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A Natural History Study of Sanfilippo Syndrome Type D

NCT05648851 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 10

Last updated 2025-08-01

No results posted yet for this study

Summary

Sanfilippo syndrome type D is a ultra rare syndrome with limited available natural history data. This study is planned to document, through retrospective and prospective data collection, syndrome progression in children and young adults with Sanfilippo syndrome type D. The results from this study may inform future clinical studies in targeted therapies for patients with Sanfilippo syndrome type D and may serve as an external control since there are very few patients with Sanfilippo syndrome type D.

Conditions

  • Sanfilippo Syndrome Type D

Sponsors & Collaborators

  • National Institute of Neurological Disorders and Stroke (NINDS)

    collaborator NIH

  • Phoenix Nest

    lead INDUSTRY

Principal Investigators

  • Wendy Chung, MD · Columbia University Department of Pediatrics

  • Andres Morales, MD · Columbia University Department of Pediatrics

Eligibility

Min Age
12 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-01-22
Primary Completion
2024-11-01
Completion
2024-11-01

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 NCT05648851 on ClinicalTrials.gov