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NCT03529786 · ClinicalTrials.gov registry record
Mucopolysaccharidosis Type II Natural History
A clinical trial, sponsored by REGENXBIO.
- Completed
- Registry status
- 36
- Enrollment target
NCT03529786: Completed study, sponsored by REGENXBIO.
NCT03529786 is a clinical trial that has completed, run by REGENXBIO. The registered enrollment target is 36 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03529786 has completed, sponsored by REGENXBIO.
- COMPLETED
- Registry status
- 36 participants
- Enrollment target
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.
Primary Outcome
There are 10 neurocognitive measures that provide intelligence quotients (IQ) scores and/or developmental quotients (DQ) scores.
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 36 participants |
| Start Date | 2017-09-27 |
| Est. Completion | 2022-03-22 |
What the finished NCT03529786 record still lists
NCT03529786 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 36 participants, a relatively small participant target.
The record links to 0 conditions, and to 0 interventions.
NCT03529786 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03529786 about?
NCT03529786 is a clinical study titled "Mucopolysaccharidosis Type II Natural History". 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 pati...
What is the current status of trial NCT03529786?
This trial is currently completed. The enrollment target is 36 participants. The study started on 2017-09-27. Estimated completion is 2022-03-22.
Who is sponsoring clinical trial NCT03529786?
This trial is sponsored by REGENXBIO, which has 9 total clinical trials registered on ClinicalTrials.gov.
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