Pediatric Pulmonary Hypertension Network (PPHNet) Informatics Registry

NCT ID: NCT02249923

Last Updated: 2025-03-13

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 2500 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2014-10-31
• Study Completion Date: 2031-12-31

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")

Conditions

Pulmonary Vascular Disease; Pulmonary Arterial Hypertension

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Pulmonary Arterial Hypertension

No interventions assigned to this group

Eligibility Criteria

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

• Minimum Eligible Age: 1 Day
• Maximum Eligible Age: 21 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role: collaborator

Janssen Pharmaceuticals

INDUSTRY

Sponsor Role: collaborator

New York Medical College

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Steven H Abman, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Colorado

David D Ivy, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Colorado

Kenneth D Mandl, MD

Role: PRINCIPAL_INVESTIGATOR

Boston Children's Hospital, Harvard School of Medicine

Roberta Keller, MD

Role: PRINCIPAL_INVESTIGATOR

University California San Francisco

Rachel Hopper, MD

Role: PRINCIPAL_INVESTIGATOR

Stanford University

Angela Bates, MD

Role: PRINCIPAL_INVESTIGATOR

University of Alberta Edmonton

Catherine Avitabile, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital of Philadelphia

Mary Mullen, MD

Role: PRINCIPAL_INVESTIGATOR

Boston Children's Hospital

Eric Austin, MD

Role: PRINCIPAL_INVESTIGATOR

Vanderbilt University Medical Center

Marc Natter, MD

Role: PRINCIPAL_INVESTIGATOR

Boston Children's Hospital

Usha Krishnan, MD

Role: PRINCIPAL_INVESTIGATOR

Columbia University

Lynn A Sleeper, ScD

Role: PRINCIPAL_INVESTIGATOR

Boston Children's Hospital

Erika Rosenzweig, MD

Role: PRINCIPAL_INVESTIGATOR

Maria Fareri Children's Hospital at WMC Health/Westchester Medical Center

Jenny Schramm, MD

Role: PRINCIPAL_INVESTIGATOR

Johns Hopkins University

Lewis Romer, MD

Role: PRINCIPAL_INVESTIGATOR

Johns Hopkins University

Grace Freire, MD

Role: PRINCIPAL_INVESTIGATOR

Johns Hopkins All Children's Heart Institute

Stephanie Handler, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Wisconsin

Nidhy Varghese, MD

Role: PRINCIPAL_INVESTIGATOR

Baylor College of Medicine

Russel Hirsch, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital Medical Center, Cincinnati

Delphine Yung, MD

Role: PRINCIPAL_INVESTIGATOR

Seattle Children's Hospital

Locations

Stanford University Medical Center

Palo Alto, California, United States

Site Status: RECRUITING

University California San Francisco

San Francisco, California, United States

Site Status: RECRUITING

Children's Hospital Colorado

Aurora, Colorado, United States

Site Status: RECRUITING

Johns Hopkins All Children's Heart Institute

St. Petersburg, Florida, United States

Site Status: RECRUITING

Johns Hopkins Children's Center

Baltimore, Maryland, United States

Site Status: RECRUITING

Boston Children's Hospital

Boston, Massachusetts, United States

Site Status: RECRUITING

Columbia University Medical Center

New York, New York, United States

Site Status: RECRUITING

Maria Fareri Children's Hospital at WMC Health/Westchester Medical Center

New York, New York, United States

Site Status: NOT_YET_RECRUITING

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status: RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status: RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status: RECRUITING

Texas Children's

Houston, Texas, United States

Site Status: NOT_YET_RECRUITING

Seattle Children's Hospital

Seattle, Washington, United States

Site Status: RECRUITING

University of Alberta Edmonton

Edmonton, Alberta, Canada

Site Status: RECRUITING

Countries

United States; Canada

Central Contacts

Erika B Rosenzweig, MD

Role: CONTACT

Erika.BermanRosenzweig@wmchealth.org

914-493-6160

Robin Mascotti

Role: CONTACT

robin.mascotti@childrenscolorado.org

303-724-6513

Facility Contacts

Rachel Hopper, MD

Role: primary

rhopper@stanford.edu

Rachel Hopper, MD

Role: backup

Roberta Keller, MD

Role: primary

roberta.keller@ucsf.edu

Roberta Keller, MD

Role: backup

David D. Ivy, MD

Role: primary

Dunbar.Ivy@childrenscolorado.org

Robin Mascotti

Role: backup

Robin.mascotti@childrenscolorado.org

David D Ivy, MD

Role: backup

Grace Freire, MD

Role: primary

Gfreire1@jhmi.edu

Grace Freire, MD

Role: backup

Jenny Schramm, MD

Role: primary

schrammj@jhu.edu

Allen Everett, MD

Role: backup

Mary Mullen, MD

Role: primary

Mary.Mullen@cardio.chboston.org

Mary Mullen, MD

Role: backup

Usha Krishnan, MD

Role: primary

usk1@cumc.columbia.edu

Usha Krishnan, MD

Role: backup

Erika Rosenzweig, MD

Role: primary

Erika.bermanrosenzweig@wmchealth.org

Erika Rosenzweig

Role: backup

Russel Hirsch, MD

Role: primary

russel.hirsch@cchmc.org

Russel Hirsch, MD

Role: backup

Catherine Avitabile, MD

Role: primary

AvitabileC@email.CHOP.EDU

Catherine Avitable, MD

Role: backup

Eric Austin, MD

Role: primary

eric.austin@Vanderbilt.edu

Eric Austin, MD

Role: backup

Nidhy Varghese, MD

Role: primary

npvarghe@texaschildrens.org

Nidhy Varghese, MD

Role: backup

Delphine Yung, MD

Role: primary

delphine.yung@seattlechildrens.org

Delphine Yung, MD

Role: backup

Angela Bates, MD

Role: primary

angela.bates@albertahealthservices.ca

Angela Bates, MD

Role: backup

References

Geva A, Gronsbell JL, Cai T, Cai T, Murphy SN, Lyons JC, Heinz MM, Natter MD, Patibandla N, Bickel J, Mullen MP, Mandl KD; Pediatric Pulmonary Hypertension Network and National Heart, Lung, and Blood Institute Pediatric Pulmonary Vascular Disease Outcomes Bioinformatics Clinical Coordinating Center Investigators. A Computable Phenotype Improves Cohort Ascertainment in a Pediatric Pulmonary Hypertension Registry. J Pediatr. 2017 Sep;188:224-231.e5. doi: 10.1016/j.jpeds.2017.05.037. Epub 2017 Jun 16.

Reference Type: DERIVED

PMID: 28625502 (View on PubMed)

Related Links

http://pubmed.ncbi.nlm.nih.gov/34140292/

Related Info

http://pubmed.ncbi.nlm.nih.gov/35049414/

Related Info

Other Identifiers

U01HL121518

Identifier Type: NIH

Identifier Source: secondary_id

View Link

NOPRODPUH4017

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

14-0018

Identifier Type: -

Identifier Source: org_study_id