Clinical trial NCT02610660: Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension
Official title: Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hypertension, Pulmonary
Lead sponsor: Association for Pediatric Pulmonary Hypertension
Sponsor class: OTHER
First posted: 2015-11-20
Enrollment: 388
Sex: ALL
Minimum age (years): 3
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: 1. Children and adolescents, newly diagnosed with PH (incident PePH patients), meeting the inclusion criteria. A patient is considered an incident patient if the time elapsed between the diagnostic RHC and the initial visit at the site including the patient is less than or equal to three months and if the time between diagnostic RHC and informed consent is less than or equal to six months
2. Ex-incident patients from the previous TOPP-1 registry.

Brief summary: The TOPP-2 registry is an international, non-interventional, prospective registry including children and adolescents newly diagnosed with pulmonary hypertension (PH) to gain further insights in the disease course and long-term outcome of PH in childhood.

Patients will undergo clinical assessments and receive standard medical care, as determined by treating physicians in their daily clinical practice. The TOPP-2 registry is specifically designed to capture the variables that have been proposed as treatment goals in PePH and the reasons for changes in treatment strategy.

The TOPP-2 registry uses the new clinical classification of PH as outlined at the 5th World Symposium for Pulmonary Hypertension (WSPH) in Nice 2013 and includes new characterizations for children with PH.

The registry is planned and implemented under the scientific leadership of the Association for Pediatric Pulmonary Hypertension (PePH), independently from the financial sponsors.

All enrolled patients will have a follow-up period of 18 months.


Eligibility criteria: Inclusion Criteria:

* Patient must be an incident patient, i.e.newly diagnosed with PH
* Age at time of diagnosis is at least 3 months and less than 18 years
* Patients must present with PH belonging to one of the following categories

  * Group 1 according to updated Nice clinical classification
  * Group 3 according to updated Nice clinical classification
  * Group 4 according to updated Nice clinical classification
  * Group 5 according to updated Nice clinical classification
* PH confirmed by heart catheterisation (HC)
* At HC, the patient must present with mean pulmonary arterial pressure (PAP) of at least 25 mmHg at rest, a pulmonary vascular resistance index (PVRi) equal to or below 3 Wood units\*m\^2 and mean pulmonary arterial wedge pressure (PAWP) below or equal to 15 mmHg
* In case of congenital heart disease (CHD) patients who had undergone palliative procedure or repair to close systemic to pulmonary shunt, the diagnosis of PH must have been confirmed by HC at least 6 months after surgery/palliative procedure took place
* For patients with PAH-CHD open shunt, only those considered not operable due to advanced pulmonary vascular disease are eligible
* Patients to be included into the registry, and/or their legal guardians, must give informed consent. Where applicable patients will be asked for their written assent
* Participating sites must agree to adhere to the recommendations of the WSPH 2015 Nice, Pediatric Taskforce.

Exclusion Criteria:

* Patients belonging to Group 2 according to updated Nice clinical classification

Primary outcomes:
measure: Death
timeFrame: Over registry run-time (5.5 years)
measure: Transplantation
timeFrame: Over registry run-time (5.5 years)
measure: Adverse events
timeFrame: Over registry run-time (5.5 years)
Secondary outcomes:
measure: Hospitalisation related to pulmonary arterial hypertension (PAH)
timeFrame: Over registry run-time (5.5 years)
measure: Use/initiation of i.v./s.c. prostanoids
timeFrame: Over registry run-time (5.5 years)
measure: Atrial septostomy
timeFrame: Over registry run-time (5.5 years)
measure: Potts shunt
timeFrame: Over registry run-time (5.5 years)
measure: Time to clinical worsening
timeFrame: Over registry run-time (5.5 years)
measure: Decline in 6-minute walk test (6MWT)
timeFrame: Over registry run-time (5.5 years)
measure: Type of treatment
timeFrame: Over registry run-time (5.5 years)
measure: Switch in treatment
timeFrame: Over registry run-time (5.5 years)
measure: Escalation of treatment
timeFrame: Over registry run-time (5.5 years)
measure: Reasons for treatment change
timeFrame: Over registry run-time (5.5 years)
measure: Decline in WHO functional class
timeFrame: Over registry run-time (5.5 years)
measure: Decline in Panama functional class
timeFrame: Over registry run-time (5.5 years)
measure: Worsening of echocardiographic parameters (ECHO)
timeFrame: Over registry run-time (5.5 years)
measure: Increase in Brain Natriuretic Peptide (BNP)
timeFrame: Over registry run-time (5.5 years)
measure: Increase in N-terminal-proBNP (NT-proBNP)
timeFrame: Over registry run-time (5.5 years)
Other outcomes:
Locations:
city: Palo Alto
country: United States
facility: Stanford Children's Hospital
state: California
city: Aurora
country: United States
facility: University of Colorado Denver School of Medicine, Children's Hospital Colorado
state: Colorado
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Atlanta
country: United States
facility: Emory University
state: Georgia
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Saint Louis
country: United States
facility: St Louis Children's Hospital
state: Missouri
city: Omaha
country: United States
facility: University of Nebraska Medical Center
state: Nebraska
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Nashville
country: United States
facility: Vanderbilt University Children's Hospital
state: Tennessee
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Salt Lake City
country: United States
facility: Primary Children's Hospital
state: Utah
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Melbourne
country: Australia
facility: Royal Children's Hospital
city: São Paulo
country: Brazil
facility: University of São Paulo
city: Toronto
country: Canada
facility: The Hospital for Sick Children
state: Ontario
city: Beijing
country: China
facility: Fu Wai Hospital
city: Bogota
country: Colombia
facility: Hospital Pediátrico La Misericordia
city: Paris
country: France
facility: Université Paris Descartes
city: Ulm
country: Germany
facility: University Children's Hospital Ulm
city: Budapest
country: Hungary
facility: Institute of Cardiology
city: Jerusalem
country: Israel
facility: Hadassah, Hebrew University Medical Center
city: Padova
country: Italy
facility: University of Padova
city: Tokyo
country: Japan
facility: Toho Universit Omori Medical Center
city: Tokyo
country: Japan
facility: Keio University
city: Mexico City
country: Mexico
facility: National Heart Institute
city: Groningen
country: Netherlands
facility: Beatrix Children's Hospital, University Medical Center Groningen
city: Warsaw
country: Poland
facility: Children's Memorial
city: Jeddah
country: Saudi Arabia
facility: King Fahd Armed Forces Hospital
city: Gothenburg
country: Sweden
facility: The Queen Silvia's Children's Hospital
city: Geneva
country: Switzerland
facility: Hôpital des Enfants
city: Zurich
country: Switzerland
facility: University Children's Hospital
Study officials:
affiliation: Association for Pediatric Pulmonary Hypertension
name: Dunbar Ivy, Prof
role: STUDY_CHAIR
References: