Decreasing Hemorrhage Risk in Children With Alagille Syndrome

NCT05846854 | Unknown

• 1 other identifier: 66477
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Alagille Syndrome; Pulmonary Artery Stenoses; Acquired Von Willebrand Disease

Outcome Measures

Primary Outcomes (3)

• Blood product volume

  Intra-operatively through 24 hours post-operatively

• Chest tube output blood volume

  Intra-operatively through 24 hours post-operatively

• Number of participants with pulmonary hemorrhage

  Intra-operatively through 24 hours post-operatively

Secondary Outcomes (1)

• Incidence of thromboembolism events in patients with hematologic condition

  Intra-operatively through 30 days post-operatively

Study Arms (2)

Alagille syndrome

EXPERIMENTAL

Patients with complex cardiac conditions requiring cardiothoracic surgery who also have a history of Alagille syndrome.

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.

Other: Assessment for bleeding disorder with tailored post-operative care

Interventions

Assessment for bleeding disorder with tailored post-operative care OTHER

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.

Alagille syndrome; No history of Alagille syndrome

Eligibility Criteria

• Age: Up to 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• years old
• with complex cardiac condition requiring pulmonary artery reconstruction (branch pulmonary artery stenosis, MAPCAs or Tetralogy of Fallot without MAPCAs)

You may not qualify if:

• history of known bleeding disorder
• aged 18 years or older

Sponsors & Collaborators

• Stanford University: lead
• Alagille Syndrome Alliance: collaborator

Study Sites (1)

Stanford University

Palo Alto, California, 94304, United States

Location

MeSH Terms

Conditions

Alagille Syndrome; Stenosis, Pulmonary Artery

Interventions

Restraint, Physical

Condition Hierarchy (Ancestors)

Cholestasis, Intrahepatic; Cholestasis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Liver Diseases; Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Arterial Occlusive Diseases; Vascular Diseases

Intervention Hierarchy (Ancestors)

Behavior Control; Therapeutics; Immobilization; Investigative Techniques

Study Officials

• Noelle Ebel, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor, Division of Pediatric Gastroenterology, Hepatology and Nutrition

Study Record Dates

• First Submitted: April 26, 2023
• First Posted: May 6, 2023
• Study Start: April 18, 2023
• Primary Completion: November 1, 2024
• Study Completion: November 1, 2024
• Last Updated: May 6, 2023

Record last verified: 2023-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)