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Fabry Disease Registry

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00196742
Recruitment Status : Recruiting
First Posted : September 20, 2005
Last Update Posted : December 20, 2017
Information provided by (Responsible Party):

September 13, 2005
September 20, 2005
December 20, 2017
July 31, 2001
July 30, 2021   (Final data collection date for primary outcome measure)
To evaluate the long-term safety and effectiveness of Fabrazyme® [ Time Frame: 15 years ]
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Complete list of historical versions of study NCT00196742 on ClinicalTrials.gov Archive Site
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Fabry Disease Registry
Fabry Disease Registry

The Fabry Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Fabry disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician.

The objectives of the Registry are:

  • To enhance the understanding of the variability, progression, and natural history of Fabry disease, including heterozygous females with the disease;
  • To assist the Fabry medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care;
  • To characterize and describe the Fabry population as a whole; and
  • To evaluate the long-term safety and effectiveness of Fabrazyme®

The Fabry Registry is an international program; in addition to the central contact information provided under the "Location" heading, patients may contact:

  • In Asia-Pacific - Vivian Liu, +65-6431-2548, Vivian.liu@genzyme.com
  • In Europe - +31-35-699-1232, europe@FabryRegistry.com
  • In Latin America - +617-591-5500, help@FabryRegistry.com
  • In North America - +617-591-5500, help@FabryRegistry.com
Observational Model: Cohort
Time Perspective: Other
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Non-Probability Sample
All patients with a confirmed diagnosis of Fabry disease are eligible for inclusion in the Registry.
Fabry Disease
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*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
July 30, 2021
July 30, 2021   (Final data collection date for primary outcome measure)

Inclusion Criteria All patients with a confirmed diagnosis of Fabry disease who have signed the informed consent and patient authorization form(s) are eligible for inclusion. Confirmed diagnosis is defined as a documented deficiency in plasma or leukocyte αGAL (alpha-galactosidase) enzyme activity and/or mutation(s) in the gene coding for αGAL.

Exclusion Criteria There are no exclusion criteria in this Registry. Patients are allowed to participate in other clinical studies and may be receiving different therapies to treat their disease; however, enrollment in other clinical studies should be noted on the Registry case report forms (CRFs).

Sexes Eligible for Study: All
Child, Adult, Senior
Contact: Trial Transparency email recommended (Toll free number for US & Canada) 800-633-1610 ext 1 then # Contact-Us@sanofi.com
Contact: Fabry Registry HelpLine 617-591-5500 help@fabryregistry.com
Australia,   Brazil,   Chile,   France,   Hong Kong,   Italy,   Japan,   Korea, Republic of,   Malaysia,   Philippines,   Portugal,   Singapore,   Slovakia,   Taiwan,   Thailand,   United States
DIREGC07006 ( Other Identifier: Sanofi )
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Sanofi ( Genzyme, a Sanofi Company )
Genzyme, a Sanofi Company
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Study Director: Medical Monitor Genzyme, a Sanofi Company
December 2017