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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02610660




Registration number
NCT02610660
Ethics application status
Date submitted
10/11/2015
Date registered
20/11/2015
Date last updated
13/04/2023

Titles & IDs
Public title
Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension
Scientific title
Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension
Secondary ID [1] 0 0
TOPP-2
Universal Trial Number (UTN)
Trial acronym
TOPP-2
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hypertension, Pulmonary 0 0
Condition category
Condition code
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Cardiovascular 0 0 0 0
Hypertension

Intervention/exposure
Study type
Observational [Patient Registry]
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Death
Timepoint [1] 0 0
Over registry run-time (5.5 years)
Primary outcome [2] 0 0
Transplantation
Timepoint [2] 0 0
Over registry run-time (5.5 years)
Primary outcome [3] 0 0
Adverse events
Timepoint [3] 0 0
Over registry run-time (5.5 years)
Secondary outcome [1] 0 0
Hospitalisation related to pulmonary arterial hypertension (PAH)
Timepoint [1] 0 0
Over registry run-time (5.5 years)
Secondary outcome [2] 0 0
Use/initiation of i.v./s.c. prostanoids
Timepoint [2] 0 0
Over registry run-time (5.5 years)
Secondary outcome [3] 0 0
Atrial septostomy
Timepoint [3] 0 0
Over registry run-time (5.5 years)
Secondary outcome [4] 0 0
Potts shunt
Timepoint [4] 0 0
Over registry run-time (5.5 years)
Secondary outcome [5] 0 0
Time to clinical worsening
Timepoint [5] 0 0
Over registry run-time (5.5 years)
Secondary outcome [6] 0 0
Decline in 6-minute walk test (6MWT)
Timepoint [6] 0 0
Over registry run-time (5.5 years)
Secondary outcome [7] 0 0
Type of treatment
Timepoint [7] 0 0
Over registry run-time (5.5 years)
Secondary outcome [8] 0 0
Switch in treatment
Timepoint [8] 0 0
Over registry run-time (5.5 years)
Secondary outcome [9] 0 0
Escalation of treatment
Timepoint [9] 0 0
Over registry run-time (5.5 years)
Secondary outcome [10] 0 0
Reasons for treatment change
Timepoint [10] 0 0
Over registry run-time (5.5 years)
Secondary outcome [11] 0 0
Decline in WHO functional class
Timepoint [11] 0 0
Over registry run-time (5.5 years)
Secondary outcome [12] 0 0
Decline in Panama functional class
Timepoint [12] 0 0
Over registry run-time (5.5 years)
Secondary outcome [13] 0 0
Worsening of echocardiographic parameters (ECHO)
Timepoint [13] 0 0
Over registry run-time (5.5 years)
Secondary outcome [14] 0 0
Increase in Brain Natriuretic Peptide (BNP)
Timepoint [14] 0 0
Over registry run-time (5.5 years)
Secondary outcome [15] 0 0
Increase in N-terminal-proBNP (NT-proBNP)
Timepoint [15] 0 0
Over registry run-time (5.5 years)

Eligibility
Key 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.
Minimum age
3 Months
Maximum age
18 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Patients belonging to Group 2 according to updated Nice clinical classification

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/08/2015
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
31/12/2022
Sample size
Target
Accrual to date
Final
388
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Children's Hospital - Melbourne
Recruitment postcode(s) [1] 0 0
3052 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
District of Columbia
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
Country [6] 0 0
United States of America
State/province [6] 0 0
Missouri
Country [7] 0 0
United States of America
State/province [7] 0 0
Nebraska
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
Ohio
Country [10] 0 0
United States of America
State/province [10] 0 0
Tennessee
Country [11] 0 0
United States of America
State/province [11] 0 0
Texas
Country [12] 0 0
United States of America
State/province [12] 0 0
Utah
Country [13] 0 0
United States of America
State/province [13] 0 0
Washington
Country [14] 0 0
Brazil
State/province [14] 0 0
São Paulo
Country [15] 0 0
Canada
State/province [15] 0 0
Ontario
Country [16] 0 0
China
State/province [16] 0 0
Beijing
Country [17] 0 0
Colombia
State/province [17] 0 0
Bogota
Country [18] 0 0
France
State/province [18] 0 0
Paris
Country [19] 0 0
Germany
State/province [19] 0 0
Ulm
Country [20] 0 0
Hungary
State/province [20] 0 0
Budapest
Country [21] 0 0
Israel
State/province [21] 0 0
Jerusalem
Country [22] 0 0
Italy
State/province [22] 0 0
Padova
Country [23] 0 0
Japan
State/province [23] 0 0
Tokyo
Country [24] 0 0
Mexico
State/province [24] 0 0
Mexico City
Country [25] 0 0
Netherlands
State/province [25] 0 0
Groningen
Country [26] 0 0
Poland
State/province [26] 0 0
Warsaw
Country [27] 0 0
Saudi Arabia
State/province [27] 0 0
Jeddah
Country [28] 0 0
Sweden
State/province [28] 0 0
Gothenburg
Country [29] 0 0
Switzerland
State/province [29] 0 0
Geneva
Country [30] 0 0
Switzerland
State/province [30] 0 0
Zurich

Funding & Sponsors
Primary sponsor type
Other
Name
Association for Pediatric Pulmonary Hypertension
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT02610660
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Dunbar Ivy, Prof
Address 0 0
Association for Pediatric Pulmonary Hypertension
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT02610660