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	EUCTR2007-001657-26-GB (EUCTR)	29/02/2008	07/11/2007	Single Centre Phase II Pilot study of Unrelated Cord Blood Transplantation in Patients with Poor Risk Haematological Malignancies.	Single Centre Phase II Pilot study of Unrelated Cord Blood Transplantation in Patients with Poor Risk Haematological Malignancies.	1.Acute, chronic leukaemia or myelodysplastic syndrome for which allogeneic transplantation is considered as the best treatment option. 2.Acute lymphoblastic leukaemia (ALL)3.Non-Hodgkin’s lymphoma4.Hodgkin’s disease5.Chronic lymphocytic leukaemia. 6.Acquired bone marrow failure syndromes7.Other haematological malignancies for which UD bone marrow transplantation is indicated MedDRA version: 9.1;Level: LLT;Classification code 10000880;Term: Acute myeloid leukaemia MedDRA version: 9.1;Classification code 10028533;Term: Myelodysplastic syndrome MedDRA version: 9.1;Classification code 10009013;Term: Chronic myeloid leukaemia MedDRA version: 9.1;Classification code 10000844;Term: Acute lymphoblastic leukaemia MedDRA version: 9.1;Classification code 10020328;Term: Hodgkin's lymphoma MedDRA version: 9.1;Classification code 10029593;Term: Non-Hodgkin's lymphoma NOS MedDRA version: 9.1;Classification code 10003892;Term: B-cell chronic lymphocytic leukaemia/prolymphocytic leukaemia/small lymphocytic lymphoma MedDRA version: 9.1;Classification code 10002968;Term: Aplastic anaemia, unspecified	Trade Name: Fludarabine INN or Proposed INN: FLUDARABINE PHOSPHATE Trade Name: Busilvex INN or Proposed INN: BUSULFAN Trade Name: Thymoglobulin Other descriptive name: RABBIT HUMAN T LYMPHOCYTE IMMUNOGLOBULIN Trade Name: Thiotepa INN or Proposed INN: THIOTEPA Trade Name: Cyclophosphamide Other descriptive name: CYCLOPHOSPHAMIDE MONOHYDRATE Trade Name: Alkeran INN or Proposed INN: MELPHALAN	King's College Hospital NHS Foundation Trust	NULL	Not Recruiting			Female: yes Male: yes	27	Phase 2	United Kingdom
2	NCT00003816 (ClinicalTrials.gov)	October 19, 1998	1/11/1999	Combination Chemotherapy and Donor Stem Cell Transplant in Treating Patients With Aplastic Anemia or Hematologic Cancer	Allogeneic Blood or Marrow Transplantation for Hematologic Malignancy and Aplastic Anemia	Chronic Myeloproliferative Disorders;Leukemia;Lymphoma;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative Diseases;Nonmalignant Neoplasm;Unspecified Adult Solid Tumor, Protocol Specific;Unspecified Childhood Solid Tumor, Protocol Specific	Biological: anti-thymocyte globulin;Drug: busulfan;Drug: carboplatin;Drug: cyclophosphamide;Drug: etoposide;Drug: fludarabine phosphate;Drug: melphalan;Drug: thiotepa;Radiation: total-body irradiation	Roswell Park Cancer Institute	NULL	Completed	4 Years	70 Years	All	362	Phase 2;Phase 3	United States
3	NCT00002718 (ClinicalTrials.gov)	November 1995	1/11/1999	T-cell Depleted Bone Marrow and G-CSF Stimulated Peripheral Stem Cell Transplantation From Related Donors in Treating Patients With Leukemia, Lymphoblastic Lymphoma, Myelodysplastic Syndrome, or Aplastic Anemia	A Phase II Trial of T-Cell Depleted Marrow Grafts Combined With Infusions of G-CSF Stimulated, CD34 Ceprate Stem Cell Column Selected, E-Rosette Depleted Peripheral Blood Progenitor Cells Derived From HLA Haplotype Matched Related Donors for Patients With Leukemia Lacking an HLA-Matched Related or Unrelated Donor	Leukemia;Lymphoma;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative Neoplasms	Biological: anti-thymocyte globulin;Biological: filgrastim;Drug: cyclophosphamide;Drug: cytarabine;Drug: methylprednisolone;Drug: thiotepa;Procedure: in vitro-treated bone marrow transplantation;Procedure: in vitro-treated peripheral blood stem cell transplantation;Radiation: radiation therapy	Memorial Sloan Kettering Cancer Center	National Cancer Institute (NCI)	Completed	N/A	49 Years	Both	31	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

EUCTR2007-001657-26-GB
(EUCTR)

29/02/2008

07/11/2007

Single Centre Phase II Pilot study of Unrelated Cord Blood Transplantation in Patients with Poor Risk Haematological Malignancies.

