Hematopoietic Stem Cell Transplant ( DrugBank: - )


2 diseases
告示番号疾患名(ページ内リンク)臨床試験数
65原発性免疫不全症候群29
285ファンコニ貧血15

65. 原発性免疫不全症候群


臨床試験数 : 482 薬物数 : 653 - (DrugBank : 119) / 標的遺伝子数 : 92 - 標的パスウェイ数 : 212
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04691622
(ClinicalTrials.gov)
October 26, 202028/12/2020Adoptive T Lymphocyte Administration for Chronic Norovirus Treatment in Immunocompromised HostsAdoptive T Lymphocyte Administration for Chronic Norovirus Treatment in Immunocompromised HostsViral Infection;Hematopoietic Stem Cell Transplantation (HSCT);Primary Immunodeficiency Disorders (PID)Biological: Norovirus -specific T-cell (NST) therapyChildren's National Research InstituteNULLRecruiting3 Months80 YearsAll48Phase 1United States
2NCT04339777
(ClinicalTrials.gov)
September 22, 20208/4/2020Allogeneic Hematopoietic Stem Cell Transplant for People With Primary Immunodeficiency DiseasesA Phase II Study of Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immunodeficiency DiseasesLymphoproliferative Disorders;Autoimmune Lymphoproliferative;Immune System Diseases;Common Variable Immunodeficiency;Primary T-cell Immunodeficiency DisordersDrug: 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)NULLSuspended4 Years69 YearsAll10Phase 2United States
3NCT04400045
(ClinicalTrials.gov)
May 22, 202019/5/2020Low Dose Treosulfan Based Conditioning Regimen in HSCT for Nijmegen Breakage SyndromeClinical Open-label Phase 2 Study of Low Dose Treosulfan Based Conditioning Regimen Efficacy in Hematopoietic Stem Cell Transplantation for Children With Nijmegen Breakage SyndromeNijmegen Breakage SyndromeDrug: TreosulfanFederal Research Institute of Pediatric Hematology, Oncology and ImmunologyNULLRecruiting3 Months21 YearsAll10Phase 2Russian Federation
4NCT03879876
(ClinicalTrials.gov)
May 13, 202015/3/2019Safety and Efficacy Study of Human T Lymphoid Progenitor (HTLP) Injection After Partially HLA Compatible Allogeneic Hematopoietic Stem Cell Transplantation in SCID PatientsA Phase I/II Study Evaluating the Safety and the Efficacy of Human T Lymphoid Progenitor (HTLP) Injection to Accelerate Immune Reconstitution After Partially HLA Compatible Allogeneic Hematopoietic Stem Cell Transplantation in SCID PatientsPediatric Patients;Any Type of Severe Combined Immunodeficiency (SCID);Partial HLA Incompatible Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)Drug: Human T Lymphoid Progenitor (HTLP) injectionAssistance Publique - Hôpitaux de ParisNULLRecruiting2 Years18 YearsAll12Phase 1/Phase 2France
5EUCTR2018-001029-14-FR
(EUCTR)
25/10/201802/07/2018HTLPA phase I/II Study evaluating the safety and the efficacy of Human T Lymphoid Progenitor (HTLP) injection to accelerate immune reconstitution after partially HLA compatible allogeneic hematopoietic stem cell transplantation in SCID patients - HTLP NECKER SCID pediatric patients (n=12 for analysis) requiring an HLA partially compatible allogeneic HSCT.
