Pompe Disease Registry
This study is currently recruiting participants.
Verified January 2013 by Genzyme
Sponsor:
Genzyme
Information provided by (Responsible Party):
Genzyme
ClinicalTrials.gov Identifier:
NCT00231400
First received: September 30, 2005
Last updated: January 30, 2013
Last verified: January 2013
| Tracking Information | |||||||||
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| First Received Date ICMJE | September 30, 2005 | ||||||||
| Last Updated Date | January 30, 2013 | ||||||||
| Start Date ICMJE | September 2004 | ||||||||
| Estimated Primary Completion Date | September 2021 (final data collection date for primary outcome measure) | ||||||||
| Current Primary Outcome Measures ICMJE |
Understanding of the variability, progression , identification and natural history of the manifestations of Pompe disease [ Time Frame: 15 Years ] [ Designated as safety issue: No ] | ||||||||
| Original Primary Outcome Measures ICMJE | Not Provided | ||||||||
| Change History | Complete list of historical versions of study NCT00231400 on ClinicalTrials.gov Archive Site | ||||||||
| Current Secondary Outcome Measures ICMJE | Not Provided | ||||||||
| Original Secondary Outcome Measures ICMJE | Not Provided | ||||||||
| Current Other Outcome Measures ICMJE | Not Provided | ||||||||
| Original Other Outcome Measures ICMJE | Not Provided | ||||||||
| Descriptive Information | |||||||||
| Brief Title ICMJE | Pompe Disease Registry | ||||||||
| Official Title ICMJE | Pompe Disease Registry | ||||||||
| Brief Summary | 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:
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| Detailed Description | The Pompe Registry is an international program; in addition to the central contact information provided under the "Location" heading, patients may contact:
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| Study Type ICMJE | Observational | ||||||||
| Study Design ICMJE | Observational Model: Cohort | ||||||||
| Target Follow-Up Duration | Not Provided | ||||||||
| Biospecimen | Not Provided | ||||||||
| Sampling Method | Non-Probability Sample | ||||||||
| Study Population | Patients diagnosed with Pompe disease |
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| Condition ICMJE |
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| Intervention ICMJE | Not Provided | ||||||||
| Study Group/Cohort (s) | Not Provided | ||||||||
| Publications * | Not Provided | ||||||||
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | |||||||||
| Recruitment Status ICMJE | Recruiting | ||||||||
| Estimated Enrollment ICMJE | 2000 | ||||||||
| Estimated Completion Date | September 2021 | ||||||||
| Estimated Primary Completion Date | September 2021 (final data collection date for primary outcome measure) | ||||||||
| Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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| Gender | Both | ||||||||
| Ages | Not Provided | ||||||||
| Accepts Healthy Volunteers | No | ||||||||
| Contacts ICMJE |
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| Location Countries ICMJE | United States | ||||||||
| Administrative Information | |||||||||
| NCT Number ICMJE | NCT00231400 | ||||||||
| Other Study ID Numbers ICMJE | Pompe Registry Protocol | ||||||||
| Has Data Monitoring Committee | No | ||||||||
| Responsible Party | Genzyme | ||||||||
| Study Sponsor ICMJE | Genzyme | ||||||||
| Collaborators ICMJE | Not Provided | ||||||||
| Investigators ICMJE |
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| Information Provided By | Genzyme | ||||||||
| Verification Date | January 2013 | ||||||||
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ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |
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