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A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome)

NCT05523206 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 15

Last updated 2025-01-30

No results posted yet for this study

Summary

This protocol is a decentralized, single cohort, natural history and biomarker study enrolling up to 20 participants with MPS IIIA (Sanfilippo syndrome). At least 10 participants (\~50%) must be less than four years old at the time of the Parent/LAR consent. The study will have a screening process and 7 study visits, e.g. home visits, that will consist of serum collection and completion of a remote assessment of the Vineland Adaptive Behavior Scales 3rd Edition (Vineland-3)

MPS IIIA remains a devastating disease with a high unmet medical need. There is currently a limited number of available data to adequately characterize the progression of the disease. Analysis of blood biospecimens and adaptive behavior in this study will help researchers better understand the clinical progression of MPS IIIA. A better understanding of disease progression may assist in developing novel therapies for rare genetic disorders.

Conditions

  • Mucopolysaccharidosis III-A

Interventions

OTHER

No intervention

No intervention

Sponsors & Collaborators

  • Denali Therapeutics Inc.

    collaborator INDUSTRY

  • Sanguine Biosciences

    lead INDUSTRY

Principal Investigators

  • Houman Hemmati, MD · Sanguine Biosciences, Inc.

Eligibility

Min Age
4 Months
Max Age
13 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-09-27
Primary Completion
2024-12-19
Completion
2025-01-28

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05523206 on ClinicalTrials.gov