MedDRA version: 20.0;Level: PT;Classification code 10010099;Term: Combined immunodeficiency;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: HTLP
Product Code: HTLP
ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
12Phase 1;Phase 2France
6NCT03019809
(ClinicalTrials.gov)
June 201611/1/2017A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS PatientsA Trial of Plerixafor With G-CSF as Additional Agents in Conditioning Regimen for Prevention of Graft Failure After Transplantation With TCR Alpha/Beta Grafts Depletion in Patients With Wiskott-Aldrich Syndrome.Wiskott-Aldrich Syndrome;Hematopoietic Stem Cell Transplantation;Graft FailureBiological: G-CSF for Conditioning before HSCT.;Biological: Plerixafor for Conditioning before HSCT.Federal Research Institute of Pediatric Hematology, Oncology and ImmunologyNULLUnknown status1 Month19 YearsAll30Phase 2Russian Federation
7NCT02162420
(ClinicalTrials.gov)
January 201510/6/2014Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic AnemiaHematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic AnemiaDyskeratosis Congenita;Aplastic AnemiaDrug: Alemtuzumab;Drug: Fludarabine;Drug: Cyclophosphamide;Radiation: Total Body Irradiation;Biological: Stem Cell Transplant;Drug: Anti-thymocyte globulinMasonic Cancer Center, University of MinnesotaNULLRecruitingN/A70 YearsAll50N/AUnited States
8NCT02179359
(ClinicalTrials.gov)
September 2, 201427/6/2014Hematopoietic Stem Cell Transplant for High Risk HemoglobinopathiesMT2014-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent DisordersSickle Cell Disease;Transfusion Dependent Alpha- or Beta- Thalassemia;Diamond Blackfan Anemia;Paroxysmal Nocturnal Hemoglobinuria;Glanzmann Thrombasthenia;Severe Congenital Neutropenia;Shwachman-Diamond Syndrome;Non-Malignant Hematologic DisordersDrug: Reduced Toxicity Ablative Regimen;Drug: Reduced Intensity Preparative Regimen;Drug: Myeloablative Preparative RegimenMasonic Cancer Center, University of MinnesotaNULLRecruitingN/A55 YearsAll25N/AUnited States
9NCT02177760
(ClinicalTrials.gov)
July 201419/6/2014Sirolimus Prophylaxis for aGVHD in TME SCIDSirolimus in Prevention of aGVHD in Maternally Engrafted (TME) Severe Combined Immunodeficiency (SCID) Infants Receiving Unconditioned Hematopoietic Stem Cell Transplant (HSCT)Severe Combined Immunodeficiency;Transplacental Maternal Engraftment;Stem Cell TransplantDrug: SirolimusUniversity of California, San FranciscoNULLWithdrawnN/A1 YearBoth0Phase 2United States
10NCT01917708
(ClinicalTrials.gov)
January 201424/7/2013Bone Marrow Transplant With Abatacept for Non-Malignant DiseasesAbatacept for Post-Transplant Immune Suppression in Children and Adolescents Receiving Allogeneic Hematopoietic Stem Cell Transplants for Non-Malignant DiseasesHurler Syndrome;Fanconi Anemia;Glanzmann Thrombasthenia;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;Severe Congenital Neutropenia;Leukocyte Adhesion Deficiency;Shwachman-Diamond Syndrome;Diamond-Blackfan Anemia;Dyskeratosis-congenita;Chediak-Higashi Syndrome;Severe Aplastic Anemia;Thalassemia Major;Hemophagocytic Lymphohistiocytosis;Sickle Cell DiseaseDrug: AbataceptEmory UniversityNULLCompletedN/A21 YearsAll10Phase 1United States
11NCT01998633
(ClinicalTrials.gov)
December 201329/10/2013Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)Reduced-Intensity Conditioning for Children and Adults With Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (RICHI) (BMT CTN #1204)Hemophagocytic Lymphohistiocytosis;Chronic Active Epstein-Barr Virus Infection;Chronic Granulomatous Disease;HIGM-1;Leukocyte Adhesion Deficiency;IPEXBiological: Hematopoietic Stem Cell TransplantMedical College of WisconsinNational Heart, Lung, and Blood Institute (NHLBI);Blood and Marrow Transplant Clinical Trials Network;National Cancer Institute (NCI);National Marrow Donor ProgramCompleted4 Months45 YearsAll47Phase 2United States;Canada
12NCT01821781
(ClinicalTrials.gov)
March 201319/3/2013Immune Disorder HSCT ProtocolA Study of Hematopoietic Stem Cell Transplantation (HSCT) in Immune Function Disorders Using a Reduced Intensity Preparatory RegimeImmune Deficiency Disorders;Severe Combined Immunodeficiency;Chronic Granulomatous Disease;X-linked Agammaglobulinemia;Wiskott-Aldrich Syndrome;Hyper-IgM;DiGeorge Syndrome;Chediak-Higashi Syndrome;Common Variable Immune Deficiency;Immune Dysregulatory Disorders;Hemophagocytic Lymphohistiocytosis;IPEX;Autoimmune Lymphoproliferative Syndrome;X-linked Lymphoproliferative SyndromeDrug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalanWashington University School of MedicineNULLRecruitingN/A21 YearsAll20Phase 2United States
