Mucopolysaccharidosis Type II Observational

Withdrawn before enrolling

Conditions studied: Mucopolysaccharidosis II

In brief

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.

Key facts

Study ID

NCT04591834

Run by

REGENXBIO Inc.

People needed

0

Starts

2022-03-01

Expected to finish

2025-07-01

Last updated by the study team

2022-10-10

Who can join

Age: 0 and older, up to 8. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Meets any of the following criteria:
• Has a clinical diagnosis of severe MPS II and has a documented mutation in IDS, OR
• Has a relative clinically diagnosed with severe MPS II who has the same IDS mutation as the subject, OR
• Has documented mutation(s) in IDS that in the opinion of the investigator is known to result in a neuronopathic phenotype
• Has sufficient communication capacity to complete the required protocol testing
• Patient's legal guardian must be willing and able to provide written, signed informed consent.

You may not qualify if…

• Has had prior treatment with an AAV-based gene therapy product
• Is currently participating in a clinical trial of an investigational product for the treatment of MPS II with the exception of IT ELAPRASE trials; no investigational product may be taken starting 30 days or 5 half-lives of the investigational product prior to signing the ICF, whichever is longer

Where it is running

• University of California San Francisco, Benioff Children's Hospital — Oakland, California, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• McGill University Health Center — Montreal, Quebec, Canada

Full record on ClinicalTrials.gov

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