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 | NCT04784052 (ClinicalTrials.gov) | December 7, 2021 | 2/3/2021 | Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing JSP191 | TCRaß+ T-cell/CD19+ B-cell Depleted Hematopoietic Grafts and a Reduced Intensity Preparative Conditioning Regimen Containing JSP191 to Achieve Engraftment and Blood Reconstitution in Patients With Fanconi Anemia | Fanconi Anemia | Drug: JSP191;Device: CliniMACS Prodigy System;Biological: Depleted Stem Cell Transplant;Biological: Rabbit Anti-Thymoglobulin (rATG);Drug: Cyclophosphamide;Drug: Fludarabine;Drug: Rituximab | Rajni Agarwal | NULL | Recruiting | 2 Years | N/A | All | 12 | Phase 1/Phase 2 | United States |
| 2 | NCT04522375 (ClinicalTrials.gov) | October 2021 | 3/8/2020 | Phase I/II Study of FP-045 in Patients With Fanconi Anemia | A Phase 1/2 Multicenter, Dose Escalation and Expansion Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Activity of FP-045 in Patients With Fanconi Anemia | Fanconi Anemia | Drug: FP-045 | Foresee Pharmaceuticals Co., Ltd. | NULL | Not yet recruiting | 3 Years | 18 Years | All | 56 | Phase 1/Phase 2 | NULL |
| 3 | NCT04483544 (ClinicalTrials.gov) | August 10, 2020 | 20/7/2020 | Pembrolizumab and Olaparib in Cervical Cancer Patients | Immunotherapy in Combination With PARP Inhibition in Advanced Cervical Cancer Patients Functionally Competent or Deficient for the Fanconi Anemia Repair Pathway | Cervical Cancer;Cervical Carcinoma | Drug: pembrolizumab;Drug: olaparib | Baptist Health South Florida | Florida Department of Health;Merck Sharp & Dohme Corp. | Recruiting | 18 Years | N/A | Female | 48 | Phase 2 | United States |
| 4 | NCT04248439 (ClinicalTrials.gov) | July 15, 2020 | 24/1/2020 | Gene Therapy for Fanconi Anemia, Complementation Group A | A Phase 2 Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene in Pediatric Subjects With Fanconi Anemia Subtype A | Fanconi Anemia Complementation Group A | Biological: RP-L102 | Rocket Pharmaceuticals Inc. | NULL | Recruiting | 1 Year | N/A | All | 5 | Phase 2 | United States |
| 5 | NCT04042831 (ClinicalTrials.gov) | June 9, 2020 | 29/7/2019 | Olaparib in Treating Patients With Metastatic Biliary Tract Cancer With Aberrant DNA Repair Gene Mutations | A Phase II Study of Olaparib in Patients With Advanced Biliary Tract Cancer With Aberrant DNA Repair Gene Mutations | BRCA2 Gene Mutation;BRIP1 Gene Mutation;CHEK2 Gene Mutation;EMSY Gene Mutation;Fanconi Anemia Complementation Group Gene Mutation;Metastatic Bile Duct Carcinoma;MRE11 Gene Mutation;NBN Gene Mutation;PALB2 Gene Mutation;PTEN Gene Deletion;RAD51 Gene Mutation;ARID1A Gene Mutation;ATM Gene Mutation;ATR Gene Mutation;Bile Duct Adenocarcinoma;BRCA1 Gene Mutation | Drug: Olaparib | Academic and Community Cancer Research United | National Cancer Institute (NCI) | Recruiting | 18 Years | N/A | All | 36 | Phase 2 | United States |
| 6 | EUCTR2018-002502-31-GB (EUCTR) | 07/02/2020 | 08/10/2019 | A 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 | A 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-II | Fanconi 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 Code: RP-L102 INN or Proposed INN: CD34+CELLS Other descriptive name: CD34+CELLS | Rocket Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 2 | Spain;United Kingdom | ||
| 7 | NCT04069533 (ClinicalTrials.gov) | November 28, 2019 | 23/8/2019 | Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A | A 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 | Fanconi Anemia Complementation Group A | Biological: RP-L102 | Rocket Pharmaceuticals Inc. | NULL | Recruiting | 1 Year | 17 Years | All | 5 | Phase 2 | Spain |
| 8 | NCT03814408 (ClinicalTrials.gov) | January 11, 2019 | 14/1/2019 | A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A | A 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 A | Fanconi Anemia Complementation Group A | Biological: RP-L102 | Rocket Pharmaceuticals Inc. | NULL | Active, not recruiting | 1 Year | 12 Years | All | 2 | Phase 1 | United States |
| 9 | 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 |
