or Refractory Cytopenia of Childhood: a Single-Center Prospective Trial | aplastic anemia,Refractory Cytopenia of Childhood | 1:Fludarabine150 mg/m2 +Cyclophosphamide200 mg/kg+ Thymoglobuline10mg/kg;2:Fludarabine200 mg/m2 +Cyclophosphamide120 mg/kg+ Thymoglobuline10mg/kg+300cGy TBI;3:Fludarabine200 mg/m2 +Cyclophosphamide120 mg/kg+ Thymoglobuline10mg/kg+ Busulfan 8mg/kg; | Shanghai Children's Medical Center affiliated to Shanghai Jiao Tong University School of Medicine | NULL | Recruiting | 18 | Both | 1:50;2:50;3:50; | China | ||
| 3 | NCT03016806 (ClinicalTrials.gov) | June 2015 | 26/12/2016 | Umbilical Cord Blood Transplantation From Unrelated Donors | Umbilical Cord Blood Transplantation From Unrelated Donors | Acute Leukemia;Immune Deficiency Disorder;Congenital Hematological Disorder;Metabolism Disorder;Aplastic Anemia;Myelodysplastic Syndromes;Chronic Leukemia;Lymphoma;Multiple Myeloma;Solid Tumor | Radiation: Total Body Irradiation 1200 cGy;Radiation: Total Body Irradiation 200 cGy;Drug: Cyclophosphamide;Drug: Mesna;Procedure: Cord Blood Infusion;Drug: Busulfan;Drug: Fludarabine;Drug: Melphalan | University of Rochester | NULL | Recruiting | 2 Months | 75 Years | All | 30 | Phase 1 | United States |
| 4 | NCT01500161 (ClinicalTrials.gov) | November 2011 | 18/11/2011 | Pooled Unrelated Donor Umbilical Cord Blood Transplant For Hematologic Malignancy Needing Allogeneic Stem Cell Transplant Without Related HLA-Match | A Phase II Study Of Pooled Unrelated Donor Umbilical Cord Blood (UCB) Transplant For Patients With Hematologic Malignancies Needing Allogeneic Stem Cell Transplant But Do Not Have A Related HLA-Matched Donor | Acute Myelogenous Leukemia;Acute Lymphocytic Leukemia;Chronic Lymphocytic Leukemia;Chronic Myelogenous Leukemia;Hodgkins Disease;Non-Hodgkins Lymphoma;Aplastic Anemia;Multiple Myeloma;Myelodysplastic Syndrome | Drug: Busulfan;Drug: Clofarabine;Drug: Fludarabine;Drug: Melphalan;Drug: Carmustine;Drug: Etoposide;Drug: Cytarabine | Texas Oncology Cancer Center | NULL | Terminated | 18 Years | 65 Years | Both | 1 | Phase 2 | United States |
| 5 | NCT01384513 (ClinicalTrials.gov) | August 4, 2011 | 27/6/2011 | A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological Malignancies | A Two Step Approach to Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation for Patients With Hematologic Malignancies | 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 Nasal Type Extranodal NK/T-cell Lymphoma;Aplastic Anemia;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Myelodysplastic Syndromes;Chronic Eosinophilic Leukemia;Chronic Myelomonocytic Leukemia;Chronic Neutrophilic Leukemia;Essential Thrombocythemia;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Juvenile Myelomonocytic Leukemia;Mastocytosis;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Nodal Marginal Zone B-cell Lymphoma;Polycythemia Vera;Previously Treated Myelodysplastic Syndromes;Primary Myelofibrosis;Recurrent Adult Acute Lymphoblastic Leukemia;Recurrent Adult Acute Myeloid Leukemia;Recurrent Adult Diffuse Small Cleaved Cell Lymphoma;Recurrent Adult Grade III Lymphomatoid Granulomatosis;Recurrent Adult Hodgkin Lymphoma;Recurrent Childhood Acute Lymphoblastic Leukemia;Recurrent Childhood Acute Myeloid Leukemia;Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma;Recurrent Grade 1 Follicular Lymphoma;Recurrent Grade 2 Follicular Lymphoma;Recurrent Marginal Zone Lymphoma;Recurrent Mycosis Fungoides/Sezary Syndrome;Recurrent Small Lymphocytic Lymphoma;Recurrent/Refractory Childhood Hodgkin Lymphoma;Refractory Anemia;Refractory Anemia With Ringed Sideroblasts;Refractory Hairy Cell Leukemia;Refractory Multiple Myeloma;Secondary Myelodysplastic Syndromes;Splenic Marginal Zone Lymphoma;T-cell Large Granular Lymphocyte Leukemia;Waldenström Macroglobulinemia | Drug: Fludarabine;Drug: Busulfan;Radiation: Total Body Irradiation (TBI);Biological: Donor Lymphocyte Infusion (DLI);Drug: Cyclophosphamide (CY);Drug: Tacrolimus;Drug: Mycophenolate mofetil;Device: Allogeneic hematopoietic stem cell transplantation;Procedure: Peripheral blood stem cell transplantation (PBSCT) | Sidney Kimmel Cancer Center at Thomas Jefferson University | NULL | Active, not recruiting | 18 Years | N/A | All | 40 | Phase 2 | United States |
| 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 |
| 6 | NCT00987480 (ClinicalTrials.gov) | September 25, 2009 | 30/9/2009 | Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine | A Multicenter Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine | Aplastic Anemia;Leukemia;Myelodysplastic Syndrome | Drug: Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine.;Device: CliniMACS device | Memorial Sloan Kettering Cancer Center | Boston Children's Hospital;Children's Hospital Medical Center, Cincinnati;Children's Hospital and Health System Foundation, Wisconsin;Rockefeller University;Fred Hutchinson Cancer Research Center | Completed | N/A | N/A | All | 45 | Phase 2 | United States |
| 7 | EUCTR2007-001657-26-GB (EUCTR) | 29/02/2008 | 07/11/2007 | Single Centre Phase II Pilot study of Unrelated Cord Blood Transplantation in Patients with Poor Risk Haematological Malignancies. | Single Centre Phase II Pilot study of Unrelated Cord Blood Transplantation in Patients with Poor Risk Haematological Malignancies. | 1.Acute, chronic leukaemia or myelodysplastic syndrome for which allogeneic transplantation is considered as the best treatment option. 2.Acute lymphoblastic leukaemia (ALL)3.Non-Hodgkin’s lymphoma4.Hodgkin’s disease5.Chronic lymphocytic leukaemia. 6.Acquired bone marrow failure syndromes7.Other haematological malignancies for which UD bone marrow transplantation is indicated MedDRA version: 9.1;Level: LLT;Classification code 10000880;Term: Acute myeloid leukaemia MedDRA version: 9.1;Classification code 10028533;Term: Myelodysplastic syndrome MedDRA version: 9.1;Classification code 10009013;Term: Chronic myeloid leukaemia MedDRA version: 9.1;Classification code 10000844;Term: Acute lymphoblastic leukaemia MedDRA version: 9.1;Classification code 10020328;Term: Hodgkin's lymphoma MedDRA version: 9.1;Classification code 10029593;Term: Non-Hodgkin's lymphoma NOS MedDRA version: 9.1;Classification code 10003892;Term: B-cell chronic lymphocytic leukaemia/prolymphocytic leukaemia/small lymphocytic lymphoma MedDRA version: 9.1;Classification code 10002968;Term: Aplastic anaemia, unspecified | Trade Name: Fludarabine INN or Proposed INN: FLUDARABINE PHOSPHATE Trade Name: Busilvex INN or Proposed INN: BUSULFAN Trade Name: Thymoglobulin Other descriptive name: RABBIT HUMAN T LYMPHOCYTE IMMUNOGLOBULIN Trade Name: Thiotepa INN or Proposed INN: THIOTEPA Trade Name: Cyclophosphamide Other descriptive name: CYCLOPHOSPHAMIDE MONOHYDRATE Trade Name: Alkeran INN or Proposed INN: MELPHALAN | King's College Hospital NHS Foundation Trust | NULL | Not Recruiting | Female: yes Male: yes | 27 | Phase 2 | United Kingdom | ||
