DDrare: Database of Drug Development for Rare Diseases

285. ファンコニ貧血
［臨床試験数：56，薬物数：111（DrugBank：31），標的遺伝子数：30，標的パスウェイ数：151］

Searched query = "Fanconi 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 = AMD3100; AP1903; AP1903 A594; ATG; Abatacept; Alefacept; Anti-CD45; Anti-Thymocyte Globulin; Anti-Thymocyte Globulin (Rabbit); Anti-thymocyte globulin; BPX-501; BPX-501 T cells; BPX-501 cells; Busulfan; Busulfan, fludarabine, & cyclophosphamide with immunosuppression with ATG and cyclosporine.; CAMPATH-1H; CD34+ CELLS; CD34+ Cells; CD34+ selected cells; CD34+CELLS; Campath; Campath 1h; Chemotherapy; Cyclophosphamide; Cyclophosphamide (CY) (Plan 1); Cyclophosphamide (CY) (Plan 2); Cyclophosphamide 30; Cyclophosphamide 40; Cyclophosphamide, Fludarabine; Cyclosporine; Danazol; Device: AmCell CliniMACs; Device: CliniMACS device; Device: CliniMACs device; Device: Donor mobilized PBSC infusion; Device: The CliniMACS device; ENBREL*SC 4FL 25MG+4SIR 1ML; Eltrombopag; Enbrel; Etanercept; FP-045; Filgrastim; Fludarabine; Fludarabine (FLU); Fludarabine Phosphate; Fludarabine phosphate; G-CSF; Genetically Engineered Hematopoietic Stem Progenitor Cells; Genetically Engineered Hematopoietic Stem/Progenitor Cells; Graft-versus-tumor induction therapy; Hematopoietic stem cell transplantation; Melphalan; Metformin; Metformin HCl; Methotrexate; Methylprednisolone; Methylprednisolone (MP); Miltenyi CliniMACS; Mozobil; Mycophenolate; Mycophenolate Mofetil; Mycophenolate mofetil; Neupogen; Olaparib; Other: Laboratory Biomarker Analysis; Oxandrolone; Pembrolizumab; Peripheral blood stem cell; Phosphate; Plerixafor; Plérixafor; Prednisone; Procedure: Allogeneic Bone Marrow Transplantation; Procedure: Bone Marrow Aspiration; Procedure: Bone Marrow Transplantation; Procedure: CD34 selected haploidentical PBSCT; Procedure: Hematopoietic Stem Cell Transplant; Procedure: Hematopoietic Stem Cell Transplantation; Procedure: IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs); Procedure: Leukapheresis; Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation; Procedure: Stem cell infusion; Procedure: Thymic Shielding During Radiation; Procedure: Total Body Irradiation; Procedure: allogeneic bone marrow transplantation; Procedure: allogeneic hematopoietic stem cell transplantation; Procedure: nonmyeloablative allogeneic hematopoietic stem cell transplantation; Procedure: peripheral blood stem cell transplantation; Procedure: radiation therapy; Procedure: umbilical cord blood transplantation; Quercetin; Quercetin (dietary supplement); RP-L102; Rabbit; Rabbit ATG; Radiation: Total body irradiation (TBI); Radiation: Total-Body Irradiation; Radiation: radiation therapy; Radiation: total-body irradiation; Rimiducid; Rituximab; Rivogenlecleucel; Rivogenleucleucel; Sargramostim; Sirolimus; T cell infusion; Tacrolimus; Therapeutic allogeneic lymphocytes; Total Body Irradiation (TBI) (Plan 1); Transduced CD34+ Cells; Zarzio

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	NCT00965666 (ClinicalTrials.gov)	October 2005	24/8/2009	Pilot Study of Etanercept (Enbrel) in Children With Fanconi Anemia	Etanercept (Enbrel) in Children With Fanconi Anemia and Early Bone Marrow Failure: A Pilot Study	Fanconi Anemia	Drug: Etanercept	Children's Hospital Medical Center, Cincinnati	Amgen	Completed	4 Years	N/A	All	3	Early Phase 1	United States
2	EUCTR2004-004914-18-IT (EUCTR)	21/01/2005	27/07/2005	A pilot multicentric study for the use of the anti-TNF alfa in patients with: 1) Fanconi Anemia with marrow failure with no bone marrow compatible donor 2) In patients with acquired aplastic anemia failing immunosuppression and with no bone marrow donor	A pilot multicentric study for the use of the anti-TNF alfa in patients with: 1) Fanconi Anemia with marrow failure with no bone marrow compatible donor 2) In patients with acquired aplastic anemia failing immunosuppression and with no bone marrow donor	Marrow failure in patients refractary/non eligible to conventional treatments;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Trade Name: ENBREL *SC 4FL 25MG+4SIR 1ML INN or Proposed INN: Etanercept	ISTITUTO GIANNINA GASLINI	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes			Italy

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

NCT00965666
(ClinicalTrials.gov)

October 2005

24/8/2009

Pilot Study of Etanercept (Enbrel) in Children With Fanconi Anemia

Etanercept (Enbrel) in Children With Fanconi Anemia and Early Bone Marrow Failure: A Pilot Study

Fanconi Anemia

Drug: Etanercept

Children's Hospital Medical Center, Cincinnati

Amgen

Completed

4 Years

N/A

All

Early Phase 1

United States

EUCTR2004-004914-18-IT
(EUCTR)

21/01/2005

27/07/2005

A pilot multicentric study for the use of the anti-TNF alfa in patients with: 1) Fanconi Anemia with marrow failure with no bone marrow compatible donor 2) In patients with acquired aplastic anemia failing immunosuppression and with no bone marrow donor

Marrow failure in patients refractary/non eligible to conventional treatments;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Trade Name: ENBREL *SC 4FL 25MG+4SIR 1ML
INN or Proposed INN: Etanercept

ISTITUTO GIANNINA GASLINI

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Italy