- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01515631
Characterization of Pulmonary Artery Stenoses in Alagille Syndrome - a Medical Record Review
September 27, 2018 updated by: Jeffrey A. Feinstein, Stanford University
Characterization of Pulmonary Artery Stenoses in Alagille Syndrome - a Medical Record
Patients who have Alagille Syndrome (AGS) also frequently have blockages (or "stenoses") of their pulmonary arteries.
Little is known about the degree or variability of these stenoses, or the effect of this disease on the right ventricle (the chamber of the heart which pumps blood to the lungs).
This study will first quantify and describe pulmonary artery stenosis in patients with Alagille Syndrome.
The study will also assess the effect of these stenoses on the right ventricle.
The investigators hope to learn the degree and characteristics of pulmonary artery stenosis in Alagille Syndrome.
The investigators also hope to learn the effect of this pulmonary artery stenosis on the right ventricle in patients with Alagille Syndrome.
This information is critical in the management of patients with Alagille syndrome, as there is currently no data to guide clinicians on the management of pulmonary artery stenosis.
Furthermore, the information from this study may help physicians manage pulmonary artery stenosis in other patients as well.
Study Overview
Status
Completed
Conditions
Detailed Description
The investigators hope to learn the degree and characteristics of pulmonary artery stenosis in Alagille Syndrome.
We also hope to learn the effect of this pulmonary artery stenosis on the right ventricle in patients with Alagille Syndrome.
This information is critical in the management of patients with Alagille syndrome, as there is currently no data to guide clinicians on the management of pulmonary artery stenosis.
Furthermore, the information from this study may help physicians manage pulmonary artery stenosis in other patients as well.
For the third aim, we hope to learn about the different blood vessel abnormalities in the brain, and which abnormalities are most likely to lead to stroke.
Study Type
Observational
Enrollment (Actual)
25
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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California
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Palo Alto, California, United States, 94304
- Lucile Packard Children's Hospital at Stanford
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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
Patients with Alagille Syndrome.
The age range will be from birth through adulthood.
There will be no gender or ethnic limitations to study.
Description
Inclusion Criteria:
- Diagnosis of Alagille Syndrome
Exclusion Criteria:
- None
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
Cohorts and Interventions
Group / Cohort |
---|
Study
Patients with alagille syndrome
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Assessment of PA in Alagille Patient
Time Frame: Open
|
Open
|
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
April 1, 2007
Primary Completion (Actual)
February 1, 2018
Study Completion (Actual)
February 1, 2018
Study Registration Dates
First Submitted
January 18, 2012
First Submitted That Met QC Criteria
January 23, 2012
First Posted (Estimate)
January 24, 2012
Study Record Updates
Last Update Posted (Actual)
September 28, 2018
Last Update Submitted That Met QC Criteria
September 27, 2018
Last Verified
September 1, 2018
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Digestive System Diseases
- Pathologic Processes
- Cardiovascular Diseases
- Vascular Diseases
- Arterial Occlusive Diseases
- Disease
- Congenital Abnormalities
- Liver Diseases
- Genetic Diseases, Inborn
- Biliary Tract Diseases
- Heart Defects, Congenital
- Cardiovascular Abnormalities
- Abnormalities, Multiple
- Bile Duct Diseases
- Cholestasis, Intrahepatic
- Cholestasis
- Syndrome
- Stenosis, Pulmonary Artery
- Alagille Syndrome
Other Study ID Numbers
- AG9031
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
No
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.
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Mirum Pharmaceuticals, Inc.Childhood Liver Disease Research and Education NetworkCompletedAlagille SyndromeUnited States, Canada
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Mirum Pharmaceuticals, Inc.Clinigen, Inc.Approved for marketingAlagille SyndromeUnited States