| 10 | NCT03206086 (ClinicalTrials.gov) | November 2, 2018 | 30/6/2017 | Eltrombopag for People With Fanconi Anemia | Eltrombopag for Patients With Fanconi Anemia | Fanconi Anemia | Drug: Eltrombopag | National Heart, Lung, and Blood Institute (NHLBI) | NULL | Recruiting | 2 Years | N/A | All | 35 | Phase 2 | United States |
| 11 | EUCTR2018-002502-31-ES (EUCTR) | 31/10/2018 | 02/08/2018 | A 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 | A 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-II | Fanconi 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 INN or Proposed INN: CD34+CELLS Other descriptive name: CD34+CELLS | Rocket Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 2 | Spain | ||
| 12 | 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 |
| 13 | NCT03476330 (ClinicalTrials.gov) | May 8, 2018 | 19/3/2018 | Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia | Quercetin Chemoprevention for Squamous Cell Carcinoma in Patients With Fanconi Anemia | Fanconi Anemia;Squamous Cell Carcinoma | Drug: Quercetin (dietary supplement) | Children's Hospital Medical Center, Cincinnati | NULL | Recruiting | 2 Years | N/A | All | 55 | Phase 2 | United States |
| 14 | NCT03398824 (ClinicalTrials.gov) | March 29, 2018 | 13/12/2017 | Pilot Study of Metformin for Patients With Fanconi Anemia | Pilot Study of Metformin for Patients With Fanconi Anemia | Fanconi Anemia | Drug: metformin HCl | Boston Children's Hospital | NULL | Completed | 6 Years | 35 Years | All | 15 | Phase 2 | United States |
| 15 | EUCTR2014-000584-41-ES (EUCTR) | 08/06/2017 | 21/03/2017 | Phase 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 disorders | Phase 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 disorders - CaspaCide TCR aß haplo HSCT | Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplasticsyndromes;Congenital immune deficiencies;Severe aplastic anemia;Fanconi anemia; Osteopetrosis;Selected cases of hemoglobinopathies) MedDRA version: 19.1;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: BPX-501 cells INN or Proposed INN: rivogenleucleucel Other descriptive name: BPX-501 Product Name: AP1903 INN or Proposed INN: Rimiducid Other descriptive name: AP1903 | Bellicum Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 175 | Phase 2 | Spain;United Kingdom;Italy | ||
| 16 | NCT03609840 (ClinicalTrials.gov) | May 24, 2017 | 5/1/2018 | Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients | Population 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 Disease | Drug: Thiotepa | University of California, San Francisco | NULL | Active, not recruiting | N/A | 17 Years | All | 25 | United States | |
| 17 | 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 |
| 18 | EUCTR2016-003226-16-IT (EUCTR) | 03/02/2017 | 17/11/2016 | Follow-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 | Follow-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 HSCT | Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplasticsyndromes;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 Other descriptive name: AP1903 | Bellicum Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 175 | Phase 1;Phase 2 | Italy | ||
| 19 | NCT03733249 (ClinicalTrials.gov) | January 2017 | 2/11/2018 | Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study | Follow-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 Disorders | Acute 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 Cell | Drug: Rimiducid;Biological: rivogenlecleucel | Bellicum Pharmaceuticals | NULL | Enrolling by invitation | 1 Month | 18 Years | All | 193 | Phase 1/Phase 2 | Italy;Saudi Arabia;United Kingdom |
| 20 | 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 |
| 21 | 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 | ||
| 22 | EUCTR2014-000584-41-IT (EUCTR) | 23/10/2014 | 15/04/2014 | 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 | 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 HSCT | Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplasticsyndromes;Congenital immune deficiencies;Severe aplastic anemia;Fanconi anemia; Osteopetrosis;Selected cases of hemoglobinopathies) MedDRA version: 16.1;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: BPX-501 cells Product Name: AP1903 | Bellicum Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 1;Phase 2 | Italy;United Kingdom;Spain | ||
