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	NCT04478227 (ClinicalTrials.gov)	August 18, 2020	10/7/2020	TPO-Mimetic Use in Children for Hemotopoietic Failure	Single Arm Prospective Open Label Pilot Study Evaluating Short-Term Safety and Efficacy of Romiplostim in Children With Inherited and Acquired Disorders of Hematopoietic Failure	Bone Marrow Failure Disorders;Aplastic Anemia;Thrombocytopenia;Refractory Cytopenia of Childhood;Myelodysplastic Syndrome(MDS)	Drug: Romiplostim	Anjali Sharathkumar	Amgen	Recruiting	N/A	21 Years	All	25	Early Phase 1	United States
2	JPRN-JapicCTI-194962	03/12/2019	18/09/2019	A Phase 2/3 Study of AMG531 in Patients with Aplastic Anemia	A Phase 2/3 Study of AMG531 Combined with Ciclosporin A in Patients with Aplastic Anemia Previously Untreated with Immunosuppressive Therapy	Aplastic Anemia	Intervention name : AMG531 INN of the intervention : romiplostim Dosage And administration of the intervention : SC-administered Control intervention name : - INN of the control intervention : - Dosage And administration of the control intervention : -	Kyowa Kirin Co., Ltd	NULL	recruiting	20		BOTH	24	Phase 2;Phase 3	Japan, Asia except Japan
3	NCT04095936 (ClinicalTrials.gov)	December 3, 2019	18/9/2019	Study of AMG531 (Romiplostim) in Patients With Aplastic Anemia	A Phase 2/3 Study of AMG531 Combined With Ciclosporin A in Patients With Aplastic Anemia Previously Untreated With Immunosuppressive Therapy	Aplastic Anemia	Drug: Romiplostim	Kyowa Kirin Co., Ltd.	NULL	Recruiting	20 Years	N/A	All	24	Phase 2;Phase 3	Japan
4	NCT03957694 (ClinicalTrials.gov)	April 25, 2019	9/5/2019	Study of AMG531(Romiplostim) in Patients With Aplastic Anemia	A Phase 2/3 Study of AMG531 in Patients With Aplastic Anemia PreviouslyUntreated With Immunosuppressive Therapy	Aplastic Anemia	Drug: Romiplostim	Kyowa Kirin Co., Ltd.	NULL	Active, not recruiting	20 Years	N/A	All	14	Phase 2;Phase 3	Japan
5	JPRN-JapicCTI-194746	01/2/2019	09/05/2019	A Phase 2/3 Study of AMG531 in Patients with Aplastic Anemia	A Phase 2/3 Study of AMG531 in Patients with Aplastic Anemia Previously Untreated with Immunosuppressive Therapy	Aplastic Anemia	Intervention name : AMG531 INN of the intervention : romiplostim Dosage And administration of the intervention : SC-administered Control intervention name : - INN of the control intervention : - Dosage And administration of the control intervention : -	Kyowa Kirin Co., Ltd	NULL	complete	20		BOTH	14	Phase 2;Phase 3	Japan, Asia except Japan
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	NCT02773290 (ClinicalTrials.gov)	May 2016	9/5/2016	Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia	Phase 2/3 Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia Refractory to or Ineligible for Immunosuppressive Therapy	Aplastic Anemia	Biological: Romiplostim	Kyowa Kirin Co., Ltd.	NULL	Active, not recruiting	20 Years	N/A	All	46	Phase 2;Phase 3	Japan;Korea, Republic of
7	JPRN-JapicCTI-163243	01/2/2016	02/05/2016	Phase 2/3 Study of AMG531 in Subjects with Aplastic Anemia	Phase 2/3 Study of AMG531 in Subjects with Aplastic Anemia Refractory to or Ineligible for Immunosuppressive Therapy	Aplastic Anemia	Intervention name : AMG531 INN of the intervention : Romiplostim Dosage And administration of the intervention : Subcutaneous administration	Kyowa Hakko Kirin Co., Ltd.	NULL	complete	20		BOTH	46	Phase 2;Phase 3	NULL

