Research & Clinical Trials | Muenzer MPS Research & Treatment Center

The Muenzer MPS Research & Treatment Center has a long history of participating in clinical research to advance the understanding, diagnosis, management, and treatment of mucopolysaccharidoses (MPS).

UNC-Chapel Hill has served as a study site for a broad range of MPS research, including clinical trials, long-term extension studies, observational studies, biomarker research, and expanded-access programs involving multiple MPS types. The studies listed below highlight current and ongoing clinical research conducted at UNC-Chapel Hill.

Please note: Study status is based on information reported on ClinicalTrials.gov and may change over time. A study listed as recruiting or active does not necessarily mean that enrollment is currently available at UNC-Chapel Hill.

Patients and families interested in research participation or learning more about a specific study are encouraged to contact the Muenzer MPS Research & Treatment Center for the most current information. For questions about clinical trial participation or enrollment, please call our team at 919-228-2432 or complete the MPS Connect Form.

MPS II (Hunter Syndrome)

A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs. Idursulfase in Pediatric and Young Adult Participants With MPS II

ClinicalTrials.gov ID: NCT05371613
Status: Recruiting
Intervention: Tividenofusp alfa (DNL310)
UNC Site: UNC Children’s Research Institute

This study is evaluating the efficacy and safety of tividenofusp alfa compared with idursulfase in pediatric and young adult participants with neuronopathic or non-neuronopathic MPS II.

STARLIGHT – A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II

ClinicalTrials.gov ID: NCT04573023
Phase: Phase 3
Status: Active, Not Recruiting
Intervention: JR-141 (pabinafusp alfa)
UNC Site: University of North Carolina at Chapel Hill

The STARLIGHT study evaluates JR-141 in individuals with MPS II.

MPS IIIA (Sanfilippo Syndrome Type A)

Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA

ClinicalTrials.gov ID: NCT06181136
Status: Active, Not Recruiting
Intervention: DNL126
UNC Site: University of North Carolina at Chapel Hill

This study evaluates DNL126 in pediatric participants with MPS IIIA (Sanfilippo syndrome type A). UNC-Chapel Hill is one of the participating clinical sites.

Resources

This toolkit from Global Genes with a step-by-step guide to walk families through the process of joining and participating in a clinical trial from start to finish. One of the key aims of this guide is to help families advocate for themselves and their loved ones throughout the process. It is not MPS specific but mayprovide useful information.

The National Organization for Rare Diseases (NORD) has developed a free educational series to help teach patients and families about the steps involved in developing a new drug treatment, particularly for rare diseases. The goal of this educational series is to provide families with the knowledge to empower them to effectively engage with and contribute to the drug development process.