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	NCT00305708 (ClinicalTrials.gov)	August 2000	21/3/2006	Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First Remission	Bone Marrow Stem Cell Transplantation for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myeloid Leukemia in 1Remission	Congenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Fanconi Anemia;Leukemia;Severe Congenital Neutropenia;Thrombocytopenia	Biological: anti-thymocyte globulin;Drug: busulfan;Drug: fludarabine phosphate;Procedure: allogeneic bone marrow transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantation;Radiation: radiation therapy	University of California, San Francisco	National Cancer Institute (NCI)	Completed	N/A	17 Years	Both	40	Phase 1;Phase 2	United States
2	NCT00001749 (ClinicalTrials.gov)	July 1998	3/11/1999	Medical Treatment for Diamond Blackfan Anemia	Treatment of Diamond Blackfan Anemia With Antithymocyte Globulin and Cyclosporine A	Fanconi's Anemia;Hematologic Disease	Drug: Antithymocyte globulin;Drug: Cyclosporine	National Heart, Lung, and Blood Institute (NHLBI)	NULL	Completed	N/A	N/A	Both	25	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

NCT00305708
(ClinicalTrials.gov)

August 2000

21/3/2006

Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First Remission

Bone Marrow Stem Cell Transplantation for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myeloid Leukemia in 1Remission

Congenital Amegakaryocytic Thrombocytopenia;Diamond-blackfan Anemia;Fanconi Anemia;Leukemia;Severe Congenital Neutropenia;Thrombocytopenia

Biological: anti-thymocyte globulin;Drug: busulfan;Drug: fludarabine phosphate;Procedure: allogeneic bone marrow transplantation;Procedure: peripheral blood stem cell transplantation;Procedure: umbilical cord blood transplantation;Radiation: radiation therapy

University of California, San Francisco

National Cancer Institute (NCI)

Completed

N/A

17 Years

Both

Phase 1;Phase 2

United States

NCT00001749
(ClinicalTrials.gov)

July 1998

3/11/1999

Medical Treatment for Diamond Blackfan Anemia

Treatment of Diamond Blackfan Anemia With Antithymocyte Globulin and Cyclosporine A

Fanconi's Anemia;Hematologic Disease

Drug: Antithymocyte globulin;Drug: Cyclosporine

National Heart, Lung, and Blood Institute (NHLBI)

NULL

Completed

N/A

Both

Phase 2

United States