13NCT01652092
(ClinicalTrials.gov)
September 4, 201225/7/2012Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune DeficienciesAllogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune DeficienciesSCID;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 HistiocytosisDrug: 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: MESNAMasonic Cancer Center, University of MinnesotaNULLRecruitingN/A50 YearsAll30N/AUnited States
14NCT02327351
(ClinicalTrials.gov)
July 201218/12/2014TCR Alpha/Beta Depletion for HSCT From Haploidentical and Unrelated Donors in the Treatment of PIDPhase II/III Study of Allogeneic Hematopoietic Stem Cell Transplantation From Unrelated and Haploidentical Donors After TCR Alfa Beta Negative Selection in Pediatric Patients With Primary Immunodeficiency DiseasesPrimary Immune Deficiency Disorder;Hematopoietic Stem Cell TransplantationOther: Biological: TCR alfa beta T cell depletionFederal Research Institute of Pediatric Hematology, Oncology and ImmunologyNULLRecruiting1 Month18 YearsBoth60Phase 2/Phase 3Russian Federation
15NCT01529827
(ClinicalTrials.gov)
February 28, 20126/2/2012Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic MalignanciesA Phase II Trial of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan and Low Dose Total Body IrradiationAccelerated Phase Chronic Myelogenous Leukemia;Adult Acute Lymphoblastic Leukemia in Remission;Adult Acute Myeloid Leukemia in Remission;Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities;Adult Acute Myeloid Leukemia With Del(5q);Adult Acute Myeloid Leukemia With Inv(16)(p13;q22);Adult Acute Myeloid Leukemia With t(15;17)(q22;q12);Adult Acute Myeloid Leukemia With t(16;16)(p13;Adult Acute Myeloid Leukemia With t(8;21)(q22;Adult Grade III Lymphomatoid Granulomatosis;Adult Nasal Type Extranodal NK/T-cell Lymphoma;Anaplastic Large Cell Lymphoma;Angioimmunoblastic T-cell Lymphoma;Aplastic Anemia;Burkitt Lymphoma;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Chronic Myelogenous Leukemia;Childhood Diffuse Large Cell Lymphoma;Childhood Grade III Lymphomatoid Granulomatosis;Childhood Immunoblastic Large Cell Lymphoma;Childhood Myelodysplastic Syndromes;Childhood Nasal Type Extranodal NK/T-cell Lymphoma;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia;Congenital Amegakaryocytic Thrombocytopenia;Diamond-Blackfan Anemia;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Hepatosplenic T-cell Lymphoma;Juvenile Myelomonocytic Leukemia;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Nodal Marginal Zone B-cell Lymphoma;Paroxysmal Nocturnal Hemoglobinuria;Peripheral T-cell Lymphoma;Polycythemia Vera;Post-transplant Lymphoproliferative Disorder;Previously Treated Myelodysplastic Syndromes;Primary Myelofibrosis;Recurrent Adult Acute Lymphoblastic Leukemia;Recurrent Adult Acute Myeloid Leukemia;Recurrent Adult Burkitt Lymphoma;Recurrent Adult Diffuse Large Cell Lymphoma;Recurrent Adult Diffuse Mixed Cell Lymphoma;Recurrent Adult Diffuse Small Cleaved Cell Lymphoma;Recurrent Adult Grade III Lymphomatoid Granulomatosis;Recurrent Adult Hodgkin Lymphoma;Recurrent Adult Immunoblastic Large Cell Lymphoma;Recurrent Adult Lymphoblastic Lymphoma;Recurrent Adult T-cell Leukemia/Lymphoma;Recurrent Childhood Acute Lymphoblastic Leukemia;Recurrent Childhood Acute Myeloid Leukemia;Recurrent Childhood Anaplastic Large Cell Lymphoma;Recurrent Childhood Grade III Lymphomatoid Granulomatosis;Recurrent Childhood Large Cell Lymphoma;Recurrent Childhood Lymphoblastic Lymphoma;Recurrent Childhood Small Noncleaved Cell Lymphoma;Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma;Recurrent Grade 1 Follicular Lymphoma;Recurrent Grade 2 Follicular Lymphoma;Recurrent Grade 3 Follicular Lymphoma;Recurrent Mantle Cell Lymphoma;Recurrent Marginal Zone Lymphoma;Recurrent Mycosis Fungoides/Sezary Syndrome;Recurrent Small Lymphocytic Lymphoma;Recurrent/Refractory Childhood Hodgkin Lymphoma;Refractory Chronic Lymphocytic Leukemia;Refractory Hairy Cell Leukemia;Refractory Multiple Myeloma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndromes;Secondary Myelofibrosis;Severe Combined Immunodeficiency;Severe Congenital Neutropenia;Shwachman-Diamond Syndrome;Splenic Marginal Zone Lymphoma;T-cell Large Granular Lymphocyte Leukemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich SyndromeDrug: fludarabine phosphate;Drug: melphalan;Radiation: total-body irradiation;Drug: tacrolimus;Drug: mycophenolate mofetil;Drug: methotrexate;Other: laboratory biomarker analysis;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantationRoswell Park Cancer InstituteNULLCompleted3 Years75 YearsAll94Phase 2United States
