DDrare: Database of Drug Development for Rare Diseases

60. 再生不良性貧血
［臨床試験数：218，薬物数：362（DrugBank：81），標的遺伝子数：39，標的パスウェイ数：155］

Searched query = "Aplastic 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 = A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs); ACICLOVIR; AMG531; AMIKACIN; AMPHOTERICINE B, LIPOSOME; AP1903; AP1903 A594; ATG; ATG conditioning regimen; ATG+Rapamune+cyclosporine; ATG+cyclosporine; ATG-Fresenius; ATG-fresenius; Abatacept; Acetate; Aciclovir; Alefacept; Alemtuzumab; Alemtuzumab (Campath ); Alemtuzumab and Rituximab; Alkeran; Allogeneic donor derived B-lymphocytes; Alpha beta depletion; Amikacin; Androgens; Anti-CD45; Anti-Thymocyte Globulin; Anti-Thymocyte Globulin (Rabbit); Anti-thymocyte globulin; Anti-thymocyte globulin (horse); Anti-thymocyte globulin (rabbit); Antilymphocyte serum; Antithymocyte Globulin; Antithymocyte Globulin (ATG); Antithymocyte globulin; Antithymocyte globulin (Rabbit); Atgam; BL-8040; BPX-501; BPX-501 cells; BUSULFAN; Bone marrow derived mesenchymal stem cells; Busilvex; Busulfan; Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine.; Busulfan/Fludarabine phosphate/Tacrolimus/Methotrexate/G-CSF; CAMPATH-1H; CAMPATH-1H (Alemtuzumab); CASPOFUNGIN ACETATE; CD34 Stem Cell Selection Therapy; CD45RA-depleted DLI; CD62L- Tem; CEFEPIME DIHYDROCHLORIDE MONOHYDRATE; CICLOSPORIN; CILASTATIN; CIPROFLOXACIN; CYCLOPHOSPHAMIDE MONOHYDRATE; Campath; Campath 1H; Campath, Chemo and/or TBI Allo SCT; Campath-1H; Carboplatin; Carmustine; Caspofungin; Cefepime; Chelation; Chemotherapy; Chinese herbal concoction twice a day for 6 months; Chloride; Ciclosporin; Ciclosporine; Cilastatin; Cilastatine; Ciprofloxacin; Clofarabine; Cohort 1: hATG, CsA, EPAG Day 14 to Month 6; Cohort 2: hATG, CsA, EPAG Day 14 to Month 3; Cohort 3: hATG, CsA (dose reduced), EPAG day 1 to month 6; Combination of thrombopoietin mimetic and cyclosporin A; Cord Blood Transplant; Cord Blood Units; Cord blood; CordIn; Creatinine; CsA; Cy; Cy-ATG; Cyclophosphamide; Cyclophosphamide (CY); Cyclophosphamide (CY) (Plan 1); Cyclophosphamide (CY) (Plan 2); Cyclophosphamide 100mg; Cyclophosphamide 150mg; Cyclophosphamide 50mg; Cyclophosphamide, Fludarabine, Rabbit ATG; Cyclophosphamide, Fludarabine, Thymoglobulin; Cyclophosphamide, fludarabine , thymoglobulin; Cyclophosphamide,Campath IH and TBI; Cyclophosphamide,cyclosporine A; Cyclosporin; Cyclosporin A; Cyclosporin A+Glucocorticoids; Cyclosporine; Cyclosporine (Gengraf ); Cyclosporine A; Cyclosporine A (CsA); Cyclosporine Oral Product; Cyclosporins; Cytarabine; Cytokine-treated Veto Cells; Cytoxan; Daclizumab; Danazol; Dapsone; Desferasirox; Device: Allogeneic hematopoietic stem cell transplantation; Device: CliniMACS; Device: CliniMACS device; Device: Donor mobilized PBSC infusion; Device: Hematopoietic stem cell transplantation (HSCT); Device: Miltenyi CD34 Reagent System; Dextrose; Donor Lymphocyte Infusion (DLI); EDTA; ELTROMBOPAG; ELTROMBOPAG OLAMINE; ENBREL*SC 4FL 25MG+4SIR 1ML; ETB115; Early retreatment with ATG; Eltrombopag; Eltrombopag 12.5 mg; Eltrombopag 25 mg; Enbrel; Etanercept; Etoposide; Ex vivo immunotherapy; Extension Cohort; FK-506; FLUDARABINE PHOSPHATE; Filgrastim; Flu-ATG; Fludarabine; Fludarabine (FLU); Fludarabine Phosphate; Fludarabine phosphate; Fludarabine, Cyclophosphamide; Fludarabine/ melphalan; Fludarabine/busulfan; Foscarnet; Foscarnet sodium; G-CSF; GLYCINE; GM-CSF; GVHD Prophylaxis; Ganciclovir; Genetic: DNA analysis; Genetic: Mesenchymal stromal cells; Genetic: RNA analysis; Genetic: polymerase chain reaction; Genetic: polymorphism analysis; Genetic: protein expression analysis; Glucocorticoids; Glycine; Graft-versus-tumor induction therapy; H-ATG (ATGAM ); HATG; HPC, A Infusion; HUMAN IGG4 ISOTYPE MONOCLONAL (IL2-RG) ANTIBODY; Haplo/cord transplant; Haploidentical HSCT using TBI free regimen, ''ATG'' with ''Post transplant cyclophosphamide''; Haploidentical Hematopoietic Cell Transplantation; Heparin; Hetrombopag; Hetrombopag Olamine; Hetrombopag Olamine+Standard Therapy; Horse anti-thymocyte globulin; Horse anti-thymocyte globulin (ATG); Horse anti-thymocyte globulin (hATG); Horse-Anti-thymocyte-Globulin; Human Placental Derived Stem Cell; ICL670; IL2; IMIPENEM; IST (ATG + CsA); Ibrutinib; Imipenem; Imipenem / cilastatine; Imipenem-cilastatine; Intravenous bone marrow mesenchymal stem cells infusion; Itacitinib; Levamisole; Levamisole hydrochloride; Levamisole+cyclosporin A+Glucocorticoids; Lo-Ovral Oral Contraceptive