285. ファンコニ貧血 Fanconi anemia Clinical trials / Disease details


臨床試験数 : 62 薬物数 : 93 - (DrugBank : 30) / 標的遺伝子数 : 30 - 標的パスウェイ数 : 144

  
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PhaseCountries
1NCT04784052
(ClinicalTrials.gov)
December 7, 20212/3/2021Depleted Donor Stem Cell Transplant in Children and Adults With Fanconi Anemia After Being Conditioned With a Regimen Containing JSP191 AntibodyTCRaß+ 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 AnemiaFanconi AnemiaDrug: JSP191;Device: CliniMACS Prodigy System;Biological: Depleted Stem Cell Transplant;Biological: Rabbit Anti-Thymoglobulin (rATG);Drug: Cyclophosphamide;Drug: Fludarabine;Drug: RituximabRajni AgarwalNULLRecruiting2 YearsN/AAll12Phase 1/Phase 2United States
2NCT04483544
(ClinicalTrials.gov)
December 3, 202020/7/2020Pembrolizumab and Olaparib in Cervical Cancer PatientsImmunotherapy in Combination With PARP Inhibition in Advanced Cervical Cancer Patients Functionally Competent or Deficient for the Fanconi Anemia Repair PathwayCervical Cancer;Cervical CarcinomaDrug: pembrolizumab;Drug: olaparibBaptist Health South FloridaFlorida Department of Health;Merck Sharp & Dohme LLCRecruiting18 YearsN/AFemale48Phase 2United States
3NCT04248439
(ClinicalTrials.gov)
July 15, 202024/1/2020Gene Therapy for Fanconi Anemia, Complementation Group AA 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 AFanconi Anemia Complementation Group ABiological: RP-L102Rocket Pharmaceuticals Inc.NULLRecruiting1 YearN/AAll5Phase 2United States
4NCT04042831
(ClinicalTrials.gov)
June 9, 202029/7/2019Olaparib in Treating Patients With Metastatic Biliary Tract Cancer With Aberrant DNA Repair Gene MutationsA Phase II Study of Olaparib in Patients With Advanced Biliary Tract Cancer With Aberrant DNA Repair Gene MutationsARID1A Gene Mutation;ATM Gene Mutation;ATR Gene Mutation;Bile Duct Adenocarcinoma;BRCA1 Gene Mutation;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 MutationDrug: OlaparibAcademic and Community Cancer Research UnitedNational Cancer Institute (NCI)Recruiting18 YearsN/AAll36Phase 2United States
5EUCTR2018-002502-31-GB
(EUCTR)
07/02/202008/10/2019A 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-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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
5Phase 2Spain;United Kingdom
6NCT04069533
(ClinicalTrials.gov)
November 28, 201923/8/2019Lentiviral-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.NULLRecruiting1 Year17 YearsAll5Phase 2Spain
7NCT03814408
(ClinicalTrials.gov)
January 11, 201914/1/2019A 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.NULLActive, not recruiting1 Year12 YearsAll2Phase 1United States
8NCT03579875
(ClinicalTrials.gov)
November 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
9NCT03206086
(ClinicalTrials.gov)
November 2, 201830/6/2017Eltrombopag for People With Fanconi AnemiaEltrombopag for Patients With Fanconi AnemiaFanconi AnemiaDrug: EltrombopagNational Heart, Lung, and Blood Institute (NHLBI)NULLRecruiting2 YearsN/AAll35Phase 2United States
10EUCTR2018-002502-31-ES
(EUCTR)
31/10/201802/08/2018A 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-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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
5Phase 2Spain
11NCT03600909
(ClinicalTrials.gov)
May 15, 201817/7/2018A Study of the Effect of Blood Stem Cell Transplant After Chemotherapy Alone in Patients With Fanconi AnemiaA Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Risk-Adjusted Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and FludarabineFanconi Anemia;Myelodysplastic Syndrome (MDS);Acute Myelogenous Leukemia (AML)Drug: Busulfan;Drug: Fludarabine;Drug: Cyclophosphamide;Drug: Anti-Thymocyte Globulin (Rabbit);Device: The CliniMACS device;Drug: G-CSFMemorial Sloan Kettering Cancer CenterPediatric Brain Tumor ConsortiumCompleted1 MonthN/AAll3Phase 2United States