1.Acute, chronic leukaemia or myelodysplastic syndrome for which allogeneic transplantation is considered as the best treatment option. 2.Acute lymphoblastic leukaemia (ALL)3.Non-Hodgkin’s lymphoma4.Hodgkin’s disease5.Chronic lymphocytic leukaemia. 6.Acquired bone marrow failure syndromes7.Other haematological malignancies for which UD bone marrow transplantation is indicated
MedDRA version: 9.1;Level: LLT;Classification code 10000880;Term: Acute myeloid leukaemia
MedDRA version: 9.1;Classification code 10028533;Term: Myelodysplastic syndrome
MedDRA version: 9.1;Classification code 10009013;Term: Chronic myeloid leukaemia
MedDRA version: 9.1;Classification code 10000844;Term: Acute lymphoblastic leukaemia
MedDRA version: 9.1;Classification code 10020328;Term: Hodgkin's lymphoma
MedDRA version: 9.1;Classification code 10029593;Term: Non-Hodgkin's lymphoma NOS
MedDRA version: 9.1;Classification code 10003892;Term: B-cell chronic lymphocytic leukaemia/prolymphocytic leukaemia/small lymphocytic lymphoma
MedDRA version: 9.1;Classification code 10002968;Term: Aplastic anaemia, unspecified

Trade Name: Fludarabine
INN or Proposed INN: FLUDARABINE PHOSPHATE
Trade Name: Busilvex
INN or Proposed INN: BUSULFAN
Trade Name: Thymoglobulin
Other descriptive name: RABBIT HUMAN T LYMPHOCYTE IMMUNOGLOBULIN
Trade Name: Thiotepa
INN or Proposed INN: THIOTEPA
Trade Name: Cyclophosphamide
Other descriptive name: CYCLOPHOSPHAMIDE MONOHYDRATE
Trade Name: Alkeran
INN or Proposed INN: MELPHALAN

King's College Hospital NHS Foundation Trust

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

United Kingdom

NCT00003816
(ClinicalTrials.gov)

October 19, 1998

1/11/1999

Combination Chemotherapy and Donor Stem Cell Transplant in Treating Patients With Aplastic Anemia or Hematologic Cancer

Allogeneic Blood or Marrow Transplantation for Hematologic Malignancy and Aplastic Anemia

Chronic Myeloproliferative Disorders;Leukemia;Lymphoma;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative Diseases;Nonmalignant Neoplasm;Unspecified Adult Solid Tumor, Protocol Specific;Unspecified Childhood Solid Tumor, Protocol Specific

Biological: anti-thymocyte globulin;Drug: busulfan;Drug: carboplatin;Drug: cyclophosphamide;Drug: etoposide;Drug: fludarabine phosphate;Drug: melphalan;Drug: thiotepa;Radiation: total-body irradiation

Roswell Park Cancer Institute

NULL

Completed

4 Years

70 Years

All

362

Phase 2;Phase 3

United States

NCT00002718
(ClinicalTrials.gov)

November 1995

1/11/1999

T-cell Depleted Bone Marrow and G-CSF Stimulated Peripheral Stem Cell Transplantation From Related Donors in Treating Patients With Leukemia, Lymphoblastic Lymphoma, Myelodysplastic Syndrome, or Aplastic Anemia

A Phase II Trial of T-Cell Depleted Marrow Grafts Combined With Infusions of G-CSF Stimulated, CD34 Ceprate Stem Cell Column Selected, E-Rosette Depleted Peripheral Blood Progenitor Cells Derived From HLA Haplotype Matched Related Donors for Patients With Leukemia Lacking an HLA-Matched Related or Unrelated Donor

Leukemia;Lymphoma;Myelodysplastic Syndromes;Myelodysplastic/Myeloproliferative Neoplasms

Biological: anti-thymocyte globulin;Biological: filgrastim;Drug: cyclophosphamide;Drug: cytarabine;Drug: methylprednisolone;Drug: thiotepa;Procedure: in vitro-treated bone marrow transplantation;Procedure: in vitro-treated peripheral blood stem cell transplantation;Radiation: radiation therapy

Memorial Sloan Kettering Cancer Center

National Cancer Institute (NCI)

Completed

N/A

49 Years

Both

Phase 2

United States