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	NCT04558736 (ClinicalTrials.gov)	November 2020	9/9/2020	Haploidentical HCT for Severe Aplastic Anemia	Haploidentical Donor Hematopoietic Cell Transplantation for Patients With Severe Aplastic Anemia	Aplastic Anemia;Bone Marrow Failure Syndrome;Dyskeratosis Congenita	Drug: Anti-Thymocyte Globulin (Rabbit);Drug: Fludarabine;Drug: Cyclophosphamide;Drug: Mesna;Drug: G-CSF;Radiation: Total Lymphoid Irradiation (TLI);Device: CliniMACS;Biological: HPC, A Infusion;Biological: CD45RA-depleted DLI	St. Jude Children's Research Hospital	NULL	Recruiting	N/A	21 Years	All	21	Phase 2	United States
2	EUCTR2007-000902-55-DE (EUCTR)	17/12/2009	29/09/2009	Prospective Phase II Pilot study of Rabbit Antithymocyte globulin(ATG, Thymoglobuline®, Genzyme) with Ciclosporin for Patients withAcquired Aplastic Anaemia and comparison with matched historicalpatients treated with horse ATG and Ciclosporin: a Study from theEuropean Blood and Marrow Transplant (EBMT) Severe AplasticAnaemia Working Party	Prospective Phase II Pilot study of Rabbit Antithymocyte globulin(ATG, Thymoglobuline®, Genzyme) with Ciclosporin for Patients withAcquired Aplastic Anaemia and comparison with matched historicalpatients treated with horse ATG and Ciclosporin: a Study from theEuropean Blood and Marrow Transplant (EBMT) Severe AplasticAnaemia Working Party	Acquired severe aplastic anaemia and transfusion dependent non-severe aplastic anaemia MedDRA version: 12.0;Level: LLT;Classification code 10002969;Term: Aplastic anemia	Trade Name: Thymoglobulin Product Name: Thymoglobuline ®/Thymoglobulin® Product Code: anti-thymocyte globulin (rabbit)	EBMT (European Group for Blood and Marrow Transplantation)	NULL	Not Recruiting			Female: yes Male: yes	35	Phase 2	France;Germany;United Kingdom
3	EUCTR2007-000902-55-FR (EUCTR)	23/04/2008	04/10/2007	Prospective Phase II Pilot study of Rabbit Antithymocyte globulin (ATG, Thymoglobuline®, Genzyme) with Ciclosporin for Patients with Acquired Aplastic Anaemia and comparison with matched historical patients treated with horse ATG and Ciclosporin	Prospective Phase II Pilot study of Rabbit Antithymocyte globulin (ATG, Thymoglobuline®, Genzyme) with Ciclosporin for Patients with Acquired Aplastic Anaemia and comparison with matched historical patients treated with horse ATG and Ciclosporin	Aquired aplastic anaemia and transfusion dependent non-severe aplastic anaemia MedDRA version: 9.1;Level: LLT;Classification code 10002274;Term: Anemia aplastic	Trade Name: Thymoglobuline Product Name: Thymoglobuline ®/Thymoglobulin® Product Code: anti-thymocyte globulin (rabbit) Other descriptive name: GLYCINE Other descriptive name: SODIUM CHLORIDE SOLUTION 0.9% Other descriptive name: Mannitol Trade Name: Neoral Product Name: Ciclosporine INN or Proposed INN: Cyclosporin A Other descriptive name: Ciclosporine	EBMT (European group for Blood and Marrow Transplantation)	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	35	Phase 2	Germany;United Kingdom;France
4	NCT00260689 (ClinicalTrials.gov)	November 28, 2005	1/12/2005	Three Immunosuppressive Treatment Regimens for Severe Aplastic Anemia	A Randomized Study of Three Immunosuppressive Regimens in Treatment Naive Patients With Severe Aplastic Anemia: Horse ATG/CsA Taper vs Rabbit-ATG/CsA vs Alemtuzumab	Immunosuppresion;Thrombocytopenia;Pancytopenia;Neutropenia	Biological: Anti-thymocyte globulin (rabbit);Biological: Anti-thymocyte globulin (horse);Drug: Cyclosporine;Drug: Alemtuzumab	National Heart, Lung, and Blood Institute (NHLBI)	NULL	Completed	2 Years	N/A	All	136	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

