Disease Registry for patients with Fabry disease.An observational program for patients with Fabry disease that was designed to track the natural history and outcomes of patients.
Not Applicable
- Conditions
- Health Condition 1: null- Fabry DiseaseHealth Condition 2: E752- Other sphingolipidosis
- Registration Number
- CTRI/2018/03/012268
- 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 Fabry 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 enzyme replacement therapy (ERT) (such as agalsidase beta) and irrespective of the commercial product with which they are being treated.
3. A confirmed diagnosis is defined as a documented deficiency in plasma or leukocyte αGAL enzyme activity and/or mutation(s) in the gene coding for αGA
Exclusion Criteria
No exclusion criteria, its a disease registry
Study & Design
- Study Type
- Observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 1) To enhance the understanding of the variability, progression, and natural history of Fabry disease, including heterozygous females with the disease; <br/ ><br>2) To assist the Fabry medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient <br/ ><br>care; <br/ ><br>3) To characterize and describe the Fabry population as a whole; and <br/ ><br>4) To evaluate the long-term safety and effectiveness of Fabrazyme® (agalsidase beta).Timepoint: 15 years
- Secondary Outcome Measures
Name Time Method o Secondary OutcomeTimepoint: NA