| 8 | NCT00295997 (ClinicalTrials.gov) | May 2005 | 23/2/2006 | Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer, Metastatic Kidney Cancer, or Aplastic Anemia | Non-myeloablative Allogeneic Stem Cell Transplantation With Match Unrelated Donors for Treatment of Hematologic Malignancies and Renal Cell Carcinoma and Aplastic Anemia | Chronic Myeloproliferative Disorders;Kidney Cancer;Leukemia;Lymphoma;Multiple Myeloma and Plasma Cell Neoplasm;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative Neoplasms | Biological: anti-thymocyte globulin;Biological: filgrastim;Biological: graft-versus-tumor induction therapy;Biological: therapeutic allogeneic lymphocytes;Drug: busulfan;Drug: cyclophosphamide;Drug: fludarabine phosphate;Drug: methotrexate;Drug: mycophenolate mofetil;Drug: tacrolimus;Procedure: allogeneic bone marrow transplantation;Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantation | University of California, San Francisco | National Cancer Institute (NCI) | Active, not recruiting | N/A | 74 Years | Both | 35 | N/A | United States |
| 9 | NCT00516152 (ClinicalTrials.gov) | November 2002 | 13/8/2007 | Phase II Study Evaluating Busulfan and Fludarabine as Preparative Therapy in Adults With Hematopoietic Disorders Undergoing MUD SCT | Phase II Study Evaluating Busulfan and Fludarabine as Preparative Therapy in Adults With Hematopoietic Disorders Undergoing Matched Unrelated Donor Stem Cell Transplantation | Chronic Myeloid Leukemia;Acute Myelogenous Leukemia;Myelodysplasia;Acute Lymphocytic Leukemia;Severe Aplastic Anemia;Non-Hodgkin's Lymphoma;Lymphoproliferative Disease;Multiple Myeloma;Advanced Myeloproliferative Disease | Drug: Busulfan/Fludarabine phosphate/Tacrolimus/Methotrexate/G-CSF | University of California, San Francisco | NULL | Completed | 15 Years | 61 Years | Both | 36 | Phase 2 | United States |
| 10 | NCT01499147 (ClinicalTrials.gov) | February 2000 | 23/11/2011 | Fludarabine Based Conditioning for Allogeneic Transplantation for Advanced Hematologic Malignancies | Fludarabine Based Conditioning for Allogeneic Transplantation for Advanced Hematologic Malignancies | Acute Myeloid Leukemia;Acute Leukemia;Chronic Myelogenous Leukemia;Malignant Lymphoma;Hodgkin's Disease;Multiple Myeloma;Lymphocytic Leukemia;Myeloproliferative Disorder;Polycythemia Vera;Myelofibrosis;Aplastic Anemia | Drug: fludarabine/busulfan;Drug: fludarabine/ melphalan;Drug: ATG | University of Illinois at Chicago | NULL | Completed | 10 Years | 65 Years | All | 100 | N/A | United States |
| 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 |
| 11 | NCT00003816 (ClinicalTrials.gov) | October 19, 1998 | 1/11/1999 | Combination Chemotherapy and Donor Stem Cell Transplant in Treating Patients With Aplastic Anemia or Hematologic Cancer | Allogeneic Blood or Marrow Transplantation for Hematologic Malignancy and Aplastic Anemia | Chronic Myeloproliferative Disorders;Leukemia;Lymphoma;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative Diseases;Nonmalignant Neoplasm;Unspecified Adult Solid Tumor, Protocol Specific;Unspecified Childhood Solid Tumor, Protocol Specific | Biological: anti-thymocyte globulin;Drug: busulfan;Drug: carboplatin;Drug: cyclophosphamide;Drug: etoposide;Drug: fludarabine phosphate;Drug: melphalan;Drug: thiotepa;Radiation: total-body irradiation | Roswell Park Cancer Institute | NULL | Completed | 4 Years | 70 Years | All | 362 | Phase 2;Phase 3 | United States |
| 12 | NCT00003336 (ClinicalTrials.gov) | January 1998 | 1/11/1999 | Pilot Study Of Unrelated UCB Transplant for Non-Malignant Hematologic Conditions | A Pilot Study of Unrelated Umbilical Cord Blood Transplantation in Patients With Severe Aplastic Anemia, Inborn Errors in Metabolism, or Inherited Hematologic Stem Cell Disorders | Leukemia;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative Diseases | Biological: anti-thymocyte globulin;Drug: busulfan;Drug: cyclophosphamide;Drug: melphalan;Drug: methylprednisolone;Procedure: bone marrow ablation with stem cell support;Procedure: umbilical cord blood transplantation;Radiation: radiation therapy | Case Comprehensive Cancer Center | National Cancer Institute (NCI) | Completed | N/A | 55 Years | Both | 6 | Phase 2 | United States |
| 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 | NCT04370795 (ClinicalTrials.gov) | December 17, 2020 | 30/4/2020 | Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus | Matched Related and Unrelated Donor Stem Cell Transplantation for Severe Combined Immune Deficiency (SCID): Busulfan-based Conditioning With h-ATG, Radiation, and Sirolimus | Severe Combined Immunodeficiency (SCID) | Drug: Sirolimus;Drug: Busulfan;Drug: Horse -Anti-thymocyte;Drug: G-CSF;Radiation: Total Body Irradiation (TBI) | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Enrolling by invitation | 3 Years | 40 Years | All | 30 | Phase 1;Phase 2 | United States |
| 2 | NCT03597594 (ClinicalTrials.gov) | December 2020 | 19/6/2018 | Haplocompatible Transplant Using TCRa/ß Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID) | Haplocompatible Transplant Using TCRa/ß Depletion Followed by CD45RA-Depleted Donor Lymphocyte Infusions for Severe Combined Immunodeficiency (SCID) | Severe Combined Immunodeficiency | Drug: Anti-thymocyte globulin (rabbit);Drug: Busulfan;Drug: Fludarabine;Drug: Thiotepa;Device: CliniMACS;Other: Donor Lymphocyte Infusion | St. Jude Children's Research Hospital | NULL | Recruiting | 2 Months | N/A | All | 42 | Phase 1;Phase 2 | United States |
