Mucopolysaccharidosis IIIB

Clinical trial pipeline · Data from ClinicalTrials.gov

See which Mucopolysaccharidosis IIIB trials you may qualify for

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

RecruitingPhase 1/2

NCT06488924

An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB

A Phase I/ II, open-label study, designed to evaluate the safety and explore efficacy of the study drug in development for the treatment of MPS IIIB patients.

Japan10 participantsStarted 2024-11-05

Treatment: JR-446

All recent trials (16 shown)

By InvitationPhase 4

NCT05492799

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

This is a Phase 3B/4, multicenter, multinational, open label study to further evaluate intracerebroventricular (ICV) delivered AX 250 treatment in MPS IIIB subj…

United States, Colombia +2 more15 participantsStarted 2022-12-02

Treatment: AX 250

UnknownPhase 2

NCT03784287

A Treatment Extension Study of Mucopolysaccharidosis Type IIIB

The primary objectives of this study are to evaluate the long-term safety and tolerability of AX 250 administered to subjects with MPS IIIB by an implanted ICV…

United States, Colombia +2 more20 participantsStarted 2018-02-19

Treatment: AX 250

Mucopolysaccharidosis IIIB

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB

All recent trials (16 shown)

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

A Treatment Extension Study of Mucopolysaccharidosis Type IIIB

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB

All recent trials (16 shown)

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

A Treatment Extension Study of Mucopolysaccharidosis Type IIIB

A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101

Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB

Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)

A Treatment Study of Mucopolysaccharidosis Type IIIB

A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)

Neurobehavioral Phenotypes in MPS III

Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type…

Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)

A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacok…

A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB

Evaluation of Blood Brain Barrier Integrity and Structural Abnormalities in MPS IIIB Patients Using Multimodal Magnetic…

Natural History Studies of Mucopolysaccharidosis III