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	NCT03653338 (ClinicalTrials.gov)	August 2, 2018	2/8/2018	T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias	T-Cell Depleted, Alternative Donor Transplant in Pediatric and Adult Patients With Severe Sickle Cell Disease (SCD) and Other Transfusion-Dependent Anemias	Sickle Cell Anemia;Beta-thalassemia Major;Diamond-blackfan Anemia	Biological: CD3/CD19 depleted leukocytes;Biological: CD45RA depleted leukocytes;Drug: Hydroxyurea;Drug: Rituximab;Drug: Alemtuzumab;Drug: Fludarabine;Drug: Thiotepa	Paul Szabolcs	NULL	Recruiting	5 Years	40 Years	All	5	Phase 1;Phase 2	United States
2	NCT03513328 (ClinicalTrials.gov)	June 15, 2018	19/4/2018	Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation	PEDS024, Phase I/II Feasibility Study of Busulfan Fludarabine and Thiotepa Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation (HSCT) for Children With Non-Malignant Disorders	Bone Marrow Failure Syndrome;Thalassemia;Sickle Cell Disease;Diamond Blackfan Anemia;Acquired Neutropenia in Newborn;Acquired Anemia Hemolytic;Acquired Thrombocytopenia;Hemophagocytic Lymphohistiocytoses;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;Common Variable Immunodeficiency;X-linked Lymphoproliferative Disease;Severe Combined Immunodeficiency;Hurler Syndrome;Mannosidosis;Adrenoleukodystrophy	Drug: Thiotepa --single daily dose;Drug: Thiotepa --escalated dose	University of Florida	Live Like Bella Pediatric Cancer Research	Recruiting	3 Months	39 Years	All	40	Phase 1;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

NCT03653338
(ClinicalTrials.gov)

August 2, 2018

2/8/2018

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

T-Cell Depleted, Alternative Donor Transplant in Pediatric and Adult Patients With Severe Sickle Cell Disease (SCD) and Other Transfusion-Dependent Anemias

Sickle Cell Anemia;Beta-thalassemia Major;Diamond-blackfan Anemia

Biological: CD3/CD19 depleted leukocytes;Biological: CD45RA depleted leukocytes;Drug: Hydroxyurea;Drug: Rituximab;Drug: Alemtuzumab;Drug: Fludarabine;Drug: Thiotepa

Paul Szabolcs

NULL

Recruiting

5 Years

40 Years

All

Phase 1;Phase 2

United States

NCT03513328
(ClinicalTrials.gov)

June 15, 2018

19/4/2018

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

PEDS024, Phase I/II Feasibility Study of Busulfan Fludarabine and Thiotepa Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation (HSCT) for Children With Non-Malignant Disorders

Bone Marrow Failure Syndrome;Thalassemia;Sickle Cell Disease;Diamond Blackfan Anemia;Acquired Neutropenia in Newborn;Acquired Anemia Hemolytic;Acquired Thrombocytopenia;Hemophagocytic Lymphohistiocytoses;Wiskott-Aldrich Syndrome;Chronic Granulomatous Disease;Common Variable Immunodeficiency;X-linked Lymphoproliferative Disease;Severe Combined Immunodeficiency;Hurler Syndrome;Mannosidosis;Adrenoleukodystrophy

Drug: Thiotepa --single daily dose;Drug: Thiotepa --escalated dose

University of Florida

Live Like Bella Pediatric Cancer Research

Recruiting

3 Months

39 Years

All

Phase 1;Phase 2

United States