326. 大理石骨病 Osteopetrosis Clinical trials / Disease details

臨床試験数 : 18 / 薬物数 : 43 - (DrugBank : 14) / 標的遺伝子数 : 17 - 標的パスウェイ数 : 82

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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	EUCTR2014-000584-41-ES (EUCTR)	08/06/2017	21/03/2017	Phase II extension study of CaspaCIDe T cells (BPX-501) from an HLA-partially matched family donor a ...	Phase II extension study of CaspaCIDe T cells (BPX-501) from an HLA-partially matched family donor a ...	Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplasticsyndromes;Congenital immune def ...	Product Name: BPX-501 cells INN or Proposed INN: rivogenleucleucel Other descriptive name: BPX-501 Produ ...	Bellicum Pharmaceuticals, Inc.	NULL	Not Recruiting			Female: yes Male: yes	175	Phase 2	Spain;United Kingdom;Italy
2	EUCTR2016-003226-16-IT (EUCTR)	03/02/2017	17/11/2016	Follow-up of phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after ...	Follow-up of phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after ...	Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplasticsyndromes;Congenital immune def ...	Product Name: AP1903 Other descriptive name: AP1903	Bellicum Pharmaceuticals, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	175	Phase 1;Phase 2	Italy
3	NCT02666768 (ClinicalTrials.gov)	February 22, 2016	23/6/2015	ACTIMMUNE in Intermediate Osteopetrosis	Open-label Early Phase 2 Study With a Single Arm of Interferon Gamma-1b Treatment of Osteopetrosis	Osteopetrosis	Drug: Interferon gamma -1b	Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center	University of Minnesota;Horizon Pharma Ireland, Ltd., Dublin Ireland	Completed	1 Year	N/A	All	5	Phase 2	United States
4	NCT02584608 (ClinicalTrials.gov)	January 1, 2016	20/10/2015	Use of ACTIMMUNE in Patients With ADO2	Phase 2a Study of Interferon Gamma-1b for the Treatment of Autosomal Dominant Type 2 Osteopetrosis	Autosomal Dominant Osteopetrosis Type 2	Drug: ACTIMMUNE	Indiana University	Horizon Pharma Ireland, Ltd., Dublin Ireland	Completed	3 Years	65 Years	All	12	Phase 2	United States
5	EUCTR2014-000584-41-IT (EUCTR)	23/10/2014	15/04/2014	Phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative sele ...	Phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative sele ...	Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplasticsyndromes;Congenital immune def ...	Product Name: BPX-501 cells Product Name: AP1903	Bellicum Pharmaceuticals, Inc.	NULL	Not Recruiting			Female: yes Male: yes	30	Phase 1;Phase 2	Spain;United Kingdom;Italy
6	NCT02171104 (ClinicalTrials.gov)	July 10, 2014	20/6/2014	MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis	MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Sever ...	Mucopolysaccharidosis Disorders;Hurler Syndrome;Hunter Syndrome;Maroteaux Lamy Syndrome;Sly Syndrome ...	Biological: Stem Cell Transplantation;Drug: IMD Preparative Regimen;Drug: Osteopetrosis Only Preparat ...	Masonic Cancer Center, University of Minnesota	NULL	Recruiting	N/A	55 Years	All	100	Phase 2	United States
7	NCT02065869 (ClinicalTrials.gov)	April 2014	13/2/2014	Safety Study of Gene Modified Donor T-cells Following TCRaß+ Depleted Stem Cell Transplant	Phase I/II Study of CaspaCIDe T Cells (BPX-501; Rivogenlecleucel) From an HLA Partially Matched Fami ...	Acute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplas ...	Biological: BPX-501 T cells;Drug: Rimiducid	Bellicum Pharmaceuticals	NULL	Active, not recruiting	1 Month	18 Years	All	187	Phase 1/Phase 2	Italy;United Kingdom;Germany;Spain;United States
8	NCT03333200 (ClinicalTrials.gov)	January 11, 2012	24/4/2017	Longitudinal Study of Neurodegenerative Disorders	Longitudinal Study of Neurodegenerative Disorders	MLD;Krabbe Disease;ALD;MPS I;MPS II;MPS III;Vanishing White Matter Disease;GM3 Gangliosidosis;PKAN;T ...	Other: Palliative Care;Biological: Hematopoetic Stem Cell Transplantation	University of Pittsburgh	NULL	Recruiting	N/A	N/A	All	1500		United States
9	NCT01200017 (ClinicalTrials.gov)	September 2010	9/9/2010	Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem ...	An Expanded Access Study of the Feasibility of Using the CliniMACS® Device for CD34+ Cell Selection ...	Cytopenias;Leukocyte Disorders;Anemia Due to Intrinsic Red Cell Abnormality;Acute Lymphoblastic Leuk ...	Biological: CD34+ enriched, T Cell Depleted donor stem cell product	Christopher Dvorak	NULL	No longer available	2 Months	30 Years	All			United States
10	NCT01087398 (ClinicalTrials.gov)	September 2009	13/3/2010	Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis	Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis	Osteopetrosis	Drug: Busulfan , Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen);Procedure: Stem ...	Tehran University of Medical Sciences	NULL	Recruiting	N/A	5 Years	Both	10	Phase 2/Phase 3	Iran, Islamic Republic of

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