Pills; MABCAMPATH; MELPHALAN; MEROPENEM; METHOTREXATE; MMF; MPA; MSC; MSC+ATG; MTX; Mabcampath; Mannitol; Melphalan; Melphalan Hydrochloride; Meropenem; Mesenchymal Stromal Cells; Mesenchymal stem cells; Mesna; Metformin; Methotrexate; Methylprednisolone; Methylprednisolone (MP); Muromonab; Muromonab-CD3; Mycophenolate; Mycophenolate Mofetil; Mycophenolate Mofetil (MMF); Mycophenolate mofetil; Mycophenolate mofetil (MMF); Mycophenolic acid; Mycophenolic acid mofetil; Nandrolone; Nandrolone Decanoate; Nandrolone decanoate; Neoral; Non ATG Conditioning regimen; Normal saline; Noxafil; Noxafil Gastro resistant tablets; Noxafil®; Obatoclax; Obatoclax mesylate (GX15-070MS); Other: Best Practice; Other: Donor derived G-CSF mobilized PBC; Other: Human umbilical cord-derived MSCs and cyclosporin A; Other: Laboratory Biomarker Analysis; Other: cyclosporin A; Other: flow cytometry; Other: laboratory biomarker analysis; Other: medical chart review; PF-06462700; Pegfilgrastim; Phosphate; Posaconazole; Prednisolone; Procedure: AD-MSC transdifferentiated HSCs (AD-HSCs); Procedure: Adipose derived mesenchymal stem cells ( AD-MSCs); Procedure: Allogeneic Bone Marrow Transplantation; Procedure: Allogeneic Hematopoietic Stem Cell Transplantation; Procedure: Allogeneic stem cell transplant; Procedure: Allogenic Stem Cell Transplantation; Procedure: Apheresis; Procedure: Bone Marrow Transplant; Procedure: Bone marrow transplant; Procedure: Bone marrow transplantation; Procedure: CD3-depleted hematopoietic cell transplantation; Procedure: CD3±CD19 depleted hematopoietic stem cell transplantation; Procedure: Cord Blood Infusion; Procedure: Cord blood; Procedure: Ex Vivo Immunotherapy; Procedure: Haplo HSCT; Procedure: Immunosuppressive Therapy (IST); Procedure: Matched Unrelated Donor Hematopoietic Stem Cell Transplant; Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation; Procedure: Peripheral Blood Stem Cell Transplantation; Procedure: Peripheral blood stem cell transplantation (PBSCT); Procedure: Stem Cell Transplantation; Procedure: Stem cell infusion; Procedure: Total Body Irradiation; Procedure: allogeneic bone marrow transplantation; Procedure: allogeneic hematopoietic stem cell transplantation; Procedure: antiviral therapy; Procedure: assessment of therapy complications; Procedure: bone marrow ablation with stem cell support; Procedure: bone marrow aspiration; Procedure: in vitro-treated bone marrow transplantation; Procedure: in vitro-treated peripheral blood stem cell transplantation; Procedure: infection prophylaxis and management; Procedure: management of therapy complications; Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantation; Procedure: peripheral blood stem cell transplantation; Procedure: umbilical cord blood transplantation; Prograf; R-ATG; RABBIT HUMAN T LYMPHOCYTE IMMUNOGLOBULIN; RATG; REGN7257; REVOLADE; RVG 101614; RVG 105155; Rabbit; Rabbit ATG; Rabbit ATG, (Genzyme); Rabbit ATG, Cyclosporine, Levamisole; Rabbit ATG, Thymoglobuline (Genzyme); Rabbit anti-thymocyte globulin (ATG); Rabbit anti-thymocyte globulin (r-ATG); Rabbit antithymocyte globulin; Rabbit antithymoglobulin (ATG); Radiation: TBI; Radiation: Total Body Irradiation; Radiation: Total Body Irradiation (TBI); Radiation: Total Body Irradiation 1200 cGy; Radiation: Total Body Irradiation 200 cGy; Radiation: Total Lymphoid Irradiation (TLI); Radiation: Total body irradiation; Radiation: Total-Body Irradiation; Radiation: low-dose total body irradiation (TBI); Radiation: radiation therapy; Radiation: total body irradiation; Radiation: total-body irradiation; Rapamune; Rapamycin; Recombinant Methionyl Human Stem Cell Factor (r-metHuSCF); Revolade; Revolade 75 mg; RhTPO; Rimiducid; Rituxan; Rituximab; Rivogenlecleucel; Rivogenleucleucel; Romiplostim; SA; SB497115; SIROLIMUS; SODIUM CHLORIDE SOLUTION 0.9%; SULFAMETH; Sandimmun Neoral; Sargramostim; Sirolimus; Sodium chloride; Standard Vitamin D3 Supplementation; Standard therapy of septic shock; Stanozolol; Stem Cell Transplant; Sulfate; T Cell-Depleted Donor Lymphocyte Infusion; TACROLIMUS; THIOTEPA; TRIMETHOPRIM SULFATE; Tacrolimus; Testosterone; Testosterone undecanoate; Therapeutic allogeneic lymphocytes; Thiotepa; Thrombopoietin; Thymoglobulin; Thymoglobuline; Thymoglobuline®/Thymoglobulin®; Total Body Irradiation (TBI) (Plan 1); Trimethoprim; Umbilical Cord Blood; Umbilical Cord Blood After Myeloablative Conditioning; Umbilical Cord Blood After Reduced-Intensity Conditioning; Umbilical Cord Blood Transplantation; Valganciclovir; Vitamin D3; Voriconazole

