Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA

NCTID	NCT04201405 (View at clinicaltrials.gov)
Description	Patients with MPS IIIA have a clinical disorder marked by severe and progressive brain disease and neurological symptoms due to the accumulation of undigested glycosaminoglycans in all cells of the body. This study will be the first in human clinical trial to explore the safety, tolerability and clinical efficacy of ex vivo gene therapy (autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene) in MPSIIIA patients. Following treatment with the gene therapy patients will be followed up for a minimum of 3 years. (Show More)
Development Status	Active
Indication	Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)
Disease Ontology Term	DOID:0111395
Compound Name	OTL-201
Compound Description	Autologous CD34+ cells transduced with LV-CD11b-hSGSH
Sponsor	University of Manchester
Funder Type	Other
Recruitment Status	Active not recruiting
Enrollment Count	5
Results Posted	Not Available

Target Gene/Variant	SGSH
Therapy Type	Gene transfer
Therapy Route	Ex-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Autologous cells
Target Tissue/Cell	CD34+ cells
Delivery System	Viral transduction
Vector Type	LV
Editor Type	none
Dose 1	Dose range: 4.37 - 22.7E6 CD34+ cells/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	United Kingdom