| 3 | NCT04339777 (ClinicalTrials.gov) | September 22, 2020 | 8/4/2020 | Allogeneic Hematopoietic Stem Cell Transplant for People With Primary Immunodeficiency Diseases | A Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immunodeficiency Diseases | Lymphoproliferative Disorders;Autoimmune Lymphoproliferative;Immune System Diseases;Common Variable Immunodeficiency;Primary T-cell Immunodeficiency Disorders | Drug: Busulfan test dose;Drug: Fludarabine;Drug: Busulfan;Drug: Alemtuzumab;Radiation: Total body Irradiation;Procedure: Allogeneic HSCT;Drug: Tacrolimus (Tacro);Drug: Mycophenolate mofetil (MMF);Drug: Cyclophosphamide (Cytoxan) | National Cancer Institute (NCI) | NULL | Recruiting | 4 Years | 69 Years | All | 56 | Phase 2 | United States |
| 4 | NCT03910452 (ClinicalTrials.gov) | October 28, 2019 | 9/4/2019 | Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide | Haploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide | Chronic Granulomatous Disease | Drug: Busulfan;Drug: Alemtuzumab;Drug: Cyclophosphamide;Drug: Sirolimus;Radiation: Total Body Irradiation;Biological: Allogeneic peripheral blood stem cell | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Recruiting | 4 Years | 65 Years | All | 30 | Early Phase 1 | United States |
| 5 | EUCTR2018-003842-18-IT (EUCTR) | 08/01/2019 | 19/11/2018 | Gene therapy study using a frozen formulation of OTL-103 in patients with Wiskott-Aldrich Syndrome (WAS) | A Single Arm, Open Label Clinical Study of Haematopoietic Stem Cell Gene Therapy with Cryopreserved Autologous CD34+ Cells Transduced with Lentiviral Vector encoding WAS cDNA in Subjects with Wiskott-Aldrich Syndrome (WAS). - Clinical study using cryopreserved OTL-103 for treatment of WAS. | Wiskott-Aldrich Syndrome MedDRA version: 20.0;Level: PT;Classification code 10061598;Term: Immunodeficiency;System Organ Class: 10021428 - Immune system disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: OTL-103 Dispersion for Infusion Product Code: OTL-103 INN or Proposed INN: Other hematological Agents Other descriptive name: Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with lentiviral vector that encodes for the human Wiskott Aldrich Syndrome (WAS) cDNA sequence Trade Name: Busilvex INN or Proposed INN: BUSULFAN Other descriptive name: NA Trade Name: Fludarabina Accord INN or Proposed INN: FLUDARABINE Other descriptive name: NA Trade Name: MabThera INN or Proposed INN: RITUXIMAB Other descriptive name: NA Trade Name: Mozobil, INN or Proposed INN: plerixafor Other descriptive name: PLERIXAFOR Trade Name: MYELOSTIM Product Name: granulocyte colony stimulating factor (G-CSF) INN or Proposed INN: | Orchard Therapeutics Ltd. | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 6 | Phase 3 | Italy | ||
| 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 |
| 6 | NCT03601286 (ClinicalTrials.gov) | December 21, 2018 | 22/2/2018 | Lentiviral Gene Therapy for X-linked Severe Combined Immunodeficiency | Phase I/II Study of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan | Severe Combined Immunodeficiency, X-Linked | Drug: Lentiviral vector transduced CD34+ cells | Great Ormond Street Hospital for Children NHS Foundation Trust | NULL | Recruiting | 8 Weeks | 5 Years | Male | 5 | Phase 1 | United Kingdom |
| 7 | NCT03619551 (ClinicalTrials.gov) | October 22, 2018 | 16/7/2018 | Conditioning SCID Infants Diagnosed Early | A Randomized Trial of Low Versus Moderate Exposure Busulfan for Infants With Severe Combined Immunodeficiency (SCID) Receiving TCRaß+/CD19+ Depleted Transplantation: A Phase II Study by the Primary Immune Deficiency Treatment Consortium (PIDTC) and Pediatric Blood and Marrow Transplant Consortium (PBMTC) | SCID | Drug: Busulfan;Device: Cell processing for TCRaß+/CD19+ depletion | Michael Pulsipher, MD | NULL | Recruiting | N/A | 2 Years | All | 64 | Phase 2 | United States |
| 8 | EUCTR2018-000673-68-GB (EUCTR) | 09/10/2018 | 27/07/2018 | A clinical trial to study the effects of genetically modified patients' CD34+ cells in patients with X-linked Severe Combined Immunodeficiency | Phase I/II study of lentiviral gene transfer for SCID-X1 with low dose targeted busulfan - Lentiviral gene therapy for SCID-X1 | Severe combined immunodeficiency disorder (SCID) is a heterogeneous group of inherited disorders characterized by a profound reduction or absence of T lymphocyte function, resulting in lack of both cellular and humoral immunity. The most common form of SCID is an X-linked form (SCID-X1), which accounts for 30-50% of all cases. Children with SCID lack virtually all immune protection from pathogens. They are prone to repeated and persistent infections that can be very serious or life threatening. MedDRA version: 20.0;Level: LLT;Classification code 10069566;Term: Severe combined immunodeficiency syndrome;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Great Ormond Street Hospital for Children NHS Trust | NULL | Authorised-recruitment may be ongoing or finished | Female: no Male: yes | 5 | Phase 1 | United Kingdom | |||
| 9 | NCT03513328 (ClinicalTrials.gov) | June 15, 2018 | 19/4/2018 | 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 | 3 Months | 39 Years | All | 40 | Phase 1;Phase 2 | United States |
| 10 | NCT03538899 (ClinicalTrials.gov) | May 31, 2018 | 3/5/2018 | Autologous Gene Therapy for Artemis-Deficient SCID | A Phase I/II Feasibility Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency (ART-SCID) Using a Self-Inactivating Lentiviral Vector (AProArt) to Transduce Autologous CD34 Hematopoietic Cells | Severe Combined Immunodeficiency | Drug: AProArt;Device: CliniMACS® CD34 Reagent System cell sorter device;Drug: Busulfan | University of California, San Francisco | NULL | Recruiting | 2 Months | N/A | All | 15 | Phase 1;Phase 2 | United States |
| 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 |
| 11 | NCT03311503 (ClinicalTrials.gov) | January 19, 2018 | 12/10/2017 | Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning | Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning | Severe Combined Immunodeficiency, X Linked;Gene Therapy | Biological: autologous CD34+ cell transduced with G2SCID vector | David Williams | NULL | Recruiting | N/A | 5 Years | Male | 10 | Phase 1;Phase 2 | United States;United Kingdom |