12NCT03476330
(ClinicalTrials.gov)
May 8, 201819/3/2018Quercetin 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, CincinnatiNULLRecruiting2 YearsN/AAll55Phase 2United States
13NCT03398824
(ClinicalTrials.gov)
March 29, 201813/12/2017Pilot Study of Metformin for Patients With Fanconi AnemiaPilot Study of Metformin for Patients With Fanconi AnemiaFanconi AnemiaDrug: metformin HClBoston Children's HospitalNULLCompleted6 Years35 YearsAll15Phase 2United States
14NCT03351868
(ClinicalTrials.gov)
December 1, 201720/11/2017FANCA Gene Transfer for Fanconi Anemia Using a High-safety, High-efficiency, Self-inactivating Lentiviral VectorGene Transfer for Fanconi Anemia Using a Self-inactivating Lentiviral VectorFanconi AnemiaGenetic: Gene-modified autologous stem cellsShenzhen Geno-Immune Medical InstituteNULLRecruiting2 Years20 YearsAll10N/AChina
15EUCTR2014-000584-41-ES
(EUCTR)
08/06/201721/03/2017Phase 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 disordersPhase 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.NULLNot RecruitingFemale: yes
Male: yes
175Phase 2Spain;United Kingdom;Italy
16NCT03609840
(ClinicalTrials.gov)
May 24, 20175/1/2018Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant PatientsPopulation Pharmacokinetics and Pharmacodynamics of Thiotepa and TEPA in Pediatric Patients Undergoing Hematopoietic Cell Transplantation (HCT).Hematologic Malignancies;Nonmalignant Diseases;Immune Deficiency;Hemoglobinopathies;Genetic Inborn Errors of Metabolism;Fanconi Anemia;Thalassemia;Sickle Cell DiseaseDrug: ThiotepaUniversity of California, San FranciscoNULLActive, not recruitingN/A17 YearsAll25United States
17NCT02678533
(ClinicalTrials.gov)
February 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 ParisEuroFancolenCompleted2 Years17 YearsAll4Phase 1/Phase 2France
18EUCTR2016-003226-16-IT
(EUCTR)
03/02/201717/11/2016Follow-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 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
175Phase 1;Phase 2Italy
19NCT03157804
(ClinicalTrials.gov)
January 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
20EUCTR2014-004272-29-GB
(EUCTR)
30/03/201515/07/2015Phase 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]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 TrustNULLNot 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): noFrance;Spain;United Kingdom
21EUCTR2014-000584-41-IT
(EUCTR)
23/10/201415/04/2014Phase 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 disordersPhase 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.NULLNot RecruitingFemale: yes
Male: yes
30Phase 1;Phase 2Spain;United Kingdom;Italy
22NCT02143830
(ClinicalTrials.gov)
April 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, CincinnatiFred Hutchinson Cancer Center;Memorial Sloan Kettering Cancer CenterRecruiting3 MonthsN/AAll70Phase 2United States
23NCT02065869
(ClinicalTrials.gov)
April 201413/2/2014Safety Study of Gene Modified Donor T-cells Following TCRaß+ Depleted Stem Cell TransplantPhase I/II Study of CaspaCIDe T Cells (BPX-501; Rivogenlecleucel) From an HLA Partially Matched Family Donor After Negative Selection of TCRaß+ T Cells in Paediatric Patients Affected by Haematological 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 PharmaceuticalsNULLActive, not recruiting1 Month18 YearsAll187Phase 1/Phase 2Italy;United Kingdom;Germany;Spain;United States
24NCT01917708
(ClinicalTrials.gov)
