AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH A LENTIVIRAL VECTOR CONTAINING THE HUMAN SGSH GENE

( DrugBank: - / KEGG DRUG: - )

1 disease

告示番号	疾患名（ページ内リンク）	臨床試験数
19	ライソゾーム病	2

19. ライソゾーム病

臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 182

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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	NCT04201405 (ClinicalTrials.gov)	January 7, 2020	5/12/2019	Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidos ...	A Phase I-II, Study of Autologous CD34+ Haematopoietic Stem Cells Transduced ex Vivo With CD11b Lent ...	Mucopolysaccharidosis Type IIIA	Drug: Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene	University of Manchester	Orchard Therapeutics;CTI Clinical Trial and Consulting Services;University College, London;Great Orm ...	Active, not recruiting	3 Months	24 Months	All	5	Phase 1/Phase 2	United Kingdom
2	EUCTR2019-002051-42-GB (EUCTR)	25/10/2019	09/04/2020	A study testing a gene therapy to treat mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndro ...	A phase I-II, study of autologous CD34+ haematopoietic stem cells transduced ex vivo with CD11b lent ...	MPS IIIA, mucopolysaccharidosis type IIIA MedDRA version: 20.1;Level: PT;Classification code 1002809 ...	Product Name: CRYOPRESERVED AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH A LENTIVIRAL VECTOR CONTAINING HU ...	University of Manchester	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	5	Phase 1;Phase 2	United Kingdom

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