Disease Registry for patients with MPS I disease.observational program for patients with MPS I disease that was designed to track the natural history and outcomes of patients
Not Applicable
- Conditions
- Health Condition 1: null- MPS I DiseaseHealth Condition 2: E760- Mucopolysaccharidosis, type I
- Registration Number
- CTRI/2018/03/012266
- Lead Sponsor
- Sanofi Synthelabo India Private Limited
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Open to Recruitment
- Sex
- Not specified
- Target Recruitment
- 0
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
Study & Design
- Study Type
- Observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method -Evaluate the long-term effectiveness and safety of Aldurazyme® (laronidase); <br/ ><br>-Characterize and describe the mucopolysaccharidosis type I (MPS I) population as a whole, including the variability, progression, and natural history of MPS I disease; and <br/ ><br>-Assist/facilitate the MPS I medical community with the development of recommendations for monitoring patients and reports on patient outcomes to optimize patient care.Timepoint: 15 years
- Secondary Outcome Measures
Name Time Method one, its a disease registryTimepoint: None