16NCT01338675
(ClinicalTrials.gov)
January 20114/3/2011Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Chronic Granulomatous Disease(CGD)Chronic Granulomatous DiseaseDrug: BusulfanSeoul National University HospitalNULLRecruitingN/AN/ABoth5Phase 1/Phase 2Korea, Republic of
17NCT01319851
(ClinicalTrials.gov)
September 201015/9/2010Alefacept and Allogeneic Hematopoietic Stem Cell TransplantationAlefacept and Allogeneic Hematopoietic Stem Cell Transplantation for Children With Non-Malignant Diseases Who Have Been Multiply Transfused: a Pilot StudyThalassemia;Sickle Cell Disease;Glanzmann Thrombasthenia;Wiskott-Aldrich Syndrome;Chronic-granulomatous Disease;Severe Congenital Neutropenia;Leukocyte Adhesion Deficiency;Schwachman-Diamond Syndrome;Diamond-Blackfan Anemia;Fanconi Anemia;Dyskeratosis-congenita;Chediak-Higashi Syndrome;Severe Aplastic AnemiaDrug: AlefaceptEmory UniversityChildren's Healthcare of AtlantaTerminatedN/A21 YearsAll3N/AUnited States
18NCT01182675
(ClinicalTrials.gov)
August 20109/8/2010Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & FilgrastimHematopoietic Stem Cell Transplantation for Children With Severe Combined Immunodeficiency Disease Utilizing Alemtuzumab and Mobilization With Plerixafor & FilgrastimSevere Combined ImmunodeficiencyDrug: Transplant Conditioning with Mobilization Only;Drug: Transplant Conditioning with Mobilization and AlemtuzumabUniversity of California, San FranciscoNULLTerminatedN/A3 YearsAll7Phase 2United States
19NCT00455312
(ClinicalTrials.gov)
August 200730/3/2007Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAAHematopoietic Stem Cell Transplant For Patients With Dyskeratosis Congenita and Severe Aplastic AnemiaDyskeratosis Congenita;Aplastic AnemiaDrug: Campath 1H;Drug: Cyclophosphamide;Drug: Fludarabine;Procedure: Total Body Irradiation;Procedure: Stem Cell Transplantation;Drug: antithymocyte globulin;Drug: MethylprednisoloneMasonic Cancer Center, University of MinnesotaNULLCompletedN/A70 YearsAll36Phase 2/Phase 3United States
20NCT02512679
(ClinicalTrials.gov)
February 200721/5/2015Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood CellsProtocol for Related Donor Hematopoietic Stem Cell Transplantation (HSCT) for Treatment of Symptomatic Genetic Lymphohematological DiseasesStem Cell Transplantation;Bone Marrow Transplantation;Peripheral Blood Stem Cell Transplantation;Allogeneic Transplantation;Genetic Diseases;Thalassemia;Pediatrics;Diamond-Blackfan Anemia;Combined Immune Deficiency;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;X-linked Lymphoproliferative Disease;Metabolic DiseasesDrug: Cyclophosphamide Dose Level 1;Drug: Cyclophosphamide Dose Level 2;Drug: Cyclophosphamide Dose Level 3;Drug: Cyclophosphamide Dose Level 4Children's Hospital Los AngelesLucile Packard Children's HospitalTerminated3 MonthsN/AAll20Phase 2NULL
21NCT02127892
(ClinicalTrials.gov)
January 2, 200719/10/2012SCID Bu/Flu/ATG Study With T Cell DepletionPhase I/II Trial of Hematopoietic Stem Cell Transplant (HSCT) for Children With Severe Combined Immune Deficiency (SCID) and Without an HLA-Matched Sibling DonorSevere Combined ImmunodeficiencyBiological: unrelated BM with T cell depletion;Biological: unrelated cord blood;Biological: haplo BM with T cell depletion;Device: unrelated PBSC with T cell depletionNeena Kapoor, M.D.NULLTerminatedN/A21 YearsAll9Phase 1/Phase 2United States
22NCT00358657
(ClinicalTrials.gov)
May 24, 200628/7/2006Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited DisordersHLA-Haploidentical Related Marrow Grafts for the Treatment of Primary Immunodeficiencies and Other Nonmalignant Disorders Using Conditioning With Low-Dose Cyclophosphamide, TBI and Fludarabine and Postgrafting CyclophosphamideImmunodeficiency Syndrome;Non-Cancer Diagnosis;Severe Aplastic Anemia;DonorProcedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Drug: Sirolimus;Drug: Tacrolimus;Radiation: Total-Body IrradiationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI)Active, not recruitingN/A55 YearsAll14Phase 2United States