January 201424/7/2013Bone Marrow Transplant With Abatacept for Non-Malignant DiseasesAbatacept for Post-Transplant Immune Suppression in Children and Adolescents Receiving Allogeneic Hematopoietic Stem Cell Transplants for Non-Malignant DiseasesHurler Syndrome;Fanconi Anemia;Glanzmann Thrombasthenia;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;Severe Congenital Neutropenia;Leukocyte Adhesion Deficiency;Shwachman-Diamond Syndrome;Diamond-Blackfan Anemia;Dyskeratosis-congenita;Chediak-Higashi Syndrome;Severe Aplastic Anemia;Thalassemia Major;Hemophagocytic Lymphohistiocytosis;Sickle Cell DiseaseDrug: AbataceptEmory UniversityNULLCompletedN/A21 YearsAll10Phase 1United States
25NCT01995305
(ClinicalTrials.gov)
October 201321/11/2013Use Massive Parallel Sequencing and Exome Capture Technology to Sequence the Exome of Fanconi Anemia Children and Their PatentsExome Sequencing of Fanconi Anemia Children and the Their ParentsFanconi Anemia;Autosomal or Sex Linked Recessive Genetic Disease;Bone Marrow Hematopoiesis Failure, Multiple Congenital Abnormalities, and Susceptibility to Neoplastic Diseases.;Hematopoiesis Maintainance.Genetic: human whole exome;Genetic: whole genomicXiaofan ZhuNULLAvailable1 Month18 YearsBothN/AChina
26NCT02931071
(ClinicalTrials.gov)
September 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
27EUCTR2011-006100-12-ES
(EUCTR)
12/04/201327/01/2012Clinical 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 JESUSNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
5Phase 1Spain
28NCT01720147
(ClinicalTrials.gov)
July 201225/7/2012Quercetin 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)Active, not recruitingN/AN/AAll30Phase 1United States
29NCT01331018
(ClinicalTrials.gov)
February 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 CenterNational Heart, Lung, and Blood Institute (NHLBI);Rocket Pharma LimitedActive, not recruiting4 YearsN/AAll3Phase 1United States
30NCT02127905
(ClinicalTrials.gov)
March 201119/10/2012Unrelated 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.NULLWithdrawn8 Weeks21 YearsAll0N/AUnited States
31NCT01316549
(ClinicalTrials.gov)
January 1, 201114/3/2011Study of Fludarabine Drug Exposure in Pediatric Bone Marrow TransplantationPopulation Pharmacokinetics of Fludarabine in Pediatric Patients Undergoing Hematopoietic Cell TransplantationHematologic Malignancies;Nonmalignant Diseases;Immunodeficiencies;Hemoglobinopathies;Genetic Inborn Errors of Metabolism;Fanconi Anemia;Thalassemia;Sickle Cell DiseaseDrug: FludarabineUniversity of California, San FranciscoThrasher Research FundCompletedN/A17 YearsAll67United States
32NCT01319851
(ClinicalTrials.gov)
September 201015/9/2010Alefacept and Allogeneic Hematopoietic Stem Cell TransplantationAlefacept and Allogeneic Hematopoietic Stem Cell Transplantation for Children With Non-Malignant Diseases Who Have Been Multiply Transfused: a Pilot StudyThalassemia;Sickle Cell Disease;Glanzmann Thrombasthenia;Wiskott-Aldrich Syndrome;Chronic-granulomatous Disease;Severe Congenital Neutropenia;Leukocyte Adhesion Deficiency;Schwachman-Diamond Syndrome;Diamond-Blackfan Anemia;Fanconi Anemia;Dyskeratosis-congenita;Chediak-Higashi Syndrome;Severe Aplastic AnemiaDrug: AlefaceptEmory UniversityChildren's Healthcare of AtlantaTerminatedN/A21 YearsAll3N/AUnited States
33NCT01001598
(ClinicalTrials.gov)
November 200922/10/2009Safety 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: danazolBoston Children's HospitalNULLTerminated3 YearsN/AAll5Phase 1/Phase 2United States
34NCT01082133
(ClinicalTrials.gov)
October 20095/3/2010Multicenter Transplant Study for Fanconi AnemiaA Multicenter Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and FludarabineFanconi AnemiaDrug: Chemotherapy;Biological: Miltenyi CliniMACSChildren's Hospital Medical Center, CincinnatiNULLCompletedN/AN/ABoth45Phase 2United States
35NCT00987480
