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Pompe Disease Registry
This study is currently recruiting participants.
Verified June 2017 by Sanofi ( Genzyme, a Sanofi Company )
Sponsor:
Genzyme, a Sanofi Company
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT00231400
First received: September 30, 2005
Last updated: June 13, 2017
Last verified: June 2017
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Purpose
The Pompe Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Pompe 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 the key manifestations of Pompe disease;
- To assist the Pompe 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 Pompe disease population as a whole; and
- To evaluate the long-term effectiveness and safety of available treatment options including ERT(Enzyme Replacement Therapy) with Myozyme®.
| Condition |
|---|
| Glycogen Storage Disease Type II Pompe Disease |
| Study Type: | Observational |
| Study Design: | Observational Model: Cohort Time Perspective: Other |
| Official Title: | Pompe Disease Registry |
Resource links provided by NLM:
Further study details as provided by Sanofi ( Genzyme, a Sanofi Company ):
Primary Outcome Measures:
- Understanding of the variability, progression , identification and natural history of the manifestations of Pompe disease [ Time Frame: 15 Years ]
| Estimated Enrollment: | 2000 |
| Actual Study Start Date: | August 21, 2004 |
| Estimated Study Completion Date: | September 30, 2026 |
| Estimated Primary Completion Date: | September 30, 2026 (Final data collection date for primary outcome measure) |
Eligibility| Ages Eligible for Study: | Child, Adult, Senior |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
Study Population
Patients diagnosed with Pompe disease
Criteria
Inclusion Criteria:
- Patient must have a confirmed diagnosis of Pompe disease, documented by GAA(Glucosidase Alpha Acid) enzyme deficiency or GAA gene mutation
Exclusion Criteria:
- There are no exclusion criteria
Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00231400
Show 50 Study Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00231400
Contacts
| Contact: Trial Transparency email recommended (Toll free number for US & Canada) | 800-633-1610 ext 1 then # | Contact-Us@sanofi.com | |
| Contact: Medical Information | 617-252-7832 | medinfo@genzyme.com |
Show 50 Study Locations
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
| Study Director: | Medical Monitor | Genzyme, a Sanofi Company |
More Information
| Responsible Party: | Genzyme, a Sanofi Company |
| ClinicalTrials.gov Identifier: | NCT00231400 History of Changes |
| Other Study ID Numbers: |
Pompe Registry DIREGC07005 ( Other Identifier: Sanofi ) |
| Study First Received: | September 30, 2005 |
| Last Updated: | June 13, 2017 |
Keywords provided by Sanofi ( Genzyme, a Sanofi Company ):
|
Glycogen Storage Disease Type II (GSD-II) GSD-II Pompe Disease |
Pompe Disease (late-onset) Acid Maltase Deficiency Disease Glycogenosis II |
Additional relevant MeSH terms:
|
Brain Diseases, Metabolic, Inborn Glycogen Storage Disease Type II Glycogen Storage Disease Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases |
Nervous System Diseases Metabolism, Inborn Errors Genetic Diseases, Inborn Carbohydrate Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases |
ClinicalTrials.gov processed this record on July 26, 2017