23NCT00160355
(ClinicalTrials.gov)
May 20058/9/2005Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich SyndromeHaploidentical Hematopoietic Stem Cell Transplantation for Pediatric Patients With Wiskott-Aldrich Syndrome: A Pilot StudyWiskott-Aldrich SyndromeProcedure: Hematopoietic stem cell transplantation;Device: Miltenyi CliniMACS selection system;Drug: Fludarabine, Melphalan, ThiotepaSt. Jude Children's Research HospitalNULLCompletedN/A18 YearsMale4Phase 1United States
24NCT00295971
(ClinicalTrials.gov)
April 200523/2/2006Donor Stem Cell Transplant in Treating Young Patients With Myelodysplastic Syndrome, Leukemia, Bone Marrow Failure Syndrome, or Severe Immunodeficiency DiseaseStem Cell Enriched, T Cell Depleted Haplocompatible Peripheral Blood Transplantation for Children With Myelodysplastic Disease, Leukemia, Marrow Failure Syndromes, or Severe Immunodeficiency DiseasesCongenital Amegakaryocytic Thrombocytopenia;Leukemia;Myelodysplastic Syndromes;Severe Congenital NeutropeniaBiological: anti-thymocyte globulin;Biological: therapeutic allogeneic lymphocytes;Drug: fludarabine phosphate;Drug: thiotepa;Procedure: allogeneic bone marrow transplantation;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: in vitro-treated peripheral blood stem cell transplantation;Radiation: total-body irradiationUniversity of California, San FranciscoNational Cancer Institute (NCI)Completed1 Year17 YearsBoth21Phase 1United States
25NCT00301834
(ClinicalTrials.gov)
January 20059/3/2006Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic DisordersEvaluation of Fludarabine, Busulfan and Alemtuzumab as a Reduced Toxicity Ablative Bone Marrow Stem Cell Transplant Regimen for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myelodysplastic Syndrome (MDS)/LeukemiaCongenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Leukemia;Myelodysplastic Syndromes;Severe Congenital NeutropeniaBiological: alemtuzumab;Drug: busulfan;Drug: cyclosporine;Drug: fludarabine phosphate;Drug: methotrexate;Drug: methylprednisolone;Procedure: allogeneic bone marrow transplantation;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantationUniversity of California, San FranciscoNational Cancer Institute (NCI)CompletedN/A21 YearsAll35Phase 2United States
26NCT00228852
(ClinicalTrials.gov)
April 200427/9/2005IMM 0212: Busulfan With Fludarabine and Antithymocyte Globulin as Preparative Therapy for Hematopoietic Stem Cell Transplant for the Treatment of Severe Congenital T-Cell ImmunodeficiencyPhase 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 ImmunodeficiencyT-Cell Immune Deficiency Diseases;Severe Combined ImmunodeficiencyDrug: Busulfan, Fludarabine and ATGEmory UniversityNULLCompletedN/AN/ABothPhase 1/Phase 2United States
27NCT00176852
(ClinicalTrials.gov)
June 200212/9/2005Stem Cell Transplant for HemoglobinopathyAllogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathy Using a Preparative Regimen to Achieve Stable Mixed ChimerismSickle Cell Disease;Thalassemia;Severe Congenital Neutropenia;Diamond-Blackfan Anemia;Shwachman-Diamond SyndromeDrug: Busulfan, Fludarabine, ATG, TLI;Drug: Busulfan, Cyclophosphamide, ATG, GCSF;Drug: Campath, Fludarabine, Cyclophosphamide;Radiation: Total Body Irradiation;Procedure: Stem cell infusionMasonic Cancer Center, University of MinnesotaNational Marrow Donor ProgramCompletedN/A50 YearsAll22Phase 2/Phase 3United States
28NCT00006056
(ClinicalTrials.gov)
March 20005/7/2000Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic DisordersChediak-Higashi Syndrome;Graft Versus Host Disease;X-Linked Lymphoproliferative Syndrome;Familial Erythrophagocytic Lymphohistiocytosis;Hemophagocytic Lymphohistiocytosis;Virus-Associated Hemophagocytic SyndromeDrug: anti-thymocyte globulin;Drug: busulfan;Drug: cyclophosphamide;Drug: cyclosporine;Drug: etoposide;Drug: filgrastim;Drug: methotrexate;Procedure: allogeneic hematopoietic stem cell transplantationFairview University Medical CenterNULLActive, not recruitingN/A55 YearsBoth40N/AUnited States
29NCT00008450
(ClinicalTrials.gov)
