Pediatric Pulmonary Hypertension Network (PPHNet) Informatics Registry

Pulmonary Arterial Hypertension Clinical Trial

— PPHNet  

Official title:

Pediatric Pulmonary Hypertension Network (PPHNet) Informatics Registry

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02249923
• Other study ID #: 14-0018
• Secondary ID: U01HL121518
• Status: Recruiting
• Start date: October 2014
• Est. completion date: December 2031

Study information

• Verified date: August 2022
• Source: University of Colorado, Denver
• Contact: Steven Abman, MD
• Phone: 303-881-9765
• Email: steven.abman[@]uanschutz.org
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

Patients are being asked to be in this research study because medical researchers hope that by gathering information about a large number of children with pulmonary hypertension over time, their understanding of the disease process will increase and lead to better treatment. Investigators believe that pulmonary hypertension in children is different than pulmonary hypertension in adults and this study will help us understand those differences.

Description:

Pulmonary Hypertension (PH) is a syndrome characterized by vasoconstriction and abnormal growth and function of endothelial and smooth muscle cells and other components within the pulmonary vessels, which leads to elevation of the pulmonary artery pressure. PH may be idiopathic (primary) without any known cause. Some cases of PH are familial. PH may also be secondary to a specific disease process such as portal hypertension, congenital heart disease, chronic lung disease, thromboembolic disease, connective tissue disease, human immunodeficiency virus (HIV), and use of anorexigens. Left untreated, PH is often progressive and fatal. There is no cure for PH. Therapy focuses upon treatment of secondary causes if present, and reduction of the pulmonary artery pressure through medical therapy. There have been many new developments within the past few years in the management of patients with PH. While there is no cure for PH early detection and treatment are important for survival of patients. Limited data is available that describes the etiologies, clinical course and prognosis of pediatric pulmonary hypertension.

Objectives

Aim 1: Clinical Research

1. To provide a mechanism to store information about newborns, infants and children with PH;

2. To determine the incidence and natural history of the various etiologies of pediatric PH;

3. To define the investigator current diagnostic and therapeutic approaches to the diverse conditions associated with pediatric PH;

4. To determine the response of children with PH to chronic therapies.

Aim 2: Research Infrastructure To create a robust scalable data architecture, to combine traditional registry data, electronic Health Record (EHR), and PRO (Patient Reported Outcome) data in a single resource.

Aim 3: Informatics Address three classes of unanswered questions crucial for the characterization and management of PH, comparing the information value of registry vs. EHR vs. fused data across registry/EHR/PROs, in the domains of spectrum of PH comorbidities, PH indicators and endpoints of morbidity and mortality, and response to therapies in PH.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 1000
• Est. completion date: December 2031
• Est. primary completion date: December 2030
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Day to 21 Years

Eligibility

Inclusion Criteria:

• The subject's age of onset of pulmonary hypertension must be prior to age 18 years

• The person providing consent must be able to read either Spanish or English.

• The subject (and/or parent/legal guardian) must be able to provide informed consent

Exclusion Criteria:

• Diagnosed with pulmonary hypertension after age 18

• Refusal to sign informed consent

Related Conditions & MeSH terms

• Hypertension
• Hypertension, Pulmonary
• Pulmonary Arterial Hypertension
• Pulmonary Vascular Disease
• Vascular Diseases

Locations

Country	Name	City	State
Canada	University of Alberta Edmonton	Edmonton	Alberta
United States	Children's Hospital Colorado	Aurora	Colorado
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Vanderbilt University Medical Center	Nashville	Tennessee
United States	Columbia University Medical Center	New York	New York
United States	Stanford University Medical center	Palo Alto	California
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	University California San Francisco	San Francisco	California

Sponsors (3)

Lead Sponsor	Collaborator
University of Colorado, Denver	Children's Hospital Colorado, National Heart, Lung, and Blood Institute (NHLBI)

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Time to clinical worsening	Death	36 months
Secondary	Escalation of Pulmonary Hypertension Therapy	The addition of patients baseline medication therapy, this can include going from mono therapy to dual therapy, or dual therapy to triple therapy	36 months
Secondary	Right Heart Failure	Elevated Right atrial pressure greater than 10 by right heart catheterization	36 months