| 23 | NCT02065869 (ClinicalTrials.gov) | April 2014 | 13/2/2014 | Safety Study of Gene Modified Donor T-cells Following TCR Alpha Beta Depleted Stem Cell Transplant | Phase 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 Disorders | Acute 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 Cell | Biological: BPX-501 T cells;Drug: rimiducid | Bellicum Pharmaceuticals | NULL | Active, not recruiting | 1 Month | 18 Years | All | 193 | Phase 2 | Italy;United Kingdom;Germany;Spain;United States |
| 24 | 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 |
| 25 | NCT01917708 (ClinicalTrials.gov) | January 2014 | 24/7/2013 | Bone Marrow Transplant With Abatacept for Non-Malignant Diseases | Abatacept for Post-Transplant Immune Suppression in Children and Adolescents Receiving Allogeneic Hematopoietic Stem Cell Transplants for Non-Malignant Diseases | Hurler 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 Disease | Drug: Abatacept | Emory University | NULL | Completed | N/A | 21 Years | All | 10 | Phase 1 | United States |
| 26 | 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 |
| 27 | EUCTR2011-006100-12-ES (EUCTR) | 12/04/2013 | 27/01/2012 | Clinical 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-1 | Fanconi 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 INN or Proposed INN: CD34+ CELLS Other descriptive name: CD34+ CELLS | FUNDACION PARA LA INVESTIGACION BIOMEDICA DEL HOSPITAL UNIVERSITARIO NIÑO JESUS | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 1 | Spain | ||
| 28 | NCT01720147 (ClinicalTrials.gov) | July 2012 | 25/7/2012 | Quercetin in Children With Fanconi Anemia; a Pilot Study | Quercetin in Children With Fanconi Anemia; a Pilot Study | Fanconi Anemia | Drug: Quercetin (dietary supplement) | Children's Hospital Medical Center, Cincinnati | Food and Drug Administration (FDA) | Recruiting | N/A | N/A | All | 32 | Phase 1 | United States |
| 29 | 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 |
| 30 | NCT02127905 (ClinicalTrials.gov) | March 2011 | 19/10/2012 | Unrelated HSCT in Patients With Fanconi Anemia | A Study of Total Body Irradiation, Cyclophosphamide and Fludarabine Followed by Alternated Donor Hematopoietic Cell Transplantation in Patients With Fanconi Anemia | Fanconi Anemia | Biological: CD34+ selected cells | Neena Kapoor, M.D. | NULL | Withdrawn | 8 Weeks | 21 Years | All | 0 | N/A | United States |
| 31 | NCT01316549 (ClinicalTrials.gov) | January 1, 2011 | 14/3/2011 | Study of Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation | Population Pharmacokinetics of Fludarabine in Pediatric Patients Undergoing Hematopoietic Cell Transplantation | Hematologic Malignancies;Nonmalignant Diseases;Immunodeficiencies;Hemoglobinopathies;Genetic Inborn Errors of Metabolism;Fanconi Anemia;Thalassemia;Sickle Cell Disease | Drug: Fludarabine | University of California, San Francisco | Thrasher Research Fund | Completed | N/A | 17 Years | All | 67 | United States | |
| 32 | NCT01319851 (ClinicalTrials.gov) | September 2010 | 15/9/2010 | Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation | Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation for Children With Non-Malignant Diseases Who Have Been Multiply Transfused: a Pilot Study | Thalassemia;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 Anemia | Drug: Alefacept | Emory University | Children's Healthcare of Atlanta | Terminated | N/A | 21 Years | All | 3 | N/A | United States |
| 33 | NCT01001598 (ClinicalTrials.gov) | November 2009 | 22/10/2009 | Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita | Phase I/II Dose Escalation Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita | Fanconi Anemia;Dyskeratosis Congenita | Drug: danazol | Boston Children's Hospital | NULL | Terminated | 3 Years | N/A | All | 5 | Phase 1/Phase 2 | United States |
| 34 | NCT01082133 (ClinicalTrials.gov) | October 2009 | 5/3/2010 | Multicenter Transplant Study for Fanconi Anemia | A Multicenter Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine | Fanconi Anemia | Drug: Chemotherapy;Biological: Miltenyi CliniMACS | Children's Hospital Medical Center, Cincinnati | NULL | Completed | N/A | N/A | Both | 45 | Phase 2 | United States |
