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Fabry Disease Registry & Pregnancy Sub-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 : May 22, 2020
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Tracking Information
First Submitted Date September 13, 2005
First Posted Date September 20, 2005
Last Update Posted Date May 22, 2020
Actual Study Start Date July 31, 2001
Estimated Primary Completion Date January 31, 2034   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: May 20, 2020)
  • Fabry Registry: To evaluate the long-term safety and effectiveness of Fabrazyme® [ Time Frame: 33 years ]
    The primary purpose of this Registry is to describe the development and progression of Fabry disease in a representative global population. As Fabry is not a well-described disease this longitudinal program has a wide variety of "primary" outcomes including antibody testing as well as complication outcomes (eg, QoL, cognitive testing) which are measured over time. Additionally, as it is subject to what is collected by clinical sites at the time of visit it is unknown the amount of data that will be available for analyses.
  • Fabry Pregnancy Sub-registry: pregnancy outcomes, including complications and infant growth [ Time Frame: 33 years ]
    The primary objective of this Sub-registry is to track pregnancy outcomes, including complications and infant growth, in all women with Fabry disease during pregnancy, regardless of whether they receive disease-specific therapy, such as ERT with agalsidase beta. This Sub-registry augments the routine monitoring and data collection recommended by the Fabry Registry. The Sub-registry aims to collect patient assessments from the ante- and perinatal periods and postpartum follow-up. Neonatal assessments and periodic pediatric assessments will also be collected.
Original Primary Outcome Measures Not Provided
Change History
Current Secondary Outcome Measures
 (submitted: May 20, 2020)
Fabry Register: Monitor factors associated with the efficacy of Fabry disease treatments [ Time Frame: 33 years ]
A secondary endpoint is to monitor those factors associated with the efficacy of Fabry disease treatments.
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Fabry Disease Registry & Pregnancy Sub-registry
Official Title Fabry Disease Registry Protocol
Brief Summary

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 primary 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;
  • To evaluate the long-term safety and effectiveness of Fabrazyme®

Fabry Pregnancy Sub-registry: This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woman enrolled in the Fabry Registry, regardless of whether she is receiving disease-specific therapy (such as enzyme replacement therapy with agalsidase beta) and irrespective of the commercial product with which she may be treated. Data from the Sub-registry are also used to fulfill various global regulatory requirements, to support product development/reimbursement, and for other research and non-research-related purposes. 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. If a patient consents to this Sub-registry, information about the patient's medical and obstetric history, pregnancy, and birth will be collected, and, if a patient consents to data collection for her infant, data on infant growth through month 36 postpartum will be collected.

Detailed Description Not Provided
Study Type Observational [Patient Registry]
Study Design Observational Model: Cohort
Time Perspective: Other
Target Follow-Up Duration 52 Weeks
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population

All patients with a confirmed diagnosis of Fabry disease are eligible for inclusion in the Registry.

For Fabry Pregnancy Sub-registry: It is recommended that pregnancy data be collected on eligible women regardless of infant enrollment. In the event of patients having multiple pregnancies, data collection for each individual pregnancy is encouraged for all women enrolled in this Sub-Registry.

Condition Fabry Disease
Intervention Not Provided
Study Groups/Cohorts
  • Patients with Fabry disease
    No experimental intervention is given. A patient with Fabry Disease will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician.
  • Pregnant women with confirmed diagnosis of Fabry
    No experimental intervention is given. Pregnant women with confirmed diagnosis of Fabry that are participating in the Fabry Registry and consented to participate in the Fabry Sub-registry, regardless of whether she is receiving disease-specific therapy (such as ERT with agalsidase beta) and irrespective of the commercial product with which she may be treated.
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: March 18, 2013)
9000
Original Enrollment Not Provided
Estimated Study Completion Date January 31, 2034
Estimated Primary Completion Date January 31, 2034   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria

  • Fabry Registry: 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.
  • Fabry Pregnancy Sub-registry:

    • Eligible women must:

      • be enrolled in the Fabry Registry.
      • be pregnant, or have been pregnant with appropriate medical documentation available.
      • provide a signed informed consent and authorization form(s) to participate in the Sub-Registry prior to any Sub-Registry-related data collection being performed.

Exclusion Criteria Fabry Registry: There are no exclusion criteria. Fabry Pregnancy Sub-registry: There are no exclusion criteria.

Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
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
Listed Location Countries Argentina,   Australia,   Belgium,   Brazil,   Bulgaria,   Canada,   Chile,   China,   Colombia,   Croatia,   Czechia,   Denmark,   Estonia,   Finland,   France,   Germany,   Hong Kong,   Hungary,   India,   Indonesia,   Italy,   Japan,   Korea, Republic of,   Kuwait,   Lithuania,   Malaysia,   Netherlands,   Norway,   Peru,   Philippines,   Poland,   Portugal,   Romania,   Russian Federation,   Singapore,   Slovakia,   Spain,   Sweden,   Taiwan,   Thailand,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT00196742
Other Study ID Numbers Fabry Disease Registry
DIREGC07006 ( Other Identifier: Genzyme, a Sanofi cmpany )
AGAL19211 ( Other Identifier: Genzyme, a Sanofi cmpany )
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Sanofi ( Genzyme, a Sanofi Company )
Study Sponsor Genzyme, a Sanofi Company
Collaborators Not Provided
Investigators
Study Director: Study Director Genzyme, a Sanofi Company
PRS Account Sanofi
Verification Date May 2020