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 285. ファンコニ貧血 [臨床試験数:46,薬物数:65(DrugBank:27),標的遺伝子数:25,標的パスウェイ数:32] 

Searched query = "Fanconi anemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
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1NCT04069533October 201911 November 2019Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype AA Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients With Fanconi Anemia Subtype AFanconi Anemia Complementation Group ABiological: RP-L102Rocket Pharmaceuticals Inc.Recruiting1 Year17 YearsAll5Phase 2Spain
2NCT03814408January 11, 201915 July 2019A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype AA Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene in Pediatric Subjects With Fanconi Anemia Subtype AFanconi Anemia Complementation Group ABiological: RP-L102Rocket Pharmaceuticals Inc.Not recruiting1 Year12 YearsAll2Phase 1United States
3NCT03579875November 13, 20181 April 2019T Cell Receptor a/ß TCD HCT in Patients With Fanconi AnemiaT 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 SyndromesDrug: 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: RituximabMasonic Cancer Center, University of MinnesotaRecruitingN/A65 YearsAll48Phase 2United States
4NCT03206086November 2, 201811 November 2019Eltrombopag for People With Fanconi AnemiaEltrombopag for Patients With Fanconi AnemiaFanconi's AnemiaDrug: EltrombopagNational Heart, Lung, and Blood Institute (NHLBI)Recruiting4 YearsN/AAll25Phase 2United States
5EUCTR2018-002502-31-ES31/10/20187 January 2019A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemia Subtype AA Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemia Subtype A - FANCOLEN-IIFanconi anemia (subtype A)
MedDRA version: 20.0 Level: LLT Classification code 10055206 Term: Fanconi's anemia System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: RP-L102
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: CD34+CELLS
Other descriptive name: CD34+CELLS
Concentration unit: IU/kg international unit(s)/kilogram
Concentration type: not less then
Concentration number: 500000-
Rocket Pharmaceuticals, Inc.AuthorisedFemale: yes
Male: yes
5Phase 2Spain
No.TrialIDDate_
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6NCT03600909May 15, 201822 October 2019A 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 ConsortiumRecruiting1 MonthN/AAll70Phase 2United States
7NCT03476330May 8, 20181 April 2019Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi AnemiaQuercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi AnemiaFanconi Anemia;Squamous Cell CarcinomaDrug: Quercetin (dietary supplement)Children's Hospital Medical Center, CincinnatiRecruiting2 YearsN/AAll55Phase 2United States
8NCT03398824March 29, 201826 August 2019Pilot Study of Metformin for Patients With Fanconi AnemiaPilot Study of Metformin for Patients With Fanconi AnemiaFanconi AnemiaDrug: metformin HClBoston Children’s HospitalRecruiting6 Years35 YearsAll24Phase 2United States
9NCT02678533February 10, 201711 June 2019Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and PlerixaforPilot Study Assessing the Feasibility of CD34+ Cells Mobilization and Collection After Treatment With G-CSF and Plerixafor in Patients With Fanconi Anemia for Subsequent Treatment by Gene TherapyFanconi AnemiaDrug: G-CSF;Drug: PlerixaforAssistance Publique - Hôpitaux de ParisEuroFancolenRecruiting2 Years17 YearsAll8Phase 1/Phase 2France
10EUCTR2016-003226-16-IT03/02/20175 February 2018Follow-up of phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR aß+ T cells in pediatric patients affected by hematological disordersFollow-up of phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR aß+ T cells in pediatric patients affected by hematological disorders - CaspaCide TCR aß haplo HSCTHematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplastic syndromes;Congenital immune deficiencies;Severe aplastic anemia;Fanconi anemia; Osteopetrosis;Selected cases of hemoglobinopathies)
MedDRA version: 19.0 Level: HLGT Classification code 10018849 Term: Haematological disorders NEC System Organ Class: 10005329 - Blood and lymphatic system disorders ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: AP1903
Pharmaceutical Form: Solution for infusion
CAS Number: 195514-63-7
Current Sponsor code: AP1903
Other descriptive name: AP1903
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 0.4-
Bellicum Pharmaceuticals, Inc.AuthorisedFemale: yes
Male: yes
175Phase 1;Phase 2Italy
No.TrialIDDate_
enrollement