| 35 | NCT00987480 (ClinicalTrials.gov) | September 25, 2009 | 30/9/2009 | Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine | A Multicenter Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine | Aplastic Anemia;Leukemia;Myelodysplastic Syndrome | Drug: Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine.;Device: CliniMACS device | Memorial Sloan Kettering Cancer Center | Boston Children's Hospital;Children's Hospital Medical Center, Cincinnati;Children's Hospital and Health System Foundation, Wisconsin;Rockefeller University;Fred Hutchinson Cancer Research Center | Completed | N/A | N/A | All | 45 | Phase 2 | United States |
| 36 | NCT01071239 (ClinicalTrials.gov) | April 2009 | 20/5/2009 | Hematopoietic Stem Cell Transplant for Fanconi Anemia | A Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine | Fanconi Anemia | Device: CliniMACs device;Drug: Busulfan;Drug: Fludarabine;Drug: Cyclophosphamide;Drug: ATG | Medical College of Wisconsin | Memorial Sloan Kettering Cancer Center | Completed | N/A | N/A | All | 1 | Phase 2 | United States |
| 37 | NCT00856388 (ClinicalTrials.gov) | January 14, 2009 | 4/3/2009 | Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders | A Pilot Trial Of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan, And Low Dose Total Body Irradiation | Accelerated 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 Macroglobulinemia | Drug: fludarabine phosphate;Drug: melphalan;Radiation: total-body irradiation;Procedure: allogeneic hematopoietic stem cell transplantation;Biological: anti-thymocyte globulin | Roswell Park Cancer Institute | National Cancer Institute (NCI) | Completed | 3 Years | 75 Years | All | 62 | N/A | United States |
| 38 | 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 |
| 39 | NCT00453388 (ClinicalTrials.gov) | February 2007 | 27/3/2007 | Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant, Mycophenolate Mofetil, and Cyclosporine in Treating Patients With Fanconi Anemia | Nonmyeloablative Hematopoietic Cell Transplantation for Patients With Fanconi Anemia Using Alternative Marrow Donors: A Phase II Dose-Finding Study | Acute Myeloid Leukemia in Remission;de Novo Myelodysplastic Syndrome;Fanconi Anemia;Previously Treated Myelodysplastic Syndrome | Procedure: 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 Irradiation | Fred Hutchinson Cancer Research Center | National Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI) | Completed | N/A | N/A | All | 6 | Phase 2 | United States;Brazil |
| 40 | NCT00352976 (ClinicalTrials.gov) | May 18, 2006 | 14/7/2006 | TBI Dose De-escalation for Fanconi Anemia | Total Body Irradiation Dose De-escalation Study in Patients With Fanconi Anemia Undergoing Alternate Donor Hematopoietic Cell Transplantation | Fanconi Anemia | Drug: Cyclophosphamide;Drug: Fludarabine;Procedure: Total Body Irradiation;Procedure: Bone Marrow Transplantation;Drug: Mycophenolate Mofetil;Drug: Sirolimus | Masonic Cancer Center, University of Minnesota | NULL | Completed | N/A | N/A | All | 88 | Phase 2/Phase 3 | United States |
| 41 | NCT00965666 (ClinicalTrials.gov) | October 2005 | 24/8/2009 | Pilot Study of Etanercept (Enbrel) in Children With Fanconi Anemia | Etanercept (Enbrel) in Children With Fanconi Anemia and Early Bone Marrow Failure: A Pilot Study | Fanconi Anemia | Drug: Etanercept | Children's Hospital Medical Center, Cincinnati | Amgen | Completed | 4 Years | N/A | All | 3 | Early Phase 1 | United States |
| 42 | EUCTR2004-004914-18-IT (EUCTR) | 21/01/2005 | 27/07/2005 | A 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 donor | A 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 donor | Marrow 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 INN or Proposed INN: Etanercept | ISTITUTO GIANNINA GASLINI | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Italy | ||||
| 43 | NCT00243399 (ClinicalTrials.gov) | July 2004 | 20/10/2005 | Oxandrolone for the Treatment of Bone Marrow Aplasia in Fanconi Anemia | A Pilot Trial of Oxandrolone for the Treatment of Bone Marrow Aplasia in Patients With Fanconi Anemia | Fanconi Anemia | Drug: Oxandrolone | Children's Hospital Medical Center, Cincinnati | FDA Office of Orphan Products Development | Completed | N/A | N/A | Both | 10 | Phase 1 | United States |
