AX 250

DRUG3 trials

Sponsors

Allievex Corporation

Conditions

MPS III BMucopolysaccharidosis Type IIIB

Phase 1

A Treatment Study of Mucopolysaccharidosis Type IIIB

CompletedNCT02754076

Allievex CorporationMPS III B, Mucopolysaccharidosis Type IIIB

Start: 2016-04-30End: 2020-07-31Updated: 2020-08-06

Phase 2

A Treatment Extension Study of Mucopolysaccharidosis Type IIIB

Allievex CorporationMPS III B, Mucopolysaccharidosis Type IIIB

Start: 2018-02-19End: 2025-02-28Updated: 2023-01-18

Phase 4

Safety, Tolerability and Efficacy of ICV AX 250 Treatment in MPS IIIB -OLE

Enrolling by invitationNCT05492799

Allievex CorporationMPS III B

Start: 2022-12-02End: 2027-12-31Target: 15Updated: 2023-08-09

Related Papers

A phase I/II study on intracerebroventricular tralesinidase alfa in patients with Sanfilippo syndrome type B

Journal of Clinical Investigation2022-11-2219 citations

Longitudinal Natural History of Pediatric Subjects Affected with Mucopolysaccharidosis IIIB

The Journal of Pediatrics2022-06-139 citations