Alagille SyndromeNCT05846854
Decreasing Hemorrhage Risk in Children With Alagille Syndrome
A study testing Assessment for bleeding disorder with tailored post-operative care in people with Alagille Syndrome and Pulmonary Artery Stenoses, run by Stanford University at 1 site in United States.
Trial summary
The goal of this interventional study is to test a hemostasis screening protocol and cardiac peri-procedural and post-operative hemostasis pathway to improving bleeding complications and improve patient survival for children with Alagille syndrome and complex cardiac conditions. Read more about this study
- Who is it for?
- Ages Up to 17 years
- Conditions the study is for
- Alagille Syndrome, Pulmonary Artery Stenoses, Acquired Von Willebrand Disease
- What is being tested?
- Assessment for bleeding disorder with tailored post-operative care
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Apr 2023to Nov 2024
- Phase
- Interventional
- 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 Up to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 0-17 years old
- with complex cardiac condition requiring pulmonary artery reconstruction (branch pulmonary artery stenosis, MAPCAs or Tetralogy of Fallot without MAPCAs)
Who cannot join
- history of known bleeding disorder
- aged 18 years or older
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Novel Protocol to Decrease Peri-procedural and Intra-operative Hemorrhage in Children With Alagille Syndrome
The goal of this interventional study is to test a hemostasis screening protocol and cardiac peri-procedural and post-operative hemostasis pathway to improving bleeding complications and improve patient survival for children with Alagille syndrome and complex cardiac conditions. The main questions it aims to answer are:
- Are children with Alagille syndrome with cardiac anomalies more likely to have acquired von Willebrand syndrome (a condition that causes increased bleeding)
- Does implementation of a novel screening protocol to detect pre-operative bleeding conditions decrease intra-operative and/or post-operative bleeding complications and mortality risk?
- Does implementation of a novel screening protocol to detect and treat bleeding conditions cause thrombotic complications?
Read more
Participants will undergo additional hematology and bleeding disorder screening prior to cardiac surgery. They will additionally undergo a detailed family screening for a history of bleeding by a genetic counselor.
Researchers will compare these findings with children who have similar complex cardiac conditions requiring surgery, but who do not have Alagille syndrome to see if bleeding conditions and complications are more or less common in children with Alagille syndrome.
Read the detailed description
This study aims to develop and implement a pre-operative screening protocol prior to major procedures or surgeries for children with Alagille syndrome, including cardiac catheterization and cardiothoracic surgery (pulmonary artery reconstruction).
Secondary Objectives
- Develop and implement an intra-operative protocol for children with Alagille syndrome and acquired von willebrand syndrome.
- Compare cardiac intra- and up to 48 hour post-operative bleeding complications, post-operative thrombotic complications within 30 days of surgery, and mortality for (a) children with Alagille syndrome with and without acquired von willebrand syndrome, and (b) children without Alagille syndrome.
This is a single-site study with the goal to enroll 40 patients over the course of the study period. All patients that are referred for Pulmonary Artery Reconstruction will be screened for eligibility. All patients will undergo standard of care pre-operative hematologic screening to evaluate for bleeding disorders (specifically platelet aggregation disorders and acquired von Willebrand Factor deficiency).
The study includes a pre-screening period of up to 4 weeks followed by a 12-months follow-up period as part of the standard of care following pulmonary artery reconstruction surgery for children with Alagille syndrome and a research related protocol for children without Alagille syndrome.
Patients would have blood drawn one time at least 2 weeks in advance of their cardiothoracic surgery. This is the screening protocol to understand if they have a bleeding disorder that would change their management in the operating room and after their surgery. For patients that are found to have a bleeding disorder requiring treatment, they would need post-operatively a blood test daily for up to 7 days total and another blood test weekly for 2 weeks and then once prior to discharge.
- Being tested
- Assessment for bleeding disorder with tailored post-operative care
- Medical subject headings
- Alagille Syndrome, Stenosis, Pulmonary Artery
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Assessment for bleeding disorder with tailored post-operative careOther
Intervention will be expanded lab work-up for hematologic conditions, consultation by Hematology and tailored intra-operative and post-operative plan with aim to mitigate bleeding and hemorrhage risk, balancing thrombotic risk with this intervention.
| Study group | Role | What they receive |
|---|---|---|
| Alagille syndrome | Experimental | Patients with complex cardiac conditions requiring cardiothoracic surgery who also have a history of Alagille syndrome. Receives: Other: Assessment for bleeding disorder with tailored post-operative care |
| No history of Alagille syndrome | Experimental | Patients with complex cardiac conditions requiring cardiothoracic surgery who do not have a diagnosis of Alagille syndrome. Receives: Other: Assessment for bleeding disorder with tailored post-operative care |
What is being measured
The primary outcome is the main question the study is designed to answer.
Blood product volume
Measured: Intra-operatively through 24 hours post-operatively
Chest tube output blood volume
Measured: Intra-operatively through 24 hours post-operatively
Number of participants with pulmonary hemorrhage
Measured: Intra-operatively through 24 hours post-operatively
Secondary outcomes (1)
Incidence of thromboembolism events in patients with hematologic condition
This outcome is to evaluate thrombosis risk for patients identified to have a hematologic condition who receive medication intra-operatively and post-operatively to decrease their risk of bleeding. Occurrence of thromboembolism (including blood vessel, intracardiac, stroke, mediastinal thrombosis events) will be assessed based on imaging.