| 44 | NCT00167206 (ClinicalTrials.gov) | March 2004 | 9/9/2005 | Stem Cell Transplantation for Fanconi Anemia | A 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 Anemia | Fanconi Anemia | Procedure: Hematopoietic Stem Cell Transplant;Procedure: Thymic Shielding During Radiation;Procedure: Total Body Irradiation;Drug: Cyclophosphamide, Fludarabine | Masonic Cancer Center, University of Minnesota | NULL | Terminated | N/A | 18 Years | All | 16 | Phase 1/Phase 2 | United States |
| 45 | NCT00084695 (ClinicalTrials.gov) | September 2003 | 10/6/2004 | Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant Diseases | The Use Of Umbilical Cord Blood As A Source Of Hematopoietic Stem Cells | Childhood Langerhans Cell Histiocytosis;Fanconi Anemia;Leukemia;Lymphoma;Myelodysplastic Syndromes;Neuroblastoma;Sarcoma;Unspecified Childhood Solid Tumor, Protocol Specific | Biological: anti-thymocyte globulin;Drug: busulfan;Drug: cyclophosphamide;Drug: fludarabine phosphate;Drug: melphalan;Drug: methylprednisolone;Radiation: radiation therapy | Milton S. Hershey Medical Center | NULL | Recruiting | N/A | 21 Years | Both | 25 | Phase 2 | United States |
| 46 | NCT01019876 (ClinicalTrials.gov) | June 2002 | 23/11/2009 | Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases | Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Selected Non-Malignant Diseases | Bone Marrow Failure;Osteopetrosis;Fanconi Anemia;Severe Combined Immunodeficiency | Drug: Fludarabine;Drug: Cyclophosphamide;Drug: Cyclophosphamide 40;Drug: Cyclophosphamide 30 | Columbia University | NULL | Recruiting | N/A | 30 Years | Both | 50 | Phase 2/Phase 3 | United States |
| 47 | 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 |
| 48 | NCT00586274 (ClinicalTrials.gov) | March 2002 | 21/12/2007 | Use of Rft5-Dga to Deplete Alloreactive Cells for Pts With Fanconi Anemia After Haploidentical SCT | A Phase I Study Evaluating The Use Of Rft5-Dga To Deplete Alloreactive Cells For Patients With Fanconi Anemia After Haploidentical Stem Cell Transplantation | FANCONI ANEMIA | Procedure: CD34 selected haploidentical PBSCT;Drug: Fludarabine;Biological: T cell infusion;Biological: Campath 1h;Biological: anti-CD45 | Baylor College of Medicine | UTSW-Dallas;Center for Cell and Gene Therapy, Baylor College of Medicine | Terminated | N/A | 64 Years | Both | 1 | Phase 1 | United States |
| 49 | NCT00053989 (ClinicalTrials.gov) | January 29, 2002 | 5/2/2003 | NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders | Non-Myeloablative Allogeneic Hematopoietic Peripheral Blood Stem Cell Transplantation for Hematologic Malignancies and Disorders | Chronic Myeloproliferative Disorders;Leukemia;Lymphoma;Multiple Myeloma and Plasma Cell Neoplasm;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative Diseases;Fanconi Anemia;Aplastic Anemia | Biological: anti-thymocyte globulin;Biological: graft-versus-tumor induction therapy;Biological: sargramostim;Biological: therapeutic allogeneic lymphocytes;Drug: cyclophosphamide;Drug: fludarabine phosphate;Drug: methylprednisolone;Drug: mycophenolate mofetil;Drug: tacrolimus;Procedure: allogeneic bone marrow transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantation | Roswell Park Cancer Institute | NULL | Completed | 4 Years | 75 Years | All | 41 | Phase 2 | United States |
| 50 | NCT00590460 (ClinicalTrials.gov) | July 2001 | 26/12/2007 | Antibody Conditioning Regimen For Allogeneic Donor Stem Cell Transplantation Of Patients With Fanconi Anemia | Cd45 (Yth-24 and Yth 54) and Cd52 (Campath-1H) Monoclonal Antibody Conditioning Regimen for Allogeneic Donor Stem Cell Transplantation of Patients With Fanconi Anemia | Fanconi Anemia;Severe Aplastic Anemia | Biological: CAMPATH-1H;Biological: Anti-CD45;Drug: Fludarabine;Procedure: Stem cell infusion | Baylor College of Medicine | The Methodist Hospital System;Center for Cell and Gene Therapy, Baylor College of Medicine | Terminated | N/A | N/A | All | 5 | Phase 1/Phase 2 | United States |