(ClinicalTrials.gov)
September 25, 200930/9/2009Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and FludarabineA Multicenter Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and FludarabineAplastic Anemia;Leukemia;Myelodysplastic SyndromeDrug: Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine.;Device: CliniMACS deviceMemorial Sloan Kettering Cancer CenterBoston Children's Hospital;Children's Hospital Medical Center, Cincinnati;Children's Hospital and Health System Foundation, Wisconsin;Rockefeller University;Fred Hutchinson Cancer Research CenterCompletedN/AN/AAll45Phase 2United States
36NCT01071239
(ClinicalTrials.gov)
April 200920/5/2009Hematopoietic Stem Cell Transplant for Fanconi AnemiaA Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and FludarabineFanconi AnemiaDevice: CliniMACs device;Drug: Busulfan;Drug: Fludarabine;Drug: Cyclophosphamide;Drug: ATGMedical College of WisconsinMemorial Sloan Kettering Cancer CenterCompletedN/AN/AAll1Phase 2United States
37NCT00856388
(ClinicalTrials.gov)
January 14, 20094/3/2009Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure DisordersA Pilot Trial Of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan, And Low Dose Total Body IrradiationAccelerated Phase Chronic Myelogenous Leukemia;Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome;Adult Acute Lymphoblastic Leukemia in Remission;Adult Acute Myeloid Leukemia in Remission;Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities;Adult Acute Myeloid Leukemia With Del(5q);Adult Acute Myeloid Leukemia With Inv(16)(p13;q22);Adult Acute Myeloid Leukemia With t(15;17)(q22;q12);Adult Acute Myeloid Leukemia With t(16;16)(p13;Adult Acute Myeloid Leukemia With t(8;21)(q22;Adult Nasal Type Extranodal NK/T-cell Lymphoma;Anaplastic Large Cell Lymphoma;Angioimmunoblastic T-cell Lymphoma;Aplastic Anemia;Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Chronic Myelogenous Leukemia;Childhood Diffuse Large Cell Lymphoma;Childhood Immunoblastic Large Cell Lymphoma;Childhood Myelodysplastic Syndromes;Childhood Nasal Type Extranodal NK/T-cell Lymphoma;Chronic Eosinophilic Leukemia;Chronic Myelomonocytic Leukemia;Chronic Neutrophilic Leukemia;Chronic Phase Chronic Myelogenous Leukemia;de Novo Myelodysplastic Syndromes;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Fanconi Anemia;Juvenile Myelomonocytic Leukemia;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Nodal Marginal Zone B-cell Lymphoma;Noncontiguous Stage II Adult Burkitt Lymphoma;Noncontiguous Stage II Adult Diffuse Large Cell Lymphoma;Noncontiguous Stage II Adult Diffuse Mixed Cell Lymphoma;Noncontiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma;Noncontiguous Stage II Adult Immunoblastic Large Cell Lymphoma;Noncontiguous Stage II Adult Lymphoblastic Lymphoma;Noncontiguous Stage II Grade 1 Follicular Lymphoma;Noncontiguous Stage II Grade 2 Follicular Lymphoma;Noncontiguous Stage II Grade 3 Follicular Lymphoma;Noncontiguous Stage II Mantle Cell Lymphoma;Noncontiguous Stage II Marginal Zone Lymphoma;Noncontiguous Stage II Small Lymphocytic Lymphoma;Paroxysmal Nocturnal Hemoglobinuria;Previously Treated Myelodysplastic Syndromes;Primary Myelofibrosis;Recurrent Adult Acute Lymphoblastic Leukemia;Recurrent Adult Acute Myeloid Leukemia;Recurrent Adult Burkitt Lymphoma;Recurrent Adult Diffuse Large Cell Lymphoma;Recurrent Adult Diffuse Mixed Cell Lymphoma;Recurrent Adult Diffuse Small Cleaved Cell Lymphoma;Recurrent Adult Grade III Lymphomatoid Granulomatosis;Recurrent Adult Hodgkin Lymphoma;Recurrent Adult Immunoblastic Large Cell Lymphoma;Recurrent Adult Lymphoblastic Lymphoma;Recurrent Adult T-cell Leukemia/Lymphoma;Recurrent Childhood Acute Lymphoblastic Leukemia;Recurrent Childhood Acute