MedTrace Logo

Mucopolysaccharidosis Type II Natural History

NCT03529786 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 36

Last updated 2022-04-26

No results posted yet for this study

Summary

Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, is caused by a deficiency of iduronate-2-sulfatase (IDS) leading to an accumulation of glycosaminoglycans (GAGs) in tissues of MPS II patients, resulting in characteristic storage lesions and diverse disease sequelae, and in patients with the more severe form of the disease, irreversible neurocognitive decline and higher morbidity and mortality than in patients with the attenuated form of the disease.

There is currently limited information on the natural history of MPS II, especially with respect to neurocognitive decline in patients with the more severe form of the disease. This study is planned to be an observational medical records review study (data collected retrospectively and no investigational product treatment or procedures) in subjects with the severe form of MPS II. Collectively, the data may inform the design of future MPS II gene therapy treatment studies and may be utilized as historical comparative control data.

Conditions

  • Mucopolysaccharidosis II

Sponsors & Collaborators

Eligibility

Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-09-27
Primary Completion
2022-03-22
Completion
2022-03-22

Countries

  • United States
  • Brazil
  • United Kingdom

Study Locations

More Related Trials

Entities

Companies

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 NCT03529786 on ClinicalTrials.gov