Glycogen Storage Disease Type IV Database (GSDIVDB)
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|ClinicalTrials.gov Identifier: NCT02683512|
Recruitment Status : Recruiting
First Posted : February 17, 2016
Last Update Posted : December 6, 2017
|Condition or disease||Intervention/treatment|
|Glycogen Storage Disease Type IV||Other: No intervention|
The long term goal of the proposed project is to develop a repository of information with the long term goal of expanding the understanding of the disease manifestation and natural history of GSD IV.
Specific Aims: To establish a repository of clinical, laboratory, and biochemical information on individuals diagnosed with glycogen brancher enzyme deficiency. In the long term, this information will permit a better understanding of the long term complications and clinical course in GBE deficiency.
The medical records will be reviewed and participant information will be collected using REDCap™ software, hosted through the Duke Translational Medicine Institute (DTMI). The database will serve as the repository for retrospective and prospective clinical information and results on all participants.
Data to be collected: Name, DOB, Sex, Age, Method of diagnosis, Liver, skin, or muscle biopsy histology, Initial symptoms and/or reason for referral, Past medical history, Past surgical history, Family history, Pregnancy history, Developmental history, Developmental assessments, and Results of liver function testing. All patients with GSD-IV regardless of age, gender or ethnicity, are eligible for the database.
This research project will enroll individuals from outside institutions. The goal is increase the robustness of data collection and analysis with a large number of participants.
|Study Type :||Observational [Patient Registry]|
|Estimated Enrollment :||200 participants|
|Target Follow-Up Duration:||10 Years|
|Official Title:||Database for Information on Individuals Affected With Glycogen Brancher Deficiency, Also Known as Glycogen Storage Disease Type IV|
|Study Start Date :||December 2015|
|Estimated Primary Completion Date :||December 2025|
|Estimated Study Completion Date :||December 2025|
- Other: No intervention
This is an observational study that consists of data abstraction.
- Progression of disease [ Time Frame: Duration of study, approximately 10 years ]
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02683512
|Contact: Mimi McCartyemail@example.com|
|Contact: Loren Pena, MD, PhDfirstname.lastname@example.org|
|United States, North Carolina|
|Duke University Medical Center||Recruiting|
|Durham, North Carolina, United States, 27710|
|Contact: Mimi McCarty 919-684-0398 email@example.com|
|Contact: Loren Pena, MD, PhD 9196819873 firstname.lastname@example.org|
|Principal Investigator: Loren Pena, MD, PhD|
|Principal Investigator:||Loren Pena, MD, PhD||Duke University|