August 11, 19976/1/2001Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow TransplantInduction of Mixed Hematopoietic Chimerism in Patients With Severe Combined Immunodeficiency Disorders Using Allogeneic Bone Marrow and Post-Transplant Immunosuppression With Cyclosporine and Mycophenolate MofetilAdenosine Deaminase Deficiency;Autosomal Recessive Disorder;Immune System Disorder;Purine-Nucleoside Phosphorylase Deficiency;Severe Combined Immunodeficiency;Severe Combined Immunodeficiency With Absence of T and B Cells;X-Linked Severe Combined ImmunodeficiencyProcedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclosporine;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Radiation: Total-Body IrradiationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI)CompletedN/AN/AAll6Phase 1United States

285. ファンコニ貧血


臨床試験数 : 59 薬物数 : 118 - (DrugBank : 32) / 標的遺伝子数 : 31 - 標的パスウェイ数 : 155
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03600909
(ClinicalTrials.gov)
May 15, 201817/7/2018A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi AnemiaA 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 FludarabineFanconi 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-CSFMemorial Sloan Kettering Cancer CenterPediatric Brain Tumor ConsortiumCompleted1 MonthN/AAll3Phase 2United States
2NCT03609840
(ClinicalTrials.gov)
May 24, 20175/1/2018Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant PatientsPopulation Pharmacokinetics and Pharmacodynamics of Thiotepa and TEPA in Pediatric Patients Undergoing Hematopoietic Cell Transplantation (HCT).Hematologic Malignancies;Nonmalignant Diseases;Immune Deficiency;Hemoglobinopathies;Genetic Inborn Errors of Metabolism;Fanconi Anemia;Thalassemia;Sickle Cell DiseaseDrug: ThiotepaUniversity of California, San FranciscoNULLActive, not recruitingN/A17 YearsAll25United States
3NCT01917708
(ClinicalTrials.gov)
January 201424/7/2013Bone Marrow Transplant With Abatacept for Non-Malignant DiseasesAbatacept for Post-Transplant Immune Suppression in Children and Adolescents Receiving Allogeneic Hematopoietic Stem Cell Transplants for Non-Malignant DiseasesHurler Syndrome;Fanconi Anemia;Glanzmann Thrombasthenia;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;Severe Congenital Neutropenia;Leukocyte Adhesion Deficiency;Shwachman-Diamond Syndrome;Diamond-Blackfan Anemia;Dyskeratosis-congenita;Chediak-Higashi Syndrome;Severe Aplastic Anemia;Thalassemia Major;Hemophagocytic Lymphohistiocytosis;Sickle Cell DiseaseDrug: AbataceptEmory UniversityNULLCompletedN/A21 YearsAll10Phase 1United States
4NCT01319851
(ClinicalTrials.gov)
September 201015/9/2010Alefacept and Allogeneic Hematopoietic Stem Cell TransplantationAlefacept and Allogeneic Hematopoietic Stem Cell Transplantation for Children With Non-Malignant Diseases Who Have Been Multiply Transfused: a Pilot StudyThalassemia;Sickle Cell Disease;Glanzmann Thrombasthenia;Wiskott-Aldrich Syndrome;Chronic-granulomatous Disease;Severe Congenital Neutropenia;Leukocyte Adhesion Deficiency;Schwachman-Diamond Syndrome;Diamond-Blackfan Anemia;Fanconi Anemia;Dyskeratosis-congenita;Chediak-Higashi Syndrome;Severe Aplastic AnemiaDrug: AlefaceptEmory UniversityChildren's Healthcare of AtlantaTerminatedN/A21 YearsAll3N/AUnited States
5NCT01082133
(ClinicalTrials.gov)
October 20095/3/2010Multicenter Transplant Study for Fanconi AnemiaA 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 FludarabineFanconi AnemiaDrug: Chemotherapy;Biological: Miltenyi CliniMACSChildren's Hospital Medical Center, CincinnatiNULLCompletedN/AN/ABoth45Phase 2United States
6NCT00987480
(ClinicalTrials.gov)
September 25, 200930/9/2009Hematopoietic 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 FludarabineA 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 FludarabineAplastic Anemia;Leukemia;Myelodysplastic SyndromeDrug: Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine.;Device: CliniMACS deviceMemorial Sloan Kettering Cancer CenterBoston Children's Hospital;Children's Hospital Medical Center, Cincinnati;Children's Hospital and Health System Foundation, Wisconsin;Rockefeller University;Fred Hutchinson Cancer Research CenterCompletedN/AN/AAll45Phase 2United States
7NCT01071239
(ClinicalTrials.gov)
April 200920/5/2009Hematopoietic Stem Cell Transplant for Fanconi AnemiaA 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 FludarabineFanconi AnemiaDevice: CliniMACs device;Drug: Busulfan;Drug: Fludarabine;Drug: Cyclophosphamide;Drug: ATGMedical College of WisconsinMemorial Sloan Kettering Cancer CenterCompletedN/AN/AAll1Phase 2United States
