DDrare: Database of Drug Development for Rare Diseases

326. 大理石骨病
［臨床試験数：20，薬物数：52（DrugBank：14），標的遺伝子数：16，標的パスウェイ数：78］

Searched query = "Osteopetrosis"
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 = ACTIMMUNE; AP1903; AP1903 A594; Actimmune; Actimmune Registry; BPX-501; BPX-501 T cells; BPX-501 cells; Busulfan; Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen); CALD HR-D (High-Risk, Regimen C); CALD HR-D (High-Risk, Regimen D); CALD SR-A (Standard-Risk, Regimen A); CALD SR-B (Standard-Risk, Regimen B); CD34+ enriched, T Cell Depleted donor stem cell product; Calcitriol; Campath; Campath, Busulfan, Clofarabine; Campath-1H; Clofarabine; Cyclophosphamide; Cyclophosphamide 30; Cyclophosphamide 40; Cyclosporin; Cyclosporin, Methotrexate (GVHD prophylaxis); Device: Miltenyi Biotec CliniMACS; Fludarabine; Fludarabine monophosphate; Hematopoetic Stem Cell Transplantation; IMD; IMD Preparative Regimen; Interferon Gamma; Interferon gamma; Interferon gamma-1b; Methotrexate; Osteopetrosis Haploidentical Only Preparative Regimen; Osteopetrosis Only Preparative Regimen; Other: Palliative Care; Procedure: Stem Cell Transplantation; Procedure: Stem Cell or Umbilical Cord Blood Transplantation; Procedure: Total Lymphoid Irradiation; Procedure: marrow graft transplantation; Procedure: umbilical cord blood transplantation; RP-L401; Radiation: Total Lymphoid Irradiation; RhPTH; Rimiducid; Rivogenlecleucel; Rivogenleucleucel; Stem Cell Transplantation; Systemic chemotherapy and antibodies; Thymoglobulin

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	NCT01200017 (ClinicalTrials.gov)	August 2010	9/9/2010	Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant	An Expanded Access Study of the Feasibility of Using the CliniMACS® Device for CD34+ Cell Selection and T Cell Depletion for Graft-versus-Host Disease Prophylaxis in Alternative Donor Stem Cell Transplant Recipients	Acute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Chronic Myeloid Leukemia;Myelodysplastic Syndrome;Lymphomas;Bone Marrow Failure;Hemoglobinopathy;Immune Deficiency;Osteopetrosis;Cytopenias;Leukocyte Disorders;Anemia Due to Intrinsic Red Cell Abnormality	Biological: CD34+ enriched, T Cell Depleted donor stem cell product	Christopher Dvorak	NULL	Available	2 Months	30 Years	All			United States

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agemin

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NCT01200017
(ClinicalTrials.gov)

August 2010

9/9/2010

Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant

An Expanded Access Study of the Feasibility of Using the CliniMACS® Device for CD34+ Cell Selection and T Cell Depletion for Graft-versus-Host Disease Prophylaxis in Alternative Donor Stem Cell Transplant Recipients

Acute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Chronic Myeloid Leukemia;Myelodysplastic Syndrome;Lymphomas;Bone Marrow Failure;Hemoglobinopathy;Immune Deficiency;Osteopetrosis;Cytopenias;Leukocyte Disorders;Anemia Due to Intrinsic Red Cell Abnormality

Biological: CD34+ enriched, T Cell Depleted donor stem cell product

Christopher Dvorak

NULL

Available

2 Months

30 Years

All

United States