Sanofi Genzyme Rare Disease Registry - A Multi-Center, International, Longitudinal, Observational program designed to track natural history and outcomes of patients with Gaucher, Fabry, MPSI, and Pompe Disease (Gaucher registry, Fabry registry, MPSI registry, Pompe registry)
International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
ClinicalTrials.gov ID NCT00358943
Sponsor Genzyme, a Sanofi Company
Information provided by Sanofi (Genzyme, a Sanofi Company) (Responsible Party)
Last Update Posted 2024-04-17
Study Overview
The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher 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 understanding of the variability, progression, identification, and natural history of Gaucher disease, with the ultimate goal of better guiding and assessing therapeutic intervention.
- To assist the Gaucher medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care.
- To characterize the Gaucher disease population.
- To evaluate the long-term effectiveness of imiglucerase and of eliglustat.
Gaucher Pregnancy Sub-registry: The primary objective of this Sub-registry is to track pregnancy outcomes, including complications and infant growth, in all women with Gaucher disease during pregnancy, regardless of whether they receive disease-specific therapy. 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.
Fabry Disease Registry & Pregnancy Sub-registry
ClinicalTrials.gov ID NCT00196742
Sponsor Genzyme, a Sanofi Company
Information provided by Sanofi (Genzyme, a Sanofi Company) (Responsible Party)
Last Update Posted 2024-04-17
Study Overview
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:
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To enhance the understanding of the variability, progression, and natural history of Fabry disease, including heterozygous females with the disease;
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To assist the Fabry medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care;
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To characterize and describe the Fabry population as a whole;
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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.
Mucopolysaccharidosis I (MPS I) Registry
ClinicalTrials.gov ID NCT00144794
Sponsor Genzyme, a Sanofi Company
Information provided by Sanofi (Genzyme, a Sanofi Company) (Responsible Party)
Last Update Posted 2024-06-10
Study Overview
The Mucopolysaccharidosis I (MPS I) Registry is an ongoing, observational database that tracks the outcomes of patients with MPS I. The data collected by the MPS I Registry will provide information to better characterize the natural history and progression of MPS I as well as the clinical responses of patients receiving enzyme replacement therapy, such as Aldurazyme (Recombinant Human Alpha-L-Iduronidase), or other treatment modalities.
The objectives of the Registry are:
- To evaluate the long-term effectiveness and safety of Aldurazyme® (laronidase)
- To characterize and describe the MPS I population as a whole, including the variability, progression, and natural history of MPS I
- To help the MPS I medical community with the development of recommendations for monitoring patients and reports on patient outcomes to optimize patient care
Pompe Disease Registry Protocol
ClinicalTrials.gov ID NCT00231400
Sponsor Genzyme, a Sanofi Company
Information provided by Sanofi (Genzyme, a Sanofi Company) (Responsible Party)
Last Update Posted 2024-02-01
Study Overview
The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outcomes in patients, both treated and not. Data from the Registry are also used to fulfill various global regulatory commitments, to support product development/reimbursement, and for other research and non-research related purposes.
The objectives of the Registry are:
- To enhance understanding of the variability, progression, identification, and natural history of Pompe disease, with the ultimate goal of better guiding and assessing therapeutic intervention.
- To assist the Pompe medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care.
- To characterize the Pompe disease population.
- To evaluate the long-term effectiveness of alglucosidase alfa.