| 12 | JPRN-UMIN000030806 | 2018/01/17 | 15/01/2018 | A phase I/II clinical trial of hematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome | Wiskott-Aldrich syndrome | WASP cDNA-transduced autologous hematopoietic stem cells are administered to patients affected by WAS after the administration of rituximab and preconditioning chemotherapy including Fludarabine and Busulfan. 1. Rituximab (day-22) 375 mg/m2 2. Preconditioning chemotherapy Fludarabine 30mg/m2 x 2 (day-3, day-2) Busulfan cumulative target AUC 48000 ng/mL*h (day-3 to -1, every 6 hours) 3. Infusion of WASP cDNA-transduced CD34 positive HSC 5 x 10^6/kg (at least 3 x 10^6/kg) | National Center for Child Heath and Development | NULL | Recruiting | Not applicable | Not applicable | Male | 3 | Phase 1;Phase 2 | Japan | |
| 13 | NCT03765632 (ClinicalTrials.gov) | January 3, 2018 | 8/8/2018 | Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID | Efficacy and Safety of a Cryopreserved Formulation of Autologous CD34+ Haematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1a Short Form (EFS) Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency (SCID) Due to Adenosine Deaminase Deficiency | Severe Combined Immunodeficiency Due to ADA Deficiency | Genetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101);Drug: Busulfan;Drug: Peg-Ada | Great Ormond Street Hospital for Children NHS Foundation Trust | Orchard Therapeutics | Recruiting | N/A | 17 Years | All | 10 | Phase 1;Phase 2 | United Kingdom |
| 14 | JPRN-jRCTs031180398 | 30/12/2017 | 20/03/2019 | A phase II study of RIC-SCT for CGD | Reduced intensity conditioning allogeneic stem cell transplantation with dose-adjusted busulfan and anti-thymocyte globulin for chronic granulomatous disease: a multicenter phase II trial - CGD-RIST2 | Chronic granulomatous disease Primary immunodeficiency;D006105 | Conditioning regimen with targeted-busulfan and fludarabin Total body irradiation (3Gy) at day -1 Stem cell transplantation at day 0 | Kato Motohiro | NULL | Recruiting | Not applicable | < 25age old | Both | 22 | Phase 2 | None (Japan only);Japan |
| 15 | JPRN-UMIN000030647 | 2017/12/30 | 31/12/2017 | Reduced intensity conditioning allogeneic stem cell transplantation with dose-adjusted busulfan and anti-thymocyte globulin for chronic granulomatous disease: a multicenter phase II trial (CGD-RIST2) | Chronic granulomatous disease | Conditioning regimen with targeted-busulfan and fludarabin Total body irradiation (3Gy) at day -1 Stem cell transplantation at day 0 | National Center for Child Health and Development | NULL | Recruiting | Not applicable | 25years-old | Male and Female | 22 | Phase 2 | Japan | |
| 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 |
| 16 | NCT02999984 (ClinicalTrials.gov) | December 16, 2016 | 19/12/2016 | Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID | Efficacy and Safety of Cryopreserved Formulation of Autologous CD34+ Hematopoietic Stem Cells Transduced Ex Vivo With EFS Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency Due to ADA Deficiency | Severe Combined Immunodeficiency Due to ADA Deficiency | Genetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101);Drug: busulfan;Drug: PEG-ADA ERT | Orchard Therapeutics | California Institute for Regenerative Medicine (CIRM);University of California, Los Angeles | Completed | N/A | 17 Years | All | 10 | Phase 1;Phase 2 | United States |
| 17 | NCT01512888 (ClinicalTrials.gov) | August 17, 2016 | 13/1/2012 | Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants | A Pilot Feasibility Study of Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants Using a Self-Inactivating Lentiviral Vector to Transduce Autologous CD34+ Hematopoietic Cells | Severe Combined Immunodeficiency Disease, X-linked | Genetic: CL20-i4-EF1a-h?c-OPT;Drug: Busulfan;Device: CliniMacs | St. Jude Children's Research Hospital | National Heart, Lung, and Blood Institute (NHLBI);Assisi Foundation;California Institute for Regenerative Medicine (CIRM) | Recruiting | N/A | 24 Months | Male | 28 | Phase 1;Phase 2 | United States |
| 18 | JPRN-UMIN000022688 | 2016/06/10 | 13/06/2016 | A pilot study of reduced intensity conditioning allogeneic stem cell transplantation with dose-adjusted busulfan for chronic granulomatous disease | Chronic granulomatous disease | Conditioning regimen with targeted-busulfan and fludarabin Total body irradiation (3Gy) at day -1 Stem cell transplantation at day 0 | National Center for Child Health and Development | NULL | Recruiting | Not applicable | 25years-old | Male and Female | 9 | Not selected | Japan | |
| 19 | NCT02629120 (ClinicalTrials.gov) | December 17, 2015 | 10/12/2015 | High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease | High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease | Chronic Granulomatous Disease Transplant | Drug: Alentuzumab (Campath);Drug: Busulfan IV;Drug: Sirolimus;Drug: Cyclophosphamide;Radiation: Total Body Irradiation;Biological: Peripheral blood stem cells | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Recruiting | 4 Years | 65 Years | All | 50 | Phase 1;Phase 2 | United States |
| 20 | EUCTR2013-005508-33-IT (EUCTR) | 13/07/2015 | 21/05/2015 | Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseases | Clinical phase II trial to compare Treosulfan-based conditioning therapy with Busulfan-based conditioning prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients with non-malignant diseases - Treosulfan-based versus Busulfan-based conditioning in paediatric patients with non-malignant diseas | Male and female children with non-malignant diseases requiring myeloablative conditioning treatment with following allogeneic haematopoietic stem cell transplantation (allo-HSCT) – i.e. primary immunodeficiencies, inborn errors of metabolism, haemoglobinopathies and bone marrow failure syndromes. MedDRA version: 18.0;Level: HLT;Classification code 10021606;Term: Inborn errors of metabolism NEC;System Organ Class: 100000004850 MedDRA version: 18.0;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870 MedDRA version: 18.0;Classification code 10018903;Term: Haemoglobinopathies congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Ovastat 1000 (Treosulfan injection) INN or Proposed INN: TREOSULFAN Trade Name: Ovastat 5000 (Treosulfan injection) INN or Proposed INN: TREOSULFAN Trade Name: Busilvex INN or Proposed INN: BUSULFAN | medac GmbH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 2 | Czech Republic;Poland;Austria;Germany;Italy | ||
| 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 |
