285. ファンコニ貧血 Fanconi anemia Clinical trials / Disease details
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. | 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 | 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 | Completed | 1 Month | N/A | All | 3 | Phase 2 | United States |
| 3 | NCT02678533 (ClinicalTrials.gov) | February 10, 2017 | 5/2/2016 | Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and Plerixafor | Pilot 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 Therapy | Fanconi Anemia | Drug: G-CSF;Drug: Plerixafor | Assistance Publique - Hôpitaux de Paris | EuroFancolen | Completed | 2 Years | 17 Years | All | 4 | Phase 1/Phase 2 | France |
| 4 | NCT03157804 (ClinicalTrials.gov) | January 7, 2016 | 26/4/2017 | Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients 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 Gene FANCA in Patients With FA Subtype A (FANCOLEN-1) | Fanconi Anemia | Procedure: 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 Aspiration | Hospital Infantil Universitario Niño Jesús, Madrid, Spain | Centro 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 Barcelona | Active, not recruiting | 1 Year | 21 Years | All | 9 | Phase 1/Phase 2 | Spain |
| 5 | EUCTR2014-004272-29-GB (EUCTR) | 30/03/2015 | 15/07/2015 | Phase 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 | Phase 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] | Trade Name: Mozobil Product Name: Mozobil INN or Proposed INN: Mozobil Other descriptive name: Plerixafor Trade Name: Neupogen Product Name: Neupogen INN or Proposed INN: Neupogen Other descriptive name: Filgrastim | Great Ormond Street Hospital NHS foundation Trust | NULL | Not Recruiting | Female: yes Male: yes | 20 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;Spain;United Kingdom | ||
| 6 | 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 |
| 7 | NCT02931071 (ClinicalTrials.gov) | September 2013 | 6/9/2016 | Clinical 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-1 | Clinical 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 Patient | Fanconi Anemia | Drug: filgrastim;Drug: plerixafor | Hospital Universitari Vall d'Hebron Research Institute | CIEMAT;CIBERER | Completed | 2 Years | 64 Years | All | 13 | Phase 2 | Spain |
| 8 | NCT01331018 (ClinicalTrials.gov) | February 22, 2012 | 16/3/2011 | Gene Therapy for Fanconi Anemia | Gene Transfer for Patients With Fanconi Anemia Complementation Group A (FANCA) | Fanconi Anemia | Procedure: Bone Marrow Aspiration;Biological: Filgrastim;Biological: Genetically Engineered Hematopoietic Stem Progenitor Cells;Other: Laboratory Biomarker Analysis;Procedure: Leukapheresis;Drug: Methylprednisolone;Drug: Plerixafor;Drug: Prednisone | Fred Hutchinson Cancer Research Center | National Heart, Lung, and Blood Institute (NHLBI);Rocket Pharma Limited | Active, not recruiting | 4 Years | N/A | All | 3 | Phase 1 | United States |
| 9 | NCT00479115 (ClinicalTrials.gov) | May 2007 | 23/5/2007 | Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and AMD3100 | AMD3100 in Combination With G-CSF to Mobilize Peripheral Blood Stem Cells in Patients With Fanconi Anemia(FA): A Phase I/II Study | Fanconi Anemia | Drug: AMD3100;Device: AmCell CliniMACs | Children's Hospital Medical Center, Cincinnati | National Heart, Lung, and Blood Institute (NHLBI) | Completed | 1 Year | 30 Years | All | 1 | Phase 1/Phase 2 | United States |
| 10 | 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 |
| 11 | NCT00630253 (ClinicalTrials.gov) | February 17, 2000 | 5/3/2008 | Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia | A Study of Cyclophosphamide, Fludarabine, and Antithymocyte Globulin Followed by Matched Sibling Donor Hematopoietic Cell Transplantation in Patients With Fanconi Anemia | Fanconi Anemia | Biological: Anti-Thymocyte Globulin;Drug: Cyclophosphamide;Drug: Fludarabine;Procedure: Hematopoietic Stem Cell Transplantation;Drug: Methylprednisolone;Drug: Filgrastim;Drug: Cyclosporine;Drug: Mycophenolate Mofetil | Masonic Cancer Center, University of Minnesota | NULL | Completed | N/A | 59 Years | All | 31 | Phase 1/Phase 2 | United States |
| 12 | 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 |
| 13 | EUCTR2014-005264-14-FR (EUCTR) | 19/06/2015 | FancoMob: Pilote study for a combined treatment helping to collect stem cells in patient suffering Fanconi anemia | NA - EUROFANCOLEN | Fanconi 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 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) | NULL | NA | Female: yes Male: yes | Phase 2 | France |