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11NCT03733249January 201728 October 2019Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical StudyFollow-up of Phase 1/2 Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR aß+T Cells in Pediatric Patients Affected by Hematological DisordersAcute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplastic Syndromes;Primary Immunodeficiency;Anemia, Aplastic;Hemoglobinopathies;Cytopenia;Fanconi Anemia;Diamond Blackfan Anemia;Thalassemia;Anemia, Sickle CellDrug: Rimiducid;Biological: rivogenlecleucelBellicum PharmaceuticalsRecruiting1 Month18 YearsAll193Phase 1/Phase 2Italy;Saudi Arabia;United Kingdom
12NCT03157804January 7, 201615 July 2019Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype AClinical Trial Phase I / II to Evaluate the Safety and Efficacy of the Infusion of Autologous CD34 + Cells Transduced With a Lentiviral Vector Carrying the Gene FANCA in Patients With FA Subtype A (FANCOLEN-1)Fanconi AnemiaProcedure: IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs);Biological: Genetically Engineered Hematopoietic Stem/Progenitor Cells;Other: Laboratory Biomarker Analysis;Biological: Filgrastim;Drug: Plerixafor;Procedure: Bone Marrow AspirationHospital Infantil Universitario Niño Jesús, Madrid, SpainCentro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT);Centro de Investigación en Red de Enfermedades Raras (CIBERER);Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz;Hospital Vall d'Hebron;Universitat Autonoma de BarcelonaNot recruiting1 Year21 YearsAll10Phase 1/Phase 2Spain
13EUCTR2014-004272-29-GB30/03/201528 February 2019Phase II clinical trial to evaluate safety and efficacy of mobilisation and collection of CD34+ cells after treatment with plerixafor and filgrastim in patients with Fanconi anaemia for subsequent transduction with a lentiviral vector carrying the FANCA gene and reinfusion into the patient - Fancostem-Plerixafor & Filgrastim mobilisation in Fanconi AnaemiaPhase II clinical trial to evaluate safety and efficacy of mobilisation and collection of CD34+ cells after treatment with plerixafor and filgrastim in patients with Fanconi anaemia for subsequent transduction with a lentiviral vector carrying the FANCA gene and reinfusion into the patient - Fancostem-Plerixafor & Filgrastim mobilisation in Fanconi AnaemiaFanconi Anaemia;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]Trade Name: Mozobil
Product Name: Mozobil
Pharmaceutical Form: Solution for injection
INN or Proposed INN: Mozobil
Current Sponsor code: 14-MI-10
Other descriptive name: Plerixafor
Concentration unit: mg/ml milligram(s)/millilitre
Concentration number: 20mg/ml-
Trade Name: Neupogen
Product Name: Neupogen
Pharmaceutical Form: Solution for injection in pre-filled syringe
INN or Proposed INN: Neupogen
Current Sponsor code: 14-MI-10
Other descriptive name: Filgrastim
Concentration unit: Munit million units
Concentration number: 30 Million Units -
Great Ormond Street Hospital NHS foundation TrustNot Recruiting Female: yes
Male: yes
20Phase 2France;Spain;United Kingdom
14NCT02065869April 20144 November 2019Safety Study of Gene Modified Donor T-cells Following TCR Alpha Beta Depleted Stem Cell TransplantPhase II Extension Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR aß+T Cells in Pediatric Patients Affected by Hematological DisordersAcute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplastic Syndrome;Primary Immunodeficiency;Anemia, Aplastic;Osteopetrosis;Hemoglobinopathies;Cytopenia;Fanconi Anemia;Diamond Blackfan Anemia;Thalassemia;Anemia, Sickle CellBiological: BPX-501 T cells;Drug: rimiducidBellicum PharmaceuticalsNot recruiting1 Month18 YearsAll193Phase 1/Phase 2Italy;United Kingdom;Germany;Spain;United States
15NCT02143830April 201422 July 2019HSCT for Patients With Fanconi Anemia Using Risk-Adjusted ChemotherapyA 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 FludarabineFanconi 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 cellChildren's Hospital Medical Center, CincinnatiMemorial Sloan Kettering Cancer Center;Fred Hutchinson Cancer Research CenterRecruiting3 MonthsN/AAll70Phase 2United States
No.TrialIDDate_
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PhaseCountries
