Alagille SyndromeNCT01515631
Characterization of Pulmonary Artery Stenoses in Alagille Syndrome - a Medical Record Review
An observational study of Alagille Syndrome, run by Stanford University at 1 site in United States.
Trial summary
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). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Alagille Syndrome
- How many people
- 25 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2007to Feb 2018
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- Diagnosis of Alagille Syndrome
Who cannot join
- None
Trial locations and contacts
1 site in 1 country.
United States1 site
Lucile Packard Children's Hospital at Stanford
Palo Alto, California, 94304
About this study
Official title: 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.
Read the 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.
- Conditions
- Alagille Syndrome
- Medical subject headings
- Alagille Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Study | Patients with alagille syndrome |
What is being measured
The primary outcome is the main question the study is designed to answer.
Assessment of PA in Alagille Patient
Measured: Open
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jan 2012 (estimated)
- Study startApr 2007
- Primary completionFeb 2018
- Study completionFeb 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alagille SyndromeStudies of the same condition, open and under way.
- More trials run by Stanford UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Retrospective
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jeffrey A. Feinstein, Stanford University
- Investigators
- Jeffrey Feinstein, MD, MPH, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01515631
- Sponsor's protocol ID
- AG9031
- Status last verified
- Sep 2018
- First submitted
- 18 Jan 2012
- Last updated
- 28 Sep 2018
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Characterization of Pulmonary Artery Stenoses in Alagille Syndrome - a Medical Record
- ClinicalTrials.gov ID
- NCT01515631
- Acronym / sponsor ID
- AG9031
- Conditions
- Alagille Syndrome
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Study start
- Apr 2007
- Primary completion
- Feb 2018 (actual)
- Study completion
- Feb 2018 (actual)
- First posted
- 24 Jan 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Observational model
- Case only
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Study | – | –Patients with alagille syndrome |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (1 listed)
- Diagnosis of Alagille Syndrome
Key exclusion criteria (1 listed)
- None
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Assessment of PA in Alagille Patient
Time frame: Open
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Sep 2018.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jeffrey Feinstein, MD, MPH principal investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Alagille Syndrome, run by Stanford University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Sep 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Assessment of PA in Alagille Patient (time frame: Open). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Sep 2018.