65. 原発性免疫不全症候群 Primary immunodeficiency Clinical trials / Disease details

臨床試験数 : 482 / 薬物数 : 653 - (DrugBank : 119) / 標的遺伝子数 : 92 - 標的パスウェイ数 : 212

15 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	NCT04140539 (ClinicalTrials.gov)	October 15, 2019	24/10/2019	A Clinical Study to Enable Process Validation of Commercial Grade OTL-101	A Single Arm, Open Label Clinical Study to Enable Process Validation of Commercial Grade Ex Vivo Hem ...	Severe Combined Immunodeficiency Due to ADA Deficiency	Biological: OTL-101	Orchard Therapeutics	University of California, Los Angeles	Terminated	N/A	17 Years	All	3	Phase 2/Phase 3	United States
2	NCT04049084 (ClinicalTrials.gov)	September 26, 2019	6/8/2019	An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ...	An Observational Long-term Follow-up Study for Patients Previously Treated With Autologous ex Vivo G ...	Adenosine Deaminase Deficiency;Severe Combined Immunodeficiency (SCID)	Biological: autologous ex vivo gene therapy products based on the EFS LV encoding for the human aden ...	Orchard Therapeutics	University of California, Los Angeles;Great Ormond Street Hospital for Children NHS Foundation Trust ...	Enrolling by invitation	N/A	N/A	All	70		United States;United Kingdom
3	NCT03878069 (ClinicalTrials.gov)	June 25, 2019	12/3/2019	Registry Study of Revcovi Treatment in Patients With ADA-SCID	Single Arm, Open-Label, Multicenter, Registry Study of Revcovi (Elapegademase-lvlr) Treatment in ADA ...	Adenosine Deaminase Deficiency;Severe Combined Immunodeficiency	Biological: elapegademase -lvlr	Chiesi Farmaceutici S.p.A.	NULL	Recruiting	N/A	65 Years	All	20		United States
4	NCT03765632 (ClinicalTrials.gov)	January 3, 2018	8/8/2018	Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID	Efficacy and Safety of a Cryopreserved Formulation of Autologous CD34+ Haematopoietic Stem Cells Tra ...	Severe Combined Immunodeficiency Due to ADA Deficiency	Genetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101);Drug: Busulfan;Drug: ...	Great Ormond Street Hospital for Children NHS Foundation Trust	Orchard Therapeutics	Active, not recruiting	N/A	17 Years	All	13	Phase 1/Phase 2	United Kingdom
5	EUCTR2017-001731-39-IT (EUCTR)	12/10/2017	02/07/2020	Retroviral insertion site methodology study	Methodology study to investigate the utility of retroviral insertion site analysis in samples from s ...	Adenosinedeaminase (ADA) deficiency severe combined immunodeficiency MedDRA version: 20.1;Level: LLT ...	Trade Name: Strimvelis Product Name: Strimvelis Product Code: [NA]	Orchard Therapeutics (Europe) Ltd	NULL	Not Recruiting			Female: yes Male: yes	15	Phase 4	Turkey;Switzerland;Italy
6	EUCTR2017-001275-23-GB (EUCTR)	21/09/2017	03/07/2017	A clinical trial to study the effects of genetically modified patients' CD34+ cells	Efficacy and safety of a cryopreserved formulation of autologous CD34+ haematopoietic stem cells tra ...	Adenosinedeaminase (ADA) deficiency is an inherited disorder that damages the immune system and caus ...	Product Name: cryopreserved EFS-ADA LV transduced patient CD34+ cells Product Code: OTL-101 INN or Pro ...	Great Ormond Street Hospital for Children NHS Trust	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	10	Phase 2	United Kingdom
7	NCT01420627 (ClinicalTrials.gov)	January 24, 2014	18/8/2011	EZN-2279 in Patients With ADA-SCID	A Study of EZN-2279 (Polyethylene Glycol Recombinant AdenosineDeaminase [PEG-rADA]) Administered as ...	ADA-SCID;Adenosine Deaminase Deficiency;Severe Combined Immunodeficiency	Biological: EZN-2279;Biological: Adagen	Leadiant Biosciences, Inc.	NULL	Completed	N/A	N/A	All	7	Phase 3	United States
8	NCT01852071 (ClinicalTrials.gov)	August 2, 2013	7/5/2013	Autologous CD34+ Hematopoietic Stem Cells Transduced ex Vivo With EFS Lentiviral Vector Encoding for ...	Autologous Transplantation of Bone Marrow CD34+ Stem/Progenitor Cells After Addition of a Normal Hum ...	ADA-SCID	Genetic: Infusion of autologous EFS-ADA LV CD34+ (OTL-101);Drug: busulfan;Drug: PEG -ADA ERT	Orchard Therapeutics	National Institute of Allergy and Infectious Diseases (NIAID);National Human Genome Research Institu ...	Completed	1 Month	17 Years	All	20	Phase 1/Phase 2	United States
9	NCT01380990 (ClinicalTrials.gov)	November 15, 2012	23/6/2011	Lentiviral (LV) Gene Therapy for Adenosine Deaminase (ADA) Deficiency	Phase I/II, Historical Controlled, Open-label, Non-randomised, Single-centre Trial to Assess the Saf ...	Adenosine Deaminase Deficiency;Severe Combined Immunodeficiencies (SCID)	Genetic: Infusion of autologous EFS-ADA LV CD34+ cells;Other: Haematopoietic Stem Cell Transplantati ...	Great Ormond Street Hospital for Children NHS Foundation Trust	Orchard Therapeutics	Completed	N/A	15 Years	All	36	Phase 1/Phase 2	United Kingdom
10	EUCTR2010-024253-36-GB (EUCTR)	03/08/2012	09/05/2012	A clinical trial to study the effects of genetically modified patients' CD34+ cells	Phase I/II, historical controlled, open-label, non-randomised, single-centre trial to assess the saf ...	Adenosinedeaminase (ADA) deficiency is an inherited disorder that damages the immune system and caus ...	Product Name: EF1aS-ADA lentiviral vector transduced patient CD34+ cells Product Code: transduced pat ...	Great Ormond Street Hospital for Children NHS Trust	NULL	Not Recruiting			Female: no Male: yes	10	Phase 1;Phase 2	United Kingdom

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