Myeloid Leukemia;Recurrent Childhood Anaplastic Large Cell Lymphoma;Recurrent Childhood Grade III Lymphomatoid Granulomatosis;Recurrent Childhood Large Cell Lymphoma;Recurrent Childhood Lymphoblastic Lymphoma;Recurrent Childhood Small Noncleaved Cell Lymphoma;Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma;Recurrent Grade 1 Follicular Lymphoma;Recurrent Grade 2 Follicular Lymphoma;Recurrent Grade 3 Follicular Lymphoma;Recurrent Mantle Cell Lymphoma;Recurrent Marginal Zone Lymphoma;Recurrent Mycosis Fungoides/Sezary Syndrome;Recurrent Small Lymphocytic Lymphoma;Recurrent/Refractory Childhood Hodgkin Lymphoma;Refractory Chronic Lymphocytic Leukemia;Refractory Multiple Myeloma;Relapsing Chronic Myelogenous Leukemia;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndromes;Splenic Marginal Zone Lymphoma;Stage III Adult Diffuse Small Cleaved Cell Lymphoma;Stage III Adult Immunoblastic Large Cell Lymphoma;Stage III Adult Lymphoblastic Lymphoma;Stage III Grade 1 Follicular Lymphoma;Stage III Grade 2 Follicular Lymphoma;Stage III Grade 3 Follicular Lymphoma;Stage III Mantle Cell Lymphoma;Stage III Marginal Zone Lymphoma;Stage III Small Lymphocytic Lymphoma;Stage IV Adult Burkitt Lymphoma;Stage IV Adult Diffuse Small Cleaved Cell Lymphoma;Stage IV Adult Immunoblastic Large Cell Lymphoma;Stage IV Adult Lymphoblastic Lymphoma;Stage IV Grade 1 Follicular Lymphoma;Stage IV Grade 2 Follicular Lymphoma;Stage IV Grade 3 Follicular Lymphoma;Stage IV Mantle Cell Lymphoma;Stage IV Marginal Zone Lymphoma;Stage IV Small Lymphocytic Lymphoma;Waldenström MacroglobulinemiaDrug: fludarabine phosphate;Drug: melphalan;Radiation: total-body irradiation;Procedure: allogeneic hematopoietic stem cell transplantation;Biological: anti-thymocyte globulinRoswell Park Cancer InstituteNational Cancer Institute (NCI)Completed3 Years75 YearsAll62N/AUnited States
38NCT00479115
(ClinicalTrials.gov)
May 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
39NCT00453388
(ClinicalTrials.gov)
February 200727/3/2007Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant, Mycophenolate Mofetil, and Cyclosporine in Treating Patients With Fanconi AnemiaNonmyeloablative Hematopoietic Cell Transplantation for Patients With Fanconi Anemia Using Alternative Marrow Donors: A Phase II Dose-Finding StudyAcute Myeloid Leukemia in Remission;de Novo Myelodysplastic Syndrome;Fanconi Anemia;Previously Treated Myelodysplastic SyndromeProcedure: Allogeneic Bone Marrow Transplantation;Drug: Cyclophosphamide;Drug: Cyclosporine;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Radiation: Total-Body IrradiationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI)CompletedN/AN/AAll6Phase 2United States;Brazil
40NCT00499070
(ClinicalTrials.gov)
January 200710/7/2007Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to TreatmentTCR Vbeta Repertoire and PNH Clones in Children With Refractory Cytopenia (RC). An Open Nonrandomised Multi-Center Prospective StudyDyskeratosis Congenita;Fanconi Anemia;Myelodysplastic Syndromes;Pearson Marrow-pancreas Syndrome;Shwachman-diamond SyndromeGenetic: polymerase chain reaction;Other: flow cytometry;Other: immunologic technique;Procedure: biopsyUniversity Hospital FreiburgNULLCompletedN/A17 YearsBoth119N/AAustria;Belgium;Czech Republic;Denmark;Germany;Ireland;Italy;Netherlands;Spain;Switzerland;Poland
41NCT00352976
(ClinicalTrials.gov)
May 18, 200614/7/2006TBI 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 MinnesotaNULLCompletedN/AN/AAll88Phase 2/Phase 3United States
42NCT00965666
(ClinicalTrials.gov)
October 200524/8/2009Pilot 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, CincinnatiAmgenCompleted4 YearsN/AAll3Early Phase 1United States
43EUCTR2004-004914-18-IT
(EUCTR)