Measured: Intra-operatively through 30 days post-operatively
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 May 2023
- Study start18 Apr 2023
- Primary completionNov 2024 (estimated)
- Study completionNov 2024 (estimated)
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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Prevention
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Collaborators
- Alagille Syndrome Alliance
- Responsible party
- Principal investigator, Noelle Hanako, Stanford University
- Investigators
- Noelle Ebel, MD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05846854
- Sponsor's protocol ID
- 66477
- Status last verified
- May 2023
- First submitted
- 26 Apr 2023
- Last updated
- 6 May 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Novel Protocol to Decrease Peri-procedural and Intra-operative Hemorrhage in Children With Alagille Syndrome
- ClinicalTrials.gov ID
- NCT05846854
- Acronym / sponsor ID
- 66477
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Collaborators
- Alagille Syndrome Alliance
- Study start
- 18 Apr 2023 (actual)
- Primary completion
- Nov 2024 (estimated)
- Study completion
- Nov 2024 (estimated)
- First posted
- 6 May 2023 (actual)
- 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.
The goal of this interventional study is to test a hemostasis screening protocol and cardiac peri-procedural and post-operative hemostasis pathway to improving bleeding complications and improve patient survival for children with Alagille syndrome and complex cardiac conditions. The main questions it aims to answer are:
- Are children with Alagille syndrome with cardiac anomalies more likely to have acquired von Willebrand syndrome (a condition that causes increased bleeding)
- Does implementation of a novel screening protocol to detect pre-operative bleeding conditions decrease intra-operative and/or post-operative bleeding complications and mortality risk?
- Does implementation of a novel screening protocol to detect and treat bleeding conditions cause thrombotic complications?
Participants will undergo additional hematology and bleeding disorder screening prior to cardiac surgery. They will additionally undergo a detailed family screening for a history of bleeding by a genetic counselor.
Researchers will compare these findings with children who have similar complex cardiac conditions requiring surgery, but who do not have Alagille syndrome to see if bleeding conditions and complications are more or less common in children with Alagille syndrome.
Detailed description
This study aims to develop and implement a pre-operative screening protocol prior to major procedures or surgeries for children with Alagille syndrome, including cardiac catheterization and cardiothoracic surgery (pulmonary artery reconstruction).
Secondary Objectives
- Develop and implement an intra-operative protocol for children with Alagille syndrome and acquired von willebrand syndrome.
- Compare cardiac intra- and up to 48 hour post-operative bleeding complications, post-operative thrombotic complications within 30 days of surgery, and mortality for (a) children with Alagille syndrome with and without acquired von willebrand syndrome, and (b) children without Alagille syndrome.
This is a single-site study with the goal to enroll 40 patients over the course of the study period. All patients that are referred for Pulmonary Artery Reconstruction will be screened for eligibility. All patients will undergo standard of care pre-operative hematologic screening to evaluate for bleeding disorders (specifically platelet aggregation disorders and acquired von Willebrand Factor deficiency).
The study includes a pre-screening period of up to 4 weeks followed by a 12-months follow-up period as part of the standard of care following pulmonary artery reconstruction surgery for children with Alagille syndrome and a research related protocol for children without Alagille syndrome.
Patients would have blood drawn one time at least 2 weeks in advance of their cardiothoracic surgery. This is the screening protocol to understand if they have a bleeding disorder that would change their management in the operating room and after their surgery. For patients that are found to have a bleeding disorder requiring treatment, they would need post-operatively a blood test daily for up to 7 days total and another blood test weekly for 2 weeks and then once prior to discharge.
Study design
- Primary purpose
- Prevention
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Alagille syndrome | Experimental | Other: Assessment for bleeding disorder with tailored post-operative carePatients with complex cardiac conditions requiring cardiothoracic surgery who also have a history of Alagille syndrome. |
| No history of Alagille syndrome | Experimental | Other: Assessment for bleeding disorder with tailored post-operative carePatients with complex cardiac conditions requiring cardiothoracic surgery who do not have a diagnosis of Alagille syndrome. |
Who may qualify
- Ages
- Up to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- 0-17 years old
- with complex cardiac condition requiring pulmonary artery reconstruction (branch pulmonary artery stenosis, MAPCAs or Tetralogy of Fallot without MAPCAs)
Key exclusion criteria (2 listed)
- history of known bleeding disorder
- aged 18 years or older
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Assessment for bleeding disorder with tailored post-operative careIntervention will be expanded lab work-up for hematologic conditions, consultation by Hematology and tailored intra-operative and post-operative plan with aim to mitigate bleeding and hemorrhage risk, balancing thrombotic risk with this intervention. | Other | Alagille syndrome, No history of Alagille syndrome |
Primary outcome
Blood product volume
Time frame: Intra-operatively through 24 hours post-operatively
Chest tube output blood volume
Time frame: Intra-operatively through 24 hours post-operatively
Number of participants with pulmonary hemorrhage
Time frame: Intra-operatively through 24 hours post-operatively
1 secondary outcome
Incidence of thromboembolism events in patients with hematologic condition
Time frame: Intra-operatively through 30 days post-operatively
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 May 2023.
- 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.
Noelle Ebel, MD 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?
A study testing Assessment for bleeding disorder with tailored post-operative care in people with Alagille Syndrome and Pulmonary Artery Stenoses, 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 2 inclusion and 2 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: status not confirmed, as of the sponsor's last update on 6 May 2023. 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?
Results are not available in the source record.
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?
Blood product volume (time frame: Intra-operatively through 24 hours post-operatively). The study lists 3 primary outcomes in all. 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 6 May 2023.