16NCT01917708January 201419 November 2018BMT Abatacept for Non-Malignant DiseasesAbatacept for Post-Transplant Immune Suppression in Children and Adolescents Receiving Allogeneic Hematopoietic Stem Cell Transplants for Non-Malignant DiseasesHurler Syndrome;Fanconi Anemia;Glanzmann Thrombasthenia;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;Severe Congenital Neutropenia;Leukocyte Adhesion Deficiency;Shwachman-Diamond Syndrome;Diamond-Blackfan Anemia;Dyskeratosis-congenita;Chediak-Higashi Syndrome;Severe Aplastic Anemia;Thalassemia Major;Hemophagocytic Lymphohistiocytosis;Sickle Cell DiseaseDrug: AbataceptEmory UniversityNot recruitingN/A21 YearsAll10Phase 1United States
17EUCTR2014-005264-14-FR2 October 2017FancoMob: Pilote study for a combined treatment helping to collect stem cells in patient suffering Fanconi anemiaNA - EUROFANCOLENFanconi Anemia
MedDRA version: 18.1 Level: LLT Classification code 10055206 Term: Fanconi's anemia System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Zarzio
Product Name: Zarzio
Pharmaceutical Form: Solution for injection
INN or Proposed INN: filgrastim
Other descriptive name: G-CSF
Concentration unit: IU/ml international unit(s)/millilitre
Concentration type: range
Concentration number: 30MU/0,5ml- 48MU/0,5ml
Trade Name: Mozobil
Product Name: Mozobil
Pharmaceutical Form: Solution for injection
INN or Proposed INN: plérixafor
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 20-
ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)Not AvailableFemale: yes
Male: yes
Phase 2France
18EUCTR2011-006100-12-ES12/04/201313 May 2013Clinical Trial Phase I / II to evaluate the safety and efficacy of the infusion of cells transduced with a therapeutic lentiviral vector for patients with Fanconi Anemia Subtype A.Clinical Trial Phase I / II to evaluate the safety and efficacy of the infusion of autologous CD34+ cells transduced with a lentiviral vector carrying the FANCA gene (orphan drug) for patients with Fanconi Anemia Subtype A. - Fancolen-1Fanconi anemia (Subtype A)
MedDRA version: 15.1 Level: LLT Classification code 10055206 Term: Fanconi's anemia System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: CD34+ Cells
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: CD34+ CELLS
Other descriptive name: CD34+ CELLS
Concentration unit: IU/kg international unit(s)/kilogram
Concentration type: not less then
Concentration number: 100.000-
FUNDACION PARA LA INVESTIGACION BIOMEDICA DEL HOSPITAL UNIVERSITARIO NIÑO JESUSAuthorisedFemale: yes
Male: yes
5Phase 1Spain
19NCT01720147July 201222 July 2019Quercetin in Children With Fanconi Anemia; a Pilot StudyQuercetin in Children With Fanconi Anemia; a Pilot StudyFanconi AnemiaDrug: Quercetin (dietary supplement)Children's Hospital Medical Center, CincinnatiFood and Drug Administration (FDA)RecruitingN/AN/AAll32Phase 1United States
20NCT01331018February 22, 20128 April 2019Gene Therapy for Fanconi AnemiaGene Transfer for Patients With Fanconi Anemia Complementation Group A (FANCA)Fanconi AnemiaProcedure: Bone Marrow Aspiration;Biological: Filgrastim;Biological: Genetically Engineered Hematopoietic Stem Progenitor Cells;Other: Laboratory Biomarker Analysis;Procedure: Leukapheresis;Drug: Methylprednisolone;Drug: Plerixafor;Drug: PrednisoneFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI);Rocket Pharma LimitedNot recruiting4 YearsN/AAll3Phase 1United States
No.TrialIDDate_
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21NCT02127905March 201116 December 2017Unrelated HSCT in Patients With Fanconi AnemiaA Study of Total Body Irradiation, Cyclophosphamide and Fludarabine Followed by Alternated Donor Hematopoietic Cell Transplantation in Patients With Fanconi AnemiaFanconi AnemiaBiological: CD34+ selected cellsNeena Kapoor, M.D.Not recruiting8 Weeks21 YearsAll0N/AUnited States
22NCT01319851September 201016 December 2017Alefacept and Allogeneic Hematopoietic Stem Cell TransplantationAlefacept and Allogeneic Hematopoietic Stem Cell Transplantation for Children With Non-Malignant Diseases Who Have Been Multiply Transfused: a Pilot StudyThalassemia;Sickle Cell Disease;Glanzmann Thrombasthenia;Wiskott-Aldrich Syndrome;Chronic-granulomatous Disease;Severe Congenital Neutropenia;Leukocyte Adhesion Deficiency;Schwachman-Diamond Syndrome;Diamond-Blackfan Anemia;Fanconi Anemia;Dyskeratosis-congenita;Chediak-Higashi Syndrome;Severe Aplastic AnemiaDrug: AlefaceptEmory UniversityChildren's Healthcare of AtlantaNot recruitingN/A21 YearsAll3N/AUnited States
23NCT01001598November 200919 February 2015Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis CongenitaPhase I/II Dose Escalation Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis CongenitaFanconi Anemia;Dyskeratosis CongenitaDrug: danazolChildren's Hospital BostonNot recruiting3 YearsN/ABoth5Phase 1/Phase 2United States
