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	NCT00455312 (ClinicalTrials.gov)	August 2007	30/3/2007	Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA	Hematopoietic Stem Cell Transplant For Patients With Dyskeratosis Congenita and Severe Aplastic Anemia	Dyskeratosis Congenita;Aplastic Anemia	Drug: Campath 1H;Drug: Cyclophosphamide;Drug: Fludarabine;Procedure: Total Body Irradiation;Procedure: Stem Cell Transplantation;Drug: antithymocyte globulin;Drug: Methylprednisolone	Masonic Cancer Center, University of Minnesota	NULL	Completed	N/A	70 Years	All	36	Phase 2;Phase 3	United States
2	NCT00195624 (ClinicalTrials.gov)	September 15, 2005	16/9/2005	Alemtuzumab to Treat Severe Aplastic Anemia	A Pilot Study of Alemtuzumab (Campath) in Patients With Relapsed or Refractory Severe Aplastic Anemia	Severe Aplastic Anemia, Refractory;Severe Aplastic Anemia, Relapse	Biological: Alemtuzumab (Campath );Drug: Cyclosporine	National Heart, Lung, and Blood Institute (NHLBI)	NULL	Completed	2 Years	110 Years	All	47	Phase 2	United States
3	NCT00065260 (ClinicalTrials.gov)	July 2003	18/7/2003	Rabbit Antithymocyte Globulin Versus Campath-1H for Treating Severe Aplastic Anemia	A Randomized Trial of Immunosuppression in Aplastic Anemia Patients With Refractory Pancytopenia or Suboptimal Hematologic Response After h-ATG/CsA Treatment	Aplastic Anemia	Drug: Campath -1H;Drug: r-ATG;Drug: CsA	National Heart, Lung, and Blood Institute (NHLBI)	NULL	Completed	2 Years	N/A	All	54	Phase 2	United States
4	NCT00533923 (ClinicalTrials.gov)	December 2002	20/9/2007	Nonmyeloablative Allogeneic Stem Cell Transplantation From HLA-Matched Unrelated Donor for the Treatment of Hematologic Disorders	Nonmyeloablative Allogeneic Stem Cell Transplantation From HLA-Matched Unrelated Donor for the Treatment of Hematologic Disorders	AML;ALL;CLL;Myelodysplastic Syndrome;Non-Hodgkin's Lymphoma;Hodgkin's Lymphoma;Multiple Myeloma;Aplastic Anemia;Myeloproliferative Disorder	Drug: Cyclophosphamide; Fludarabine; Cyclosporin; CAMPATH-1H (Alemtuzumab); GM-CSF	Beth Israel Deaconess Medical Center	Bayer	Completed	N/A	65 Years	Both	25	Phase 2	United States
5	NCT00578903 (ClinicalTrials.gov)	February 2002	19/12/2007	Allogeneic Stem Cell Transplant for Patients With Severe Aplastic Anemia	Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia, Using Matched Unrelated Donors and Mismatched Related Donors (SAA MUD)	Aplastic Anemia	Drug: Cytoxan;Drug: Campath;Radiation: Total Body Irradiation (TBI);Drug: FK-506;Drug: Methotrexate;Procedure: Stem cell infusion	Baylor College of Medicine	The Methodist Hospital System;Texas Children's Hospital;Center for Cell and Gene Therapy, Baylor College of Medicine	Terminated	N/A	60 Years	All	22	Phase 2	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
6	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
7	NCT00004143 (ClinicalTrials.gov)	September 1999	10/12/1999	Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes	Allogeneic Mixed Chimerism Stem Cell Transplantation Utilizing In Vivo and In Vitro Campath for Hemoglobinopathies and Bone Marrow Failure Syndromes	Sickle Cell Anemia;Severe Aplastic Anemia;Paroxysmal Nocturnal Hemoglobinuria (PNH);Pure Red Cell Aplasia	Drug: Campath , Chemo and/or TBI Allo SCT	David Rizzieri, MD	NULL	Completed	18 Years	N/A	All	2	Phase 2	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

