Fabry Disease Registry & Pregnancy Sub-registry
-
- STATUS
- Recruiting
-
- End date
- Jan 31, 2034
-
- participants needed
- 9000
-
- sponsor
- Genzyme, a Sanofi Company
Updated on 5 August 2024
deficiency
alpha-galactosidase
agalsidase beta
fabrazyme
replacement therapy
fabry's disease
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.
Description
Details
| Condition | fabry disease |
|---|---|
| Age | 100years or below |
| Clinical Study Identifier | NCT00196742 |
| Sponsor | Genzyme, a Sanofi Company |
| Last Modified on | 5 August 2024 |
How understandable was the trial content above?
Hard to understand
Easy to understand
Eligibility
No information provided
How to participate?
Step
2
Get screened
Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.
Learn more
Step
3
Enroll in the clinical study
If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
Learn more
Step
4
Get your study results
Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.
Learn moreSimilar trials to consider
Loading...
Browse trials for
Not finding what you're looking for?
Sign up as a volunteer to stay informed
Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.
Sign up as volunteerStudy Definition
WikipediaAdd a private note
- Select a piece of text.
- Add notes visible only to you.
- Send it to people through a passcode protected link.