19. Lysosomal storage disease Clinical trials / Disease details

Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182

2 trials found

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	NCT05682144 (ClinicalTrials.gov)	January 15, 2023	12/12/2022	ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I	A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Adult Patients With Mucopolysaccharidosis Type I Hurler-Scheie and Scheie	Mucopolysaccharidosis IH/S;Mucopolysaccharidosis IS	Biological: Autologous Plasmablasts (B cells)	Immusoft Corporation	NULL	Not yet recruiting	18 Years	N/A	All	2	Phase 1	United States
2	NCT04284254 (ClinicalTrials.gov)	December 2022	11/11/2019	MT2018-18: Sleeping Beauty Transposon-Engineered Plasmablasts for Hurler Syndrome Post Allo HSCT	Sleeping Beauty Transposon-Engineered Plasmablasts for Expression and Delivery of Alpha-L-iduronidase in Patients With Hurler Syndrome That Have Previously Undergone Allogeneic Transplantation	Mucopolysaccharidosis Type IH (MPS IH, Hurler Syndrome);Mucopolysaccharidosis Type IH;MPS IH, Hurler Syndrome	Drug: Autologous Plasmablasts	Masonic Cancer Center, University of Minnesota	NULL	Withdrawn	3 Years	8 Years	All	0	Phase 1/Phase 2	NULL