8NCT00856388
(ClinicalTrials.gov)
January 14, 20094/3/2009Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure DisordersA Pilot Trial Of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan, And Low Dose Total Body IrradiationAccelerated Phase Chronic Myelogenous Leukemia;Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome;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;Anaplastic Large Cell Lymphoma;Angioimmunoblastic T-cell Lymphoma;Aplastic Anemia;Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Chronic Myelogenous Leukemia;Childhood Diffuse Large Cell Lymphoma;Childhood Immunoblastic Large Cell Lymphoma;Childhood Myelodysplastic Syndromes;Childhood Nasal Type Extranodal NK/T-cell Lymphoma;Chronic Eosinophilic Leukemia;Chronic Myelomonocytic Leukemia;Chronic Neutrophilic Leukemia;Chronic Phase Chronic Myelogenous Leukemia;de Novo Myelodysplastic Syndromes;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Fanconi Anemia;Juvenile Myelomonocytic Leukemia;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Nodal Marginal Zone B-cell Lymphoma;Noncontiguous Stage II Adult Burkitt Lymphoma;Noncontiguous Stage II Adult Diffuse Large Cell Lymphoma;Noncontiguous Stage II Adult Diffuse Mixed Cell Lymphoma;Noncontiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma;Noncontiguous Stage II Adult Immunoblastic Large Cell Lymphoma;Noncontiguous Stage II Adult Lymphoblastic Lymphoma;Noncontiguous Stage II Grade 1 Follicular Lymphoma;Noncontiguous Stage II Grade 2 Follicular Lymphoma;Noncontiguous Stage II Grade 3 Follicular Lymphoma;Noncontiguous Stage II Mantle Cell Lymphoma;Noncontiguous Stage II Marginal Zone Lymphoma;Noncontiguous Stage II Small Lymphocytic Lymphoma;Paroxysmal Nocturnal Hemoglobinuria;Previously Treated Myelodysplastic Syndromes;Primary Myelofibrosis;Recurrent Adult Acute Lymphoblastic Leukemia;Recurrent Adult Acute Myeloid Leukemia;Recurrent Adult Burkitt Lymphoma;Recurrent Adult Diffuse Large Cell Lymphoma;Recurrent Adult Diffuse Mixed Cell Lymphoma;Recurrent Adult Diffuse Small Cleaved Cell Lymphoma;Recurrent Adult Grade III Lymphomatoid Granulomatosis;Recurrent Adult Hodgkin Lymphoma;Recurrent Adult Immunoblastic Large Cell Lymphoma;Recurrent Adult Lymphoblastic Lymphoma;Recurrent Adult T-cell Leukemia/Lymphoma;Recurrent Childhood Acute Lymphoblastic Leukemia;Recurrent Childhood Acute Myeloid Leukemia;Recurrent Childhood Anaplastic Large Cell Lymphoma;Recurrent Childhood Grade III Lymphomatoid Granulomatosis;Recurrent Childhood Large Cell Lymphoma;Recurrent Childhood Lymphoblastic Lymphoma;Recurrent Childhood Small Noncleaved Cell Lymphoma;Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma;Recurrent Grade 1 Follicular Lymphoma;Recurrent Grade 2 Follicular Lymphoma;Recurrent Grade 3 Follicular Lymphoma;Recurrent Mantle Cell Lymphoma;Recurrent Marginal Zone Lymphoma;Recurrent Mycosis Fungoides/Sezary Syndrome;Recurrent Small Lymphocytic Lymphoma;Recurrent/Refractory Childhood Hodgkin Lymphoma;Refractory Chronic Lymphocytic Leukemia;Refractory Multiple Myeloma;Relapsing Chronic Myelogenous Leukemia;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndromes;Splenic Marginal Zone Lymphoma;Stage III Adult Diffuse Small Cleaved Cell Lymphoma;Stage III Adult Immunoblastic Large Cell Lymphoma;Stage III Adult Lymphoblastic Lymphoma;Stage III Grade 1 Follicular Lymphoma;Stage III Grade 2 Follicular Lymphoma;Stage III Grade 3 Follicular Lymphoma;Stage III Mantle Cell Lymphoma;Stage III Marginal Zone Lymphoma;Stage III Small Lymphocytic Lymphoma;Stage IV Adult Burkitt Lymphoma;Stage IV Adult Diffuse Small Cleaved Cell Lymphoma;Stage IV Adult Immunoblastic Large Cell Lymphoma;Stage IV Adult Lymphoblastic Lymphoma;Stage IV Grade 1 Follicular Lymphoma;Stage IV Grade 2 Follicular Lymphoma;Stage IV Grade 3 Follicular Lymphoma;Stage IV Mantle Cell Lymphoma;Stage IV Marginal Zone Lymphoma;Stage IV Small Lymphocytic Lymphoma;Waldenström MacroglobulinemiaDrug: fludarabine phosphate;Drug: melphalan;Radiation: total-body irradiation;Procedure: allogeneic hematopoietic stem cell transplantation;Biological: anti-thymocyte globulinRoswell Park Cancer InstituteNational Cancer Institute (NCI)Completed3 Years75 YearsAll62N/AUnited States
