Research Study Summary
Rare disease registries for certain lysosomal storage disorders, including, but not limited to, Fabry, Gaucher, MPS I, and Pompe diseases (each, a Registry)
The Rare Disease Registry Program (including but not limited to, Gaucher, Fabry, MPS I, and Pompe diseases) is a multi-center, international, longitudinal, observational program that tracks the natural history and outcomes of patients with these rare diseases.
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CW ID: 209881
Date Last Changed:
March 8, 2016
Clinical Trial Snapshot
- Both Male and Female
- Overall Status
- Facility Type
- All patients with a confirmed diagnosis of their specific 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
- 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 patient’s participation in the clinical study) should be put on hold in the Registry during the patients’ 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
Protocol Number: Rare Disease Registry
The Registry program is a multi-center, international, observational program for patients with these rare diseases that was designed to track the natural history and outcomes of patients. No experimental intervention is given; thus, a patient will undergo clinical assessments and receive standard of-care treatment as determined by the patient’s physician. As the Registries are a voluntary program, the total number of patients with these diseases who will participate in the Registries and the associated person-years of follow-up are not pre-defined, as not all patients have been identified, nor will all patients wish to participate. The Registry databases have been designed to collect a variety of baseline and follow-up data obtained through routine clinical and laboratory assessments. All data submission is voluntary and there are no pre-determined follow-up periods. Participating sites are encouraged to enter comprehensive baseline data at enrollment and to perform follow-up assessments at regular intervals. While the protocols provide Recommended Schedules of Assessments, each physician is solely responsible for determining the appropriate clinical care for each patient.
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