Clinical trial NCT02249923: Pediatric Pulmonary Hypertension Network (PPHNet) Informatics Registry
Official title: Pediatric Pulmonary Hypertension Network (PPHNet) Informatics Registry
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Pulmonary Vascular Disease
Pulmonary Arterial Hypertension
Keywords: Pulmonary Arterial Hypertension (PAH)
Pulmonary Vascular Disease (PVD)
Registry
Pediatric
Lead sponsor: New York Medical College
Sponsor class: OTHER
Collaborators: name: National Heart, Lung, and Blood Institute (NHLBI)
name: Janssen Pharmaceuticals
First posted: 2014-09-26
Enrollment: 2500
Sex: ALL
Minimum age (years): 1
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: 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, HIV, and use of anorexigens.

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")

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

Primary outcomes:
measure: Time to clinical worsening
timeFrame: 12 months
Secondary outcomes:
measure: Escalation of Pulmonary Hypertension Therapy
timeFrame: 36 months
measure: Right Heart Failure
timeFrame: 36 months
Other outcomes:
Locations:
city: Palo Alto
country: United States
facility: Stanford University Medical Center
state: California
city: San Francisco
country: United States
facility: University California San Francisco
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Saint Petersburg
country: United States
facility: Johns Hopkins All Children's Heart Institute
state: Florida
city: Baltimore
country: United States
facility: Johns Hopkins Children's Center
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
city: New York
country: United States
facility: Maria Fareri Children's Hospital at WMC Health/Westchester Medical Center
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Philadelphia
country: United States
facility: Children's Hospital of Philadelphia
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Houston
country: United States
facility: Texas Children's
state: Texas
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Edmonton
country: Canada
facility: University of Alberta Edmonton
state: Alberta
Study officials:
affiliation: Children's Hospital Colorado
name: Steven H Abman, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Children's Hospital Colorado
name: David D Ivy, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Boston Children's Hospital, Harvard School of Medicine
name: Kenneth D Mandl, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University California San Francisco
name: Roberta Keller, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Stanford University
name: Rachel Hopper, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: University of Alberta Edmonton
name: Angela Bates, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Children's Hospital of Philadelphia
name: Catherine Avitabile, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Boston Children's Hospital
name: Mary Mullen, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Vanderbilt University Medical Center
name: Eric Austin, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Boston Children's Hospital
name: Marc Natter, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Columbia University
name: Usha Krishnan, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Boston Children's Hospital
name: Lynn A Sleeper, ScD
role: PRINCIPAL_INVESTIGATOR
affiliation: Maria Fareri Children's Hospital at WMC Health/Westchester Medical Center
name: Erika Rosenzweig, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Johns Hopkins University
name: Jenny Schramm, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Johns Hopkins University
name: Lewis Romer, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Johns Hopkins All Children's Heart Institute
name: Grace Freire, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Children's Hospital Wisconsin
name: Stephanie Handler, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Baylor College of Medicine
name: Nidhy Varghese, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Children's Hospital Medical Center, Cincinnati
name: Russel Hirsch, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Seattle Children's Hospital
name: Delphine Yung, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.