OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)

NCTID	NCT04283227 (View at clinicaltrials.gov)
Description	OTL-200 is a cryopreserved dispersion for infusion containing autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A (ARSA) gene. MLD is an autosomal recessive lysosomal storage disorder (LSD) characterized by severe and progressive demyelination affecting the central and peripheral nervous system. The aim of this clinical study is to assess the pharmacodynamic effect and long-term clinical efficacy and safety of OTL-200 in Late Juvenile MLD patients. (Show More)
Development Status	Approved
Indication	Metachromatic Leukodystrophy
Disease Ontology Term	DOID:10581
Compound Name	LENMELDY
Compound Alias	atidarsagene autotemcel, OTL-200
Sponsor	Orchard Therapeutics
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	6
Results Posted	Not Available

Target Gene/Variant	ARSA
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	VSV-G
Editor Type	none
Dose 1	Minimum dose: 4.2E6 CD34+ cells/kg
Dose 2	Maximum dose: 30E6 CD34+ cells/kg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	Italy

Has US IND	False
FDA Designations	Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates	FDA approval granted 3/18/24