NCT04558736
(ClinicalTrials.gov)

November 2020

9/9/2020

Haploidentical HCT for Severe Aplastic Anemia

Haploidentical Donor Hematopoietic Cell Transplantation for Patients With Severe Aplastic Anemia

Aplastic Anemia;Bone Marrow Failure Syndrome;Dyskeratosis Congenita

Drug: Anti-Thymocyte Globulin (Rabbit);Drug: Fludarabine;Drug: Cyclophosphamide;Drug: Mesna;Drug: G-CSF;Radiation: Total Lymphoid Irradiation (TLI);Device: CliniMACS;Biological: HPC, A Infusion;Biological: CD45RA-depleted DLI

St. Jude Children's Research Hospital

NULL

Recruiting

N/A

21 Years

All

Phase 2

United States

EUCTR2007-000902-55-DE
(EUCTR)

17/12/2009

29/09/2009

Prospective Phase II Pilot study of Rabbit Antithymocyte globulin(ATG, Thymoglobuline®, Genzyme) with Ciclosporin for Patients withAcquired Aplastic Anaemia and comparison with matched historicalpatients treated with horse ATG and Ciclosporin: a Study from theEuropean Blood and Marrow Transplant (EBMT) Severe AplasticAnaemia Working Party

Acquired severe aplastic anaemia and transfusion dependent non-severe aplastic anaemia
MedDRA version: 12.0;Level: LLT;Classification code 10002969;Term: Aplastic anemia

Trade Name: Thymoglobulin
Product Name: Thymoglobuline ®/Thymoglobulin®
Product Code: anti-thymocyte globulin (rabbit)

EBMT (European Group for Blood and Marrow Transplantation)

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

France;Germany;United Kingdom

EUCTR2007-000902-55-FR
(EUCTR)

23/04/2008

04/10/2007

Prospective Phase II Pilot study of Rabbit Antithymocyte globulin (ATG, Thymoglobuline®, Genzyme) with Ciclosporin for Patients with Acquired Aplastic Anaemia and comparison with matched historical patients treated with horse ATG and Ciclosporin

Aquired aplastic anaemia and transfusion dependent non-severe aplastic anaemia
MedDRA version: 9.1;Level: LLT;Classification code 10002274;Term: Anemia aplastic

Trade Name: Thymoglobuline
Product Name: Thymoglobuline ®/Thymoglobulin®
Product Code: anti-thymocyte globulin (rabbit)
Other descriptive name: GLYCINE
Other descriptive name: SODIUM CHLORIDE SOLUTION 0.9%
Other descriptive name: Mannitol
Trade Name: Neoral
Product Name: Ciclosporine
INN or Proposed INN: Cyclosporin A
Other descriptive name: Ciclosporine

EBMT (European group for Blood and Marrow Transplantation)

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

Germany;United Kingdom;France

NCT00260689
(ClinicalTrials.gov)

November 28, 2005

1/12/2005

Three Immunosuppressive Treatment Regimens for Severe Aplastic Anemia

A Randomized Study of Three Immunosuppressive Regimens in Treatment Naive Patients With Severe Aplastic Anemia: Horse ATG/CsA Taper vs Rabbit-ATG/CsA vs Alemtuzumab

Immunosuppresion;Thrombocytopenia;Pancytopenia;Neutropenia

Biological: Anti-thymocyte globulin (rabbit);Biological: Anti-thymocyte globulin (horse);Drug: Cyclosporine;Drug: Alemtuzumab

National Heart, Lung, and Blood Institute (NHLBI)

NULL

Completed

2 Years

N/A

All

136

Phase 2

United States