Pulmonary Vascular DiseasePPHNetNCT02249923
Pediatric Pulmonary Hypertension Network (PPHNet) Informatics Registry
An observational study of Pulmonary Vascular Disease and Pulmonary Arterial Hypertension, run by New York Medical College at 14 sites in 2 countries.
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. Read more about this study
- Who is it for?
- Ages 1 day to 21 years
- Conditions the study is for
- Pulmonary Vascular Disease, Pulmonary Arterial Hypertension
- How many people
- 2,500 participants
- Where
- 14 sitesin 2 countries
- Study dates
- Oct 2014to Dec 2031
- How long will I take part?
- 5 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 day to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
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.
Who may be able to join
- 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
Who cannot join
- Diagnosed with pulmonary hypertension after age 18
- Refusal to sign informed consent
Trial locations and contacts
14 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
14 sites in 2 countries
United States13 sites, 13 recruiting
- Recruiting
Children's Hospital Colorado
Aurora, Colorado, 80045
David D. Ivy, MD, Dunbar.Ivy@childrenscolorado.org
Robin Mascotti, Robin.mascotti@childrenscolorado.org
- Recruiting
Johns Hopkins Children's Center
Baltimore, Maryland
Jenny Schramm, MD, schrammj@jhu.edu
Allen Everett, MD
- Recruiting
Boston Children's Hospital
Boston, Massachusetts
Mary Mullen, MD, Mary.Mullen@cardio.chboston.org
Mary Mullen, MD
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio
Russel Hirsch, MD, russel.hirsch@cchmc.org
Russel Hirsch, MD
- Not yet recruiting
Texas Children's
Houston, Texas
Nidhy Varghese, MD, npvarghe@texaschildrens.org
Nidhy Varghese, MD
- Recruiting
Vanderbilt University Medical Center
Nashville, Tennessee
Eric Austin, MD, eric.austin@Vanderbilt.edu
Eric Austin, MD
- Recruiting
Columbia University Medical Center
New York, New York
Usha Krishnan, MD, usk1@cumc.columbia.edu
Usha Krishnan, MD
- Not yet recruiting
Maria Fareri Children's Hospital at WMC Health/Westchester Medical Center
New York, New York
Erika Rosenzweig, MD, Erika.bermanrosenzweig@wmchealth.org
Erika Rosenzweig
- Recruiting
Stanford University Medical Center
Palo Alto, California
Rachel Hopper, MD, rhopper@stanford.edu
Rachel Hopper, MD
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania
Catherine Avitabile, MD, AvitabileC@email.CHOP.EDU
Catherine Avitable, MD
- Recruiting
University California San Francisco
San Francisco, California
Roberta Keller, MD, roberta.keller@ucsf.edu
Roberta Keller, MD
- Recruiting
Seattle Children's Hospital
Seattle, Washington
Delphine Yung, MD, delphine.yung@seattlechildrens.org
Delphine Yung, MD
- Recruiting
Johns Hopkins All Children's Heart Institute
St. Petersburg, Florida
Grace Freire, MD, Gfreire1@jhmi.edu
Grace Freire, MD
Canada1 site, 1 recruiting
- Recruiting
University of Alberta Edmonton
Edmonton, Alberta
Angela Bates, MD, angela.bates@albertahealthservices.ca
Angela Bates, MD
About this study
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.
Read the 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
- To provide a mechanism to store information about newborns, infants and children with PH;
- To determine the incidence and natural history of the various etiologies of pediatric PH;
- To define the investigator current diagnostic and therapeutic approaches to the diverse conditions associated with pediatric PH;
- 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")
- Keywords
- Pulmonary Arterial Hypertension (PAH), Pulmonary Vascular Disease (PVD), Registry, Pediatric
- Medical subject headings
- Pulmonary Arterial Hypertension
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Pulmonary Arterial Hypertension | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time to clinical worsening
Time to clinical worsening for death, cardiac transplant, atrial septostomy, or Potts shunt.
Measured: 12 months
Secondary outcomes (2)
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
Measured: 36 months
Right Heart Failure
Elevated Right atrial pressure greater than 10 by right heart catheterization
Measured: 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Sep 2014 (estimated)
- Study startOct 2014
- Primary completionDec 2030 (estimated)
- Study completionDec 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Pulmonary Vascular DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 2,500 participants (estimated)
- Target duration
- 5 Years
Sponsor and investigators
- Lead sponsor
- New York Medical College
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI), Janssen Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Steven H Abman, MD, Principal investigator, Children's Hospital ColoradoDavid D Ivy, MD, Principal investigator, Children's Hospital ColoradoKenneth D Mandl, MD, Principal investigator, Boston Children's Hospital, Harvard School of MedicineRoberta Keller, MD, Principal investigator, University California San FranciscoRachel Hopper, MD, Principal investigator, Stanford UniversityAngela Bates, MD, Principal investigator, University of Alberta EdmontonCatherine Avitabile, MD, Principal investigator, Children's Hospital of PhiladelphiaMary Mullen, MD, Principal investigator, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02249923
- Sponsor's protocol ID
- 14-0018
- Other IDs
- U01HL121518, NOPRODPUH4017
- Acronym
- PPHNet
- Status last verified
- Feb 2025
- First submitted
- 10 Sep 2014
- Last updated
- 13 Mar 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 28625502
Study at a glance
From the registry record, last updated by the sponsor on 13 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pediatric Pulmonary Hypertension Network (PPHNet) Informatics Registry
- ClinicalTrials.gov ID
- NCT02249923
- Acronym / sponsor ID
- PPHNet / 14-0018
- Keywords
- Pulmonary Arterial Hypertension (PAH), Pulmonary Vascular Disease (PVD), Registry, Pediatric
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 2,500 participants (estimated)
- Lead sponsor
- New York Medical CollegeUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI), Janssen Pharmaceuticals
- Study start
- Oct 2014
- Primary completion
- Dec 2030 (estimated)
- Study completion
- Dec 2031 (estimated)
- First posted
- 26 Sep 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
- To provide a mechanism to store information about newborns, infants and children with PH;
- To determine the incidence and natural history of the various etiologies of pediatric PH;
- To define the investigator current diagnostic and therapeutic approaches to the diverse conditions associated with pediatric PH;
- 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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 5 Years
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pulmonary Arterial Hypertension | – | – |
Who may qualify
- Ages
- 1 day to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- 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.
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (2 listed)
- Diagnosed with pulmonary hypertension after age 18
- Refusal to sign informed consent
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Time to clinical worsening
Time frame: 12 months
Time to clinical worsening for death, cardiac transplant, atrial septostomy, or Potts shunt.
2 secondary outcomes
Escalation of Pulmonary Hypertension Therapy
Time frame: 36 months
Right Heart Failure
Time frame: 36 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Mar 2025.
- Derived from this study: PubMed 28625502Geva 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.
- Related InfoLink provided by the sponsor.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Steven H Abman, MD principal investigator
Children's Hospital Colorado
David D Ivy, MD principal investigator
Children's Hospital Colorado
Kenneth D Mandl, MD principal investigator
Boston Children's Hospital, Harvard School of Medicine
14 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Pulmonary Vascular Disease and Pulmonary Arterial Hypertension, run by New York Medical College at 14 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 13 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Erika B Rosenzweig, MD (914-493-6160, Erika.BermanRosenzweig@wmchealth.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Time to clinical worsening (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02249923, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Mar 2025.