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)
Development Status	Active
Indication	Mucopolysaccharidosis Type I (Hurler Syndrome)
Disease Ontology Term	DOID:12802
Compound Name	OTL-203
Compound Description	Autologous CD34+ cells transduced with IDUA lentiviral vector
Sponsor	Orchard Therapeutics
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	41 (ACTUAL)
Results Posted	Not Available

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	none
Dose 1	Mean dose: 20.9E6 transduced CD34+ cells/kg
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	5
Locations	Netherlands,United States,Italy,United Kingdom

Has US IND	True
FDA Designations	Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates	Last patient randomized in registrational trial July 2025