24NCT01082133October 200911 July 2016Multicenter 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, CincinnatiNot recruitingN/AN/ABoth45Phase 2United States
25NCT00987480September 25, 200916 December 2017Hematopoietic 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 CenterNot recruitingN/AN/AAll11Phase 2United States
No.TrialIDDate_
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26NCT01071239April 200916 September 2019Hematopoietic 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 CenterNot recruitingN/AN/AAll1Phase 2United States
27NCT00856388January 14, 200918 December 2018Fludarabine 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 MacroglobulinemiaProcedure: allogeneic hematopoietic stem cell transplantation;Biological: anti-thymocyte globulin;Drug: fludarabine phosphate;Drug: melphalan;Radiation: total-body irradiationRoswell Park Cancer InstituteNational Cancer Institute (NCI)Not recruiting3 Years75 YearsAll62N/AUnited States
28NCT00479115May 200719 February 2015Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100AMD3100 in Combination With G-CSF to Mobilize Peripheral Blood Stem Cells in Patients With Fanconi Anemia(FA): A Phase I/II StudyFanconi AnemiaDrug: AMD3100;Device: AmCell CliniMACsChildren's Hospital Medical Center, CincinnatiNot recruiting1 Year30 YearsBoth1Phase 1/Phase 2United States
29NCT00453388February 200727 May 2019Fludarabine 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)Not recruitingN/AN/AAll6Phase 2United States;Brazil
30NCT00352976May 18, 200628 January 2019TBI Dose De-escalation for Fanconi AnemiaTotal Body Irradiation Dose De-escalation Study in Patients With Fanconi Anemia Undergoing Alternate Donor Hematopoietic Cell TransplantationFanconi AnemiaDrug: Cyclophosphamide;Drug: Fludarabine;Procedure: Total Body Irradiation;Procedure: Bone Marrow Transplantation;Drug: Mycophenolate Mofetil;Drug: SirolimusMasonic Cancer Center, University of MinnesotaRecruitingN/AN/AAll120Phase 2/Phase 3United States
No.TrialIDDate_
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31NCT00965666October 200519 February 2015Pilot Study of Etanercept (Enbrel) in Children With Fanconi AnemiaEtanercept (Enbrel) in Children With Fanconi Anemia and Early Bone Marrow Failure: A Pilot StudyFanconi AnemiaDrug: EtanerceptChildren's Hospital Medical Center, CincinnatiAmgenNot recruiting4 YearsN/ABoth3N/AUnited States
32EUCTR2004-004914-18-IT21/01/20057 January 2013A pilot multicentric study for the use of the anti-TNF alfa in patients with: 1) Fanconi Anemia with marrow failure with no bone marrow compatible donor 2) In patients with acquired aplastic anemia failing immunosuppression and with no bone marrow donorA pilot multicentric study for the use of the anti-TNF alfa in patients with: 1) Fanconi Anemia with marrow failure with no bone marrow compatible donor 2) In patients with acquired aplastic anemia failing immunosuppression and with no bone marrow donorMarrow failure in patients refractary/non eligible to conventional treatments;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]Trade Name: ENBREL*SC 4FL 25MG+4SIR 1ML
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: Etanercept
CAS Number: 185243-69-0
Concentration unit: mg milligram(s)
Concentration number: .4-
ISTITUTO GIANNINA GASLINIAuthorisedFemale: yes
Male: yes
Italy
33NCT00243399July 200419 February 2015Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi AnemiaA Pilot Trial of Oxandrolone for the Treatment of Bone Marrow Aplasia in Patients With Fanconi AnemiaFanconi AnemiaDrug: OxandroloneChildren's Hospital Medical Center, CincinnatiFDA Office of Orphan Products DevelopmentNot recruitingN/AN/ABoth10Phase 1United States
34NCT00167206March 20042 September 2019Stem 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 MinnesotaNot recruitingN/A18 YearsAll16Phase 1/Phase 2United States
35NCT00084695September 200319 February 2015Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant DiseasesThe Use Of Umbilical Cord Blood As A Source Of Hematopoietic Stem CellsChildhood Langerhans Cell Histiocytosis;Fanconi Anemia;Leukemia;Lymphoma;Myelodysplastic Syndromes;Neuroblastoma;Sarcoma;Unspecified Childhood Solid Tumor, Protocol SpecificBiological: anti-thymocyte globulin;Drug: busulfan;Drug: cyclophosphamide;Drug: fludarabine phosphate;Drug: melphalan;Drug: methylprednisolone;Radiation: radiation therapyMilton S. Hershey Medical CenterRecruitingN/A21 YearsBoth25Phase 2United States