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

NCT04478227
(ClinicalTrials.gov)

August 18, 2020

10/7/2020

TPO-Mimetic Use in Children for Hemotopoietic Failure

Single Arm Prospective Open Label Pilot Study Evaluating Short-Term Safety and Efficacy of Romiplostim in Children With Inherited and Acquired Disorders of Hematopoietic Failure

Bone Marrow Failure Disorders;Aplastic Anemia;Thrombocytopenia;Refractory Cytopenia of Childhood;Myelodysplastic Syndrome(MDS)

Drug: Romiplostim

Anjali Sharathkumar

Amgen

Recruiting

N/A

21 Years

All

Early Phase 1

United States

JPRN-JapicCTI-194962

03/12/2019

18/09/2019

A Phase 2/3 Study of AMG531 in Patients with Aplastic Anemia

A Phase 2/3 Study of AMG531 Combined with Ciclosporin A in Patients with Aplastic Anemia Previously Untreated with Immunosuppressive Therapy

Aplastic Anemia

Intervention name : AMG531
INN of the intervention : romiplostim
Dosage And administration of the intervention : SC-administered
Control intervention name : -
INN of the control intervention : -
Dosage And administration of the control intervention : -

Kyowa Kirin Co., Ltd

NULL

recruiting

BOTH

Phase 2;Phase 3

Japan, Asia except Japan

NCT04095936
(ClinicalTrials.gov)

December 3, 2019

18/9/2019

Study of AMG531 (Romiplostim) in Patients With Aplastic Anemia

A Phase 2/3 Study of AMG531 Combined With Ciclosporin A in Patients With Aplastic Anemia Previously Untreated With Immunosuppressive Therapy

Aplastic Anemia

Drug: Romiplostim

Kyowa Kirin Co., Ltd.

NULL

Recruiting

20 Years

N/A

All

Phase 2;Phase 3

Japan

NCT03957694
(ClinicalTrials.gov)

April 25, 2019

9/5/2019

Study of AMG531(Romiplostim) in Patients With Aplastic Anemia

A Phase 2/3 Study of AMG531 in Patients With Aplastic Anemia PreviouslyUntreated With Immunosuppressive Therapy

Aplastic Anemia

Drug: Romiplostim

Kyowa Kirin Co., Ltd.

NULL

Active, not recruiting

20 Years

N/A

All

Phase 2;Phase 3

Japan

JPRN-JapicCTI-194746

01/2/2019

09/05/2019

A Phase 2/3 Study of AMG531 in Patients with Aplastic Anemia

A Phase 2/3 Study of AMG531 in Patients with Aplastic Anemia Previously Untreated with Immunosuppressive Therapy

Aplastic Anemia

Kyowa Kirin Co., Ltd

NULL

complete

BOTH

Phase 2;Phase 3

Japan, Asia except Japan

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

NCT02773290
(ClinicalTrials.gov)

May 2016

9/5/2016

Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia

Phase 2/3 Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia Refractory to or Ineligible for Immunosuppressive Therapy

Aplastic Anemia

Biological: Romiplostim

Kyowa Kirin Co., Ltd.

NULL

Active, not recruiting

20 Years

N/A

All

Phase 2;Phase 3

Japan;Korea, Republic of

JPRN-JapicCTI-163243

01/2/2016

02/05/2016

Phase 2/3 Study of AMG531 in Subjects with Aplastic Anemia

Phase 2/3 Study of AMG531 in Subjects with Aplastic Anemia Refractory to or Ineligible for Immunosuppressive Therapy

Aplastic Anemia

Intervention name : AMG531
INN of the intervention : Romiplostim
Dosage And administration of the intervention : Subcutaneous administration

Kyowa Hakko Kirin Co., Ltd.

NULL

complete

BOTH

Phase 2;Phase 3

NULL