| 21 | NCT02349906 (ClinicalTrials.gov) | April 2015 | 26/1/2015 | Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases | Clinical Phase II Trial to Compare Treosulfan-based Conditioning Therapy With Busulfan-based Conditioning Prior to Allogeneic Haematopoietic Stem Cell Transplantation (HSCT) in Paediatric Patients With Non-malignant Diseases | Primary Immunodeficiencies;Inborn Errors of Metabolism;Haemoglobinopathies;Bone Marrow Failure Syndromes | Drug: Treosulfan;Drug: Busilvex | medac GmbH | Celerion;Venn Life Sciences;Syneos Health | Active, not recruiting | N/A | 17 Years | All | 100 | Phase 2 | Czechia;Germany;Italy;Poland;Austria;Czech Republic |
| 22 | EUCTR2013-005508-33-PL (EUCTR) | 17/11/2014 | 08/08/2014 | Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseases | Clinical phase II trial to compare Treosulfan-based conditioning therapy with Busulfan-based conditioning prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients with non-malignant diseases - Treosulfan-based versus Busulfan-based conditioning in paediatric patients with non-malignant diseas | Male and female children with non-malignant diseases requiring myeloablative conditioning treatment with following allogeneic haematopoietic stem cell transplantation (allo-HSCT) – i.e. primary immunodeficiencies, inborn errors of metabolism, haemoglobinopathies and bone marrow failure syndromes. MedDRA version: 20.0;Level: HLT;Classification code 10021606;Term: Inborn errors of metabolism NEC;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870 MedDRA version: 20.0;Classification code 10018903;Term: Haemoglobinopathies congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | medac GmbH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 2 | Czech Republic;Poland;Austria;Germany;Italy | |||
| 23 | EUCTR2013-005508-33-CZ (EUCTR) | 12/11/2014 | 29/07/2014 | Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseases | Clinical phase II trial to compare Treosulfan-based conditioning therapy with Busulfan-based conditioning prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients with non-malignant diseases - Treosulfan-based versus Busulfan-based conditioning in paediatric patients with non-malignant diseas | Male and female children with non-malignant diseases requiring myeloablative conditioning treatment with following allogeneic haematopoietic stem cell transplantation (allo-HSCT) – i.e. primary immunodeficiencies, inborn errors of metabolism, haemoglobinopathies and bone marrow failure syndromes. MedDRA version: 20.0;Level: HLT;Classification code 10021606;Term: Inborn errors of metabolism NEC;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870 MedDRA version: 20.0;Classification code 10018903;Term: Haemoglobinopathies congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Ovastat 1000 mg, powder for solution for infusion Product Name: Ovastat 1000 INN or Proposed INN: TREOSULFAN Trade Name: Ovastat 5000 mg, powder for solution for infusion Product Name: Ovastat 5000 INN or Proposed INN: TREOSULFAN Trade Name: Busilvex INN or Proposed INN: BUSULFAN | medac Gesellschaft für klinische Spezialpräparate mbH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 2 | Czech Republic;Poland;Austria;Germany;Italy | ||
| 24 | NCT02282904 (ClinicalTrials.gov) | October 23, 2014 | 4/11/2014 | Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant Cyclophosphamide | Haploidentical Transplant for Patients With Chronic Granulomatous Disease (CGD) Using Post-Transplant Cyclophosphamide | Chronic Granulomatous Disease | Drug: Sirolimus;Biological: Donor peripheral blood stem cells.;Drug: Cyclophosphamide post transplant;Radiation: Total body 200cGy;Drug: Cyclophosphamide;Drug: Fludarabine;Drug: Busulfan | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Terminated | 2 Years | 65 Years | All | 7 | Phase 1;Phase 2 | United States |
| 25 | EUCTR2013-005508-33-DE (EUCTR) | 29/09/2014 | 06/06/2014 | Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseases | Clinical phase II trial to compare Treosulfan-based conditioning therapy with Busulfan-based conditioning prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients with non-malignant diseases - Treosulfan-based versus Busulfan-based conditioning in paediatric patients with non-malignant diseas | Male and female children with non-malignant diseases requiring myeloablative conditioning treatment with following allogeneic haematopoietic stem cell transplantation (allo-HSCT) – i.e. primary immunodeficiencies, inborn errors of metabolism, haemoglobinopathies and bone marrow failure syndromes. MedDRA version: 20.0;Level: HLT;Classification code 10021606;Term: Inborn errors of metabolism NEC;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870 MedDRA version: 20.0;Classification code 10018903;Term: Haemoglobinopathies congenital;System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | medac Gesellschaft für klinische Spezialpräparate mbH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 100 | Phase 2 | Czech Republic;Poland;Austria;Germany;Italy | |||
| 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 |
| 26 | EUCTR2013-005508-33-AT (EUCTR) | 08/08/2014 | 08/07/2014 | Comparison of Treosulfan-based with Busulfan-based conditioning in paediatric patients with non-malignant diseases | Clinical phase II trial to compare Treosulfan-based conditioning therapy with Busulfan-based conditioning prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients with non-malignant diseases - Treosulfan-based versus Busulfan-based conditioning in paediatric patients with non-malignant diseas | Male and female children with non-malignant diseases requiring myeloablative conditioning treatment with following allogeneic haematopoietic stem cell transplantation (allo-HSCT) – i.e. primary immunodeficiencies, inborn errors of metabolism, haemoglobinopathies and bone marrow failure syndromes. MedDRA version: 19.1;Level: HLT;Classification code 10021606;Term: Inborn errors of metabolism NEC;System Organ Class: 100000004850 MedDRA version: 19.1;Classification code 10036700;Term: Primary immunodeficiency syndromes;System Organ Class: 100000004870 MedDRA version: 19.1;Classification code 10018903;Term: Haemoglobinopathies congenital;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Trade Name: Ovastat 1000 (Treosulfan injection) INN or Proposed INN: TREOSULFAN Trade Name: Ovastat 5000 (Treosulfan injection) INN or Proposed INN: TREOSULFAN Trade Name: Busilvex INN or Proposed INN: BUSULFAN | medac Gesellschaft fuer klinische Spezialpräparate mbH | NULL | Not Recruiting | Female: yes Male: yes | 100 | Phase 2 | Czech Republic;Poland;Austria;Germany;Italy | ||
