CTRI/2018/03/012266Recruiting未知
Genzyme Rare disease registries (MPS I Disease Registry Protocol).
Sanofi Synthelabo India Private Limited0 sites0 target enrollmentStarted: TBDLast updated:
Trial Snapshot
- Phase
- 未知
- Status
- Recruiting
- Sponsor
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Observational
Eligibility Criteria
Inclusion Criteria
- •1. All age group from new born to old age allowed
- •2. All patients with a confirmed diagnosis of MPS I disease who are willing and able to provide written informed consent and any additional authorization documents required by local law to send health information to the Registry are eligible for inclusion, regardless of whether they are receiving disease therapy including ERT (such as laronidase) and irrespective of the commercial product with which they are being treated.
- •3. A confirmed diagnosis of MPS I consists of documented biochemical evidence of a deficiency in α-Liduronidase enzyme activity or mutation(s) in the gene coding for α-L-iduronidase
Exclusion Criteria
- •No Exclusion Criteria
Investigators
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