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	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

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

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

Phase 1

United States