Pediatric Pulmonary Hypertension Network (PPHNet) Informatics Registry

Recruiting

• Conditions: Pulmonary Vascular Disease; Pulmonary Arterial Hypertension

• Registration Number: NCT02249923
• Lead Sponsor: University of Colorado, Denver

Brief 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.

Detailed 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.

Aim 4: Risk Stratification To validate the Pediatric Risk Score model using an independent patient cohort, obtained by enrichment of the PPHNet Registry with phenotypic data collection from a newly enrolled cohort of 500 patients (Collaborative substudy with Johnson \& Johnson- "Children Are Not Small Adults: Validation of the Pediatric Pulmonary Hypertension Risk Score")

Study Timeline

• Study First Submitted: 2014-09-10
• Study First Submitted QC: 2014-09-23
• Study First Posted: 2014-09-26
• Study Start: 2014-10
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-08
• Last Update Posted: 2025-01-10
• Primary Completion: 2030-12
• Study Completion: 2031-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 2500

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

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Exclusion Criteria

• Diagnosed with pulmonary hypertension after age 18
• Refusal to sign informed consent

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Time to clinical worsening	36 months	Time to clinical worsening for death, cardiac transplant, atrial septostomy, or Potts shunt.

Secondary Outcome Measures

Name	Time	Method
Escalation of Pulmonary Hypertension Therapy	36 months	The addition of patients baseline medication therapy, this can include going from mono therapy to dual therapy, or dual therapy to triple therapy
Right Heart Failure	36 months	Elevated Right atrial pressure greater than 10 by right heart catheterization

Trial Locations

Locations (14)

Stanford University Medical Center; 🇺🇸 Palo Alto, California, United States

Johns Hopkins All Children's Heart Institute; 🇺🇸 Saint Petersburg, Florida, United States

Johns Hopkins Children's Center; 🇺🇸 Baltimore, Maryland, United States

Maria Fareri Children's Hospital at WMC Health/Westchester Medical Center; 🇺🇸 New York, New York, United States

Cincinnati Children's Hospital Medical Center; 🇺🇸 Cincinnati, Ohio, United States

Texas Children's; 🇺🇸 Houston, Texas, United States

Seattle Children's Hospital; 🇺🇸 Seattle, Washington, United States

University California San Francisco; 🇺🇸 San Francisco, California, United States

Children's Hospital Colorado; 🇺🇸 Aurora, Colorado, United States

Boston Children's Hospital; 🇺🇸 Boston, Massachusetts, United States

Columbia University Medical Center; 🇺🇸 New York, New York, United States

Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

Vanderbilt University Medical Center; 🇺🇸 Nashville, Tennessee, United States

University of Alberta Edmonton; 🇨🇦 Edmonton, Alberta, Canada