| 27 | NCT01852071 (ClinicalTrials.gov) | August 2, 2013 | 7/5/2013 | Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With EFS Lentiviral Vector Encoding for the Human ADA Gene | Autologous Transplantation of Bone Marrow CD34+ Stem/Progenitor Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector for Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency (ADA-SCID) | ADA-SCID | Genetic: Infusion of autologous EFS-ADA LV CD34+ (OTL-101);Drug: busulfan;Drug: PEG-ADA ERT | Orchard Therapeutics | National Institute of Allergy and Infectious Diseases (NIAID);National Human Genome Research Institute (NHGRI);National Heart, Lung, and Blood Institute (NHLBI);University of California, Los Angeles | Completed | 1 Month | 17 Years | All | 20 | Phase 1;Phase 2 | United States |
| 28 | NCT01380990 (ClinicalTrials.gov) | November 15, 2012 | 23/6/2011 | Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency | Phase I/II, Historical Controlled, Open-label, Non-randomised, Single-centre Trial to Assess the Safety and Efficacy of EF1aS-ADA Lentiviral Vector Mediated Gene Modification of Autologous CD34+ Cells From ADA-deficient Individuals | Adenosine Deaminase Deficiency;Severe Combined Immunodeficiencies (SCID) | Genetic: Infusion of autologous EFS-ADA LV CD34+ cells;Other: Haematopoietic Stem Cell Transplantation (HSCT);Drug: Busulfan;Drug: Peg-Ada | Great Ormond Street Hospital for Children NHS Foundation Trust | Orchard Therapeutics | Completed | N/A | 15 Years | All | 36 | Phase 1;Phase 2 | United Kingdom |
| 29 | NCT01306019 (ClinicalTrials.gov) | September 25, 2012 | 26/2/2011 | Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID) | Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency | X-Linked Severe Combined Immune Deficiency (XSCID) | Drug: Palifermin;Drug: Busulfan;Biological: Ex vivo culture and transduction of the patient's autologous CD34+ HSC with lentivirus vector VSV-G pseudotyped CL20- 4i-EF1a-hyc-OPT vector | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Recruiting | 2 Years | 40 Years | Male | 30 | Phase 1;Phase 2 | United States |
| 30 | 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 |
| 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 |
| 31 | NCT03315078 (ClinicalTrials.gov) | April 2012 | 16/10/2017 | Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency | Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency | X-Linked Combined Immunodeficiency Diseases | Biological: CD34+ HSCs transduced with the lentivirus vector, VSV-G pseudotyped CL20-4i-EF1a-h?c-OPT;Drug: Palifermin;Drug: Busulfan | National Institute of Allergy and Infectious Diseases (NIAID) | NULL | Recruiting | 2 Years | 40 Years | All | 13 | Phase 1;Phase 2 | United States |
| 32 | NCT01338675 (ClinicalTrials.gov) | January 2011 | 4/3/2011 | Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Chronic Granulomatous Disease(CGD) | Chronic Granulomatous Disease | Drug: Busulfan | Seoul National University Hospital | NULL | Recruiting | N/A | N/A | Both | 5 | Phase 1;Phase 2 | Korea, Republic of | |
| 33 | NCT00794508 (ClinicalTrials.gov) | November 2008 | 19/11/2008 | MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID | MND-ADA Transduction of CD34+ Cells From the Bone Marrow Of Children With Adenosine Deaminase (ADA)-Deficient Severe Combined Immunodeficiency (SCID): Effect of Discontinuation of PEG-ADA and Marrow Cytoreduction With Busulfan | Severe Combined Immunodeficiency | Biological: ADA gene transfer | Donald B. Kohn, M.D. | FDA Office of Orphan Products Development;National Institutes of Health (NIH) | Completed | 1 Month | 18 Years | All | 10 | Phase 2 | United States |
| 34 | NCT00885833 (ClinicalTrials.gov) | February 2007 | 21/4/2009 | Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome (WAS) | Phase I/II Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome | Wiskott-Aldrich Syndrome | Drug: Fludarabine, Busulfan, Thymoglobulin | The Korean Society of Pediatric Hematology Oncology | NULL | Completed | 1 Year | 25 Years | Both | 5 | Phase 1;Phase 2 | Korea, Republic of |
| 35 | 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 |
| 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 |
| 36 | NCT00228852 (ClinicalTrials.gov) | April 2004 | 27/9/2005 | IMM 0212: Busulfan With Fludarabine and Antithymocyte Globulin as Preparative Therapy for Hematopoietic Stem Cell Transplant for the Treatment of Severe Congenital T-Cell Immunodeficiency | Phase I/II Trial of De-Escalation of Busulfan With Fludarabine and Antithymocyte Globulin as Preparative Therapy for Hematopoietic Stem Cell Transplant for the Treatment of Severe Congenital T-Cell Immunodeficiency | T-Cell Immune Deficiency Diseases;Severe Combined Immunodeficiency | Drug: Busulfan, Fludarabine and ATG | Emory University | NULL | Completed | N/A | N/A | Both | Phase 1;Phase 2 | United States | |
| 37 | NCT00578643 (ClinicalTrials.gov) | March 2004 | 19/12/2007 | Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease | HLA Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease | Chronic Granulomatous Disease | Drug: Busulfan;Biological: Alemtuzumab;Drug: Cyclophosphamide;Drug: Fludarabine;Drug: Cyclosporine;Procedure: Stem Cell Infusion | Baylor College of Medicine | NULL | Completed | N/A | N/A | All | 15 | Phase 2 | United States |
| 38 | NCT00176852 (ClinicalTrials.gov) | June 2002 | 12/9/2005 | 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 | Completed | N/A | 50 Years | All | 22 | Phase 2;Phase 3 | United States |
| 39 | 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 |
| 40 | NCT00176878 (ClinicalTrials.gov) | June 2000 | 12/9/2005 | 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 | NULL | Completed | N/A | 35 Years | All | 10 | Phase 2;Phase 3 | United States |
| 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 |