21/01/200527/07/2005A 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 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 GASLININULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Italy
44NCT00272857
(ClinicalTrials.gov)
August 20044/1/2006Bone Marrow Cell Gene Transfer in Individuals With Fanconi AnemiaGene Transfer From Patients With Fanconi Anemia, Genotype A: A Pilot StudyFanconi AnemiaGenetic: Retrovirus ConstructBoston Children’s HospitalNational Heart, Lung, and Blood Institute (NHLBI);Children's Hospital Medical Center, CincinnatiCompleted1 Year35 YearsAll3Phase 1United States
45NCT00243399
(ClinicalTrials.gov)
July 200420/10/2005Oxandrolone 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 DevelopmentCompletedN/AN/ABoth10Phase 1United States
46NCT00084695
(ClinicalTrials.gov)
September 200310/6/2004Umbilical 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 CenterNULLRecruitingN/A21 YearsBoth25Phase 2United States
47NCT01019876
(ClinicalTrials.gov)
June 200223/11/2009Risk-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 UniversityNULLRecruitingN/A30 YearsBoth50Phase 2/Phase 3United States
48NCT00899795
(ClinicalTrials.gov)
June 20029/5/2009Evaluating Cell Damage in Patients With Acute Myeloid Leukemia, Myelodysplastic Syndromes, or Fanconi Anemia; in Patients Who Were Exposed to Alkylating Agents; and in Healthy VolunteersStromal Injury and Clonal Adaptation in MyelodysplasiaLeukemia;Myelodysplastic SyndromesGenetic: cytogenetic analysis;Genetic: fluorescence in situ hybridization;Other: flow cytometry;Procedure: biopsyOHSU Knight Cancer InstituteNational Cancer Institute (NCI)Completed5 Years120 YearsAll35N/AUnited States
49NCT00258427
(ClinicalTrials.gov)
March 26, 200222/11/2005Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi AnemiaHematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia MT2002-02Fanconi AnemiaBiological: anti-thymocyte globulin;Biological: filgrastim;Drug: busulfan;Drug: cyclophosphamide;Drug: fludarabine phosphate;Drug: methylprednisolone;Biological: Hematopoietic stem cell transplantationMasonic Cancer Center, University of MinnesotaNULLCompletedN/A44 YearsAll14Phase 2United States
50NCT00896740
(ClinicalTrials.gov)
March 20029/5/2009Gene Function in Bone Marrow Cells From Patients With Fanconi Anemia and From Healthy ParticipantsAnalysis of Fanconi Anemia Gene Function by Microarray Analysis of Bone Marrow CellsFanconi AnemiaGenetic: microarray analysis;Procedure: biopsyOHSU Knight Cancer InstituteNational Cancer Institute (NCI)Terminated1 Year55 YearsAll90N/AUnited States
51NCT00586274
(ClinicalTrials.gov)
March 200221/12/2007Use 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 MedicineTerminatedN/A64 YearsBoth1Phase 1United States
52NCT00053989
(ClinicalTrials.gov)
January 29, 20025/2/2003NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/DisordersNon-Myeloablative Allogeneic Hematopoietic Peripheral Blood Stem Cell Transplantation for Hematologic Malignancies and DisordersChronic Myeloproliferative Disorders;Leukemia;Lymphoma;Multiple Myeloma and Plasma Cell Neoplasm;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative Diseases;Fanconi Anemia;Aplastic AnemiaBiological: 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 transplantationRoswell Park Cancer InstituteNULLCompleted4 Years75 YearsAll41Phase 2United States
53NCT00590460
(ClinicalTrials.gov)
July 200126/12/2007Antibody 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 MedicineThe Methodist Hospital System;Center for Cell and Gene Therapy, Baylor College of MedicineTerminatedN/AN/AAll5Phase 1/Phase 2United States
54NCT00595127
(ClinicalTrials.gov)
