20. 副腎白質ジストロフィー Adrenoleukodystrophy Clinical trials / Disease details

臨床試験数 : 61 / 薬物数 : 90 - (DrugBank : 31) / 標的遺伝子数 : 23 - 標的パスウェイ数 : 126

10 trials found

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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	EUCTR2018-001145-14-DE (EUCTR)	23/01/2020	27/11/2018	Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adren ...	A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfanand Fludarabi ...	Cerebral Adrenoleukodystrophy (CALD) MedDRA version: 20.0;Level: PT;Classification code 10051260;Ter ...	Product Name: Lenti-D Drug Product INN or Proposed INN: elivaldogene autotemcel Other descriptive name ...	bluebird bio, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	35	Phase 3	France;United States;Netherlands;Germany;United Kingdom;Italy
2	EUCTR2018-001145-14-NL (EUCTR)	24/06/2019	25/02/2019	Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adren ...	A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfanand Fludarabi ...	Cerebral Adrenoleukodystrophy (CALD) MedDRA version: 20.0;Level: PT;Classification code 10051260;Ter ...	Product Name: Lenti-D Drug Product Product Code: Not Applicable INN or Proposed INN: elivaldogene auto ...	bluebird bio, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	35	Phase 3	France;United States;Germany;Netherlands;Italy;United Kingdom
3	EUCTR2018-001145-14-IT (EUCTR)	19/04/2019	02/08/2021	Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adren ...	A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfanand Fludarabi ...	Cerebral Adrenoleukodystrophy (CALD) MedDRA version: 20.0;Level: PT;Classification code 10051260;Ter ...	Product Name: Lenti-D Drug Product Product Code: [not applicable] INN or Proposed INN: elivaldogene au ...	BLUEBIRD BIO, INC.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	35	Phase 3	France;United States;Netherlands;Germany;United Kingdom;Italy
4	EUCTR2018-001145-14-GB (EUCTR)	17/04/2019	23/10/2018	Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adren ...	A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfanand Fludarabi ...	Cerebral Adrenoleukodystrophy (CALD) MedDRA version: 20.0;Level: PT;Classification code 10051260;Ter ...	Product Name: Lenti-D Drug Product INN or Proposed INN: elivaldogene autotemcel Other descriptive name ...	bluebird bio, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	35	Phase 3	United States;France;Netherlands;Germany;Italy;United Kingdom
5	EUCTR2018-001145-14-FR (EUCTR)	27/03/2019	23/10/2018	Clinical study to assess the efficacy and safety of gene therapy for the treatment of cerebral adren ...	A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfanand Fludarabi ...	Cerebral Adrenoleukodystrophy (CALD) MedDRA version: 20.0;Level: PT;Classification code 10051260;Ter ...	Product Name: Lenti-D Drug Product Other descriptive name: Autologous haematopoietic stem cells trans ...	bluebird bio, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	20	Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ...	United States;France;Netherlands;Germany;Italy;United Kingdom
6	NCT03852498 (ClinicalTrials.gov)	January 24, 2019	13/2/2019	A Clinical Study to Assess the Efficacy and Safety of Gene Therapy for the Treatment of Cerebral Adr ...	A Phase 3 Study of Lenti-D Drug Product After Myeloablative Conditioning Using Busulfanand Fludarabi ...	Cerebral Adrenoleukodystrophy (CALD)	Genetic: Lenti-D	bluebird bio	NULL	Active, not recruiting	N/A	17 Years	Male	35	Phase 3	United States;France;Germany;Italy;Netherlands;United Kingdom
7	NCT03513328 (ClinicalTrials.gov)	June 15, 2018	19/4/2018	Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation	PEDS024, Phase I/II Feasibility Study of BusulfanFludarabine and Thiotepa Conditioning Regimen for A ...	Bone Marrow Failure Syndrome;Thalassemia;Sickle Cell Disease;Diamond Blackfan Anemia;Acquired Neutro ...	Drug: Thiotepa --single daily dose;Drug: Thiotepa --escalated dose	University of Florida	Live Like Bella Pediatric Cancer Research	Active, not recruiting	3 Months	39 Years	All	6	Phase 1/Phase 2	United States
8	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
9	NCT01043640 (ClinicalTrials.gov)	December 2009	5/1/2010	Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders	Allogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic Disorders	Mucopolysaccharidosis;Hurler Syndrome;Hunter Syndrome;Maroteaux-Lamy Syndrome;Sly Syndrome;Alpha Man ...	Drug: Campath -1H;Drug: Cyclophosphamide;Drug: Busulfan;Procedure: Allogeneic stem cell transplantati ...	Masonic Cancer Center, University of Minnesota	NULL	Completed	N/A	21 Years	All	46	Phase 2	United States
10	NCT00176904 (ClinicalTrials.gov)	January 1995	12/9/2005	Stem Cell Transplant for Inborn Errors of Metabolism	Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Bone Marrow Transplantation	Adrenoleukodystrophy;Metachromatic Leukodystrophy;Globoid Cell Leukodystrophy;Gaucher's Disease;Fuco ...	Procedure: Stem Cell Transplant;Drug: Busulfan , Cyclophosphamide, Antithymocyte Globulin	Masonic Cancer Center, University of Minnesota	NULL	Completed	N/A	N/A	All	135	Phase 2/Phase 3	United States

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