DDrare: Database of Drug Development for Rare Diseases

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	NCT01586455 (ClinicalTrials.gov)	April 2013	25/4/2012	Human Placental-Derived Stem Cell Transplantation	A Single-Arm Study to Assess the Safety of Transplantation With Human Placental-Derived Stem-Cells Combined With Unrelated and Related Cord Blood in Subjects With Certain Malignant Hematologic Diseases and Non-Malignant Disorders	Mucopolysaccharidosis I;Mucopolysaccharidosis VI;Adrenoleukodystrophy;Niemann-Pick Disease;Metachromatic Leukodystrophy;Wolman Disease;Krabbe's Disease;Gaucher's Disease;Fucosidosis;Batten Disease;Severe Aplastic Anemia;Diamond-Blackfan Anemia;Amegakaryocytic Thrombocytopenia;Myelodysplastic Syndrome;Acute Myelogenous Leukemia;Acute Lymphocytic Leukemia	Drug: Human Placental Derived Stem Cell	New York Medical College	NULL	Active, not recruiting	N/A	55 Years	All	43	Phase 1	United States

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agemin

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NCT01586455
(ClinicalTrials.gov)

April 2013

25/4/2012

Human Placental-Derived Stem Cell Transplantation

A Single-Arm Study to Assess the Safety of Transplantation With Human Placental-Derived Stem-Cells Combined With Unrelated and Related Cord Blood in Subjects With Certain Malignant Hematologic Diseases and Non-Malignant Disorders

Mucopolysaccharidosis I;Mucopolysaccharidosis VI;Adrenoleukodystrophy;Niemann-Pick Disease;Metachromatic Leukodystrophy;Wolman Disease;Krabbe's Disease;Gaucher's Disease;Fucosidosis;Batten Disease;Severe Aplastic Anemia;Diamond-Blackfan Anemia;Amegakaryocytic Thrombocytopenia;Myelodysplastic Syndrome;Acute Myelogenous Leukemia;Acute Lymphocytic Leukemia

Drug: Human Placental Derived Stem Cell

New York Medical College

NULL

Active, not recruiting

N/A

55 Years

All

Phase 1

United States