9NCT00453388
(ClinicalTrials.gov)
February 200727/3/2007Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant, Mycophenolate Mofetil, and Cyclosporine in Treating Patients With Fanconi AnemiaNonmyeloablative Hematopoietic Cell Transplantation for Patients With Fanconi Anemia Using Alternative Marrow Donors: A Phase II Dose-Finding StudyAcute Myeloid Leukemia in Remission;de Novo Myelodysplastic Syndrome;Fanconi Anemia;Previously Treated Myelodysplastic SyndromeProcedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclophosphamide;Drug: Cyclosporine;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Radiation: Total-Body IrradiationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI)CompletedN/AN/AAll6Phase 2United States;Brazil
10NCT00167206
(ClinicalTrials.gov)
March 20049/9/2005Stem Cell Transplantation for Fanconi AnemiaA Study of Thymic Shielding in Recipients of Total Body Irradiation, Cyclophosphamide, and Fludarabine Followed by Alternate Donor Hematopoietic Stem Cell Transplantation in Patients With Fanconi AnemiaFanconi AnemiaProcedure: Hematopoietic Stem Cell Transplant;Procedure: Thymic Shielding During Radiation;Procedure: Total Body Irradiation;Drug: Cyclophosphamide, FludarabineMasonic Cancer Center, University of MinnesotaNULLTerminatedN/A18 YearsAll16Phase 1/Phase 2United States
11NCT00258427
(ClinicalTrials.gov)
March 26, 200222/11/2005Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi AnemiaHematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia MT2002-02Fanconi AnemiaBiological: anti-thymocyte globulin;Biological: filgrastim;Drug: busulfan;Drug: cyclophosphamide;Drug: fludarabine phosphate;Drug: methylprednisolone;Biological: Hematopoietic stem cell transplantationMasonic Cancer Center, University of MinnesotaNULLCompletedN/A44 YearsAll14Phase 2United States
12NCT00595127
(ClinicalTrials.gov)
June 20017/1/2008Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and FludarabineA Pilot Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and FludarabineFanconi AnemiaDrug: Cyclophosphamide;Drug: Fludarabine;Radiation: Total body irradiation (TBI)Memorial Sloan Kettering Cancer CenterNational Cancer Institute (NCI);Miltenyi Biotec, Inc.CompletedN/AN/AAll21N/AUnited States
13NCT00630253
(ClinicalTrials.gov)
February 17, 20005/3/2008Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi AnemiaA Study of Cyclophosphamide, Fludarabine, and Antithymocyte Globulin Followed by Matched Sibling Donor Hematopoietic Cell Transplantation in Patients With Fanconi AnemiaFanconi AnemiaBiological: Anti-Thymocyte Globulin;Drug: Cyclophosphamide;Drug: Fludarabine;Procedure: Hematopoietic Stem Cell Transplantation;Drug: Methylprednisolone;Drug: Filgrastim;Drug: Cyclosporine;Drug: Mycophenolate MofetilMasonic Cancer Center, University of MinnesotaNULLCompletedN/A59 YearsAll31Phase 1/Phase 2United States
14NCT00093743
(ClinicalTrials.gov)
January 20006/10/2004Low-Dose Total-Body Irradiation and Fludarabine Phosphate Followed by Unrelated Donor Stem Cell Transplant in Treating Patients With Fanconi AnemiaLow-Dose Total Body Irradiation and Fludarabine Followed By Unrelated Donor Stem Cell Transplantation for Patients With Fanconi Anemia - A Multicenter TrialAdult 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;Childhood Acute Myeloid Leukemia in Remission;Childhood Myelodysplastic Syndromes;Fanconi Anemia;Previously Treated Myelodysplastic SyndromesDrug: fludarabine phosphate;Drug: cyclosporine;Radiation: total-body irradiation;Procedure: allogeneic bone marrow transplantation;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantation;Drug: mycophenolate mofetilFred Hutchinson Cancer Research CenterNational Heart, Lung, and Blood Institute (NHLBI);National Cancer Institute (NCI)CompletedN/AN/AAll2Phase 1United States
15NCT00317876
(ClinicalTrials.gov)
June 199824/4/2006Cyclophosphamide in Treating Patients Who Are Undergoing a Donor Bone Marrow Transplant for Fanconi's AnemiaDose-Finding Study for Cyclophosphamide as Conditioning Regimens for Bone Marrow Transplantation From Related Donors in Patients With Fanconi AnemiaFanconi AnemiaDrug: cyclophosphamide;Drug: cyclosporine;Drug: methotrexate;Procedure: allogeneic bone marrow transplantation;Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI)CompletedN/AN/ABoth25Phase 1United States;Brazil