Clinical Trial: A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB

Study Status: Completed
Recruit Status: Completed
Study Type: Observational

Official Title: A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB

Brief Summary: The objective is to perform a retrospective chart review to generate data to evaluate the clinical characteristics and course of disease progression of MPS IIIB.

Detailed Summary:
Sponsor: Alexion Pharmaceuticals

Current Primary Outcome: Evaluation of disease presentation and the course of disease progression including the end stage of the disease in patients with MPS IIIB, assessed from medical history data obtained by retrospective chart review of deceased patients. [ Time Frame: N/A as this is a retrospective chart review of deceased patients ]

Data obtained by retrospective chart review will include medical history data including patient demographics, disease manifestation onset, clinical history, diagnostic tests and treatments for MPS IIIB, supportive interventions/medications, clinical chemistry and haematology results, anthropometric data, radiology results, and autopsy findings.


Original Primary Outcome: Evaluation of disease presentation and the course of disease progression including the end stage of the disease in patients with MPS IIIB, assessed from medical history data obtained by retrospective chart review of deceased patients. [ Time Frame: N/A as this is a retrospective chart review of deceased patients ]

Data obtained by retrospective chart review will include medical history data including patient demographics, disease manifestation onset, clinical history, diagnostic tests and treatments for MPS IIIB, supportive interventions/medications, clinical chemistry and heamatology results, anthropometric data, radiology results, and autopsy findings.


Current Secondary Outcome:

Original Secondary Outcome:

Information By: Alexion Pharmaceuticals

Dates:
Date Received: November 3, 2014
Date Started: July 2013
Date Completion:
Last Updated: February 2, 2017
Last Verified: February 2017