19. Lysosomal storage disease
784 clinical trials,   673 drugs   (DrugBank: 101 drugs),   68 drug target genes,   184 drug target pathways
Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
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.
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 |
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1 | NCT04201405 (ClinicalTrials.gov) | January 7, 2020 | 5/12/2019 | Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type IIIA | A Phase I-II, Study of Autologous CD34+ Haematopoietic Stem Cells Transduced ex Vivo With CD11b Lentiviral Vector Encoding for Human SGSH in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIa, Sanfilippo Syndrome Type A) | 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 Ormond Street Hospital for Children NHS Foundation Trust;Manchester University NHS Foundation Trust | 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 syndrome type A) | A phase I-II, study of autologous CD34+ haematopoietic stem cells transduced ex vivo with CD11b lentiviral vector encoding for human SGSH in patients with mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A) | MPS IIIA, mucopolysaccharidosis type IIIA MedDRA version: 20.1;Level: PT;Classification code 10028093;Term: Mucopolysaccharidosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Body processes [G] - Genetic Phenomena [G05] | Product Name: CRYOPRESERVED AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH A LENTIVIRAL VECTOR CONTAINING HUMAN SGSH GENE INN or Proposed INN: no recommended INN Other descriptive name: AUTOLOGOUS CD34+ CELLS TRANSDUCED WITH A LENTIVIRAL VECTOR CONTAINING THE HUMAN SGSH GENE | University of Manchester | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 5 | Phase 1;Phase 2 | United Kingdom |