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	NCT03413306 (ClinicalTrials.gov)	December 10, 2016	22/12/2017	Eltrombopag+hATG+CsA vs. hATG+CsA in Children With Severe AA	A Phase II Multicenter Randomized Study of Eltrombopag Combined With Cyclosporine and hATG Versus hATG and CsA as First Line Treatment in Pediatric Patients With Severe Acquired Aplastic Anemia	Acquired Aplastic Anemia	Drug: Eltrombopag;Drug: IST (ATG + CsA)	Federal Research Institute of Pediatric Hematology, Oncology and Immunology	NULL	Recruiting	2 Years	18 Years	All	100	Phase 3	Russian Federation

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NCT03413306
(ClinicalTrials.gov)

December 10, 2016

22/12/2017

Eltrombopag+hATG+CsA vs. hATG+CsA in Children With Severe AA

A Phase II Multicenter Randomized Study of Eltrombopag Combined With Cyclosporine and hATG Versus hATG and CsA as First Line Treatment in Pediatric Patients With Severe Acquired Aplastic Anemia

Acquired Aplastic Anemia

Drug: Eltrombopag;Drug: IST (ATG + CsA)

Federal Research Institute of Pediatric Hematology, Oncology and Immunology

NULL

Recruiting

2 Years

18 Years

All

100

Phase 3

Russian Federation