| 51 | NCT00595127 (ClinicalTrials.gov) | June 2001 | 7/1/2008 | Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine | A 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 Fludarabine | Fanconi Anemia | Drug: Cyclophosphamide;Drug: Fludarabine;Radiation: Total body irradiation (TBI) | Memorial Sloan Kettering Cancer Center | National Cancer Institute (NCI);Miltenyi Biotec, Inc. | Completed | N/A | N/A | All | 21 | N/A | United States |
| 52 | NCT00305708 (ClinicalTrials.gov) | August 2000 | 21/3/2006 | Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First Remission | Bone Marrow Stem Cell Transplantation for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myeloid Leukemia in 1Remission | Congenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Fanconi Anemia;Leukemia;Severe Congenital Neutropenia;Thrombocytopenia | Biological: anti-thymocyte globulin;Drug: busulfan;Drug: fludarabine phosphate;Procedure: allogeneic bone marrow transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantation;Radiation: radiation therapy | University of California, San Francisco | National Cancer Institute (NCI) | Completed | N/A | 17 Years | Both | 40 | Phase 1/Phase 2 | United States |
| 53 | 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 |
| 54 | NCT00093743 (ClinicalTrials.gov) | January 2000 | 6/10/2004 | Low-Dose Total-Body Irradiation and Fludarabine Phosphate Followed by Unrelated Donor Stem Cell Transplant in Treating Patients With Fanconi Anemia | Low-Dose Total Body Irradiation and Fludarabine Followed By Unrelated Donor Stem Cell Transplantation for Patients With Fanconi Anemia - A Multicenter Trial | 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;Childhood Acute Myeloid Leukemia in Remission;Childhood Myelodysplastic Syndromes;Fanconi Anemia;Previously Treated Myelodysplastic Syndromes | Drug: 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 mofetil | Fred Hutchinson Cancer Research Center | National Heart, Lung, and Blood Institute (NHLBI);National Cancer Institute (NCI) | Completed | N/A | N/A | All | 2 | Phase 1 | United States |
| 55 | 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 |
| 56 | NCT00317876 (ClinicalTrials.gov) | June 1998 | 24/4/2006 | Cyclophosphamide in Treating Patients Who Are Undergoing a Donor Bone Marrow Transplant for Fanconi's Anemia | Dose-Finding Study for Cyclophosphamide as Conditioning Regimens for Bone Marrow Transplantation From Related Donors in Patients With Fanconi Anemia | Fanconi Anemia | Drug: cyclophosphamide;Drug: cyclosporine;Drug: methotrexate;Procedure: allogeneic bone marrow transplantation;Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantation | Fred Hutchinson Cancer Research Center | National Cancer Institute (NCI) | Completed | N/A | N/A | Both | 25 | Phase 1 | United States;Brazil |
| 57 | NCT00001399 (ClinicalTrials.gov) | December 3, 1993 | 3/11/1999 | Gene Therapy for the Treatment of Fanconi's Anemia Type C | Retroviral Mediated Gene Transfer of the Fanconi Anemia Complementation Group C Gene to Hematopoietic Progenitors of Group C Patients | Fanconi's Anemia;Pancytopenia | Drug: Transduced CD34+ Cells | National Heart, Lung, and Blood Institute (NHLBI) | NULL | Completed | 5 Years | N/A | All | 9 | Phase 1 | United States |
| 58 | 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 | ||||
| 59 | EUCTR2014-000584-41-GB (EUCTR) | 15/07/2015 | Phase I/II study of CaspaCide T cells in children following aß- depleted mis-matched family donor stem cell transplantation | 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 HSCT | Hematological disorders (ALL; AML; Non-Hodgkin lymphoma; Myelodysplastic syndromes; Congenital immune deficiencies; Severe aplastic anemia; Fanconi anemia; Osteopetrosis; Selected cases of hemoglobinopathies) MedDRA version: 20.0;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: BPX-501 cells INN or Proposed INN: Rivogenlecleucel Other descriptive name: BPX-501 Product Name: rimiducid INN or Proposed INN: Rimiducid Other descriptive name: AP1903 A594 | Bellicum Pharmaceuticals, Inc. | NULL | NA | Female: yes Male: yes | 175 | Phase 2 | Spain;Italy;United Kingdom |