A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

NCTID	NCT06149403 (View at clinicaltrials.gov)
Description	A multi-center randomized clinical trial to compare OTL-203 (gene therapy) with stem cell transplant (standard of care) in patients with MPS-IH (Hurler syndrome). (Show More)
Indication	Mucopolysaccharidosis Type I (Hurler Syndrome)
Compound Name	OTL-203 (Autologous CD34+ cells transduced with IDUA lentiviral vector)
Sponsor	Orchard Therapeutics
Funder Type	Industry
Status	Recruiting
Enrollment Count	40

Target Gene/Variant	IDUA
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
Dose 1	Mean dose: 20.9 x 10^6 transduced CD34+ cells/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	7
Locations	Netherlands,United States,Italy,United Kingdom

Has US IND	True
Recent Updates	First patient randomized 2/2024