| 41 | NCT00006056 (ClinicalTrials.gov) | March 2000 | 5/7/2000 | Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic Disorders | Chediak-Higashi Syndrome;Graft Versus Host Disease;X-Linked Lymphoproliferative Syndrome;Familial Erythrophagocytic Lymphohistiocytosis;Hemophagocytic Lymphohistiocytosis;Virus-Associated Hemophagocytic Syndrome | Drug: anti-thymocyte globulin;Drug: busulfan;Drug: cyclophosphamide;Drug: cyclosporine;Drug: etoposide;Drug: filgrastim;Drug: methotrexate;Procedure: allogeneic hematopoietic stem cell transplantation | Fairview University Medical Center | NULL | Active, not recruiting | N/A | 55 Years | Both | 40 | N/A | United States | |
| 42 | NCT00006054 (ClinicalTrials.gov) | March 2000 | 5/7/2000 | Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies | Immunologic Deficiency Syndromes;Chediak-Higashi Syndrome;Common Variable Immunodeficiency;Graft Versus Host Disease;X-Linked Lymphoproliferative Syndrome;Familial Erythrophagocytic Lymphohistiocytosis;Hemophagocytic Lymphohistiocytosis;X-linked Agammaglobulinemia;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;X-linked Hyper IgM Syndrome;Severe Combined Immunodeficiency;Leukocyte Adhesion Deficiency Syndrome;Virus-Associated Hemophagocytic Syndrome | Drug: anti-thymocyte globulin;Drug: busulfan;Drug: cyclophosphamide;Drug: cyclosporine;Drug: etoposide;Drug: methotrexate;Drug: methylprednisolone;Drug: prednisone;Procedure: Allogeneic Bone Marrow Transplantation | Fairview University Medical Center | NULL | Terminated | N/A | 35 Years | Both | N/A | United States |
| 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 | 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 |
| 2 | NCT00006056 (ClinicalTrials.gov) | March 2000 | 5/7/2000 | Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic Disorders | Chediak-Higashi Syndrome;Graft Versus Host Disease;X-Linked Lymphoproliferative Syndrome;Familial Erythrophagocytic Lymphohistiocytosis;Hemophagocytic Lymphohistiocytosis;Virus-Associated Hemophagocytic Syndrome | Drug: anti-thymocyte globulin;Drug: busulfan;Drug: cyclophosphamide;Drug: cyclosporine;Drug: etoposide;Drug: filgrastim;Drug: methotrexate;Procedure: allogeneic hematopoietic stem cell transplantation | Fairview University Medical Center | NULL | Active, not recruiting | N/A | 55 Years | Both | 40 | N/A | United States | |
| 3 | NCT00006054 (ClinicalTrials.gov) | March 2000 | 5/7/2000 | Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies | Immunologic Deficiency Syndromes;Chediak-Higashi Syndrome;Common Variable Immunodeficiency;Graft Versus Host Disease;X-Linked Lymphoproliferative Syndrome;Familial Erythrophagocytic Lymphohistiocytosis;Hemophagocytic Lymphohistiocytosis;X-linked Agammaglobulinemia;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;X-linked Hyper IgM Syndrome;Severe Combined Immunodeficiency;Leukocyte Adhesion Deficiency Syndrome;Virus-Associated Hemophagocytic Syndrome | Drug: anti-thymocyte globulin;Drug: busulfan;Drug: cyclophosphamide;Drug: cyclosporine;Drug: etoposide;Drug: methotrexate;Drug: methylprednisolone;Drug: prednisone;Procedure: Allogeneic Bone Marrow Transplantation | Fairview University Medical Center | NULL | Terminated | N/A | 35 Years | Both | N/A | United States |
| 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 | NCT00705120 (ClinicalTrials.gov) | November 1995 | 23/6/2008 | Treatment of Severe Osteogenesis Imperfecta by Allogeneic Bone Marrow Transplantation | Treatment of Severe (Types II and III) Osteogenesis Imperfecta by Allogeneic Bone Marrow Transplantation | Osteogenesis Imperfecta | Other: Bone Marrow Cell Transplantation;Radiation: Irradiation, Total Body;Drug: Cyclophosphamide;Drug: Cyclosporin;Procedure: Mesenchymal Stem Cell Transplantation;Drug: Busulfan | St. Jude Children's Research Hospital | NULL | Completed | 3 Years | N/A | Both | 9 | Phase 1 | United States |
| 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 | NCT03513328 (ClinicalTrials.gov) | June 15, 2018 | 19/4/2018 | 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 | 3 Months | 39 Years | All | 40 | Phase 1;Phase 2 | United States |
| 2 | 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 |
| 3 | NCT00176852 (ClinicalTrials.gov) | June 2002 | 12/9/2005 | 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 | Completed | N/A | 50 Years | All | 22 | Phase 2;Phase 3 | United States |
| 4 | 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 |
| 5 | NCT00176878 (ClinicalTrials.gov) | June 2000 | 12/9/2005 | 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 | NULL | Completed | N/A | 35 Years | All | 10 | Phase 2;Phase 3 | United States |
| 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 |
| 6 | NCT00290628 (ClinicalTrials.gov) | October 1999 | 9/2/2006 | 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) | Terminated | N/A | 45 Years | All | 43 | N/A | United States |
| 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 |
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| 1 | NCT03579875 (ClinicalTrials.gov) | November 13, 2018 | 25/5/2018 | T Cell Receptor a/ß TCD HCT in Patients With Fanconi Anemia | T Cell Receptor Alpha/Beta T Cell Depleted (a/ß TCD) Hematopoietic Cell Transplantation in Patients With Fanconi Anemia (FA) | Fanconi Anemia;Severe Aplastic Anemia;Myelodysplastic Syndromes | Drug: Total Body Irradiation (TBI) (Plan 1);Drug: Cyclophosphamide (CY) (Plan 1);Drug: Fludarabine (FLU);Drug: Methylprednisolone (MP);Device: Donor mobilized PBSC infusion;Drug: G-CSF;Drug: Cyclophosphamide (CY) (Plan 2);Drug: Rituximab;Drug: Busulfan | Masonic Cancer Center, University of Minnesota | NULL | Recruiting | N/A | 65 Years | All | 48 | Phase 2 | United States |
| 2 | NCT03600909 (ClinicalTrials.gov) | May 15, 2018 | 17/7/2018 | A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi Anemia | A Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine | Fanconi Anemia;Myelodysplastic Syndrome (MDS);Acute Myelogenous Leukemia (AML) | Drug: Busulfan;Drug: Fludarabine;Drug: Cyclophosphamide;Drug: Anti-Thymocyte Globulin (Rabbit);Device: The CliniMACS device;Drug: G-CSF | Memorial Sloan Kettering Cancer Center | Pediatric Brain Tumor Consortium | Recruiting | 1 Month | N/A | All | 70 | Phase 2 | United States |
