Pilot Study: Urea Cycle Disorders Practice Patterns and Outcomes Assessment
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|ClinicalTrials.gov Identifier: NCT02311283|
Recruitment Status : Completed
First Posted : December 8, 2014
Last Update Posted : March 7, 2018
|Condition or disease|
|Urea Cycle Disorders|
Participants enrolled in the RDCRN UCDC Contact Registry will receive an email inviting them to participate in the study. Interested participants will be directed to the informed consent document. After agreeing to participate, participants will be directed to the study web portal. The study web portal will be developed and maintained by the Data Management and Coordinating Center at the University of South Florida (USF).
Participants will complete several questionnaires about their urea cycle disorder including diagnosis and treatment information and quality of life.
|Study Type :||Observational [Patient Registry]|
|Actual Enrollment :||87 participants|
|Target Follow-Up Duration:||1 Year|
|Official Title:||Pilot Study: Urea Cycle Disorders Practice Patterns and Outcomes Assessment|
|Study Start Date :||October 2014|
|Actual Primary Completion Date :||October 2015|
|Actual Study Completion Date :||July 1, 2016|
- Test the feasibility of collecting survey data from the UCDC Contact Registry [ Time Frame: Up to one year from study activation. ]Patient-reported survey data will be reviewed for accuracy and feasibility as a reliable data source.
- Assess patient reported outcomes on urea cycle relevant dimensions (applied cognition & subjective well-being). [ Time Frame: Up to one year from study activation. ]The PROMIS website will be used to assess the accuracy and completeness of patient reported outcomes on urea cycle relevant dimensions.
- Validate data on treatment setting (team and organization) and management (diet, pharmacologic management, transplantation) [ Time Frame: Up to one year from study activation. ]Patient-reported survey data will be linked with research data collected in clinic for the same patients to validate patient-reported data on treatment setting and disease management.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02311283
|United States, Florida|
|University of South Florida|
|Tampa, Florida, United States, 33612|
|Study Chair:||Jeffrey Krischer, PhD||University of South Florida|
|Principal Investigator:||Jennifer Seminara, MD||Children's Research Institute|
|Principal Investigator:||Vera Anastasoaie, MD||Boston Children’s Hospital|