Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension

Hypertension, Pulmonary Clinical Trial

— TOPP-2  

Official title:

Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02610660
• Other study ID #: TOPP-2
• Status: Completed
• Start date: August 2015
• Est. completion date: December 31, 2022

Study information

• Verified date: April 2023
• Source: Association for Pediatric Pulmonary Hypertension
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 388
• Est. completion date: December 31, 2022
• Est. primary completion date: December 31, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Months to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Hypertension
• Hypertension, Pulmonary

Locations

Country	Name	City	State
Australia	Royal Children's Hospital	Melbourne
Brazil	University of São Paulo	São Paulo
Canada	The Hospital for Sick Children	Toronto	Ontario
China	Fu Wai Hospital	Beijing
Colombia	Hospital Pediátrico La Misericordia	Bogota
France	Université Paris Descartes	Paris
Germany	University Children's Hospital Ulm	Ulm
Hungary	Institute of Cardiology	Budapest
Israel	Hadassah, Hebrew University Medical Center	Jerusalem
Italy	University of Padova	Padova
Japan	Keio University	Tokyo
Japan	Toho Universit Omori Medical Center	Tokyo
Mexico	National Heart Institute	Mexico City
Netherlands	Beatrix Children's Hospital, University Medical Center Groningen	Groningen
Poland	Children's Memorial	Warsaw
Saudi Arabia	King Fahd Armed Forces Hospital	Jeddah
Sweden	The Queen Silvia's Children's Hospital	Gothenburg
Switzerland	Hôpital des Enfants	Geneva
Switzerland	University Children's Hospital	Zurich
United States	Emory University	Atlanta	Georgia
United States	University of Colorado Denver School of Medicine, Children's Hospital Colorado	Aurora	Colorado
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Texas Children's Hospital	Houston	Texas
United States	Vanderbilt University Children's Hospital	Nashville	Tennessee
United States	Columbia University Medical Center	New York	New York
United States	University of Nebraska Medical Center	Omaha	Nebraska
United States	Stanford Children's Hospital	Palo Alto	California
United States	St Louis Children's Hospital	Saint Louis	Missouri
United States	Primary Children's Hospital	Salt Lake City	Utah
United States	Seattle Children's Hospital	Seattle	Washington
United States	Children's National Medical Center	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Association for Pediatric Pulmonary Hypertension

Countries where clinical trial is conducted

United States,  Australia,  Brazil,  Canada,  China,  Colombia,  France,  Germany,  Hungary,  Israel,  Italy,  Japan,  Mexico,  Netherlands,  Poland,  Saudi Arabia,  Sweden,  Switzerland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Death		Over registry run-time (5.5 years)
Primary	Transplantation		Over registry run-time (5.5 years)
Primary	Adverse events		Over registry run-time (5.5 years)
Secondary	Hospitalisation related to pulmonary arterial hypertension (PAH)		Over registry run-time (5.5 years)
Secondary	Use/initiation of i.v./s.c. prostanoids		Over registry run-time (5.5 years)
Secondary	Atrial septostomy		Over registry run-time (5.5 years)
Secondary	Potts shunt		Over registry run-time (5.5 years)
Secondary	Time to clinical worsening	Various composites of above parameters	Over registry run-time (5.5 years)
Secondary	Decline in 6-minute walk test (6MWT)		Over registry run-time (5.5 years)
Secondary	Type of treatment	Type of treatment (drug, mono/combination, administration route)	Over registry run-time (5.5 years)
Secondary	Switch in treatment		Over registry run-time (5.5 years)
Secondary	Escalation of treatment		Over registry run-time (5.5 years)
Secondary	Reasons for treatment change		Over registry run-time (5.5 years)
Secondary	Decline in WHO functional class		Over registry run-time (5.5 years)
Secondary	Decline in Panama functional class		Over registry run-time (5.5 years)
Secondary	Worsening of echocardiographic parameters (ECHO)	Parameters encompass TAPSE and RV/LV dimension ratio	Over registry run-time (5.5 years)
Secondary	Increase in Brain Natriuretic Peptide (BNP)		Over registry run-time (5.5 years)
Secondary	Increase in N-terminal-proBNP (NT-proBNP)		Over registry run-time (5.5 years)

External Links

•   Webpage of the Association for Pediatric Pulmonary Hypertension