NCT00455312
(ClinicalTrials.gov)

August 2007

30/3/2007

Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA

Hematopoietic Stem Cell Transplant For Patients With Dyskeratosis Congenita and Severe Aplastic Anemia

Dyskeratosis Congenita;Aplastic Anemia

Drug: Campath 1H;Drug: Cyclophosphamide;Drug: Fludarabine;Procedure: Total Body Irradiation;Procedure: Stem Cell Transplantation;Drug: antithymocyte globulin;Drug: Methylprednisolone

Masonic Cancer Center, University of Minnesota

NULL

Completed

N/A

70 Years

All

Phase 2;Phase 3

United States

NCT00195624
(ClinicalTrials.gov)

September 15, 2005

16/9/2005

Alemtuzumab to Treat Severe Aplastic Anemia

A Pilot Study of Alemtuzumab (Campath) in Patients With Relapsed or Refractory Severe Aplastic Anemia

Severe Aplastic Anemia, Refractory;Severe Aplastic Anemia, Relapse

Biological: Alemtuzumab (Campath );Drug: Cyclosporine

National Heart, Lung, and Blood Institute (NHLBI)

NULL

Completed

2 Years

110 Years

All

Phase 2

United States

NCT00065260
(ClinicalTrials.gov)

July 2003

18/7/2003

Rabbit Antithymocyte Globulin Versus Campath-1H for Treating Severe Aplastic Anemia

A Randomized Trial of Immunosuppression in Aplastic Anemia Patients With Refractory Pancytopenia or Suboptimal Hematologic Response After h-ATG/CsA Treatment

Aplastic Anemia

Drug: Campath -1H;Drug: r-ATG;Drug: CsA

National Heart, Lung, and Blood Institute (NHLBI)

NULL

Completed

2 Years

N/A

All

Phase 2

United States

NCT00533923
(ClinicalTrials.gov)

December 2002

20/9/2007

Nonmyeloablative Allogeneic Stem Cell Transplantation From HLA-Matched Unrelated Donor for the Treatment of Hematologic Disorders

AML;ALL;CLL;Myelodysplastic Syndrome;Non-Hodgkin's Lymphoma;Hodgkin's Lymphoma;Multiple Myeloma;Aplastic Anemia;Myeloproliferative Disorder

Drug: Cyclophosphamide; Fludarabine; Cyclosporin; CAMPATH-1H (Alemtuzumab); GM-CSF

Beth Israel Deaconess Medical Center

Bayer

Completed

N/A

65 Years

Both

Phase 2

United States

NCT00578903
(ClinicalTrials.gov)

February 2002

19/12/2007

Allogeneic Stem Cell Transplant for Patients With Severe Aplastic Anemia

Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia, Using Matched Unrelated Donors and Mismatched Related Donors (SAA MUD)

Aplastic Anemia

Drug: Cytoxan;Drug: Campath;Radiation: Total Body Irradiation (TBI);Drug: FK-506;Drug: Methotrexate;Procedure: Stem cell infusion

Baylor College of Medicine

The Methodist Hospital System;Texas Children's Hospital;Center for Cell and Gene Therapy, Baylor College of Medicine

Terminated

N/A

60 Years

All

Phase 2

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

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

All

Phase 1;Phase 2

United States

NCT00004143
(ClinicalTrials.gov)

September 1999

10/12/1999

Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes

Allogeneic Mixed Chimerism Stem Cell Transplantation Utilizing In Vivo and In Vitro Campath for Hemoglobinopathies and Bone Marrow Failure Syndromes

Sickle Cell Anemia;Severe Aplastic Anemia;Paroxysmal Nocturnal Hemoglobinuria (PNH);Pure Red Cell Aplasia

Drug: Campath , Chemo and/or TBI Allo SCT

David Rizzieri, MD

NULL

Completed

18 Years

N/A

All

Phase 2

United States