DDrare: Database of Drug Development for Rare Diseases

284. ダイアモンド・ブラックファン貧血
［臨床試験数：37，薬物数：110（DrugBank：34），標的遺伝子数：22，標的パスウェイ数：121］

Searched query = "Diamond-Blackfan 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 = (S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazo; (S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide; (S)-4,5-dihydro-2[hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-m; (S)-4,5-dihydro-2[hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide; Abatacept; Alefacept; Alemtuzumab; Alpha beta depletion; Anti-thymocyte globulin; Antithymocyte globulin; BPX-501 T cells; Busulfan; Busulfan, Cyclophosphamide, ATG, GCSF; Busulfan, Fludarabine, ATG, TLI; CD3/CD19 depleted leukocytes; CD34 Stem Cell Selection Therapy; CD45RA depleted leukocytes; Campath, Fludarabine, Cyclophosphamide; Cyclophosphamide; Cyclophosphamide Dose Level 1; Cyclophosphamide Dose Level 2; Cyclophosphamide Dose Level 3; Cyclophosphamide Dose Level 4; Cyclosporine; DFO; Daclizumab; Deferasirox; Deferitazole; Deferoxamine; Desferal; EXJADE; Eltrombopag; Enriched Hematopoetic Stem Cell Infusion; Exjade; FBS0701; FBS0701 CAPSULE; FBS0701 Capsule; FBS0701/SSP004184; Filgrastim; Fludarabine; Fludarabine monophosphate; Fludarabine phosphate; G-CSF; Human Placental Derived Stem Cell; Hydroxide; Hydroxyurea; ICL670; IRON CHELATING AGENTS; Iron; Iron chelating agents; L-leucine; Lenalidomide; Leucine; Magnesium; Magnesium hydroxide; Melphalan; Mesenchymal Stromal Cells; Methotrexate; Methylprednisolone; Mycophenolate; Mycophenolate mofetil; Myeloablative Preparative Regimen; Other: laboratory biomarker analysis; Phosphate; Prednisone; Procedure: Bone marrow transplantation; Procedure: Leukapheresis; Procedure: Stem cell infusion; Procedure: Stem cell transplant; Procedure: Total lymphoid irradiation; Procedure: allogeneic bone marrow transplantation; Procedure: allogeneic hematopoietic stem cell transplantation; Procedure: peripheral blood stem cell transplantation; Procedure: radiation therapy; Procedure: umbilical cord blood transplantation; Radiation: Total Body Irradiation; Radiation: radiation therapy; Radiation: total-body irradiation; Reduced Intensity Preparative Regimen; Reduced Toxicity Ablative Regimen; Rimiducid; Rituximab; Rivogenlecleucel; SSP-004184 100 mg; SSP-004184 100 mg Capsule; SSP-004184 250 mg; SSP-004184 250 mg Capsule; SSP-004184 375 mg; SSP-004184 375 mg Capsule; SSP-004184 50 mg; SSP-004184 50 mg Capsule; SSP-004184 500 mg; SSP-004184 500 mg Capsule; SSP-004184AQ 100 mg; SSP-004184AQ 100 mg Capsule; SSP-004184AQ 200 mg; SSP-004184AQ 200 mg Capsule; SSP-004184AQ 250 mg; SSP-004184AQ 250 mg Capsule; SSP-004184AQ 375 mg; SSP-004184AQ 375 mg Capsule; SSP-004184AQ 50 mg; SSP-004184AQ 50 mg Capsule; Sotatercept; Sotatercept with prednisone boost; Tacrolimus; Thiotepa; Thiotepa--escalated dose; Thiotepa--single daily dose; Trifluoperazine

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	NCT00176852 (ClinicalTrials.gov)	June 2002	12/9/2005	Stem Cell Transplant for Hemoglobinopathy	Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathy Using a Preparative Regimen to Achieve Stable Mixed Chimerism	Sickle Cell Disease;Thalassemia;Severe Congenital Neutropenia;Diamond-Blackfan Anemia;Shwachman-Diamond Syndrome	Drug: Busulfan , Fludarabine, ATG, TLI;Drug: Busulfan , Cyclophosphamide, ATG, GCSF;Drug: Campath, Fludarabine, Cyclophosphamide;Radiation: Total Body Irradiation;Procedure: Stem cell infusion	Masonic Cancer Center, University of Minnesota	National Marrow Donor Program	Completed	N/A	50 Years	All	22	Phase 2;Phase 3	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

NCT00176852
(ClinicalTrials.gov)

June 2002

12/9/2005

Stem Cell Transplant for Hemoglobinopathy

Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathy Using a Preparative Regimen to Achieve Stable Mixed Chimerism

Sickle Cell Disease;Thalassemia;Severe Congenital Neutropenia;Diamond-Blackfan Anemia;Shwachman-Diamond Syndrome

Drug: Busulfan , Fludarabine, ATG, TLI;Drug: Busulfan , Cyclophosphamide, ATG, GCSF;Drug: Campath, Fludarabine, Cyclophosphamide;Radiation: Total Body Irradiation;Procedure: Stem cell infusion

Masonic Cancer Center, University of Minnesota

National Marrow Donor Program

Completed

N/A

50 Years

All

Phase 2;Phase 3

United States