Pompe Disease Registry Protocol
|
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: NCT00231400 |
|
Recruitment Status :
Recruiting
First Posted : October 4, 2005
Last Update Posted : January 6, 2021
|
Sponsor:
Genzyme, a Sanofi Company
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
| Tracking Information | |||||||||
|---|---|---|---|---|---|---|---|---|---|
| First Submitted Date | September 30, 2005 | ||||||||
| First Posted Date | October 4, 2005 | ||||||||
| Last Update Posted Date | January 6, 2021 | ||||||||
| Actual Study Start Date | September 15, 2004 | ||||||||
| Estimated Primary Completion Date | January 31, 2034 (Final data collection date for primary outcome measure) | ||||||||
| Current Primary Outcome Measures |
Understanding of the variability, progression , identification and natural history of the manifestations of Pompe disease [ Time Frame: maximum 30 years ] | ||||||||
| Original Primary Outcome Measures | Not Provided | ||||||||
| Change History | |||||||||
| Current Secondary Outcome Measures | Not Provided | ||||||||
| 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 | Pompe Disease Registry Protocol | ||||||||
| Official Title | Pompe Disease Registry | ||||||||
| Brief Summary | 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:
|
||||||||
| Detailed Description | Study Design Time Perspective: Retrospective and Prospective | ||||||||
| Study Type | Observational [Patient Registry] | ||||||||
| Study Design | Observational Model: Cohort Time Perspective: Other |
||||||||
| Target Follow-Up Duration | 5 Years | ||||||||
| Biospecimen | Not Provided | ||||||||
| Sampling Method | Non-Probability Sample | ||||||||
| Study Population | All patients with a confirmed diagnosis of Pompe disease are eligible for inclusion in the Registry. | ||||||||
| Condition |
|
||||||||
| Intervention | Not Provided | ||||||||
| Study Groups/Cohorts | Not Provided | ||||||||
| 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 |
2000 | ||||||||
| 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: All patients with a confirmed diagnosis of Pompe disease who have signed the informed consent and authorization form(s) are eligible for inclusion. Confirmed diagnosis is defined as documented GAA enzyme deficiency from blood, skin, or muscle tissue and/or documentation of 2 GAA gene mutations. Exclusion Criteria: There are no exclusion criteria in this Registry |
||||||||
| Sex/Gender |
|
||||||||
| Ages | Child, Adult, Older Adult | ||||||||
| Accepts Healthy Volunteers | No | ||||||||
| Contacts |
|
||||||||
| Listed Location Countries | Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Chile, China, Colombia, Denmark, France, Germany, Greece, Hong Kong, Hungary, India, Italy, Japan, Korea, Republic of, Kuwait, Malaysia, Netherlands, Philippines, Poland, Portugal, Russian Federation, Serbia, Singapore, Slovakia, Taiwan, Thailand, United Arab Emirates, United Kingdom, United States | ||||||||
| Removed Location Countries | |||||||||
| Administrative Information | |||||||||
| NCT Number | NCT00231400 | ||||||||
| Other Study ID Numbers | DIREGC07005 | ||||||||
| 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 |
|
||||||||
| PRS Account | Sanofi | ||||||||
| Verification Date | May 2020 | ||||||||