Sanfilippo Syndrome B

Clinical trial pipeline · Data from ClinicalTrials.gov

See which Sanfilippo Syndrome B 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 (21 shown)

By InvitationNot Applicable

NCT05368038

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

ScreenPlus is a consented, multi-disorder pilot newborn screening program implemented in conjunction with the New York State Newborn Screening Program that prov…

United States100,000 participantsStarted 2021-05-10

Treatment: Confirmatory Testing

By InvitationNot Applicable

NCT03655223

Early Check: Expanded Screening in Newborns

Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approa…

United States30,000 participantsStarted 2018-10-15

Treatment: Confirmatory Testing

Sanfilippo Syndrome B

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 (21 shown)

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

Early Check: Expanded Screening in Newborns

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 (21 shown)

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

Early Check: Expanded Screening in Newborns

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

Biomarker for Sanfilippo Type A-B-C-D Disease (BioSanfilippo)

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)

Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome

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

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Disea…