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36NCT01019876June 200219 February 2015Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant DiseasesRisk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Selected Non-Malignant DiseasesBone Marrow Failure;Osteopetrosis;Fanconi Anemia;Severe Combined ImmunodeficiencyDrug: Fludarabine;Drug: Cyclophosphamide;Drug: Cyclophosphamide 40;Drug: Cyclophosphamide 30Columbia UniversityRecruitingN/A30 YearsBoth50Phase 2/Phase 3United States
37NCT00258427March 26, 200225 February 2019Hematopoietic 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 MinnesotaRecruitingN/A44 YearsAll25Phase 2United States
38NCT00586274March 200219 February 2015Use of Rft5-Dga to Deplete Alloreactive Cells for Pts With Fanconi Anemia After Haploidentical SCTA Phase I Study Evaluating The Use Of Rft5-Dga To Deplete Alloreactive Cells For Patients With Fanconi Anemia After Haploidentical Stem Cell TransplantationFANCONI ANEMIAProcedure: CD34 selected haploidentical PBSCT;Drug: Fludarabine;Biological: T cell infusion;Biological: Campath 1h;Biological: anti-CD45Baylor College of MedicineUTSW-Dallas;Center for Cell and Gene Therapy, Baylor College of MedicineNot recruitingN/A64 YearsBoth1Phase 1United States
39NCT00590460July 200111 June 2018Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Fanconi AnemiaCd45 (Yth-24 and Yth 54) and Cd52 (Campath-1H) Monoclonal Antibody Conditioning Regimen for Allogeneic Donor Stem Cell Transplantation of Patients With Fanconi AnemiaFanconi Anemia;Severe Aplastic AnemiaBiological: CAMPATH-1H;Biological: Anti-CD45;Drug: Fludarabine;Procedure: Stem cell infusionBaylor College of MedicineTexas Children's Hospital;The Methodist Hospital System;Center for Cell and Gene Therapy, Baylor College of MedicineNot recruitingN/AN/AAll5Phase 1/Phase 2United States
40NCT00595127June 200119 October 2017Hematopoietic 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.Not recruitingN/AN/AAll21N/AUnited States
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41NCT00305708August 200019 February 2015Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First RemissionBone Marrow Stem Cell Transplantation for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myeloid Leukemia in 1RemissionCongenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Fanconi Anemia;Leukemia;Severe Congenital Neutropenia;ThrombocytopeniaBiological: anti-thymocyte globulin;Drug: busulfan;Drug: fludarabine phosphate;Procedure: allogeneic bone marrow transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantation;Radiation: radiation therapyUniversity of California, San FranciscoNational Cancer Institute (NCI)Not recruitingN/A17 YearsBoth40Phase 1/Phase 2United States
42NCT00630253February 17, 200028 January 2019Cytoxan, 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 MinnesotaRecruitingN/A59 YearsAll45Phase 1/Phase 2United States
43NCT00093743January 200016 December 2017Low-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)Not recruitingN/AN/AAll2Phase 1United States
44NCT00290628October 199916 December 2017Donor Umbilical Cord Blood Transplant in Treating Patients With Hematologic CancerTransplantation of Umbilical Cord Blood From Related and Unrelated DonorsChronic Myeloproliferative Disorders;Diamond-blackfan Anemia;Fanconi Anemia;Graft Versus Host Disease;Leukemia;Lymphoma;Multiple Myeloma and Plasma Cell Neoplasm;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative DiseasesDrug: cyclophosphamide;Drug: cyclosporine;Drug: filgrastim;Drug: melphalan;Drug: methylprednisolone;Drug: mycophenolate mofetil;Procedure: radiation therapy;Procedure: umbilical cord blood transplantation;Drug: anti-thymocyte globulin;Drug: busulfanMasonic Cancer Center, University of MinnesotaNational Cancer Institute (NCI)Not recruitingN/A45 YearsAll43N/AUnited States
45NCT00317876June 199819 February 2015Cyclophosphamide 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)Not recruitingN/AN/ABoth25Phase 1United States;Brazil
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46NCT00001399December 3, 199316 December 2017Gene Therapy for the Treatment of Fanconi's Anemia Type CRetroviral Mediated Gene Transfer of the Fanconi Anemia Complementation Group C Gene to Hematopoietic Progenitors of Group C PatientsFanconi's Anemia;PancytopeniaDrug: Transduced CD34+ CellsNational Heart, Lung, and Blood Institute (NHLBI)Not recruiting5 YearsN/AAll9Phase 1United States

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