DDrare: Database of Drug Development for Rare Diseases

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

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

Drug = AMD3100; AP1903; AP1903 A594; ATG; Abatacept; Alefacept; Anti-CD45; Anti-Thymocyte Globulin; Anti-Thymocyte Globulin (Rabbit); Anti-thymocyte globulin; BPX-501; BPX-501 T cells; BPX-501 cells; Busulfan; Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine.; CAMPATH-1H; CD34+ CELLS; CD34+ Cells; CD34+ selected cells; CD34+CELLS; Campath; Campath 1h; Chemotherapy; Cyclophosphamide; Cyclophosphamide (CY) (Plan 1); Cyclophosphamide (CY) (Plan 2); Cyclophosphamide 30; Cyclophosphamide 40; Cyclophosphamide, Fludarabine; Cyclosporine; Danazol; Device: AmCell CliniMACs; Device: CliniMACS device; Device: CliniMACs device; Device: Donor mobilized PBSC infusion; Device: The CliniMACS device; ENBREL*SC 4FL 25MG+4SIR 1ML; Eltrombopag; Enbrel; Etanercept; FP-045; Filgrastim; Fludarabine; Fludarabine (FLU); Fludarabine Phosphate; Fludarabine phosphate; G-CSF; Genetically Engineered Hematopoietic Stem Progenitor Cells; Genetically Engineered Hematopoietic Stem/Progenitor Cells; Graft-versus-tumor induction therapy; Hematopoietic stem cell transplantation; Melphalan; Metformin; Metformin HCl; Methotrexate; Methylprednisolone; Methylprednisolone (MP); Miltenyi CliniMACS; Mozobil; Mycophenolate; Mycophenolate Mofetil; Mycophenolate mofetil; Neupogen; Olaparib; Other: Laboratory Biomarker Analysis; Oxandrolone; Pembrolizumab; Peripheral blood stem cell; Phosphate; Plerixafor; Plérixafor; Prednisone; Procedure: Allogeneic Bone Marrow Transplantation; Procedure: Bone Marrow Aspiration; Procedure: Bone Marrow Transplantation; Procedure: CD34 selected haploidentical PBSCT; Procedure: Hematopoietic Stem Cell Transplant; Procedure: Hematopoietic Stem Cell Transplantation; Procedure: IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs); Procedure: Leukapheresis; Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation; Procedure: Stem cell infusion; Procedure: Thymic Shielding During Radiation; Procedure: Total Body Irradiation; Procedure: allogeneic bone marrow transplantation; Procedure: allogeneic hematopoietic stem cell transplantation; Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantation; Procedure: peripheral blood stem cell transplantation; Procedure: radiation therapy; Procedure: umbilical cord blood transplantation; Quercetin; Quercetin (dietary supplement); RP-L102; Rabbit; Rabbit ATG; Radiation: Total body irradiation (TBI); Radiation: Total-Body Irradiation; Radiation: radiation therapy; Radiation: total-body irradiation; Rimiducid; Rituximab; Rivogenlecleucel; Rivogenleucleucel; Sargramostim; Sirolimus; T cell infusion; Tacrolimus; Therapeutic allogeneic lymphocytes; Total Body Irradiation (TBI) (Plan 1); Transduced CD34+ Cells; Zarzio

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	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

No.

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Public_title

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Intervention

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agemin

Inclusion_
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Inclusion_
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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

Phase 1

United States