285. ファンコニ貧血 [臨床試験数:56,薬物数:111(DrugBank:31),標的遺伝子数:30,標的パスウェイ数:151

Searched query = "Fanconi anemia"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
13 trials found
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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PhaseCountries
1NCT03579875November 13, 201825/5/2018T 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: Rituximab;Drug: BusulfanMasonic Cancer Center, University of MinnesotaNULLRecruitingN/A65 YearsAll48Phase 2United States
2NCT03600909May 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 ConsortiumRecruiting1 MonthN/AAll70Phase 2United States
3NCT02678533February 10, 20175/2/2016Mobilization 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
4NCT03157804January 7, 201626/4/2017Lentiviral-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 BarcelonaActive, not recruiting1 Year21 YearsAll9Phase 1/Phase 2Spain
5EUCTR2014-004272-29-GB30/03/201515/07/2015Phase 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 Anaemia Fanconi Anaemia;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]Great Ormond Street Hospital NHS foundation TrustNULLNot Recruiting Female: yes
Male: yes
20 Human pharmacology (Phase 1): no Therapeut1c exploratory (Phase 2): yes Therapeut1c conf1rmatory - (Phase 3): no Therapeut1c use (Phase 4): noFrance;Spain;United Kingdom
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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agemin
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PhaseCountries
6NCT02143830April 201428/4/2014HSCT 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
7NCT02931071September 20136/9/2016Clinical Phase II Trial to Evaluate CD34+ Cells Mobilization and Collection in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene. FANCOSTEM-1Clinical Phase II Trial to Evaluate Efficacy and Safety of CD34+ Cells Mobilization and Collection After Treatment With Plerixafor and Filgrastim in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene and Reinfusion in the PatientFanconi AnemiaDrug: filgrastim;Drug: plerixaforHospital Universitari Vall d'Hebron Research InstituteCIEMAT;CIBERERCompleted2 Years64 YearsAll13Phase 2Spain
8NCT01331018February 22, 201216/3/2011Gene 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 LimitedActive, not recruiting4 YearsN/AAll3Phase 1United States
9NCT00479115May 200723/5/2007Mobilization 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, CincinnatiNational Heart, Lung, and Blood Institute (NHLBI)Completed1 Year30 YearsAll1Phase 1/Phase 2United States
10NCT00258427March 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
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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agemin
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PhaseCountries
11NCT00630253February 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
12NCT00290628October 19999/2/2006Donor 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)TerminatedN/A45 YearsAll43N/AUnited States
13EUCTR2014-005264-14-FR19/06/2015FancoMob: Pilote study for a combined treatment helping to collect stem cells in patient suffering Fanconi anemiaNA - EUROFANCOLEN Fanconi Anemia
MedDRA version: 18.1Level: LLTClassification code 10055206Term: Fanconi's anemiaSystem Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Trade Name: Zarzio
Product Name: Zarzio
INN or Proposed INN: filgrastim
Other descriptive name: G-CSF
Trade Name: Mozobil
Product Name: Mozobil
INN or Proposed INN: plérixafor
ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)NULLNAFemale: yes
Male: yes
Phase 2France