| 3 | NCT02143830 (ClinicalTrials.gov) | April 2014 | 28/4/2014 | HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy | A Phase II Trial of HSCT for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine | Fanconi Anemia;Severe Marrow Failure;Myelodysplastic Syndrome (MDS);Acute Myelogenous Leukemia (AML) | Drug: Busulfan;Drug: Cyclophosphamide;Drug: Fludarabine;Drug: rabbit ATG;Drug: G-CSF;Biological: Peripheral blood stem cell | Children's Hospital Medical Center, Cincinnati | Memorial Sloan Kettering Cancer Center;Fred Hutchinson Cancer Research Center | Recruiting | 3 Months | N/A | All | 70 | Phase 2 | United States |
| 4 | NCT01082133 (ClinicalTrials.gov) | October 2009 | 5/3/2010 | Multicenter Transplant Study for Fanconi Anemia | A Multicenter Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine | Fanconi Anemia | Drug: Chemotherapy;Biological: Miltenyi CliniMACS | Children's Hospital Medical Center, Cincinnati | NULL | Completed | N/A | N/A | Both | 45 | Phase 2 | United States |
| 5 | NCT00987480 (ClinicalTrials.gov) | September 25, 2009 | 30/9/2009 | Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine | A Multicenter Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine | Aplastic Anemia;Leukemia;Myelodysplastic Syndrome | Drug: Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine.;Device: CliniMACS device | Memorial Sloan Kettering Cancer Center | Boston Children's Hospital;Children's Hospital Medical Center, Cincinnati;Children's Hospital and Health System Foundation, Wisconsin;Rockefeller University;Fred Hutchinson Cancer Research Center | Completed | N/A | N/A | All | 45 | Phase 2 | United States |
| 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 |
| 6 | NCT01071239 (ClinicalTrials.gov) | April 2009 | 20/5/2009 | Hematopoietic Stem Cell Transplant for Fanconi Anemia | A Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine | Fanconi Anemia | Device: CliniMACs device;Drug: Busulfan;Drug: Fludarabine;Drug: Cyclophosphamide;Drug: ATG | Medical College of Wisconsin | Memorial Sloan Kettering Cancer Center | Completed | N/A | N/A | All | 1 | Phase 2 | United States |
| 7 | NCT00084695 (ClinicalTrials.gov) | September 2003 | 10/6/2004 | Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant Diseases | The Use Of Umbilical Cord Blood As A Source Of Hematopoietic Stem Cells | Childhood Langerhans Cell Histiocytosis;Fanconi Anemia;Leukemia;Lymphoma;Myelodysplastic Syndromes;Neuroblastoma;Sarcoma;Unspecified Childhood Solid Tumor, Protocol Specific | Biological: anti-thymocyte globulin;Drug: busulfan;Drug: cyclophosphamide;Drug: fludarabine phosphate;Drug: melphalan;Drug: methylprednisolone;Radiation: radiation therapy | Milton S. Hershey Medical Center | NULL | Recruiting | N/A | 21 Years | Both | 25 | Phase 2 | United States |
| 8 | NCT00258427 (ClinicalTrials.gov) | March 26, 2002 | 22/11/2005 | Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia | Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia MT2002-02 | Fanconi Anemia | Biological: anti-thymocyte globulin;Biological: filgrastim;Drug: busulfan;Drug: cyclophosphamide;Drug: fludarabine phosphate;Drug: methylprednisolone;Biological: Hematopoietic stem cell transplantation | Masonic Cancer Center, University of Minnesota | NULL | Completed | N/A | 44 Years | All | 14 | Phase 2 | United States |
| 9 | 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 |
| 10 | NCT00290628 (ClinicalTrials.gov) | October 1999 | 9/2/2006 | 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) | Terminated | N/A | 45 Years | All | 43 | N/A | United States |
| 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 | NCT02171104 (ClinicalTrials.gov) | July 10, 2014 | 20/6/2014 | MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis | MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG | Mucopolysaccharidosis Disorders;Hurler Syndrome;Hunter Syndrome;Maroteaux Lamy Syndrome;Sly Syndrome;Alpha-Mannosidosis;Fucosidosis;Aspartylglucosaminuria;Glycoprotein Metabolic Disorders;Sphingolipidoses;Recessive Leukodystrophies;Globoid Cell Leukodystrophy;Metachromatic Leukodystrophy;Niemann-Pick B;Niemann-Pick C Subtype 2;Sphingomyelin Deficiency;Peroxisomal Disorders;Adrenoleukodystrophy With Cerebral Involvement;Zellweger Syndrome;Neonatal Adrenoleukodystrophy;Infantile Refsum Disease;Acyl-CoA Oxidase Deficiency;D-Bifunctional Enzyme Deficiency;Multifunctional Enzyme Deficiency;Alpha-methylacyl-CoA Racmase Deficiency;Mitochondrial Neurogastrointestingal Encephalopathy;Severe Osteopetrosis;Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation);Inherited Metabolic Disorders | Biological: Stem Cell Transplantation;Drug: IMD Preparative Regimen;Drug: Osteopetrosis Only Preparative Regimen;Drug: Osteopetrosis Haploidentical Only Preparative Regimen;Drug: cALD SR-A (Standard-Risk, Regimen A);Drug: cALD SR-B (Standard-Risk, Regimen B);Drug: cALD HR-D (High-Risk, Regimen C);Drug: cALD HR-D (High-Risk, Regimen D) | Masonic Cancer Center, University of Minnesota | NULL | Recruiting | N/A | 55 Years | All | 100 | Phase 2 | United States |
| 2 | NCT01087398 (ClinicalTrials.gov) | September 2009 | 13/3/2010 | Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis | Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis | Osteopetrosis | Drug: Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen);Procedure: Stem Cell Transplantation;Drug: Cyclosporin, Methotrexate (GVHD prophylaxis) | Tehran University of Medical Sciences | NULL | Recruiting | N/A | 5 Years | Both | 10 | Phase 2;Phase 3 | Iran, Islamic Republic of |
| 3 | NCT00775931 (ClinicalTrials.gov) | August 2008 | 16/10/2008 | Allogeneic Transplantation For Severe Osteopetrosis | Allogeneic Hematopoietic Stem Cell Transplantation For Severe Osteopetrosis | Severe Osteopetrosis | Procedure: umbilical cord blood transplantation;Drug: Campath-1H;Radiation: Total Lymphoid Irradiation;Drug: Cyclophosphamide;Drug: Busulfan;Drug: Fludarabine monophosphate;Procedure: marrow graft transplantation | Masonic Cancer Center, University of Minnesota | NULL | Completed | N/A | 45 Years | All | 7 | Phase 2;Phase 3 | United States |
| 4 | NCT00638820 (ClinicalTrials.gov) | September 2007 | 11/3/2008 | Reduced Intensity AlloTransplant For Osteopetrosis | Reduced Intensity Allogeneic Transplantation For Severe Osteopetrosis Incorporating A Second Cd34 Selected Graft | Osteopetrosis | Procedure: Stem Cell or Umbilical Cord Blood Transplantation;Drug: Campath, Busulfan, Clofarabine;Procedure: Total Lymphoid Irradiation | Masonic Cancer Center, University of Minnesota | NULL | Terminated | N/A | 45 Years | All | 3 | Phase 2 | United States |