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Mucopolysaccharidosis Type II Observational

NCT04591834 · Status: WITHDRAWN · Type: OBSERVATIONAL

Last updated 2022-10-10

No results posted yet for this study

Summary

This is an observational study planned to document prospectively disease manifestation and neurocognitive course in pediatric patients with a clinical presentation consistent with neuronopathic ("severe") MPS II undergoing current standard of care and/or intrathecal Elaprase® for their condition. Some patients may be offered the opportunity to screen for a gene therapy study conducted by the same sponsor.

Conditions

  • Mucopolysaccharidosis II

Interventions

OTHER

Observational

An observational study in subjects with the severe form of MPS II.

Sponsors & Collaborators

Eligibility

Min Age
1 Month
Max Age
8 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-03-31
Primary Completion
2025-07-31
Completion
2025-07-31

Countries

  • United States
  • Canada

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 NCT04591834 on ClinicalTrials.gov