Disease registry for patients with Gaucher disease.An observational program for patients with Gaucher disease that was designed to track the natural history and outcomes of patients.
- Conditions
- Health Condition 1: null- Gaucher DiseaseHealth Condition 2: E752- Other sphingolipidosis
- Registration Number
- CTRI/2018/03/012357
- 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
1.all age group from new born to old age allowed
2. All patients with a confirmed diagnosis of Gaucher 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, Regardless of whether they are receiving disease therapy including ERT (such as imiglucerase) and irrespective of the commercial product with which they are being treated.
3. A confirmed diagnosis is defined as a documented acid Ã?-glucosidase deficiency and/or mutations in the acid Ã?-glucosidase gene.
1. There are no exclusion criteria in this Registry. Patients are allowed to participate in clinical studies and may be receiving different therapies to treat their disease. However, data entry (with the exception of data collected prior to the patientâ??s participation in the clinical study) should be put on hold in the Registry during the patientsâ?? participation in the clinical study. When the patient has discontinued or completed the clinical study, data entry can resume. Where the clinical study was sponsored by Genzyme, the Registry team may work with
participating physicians, as necessary and appropriate, to facilitate the entry of data from the clinical study (to the extent such data are also collected by the Registry) into the Registry database.
Study & Design
- Study Type
- Observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method - To enhance the understanding of the variability, progression, and natural history of Gaucher disease, with the ultimate goal of better guiding and assessing therapeutic intervention; <br/ ><br> <br/ ><br>- To provide the Gaucher medical community with recommendations for monitoring patients and to provide reports on patient outcomes to help optimize patient care; and <br/ ><br>- To evaluate the long-term effectiveness of Cerezyme®Timepoint: 15 years
- Secondary Outcome Measures
Name Time Method one, its a disease registryTimepoint: None