- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02293382
A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB
February 2, 2017 updated by: Alexion Pharmaceuticals
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.
Study Overview
Status
Completed
Conditions
Study Type
Observational
Enrollment (Actual)
30
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Porte Alegre, Brazil
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Amsterdam, Netherlands
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Barcelona, Spain
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Birmingham, United Kingdom
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Pennsylvania
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Pittsburg, Pennsylvania, United States
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Sampling Method
Non-Probability Sample
Study Population
30 deceased patients with MPS IIIB
Description
Inclusion Criteria:
The deceased patient was diagnosed with MPS IIIB as determined by either of the following:
- Documented deficiency of NAGLU enzyme activity.
- Documented functionally-relevant mutations in both alleles of the NAGLU gene.
- Patient's parent or legal guardian provides informed consent (unless the requirement for informed consent is waived by local regulations).
- The availability of pre-defined information in the patient's clinical chart:
Exclusion Criteria:
There are no exclusion criteria for this study.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Observational Models: Case-Only
- Time Perspectives: Retrospective
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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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
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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.
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N/A as this is a retrospective chart review of deceased patients
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
July 1, 2013
Primary Completion (Actual)
January 1, 2015
Study Completion (Actual)
January 1, 2015
Study Registration Dates
First Submitted
November 3, 2014
First Submitted That Met QC Criteria
November 17, 2014
First Posted (Estimate)
November 18, 2014
Study Record Updates
Last Update Posted (Estimate)
February 3, 2017
Last Update Submitted That Met QC Criteria
February 2, 2017
Last Verified
February 1, 2017
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- NGLU-NH01
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on MPS IIIB (Sanfilippo Syndrome)
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Swedish Orphan BiovitrumCompletedSanfilippo Syndrome Type A (MPS IIIA)United States, Germany, Turkey
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ShireTerminatedSanfilippo Syndrome | Mucopolysaccharidosis (MPS)United States, United Kingdom, Spain, Germany, France, Italy, Netherlands
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ShireCompletedSanfilippo Syndrome Type BUnited States, United Kingdom, Germany