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A Natural History Study of Aspartylglucosaminuria

NCT03853876 · Status: TERMINATED · Type: OBSERVATIONAL · Enrollment: 8

Last updated 2022-04-12

No results posted yet for this study

Summary

Aspartylglucosaminuria (AGU) is a rare neurodegenerative lysosomal storage disease (LSD) characterized by developmental delay, psychomotor regression, worsening intellectual disability, gait disturbance and, ultimately, premature death, and has no available treatments.

The purpose of this study is to investigate the clinical characteristics and natural clinical progression of symptoms in individuals with AGU. This natural history study is important to better understand disease course to be able to determine clinically meaningful outcome measures for use in future clinical trials.

Conditions

  • Aspartylglucosaminuria
  • Aspartylglucosamidase (AGA) Deficiency
  • Lysosomal Storage Diseases

Sponsors & Collaborators

  • Neurogene Inc.

    lead INDUSTRY

Principal Investigators

  • Elise Beausoleil · Neurogene Inc.

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-04-18
Primary Completion
2020-10-15
Completion
2022-03-17

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 NCT03853876 on ClinicalTrials.gov