June 20017/1/2008Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and FludarabineA Pilot Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and FludarabineFanconi AnemiaDrug: Cyclophosphamide;Drug: Fludarabine;Radiation: Total body irradiation (TBI)Memorial Sloan Kettering Cancer CenterNational Cancer Institute (NCI);Miltenyi Biotec, Inc.CompletedN/AN/AAll21N/AUnited States
55NCT00305708
(ClinicalTrials.gov)
August 200021/3/2006Busulfan, 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)CompletedN/A17 YearsBoth40Phase 1/Phase 2United States
56NCT00630253
(ClinicalTrials.gov)
February 17, 20005/3/2008Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi AnemiaA Study of Cyclophosphamide, Fludarabine, and Antithymocyte Globulin Followed by Matched Sibling Donor Hematopoietic Cell Transplantation in Patients With Fanconi AnemiaFanconi AnemiaBiological: Anti-Thymocyte Globulin;Drug: Cyclophosphamide;Drug: Fludarabine;Procedure: Hematopoietic Stem Cell Transplantation;Drug: Methylprednisolone;Drug: Filgrastim;Drug: Cyclosporine;Drug: Mycophenolate MofetilMasonic Cancer Center, University of MinnesotaNULLCompletedN/A59 YearsAll31Phase 1/Phase 2United States
57NCT00093743
(ClinicalTrials.gov)
January 20006/10/2004Low-Dose Total-Body Irradiation and Fludarabine Phosphate Followed by Unrelated Donor Stem Cell Transplant in Treating Patients With Fanconi AnemiaLow-Dose Total Body Irradiation and Fludarabine Followed By Unrelated Donor Stem Cell Transplantation for Patients With Fanconi Anemia - A Multicenter TrialAdult Acute Myeloid Leukemia in Remission;Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities;Adult Acute Myeloid Leukemia With Del(5q);Adult Acute Myeloid Leukemia With Inv(16)(p13;q22);Adult Acute Myeloid Leukemia With t(15;17)(q22;q12);Adult Acute Myeloid Leukemia With t(16;16)(p13;Adult Acute Myeloid Leukemia With t(8;21)(q22;Childhood Acute Myeloid Leukemia in Remission;Childhood Myelodysplastic Syndromes;Fanconi Anemia;Previously Treated Myelodysplastic SyndromesDrug: fludarabine phosphate;Drug: cyclosporine;Radiation: total-body irradiation;Procedure: allogeneic bone marrow transplantation;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantation;Drug: mycophenolate mofetilFred Hutchinson Cancer Research CenterNational Heart, Lung, and Blood Institute (NHLBI);National Cancer Institute (NCI)CompletedN/AN/AAll2Phase 1United States
58NCT00290628
(ClinicalTrials.gov)
October 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
59NCT00317876
(ClinicalTrials.gov)
June 199824/4/2006Cyclophosphamide in Treating Patients Who Are Undergoing a Donor Bone Marrow Transplant for Fanconi's AnemiaDose-Finding Study for Cyclophosphamide as Conditioning Regimens for Bone Marrow Transplantation From Related Donors in Patients With Fanconi AnemiaFanconi AnemiaDrug: cyclophosphamide;Drug: cyclosporine;Drug: methotrexate;Procedure: allogeneic bone marrow transplantation;Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantationFred Hutchinson Cancer Research CenterNational Cancer Institute (NCI)CompletedN/AN/ABoth25Phase 1United States;Brazil
60NCT00001399
(ClinicalTrials.gov)
December 3, 19933/11/1999Gene 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)NULLCompleted5 YearsN/AAll9Phase 1United States
61EUCTR2014-005264-14-FR
(EUCTR)
19/06/2015FancoMob: Pilote study for a combined treatment helping to collect stem cells in patient suffering Fanconi anemiaNA - 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)NULLNAFemale: yes
Male: yes
Phase 2France
62EUCTR2014-000584-41-GB
(EUCTR)
15/07/2015Phase I/II study of CaspaCide T cells in children following aß- depleted mis-matched family donor stem cell transplantationPhase 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.NULLNAFemale: yes
Male: yes
175Phase 2Spain;Italy;United Kingdom