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OpenTrials
Completed

NCT Number: NCT02610660

Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension

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.

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Key information

Conditions

Hypertension, Pulmonary Cardiovascular Diseases Hypertension Lung Diseases Respiratory Tract Diseases Vascular Diseases

Age range

3 month–18 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Royal Children's Hospital, Melbourne, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Primary outcomes

  1. Death

    Time frame: Over registry run-time (5.5 years)

  2. Transplantation

    Time frame: Over registry run-time (5.5 years)

  3. Adverse events

    Time frame: Over registry run-time (5.5 years)

Secondary outcomes

  1. Hospitalisation related to pulmonary arterial hypertension (PAH)

    Time frame: Over registry run-time (5.5 years)

  2. Use/initiation of i.v./s.c. prostanoids

    Time frame: Over registry run-time (5.5 years)

  3. Atrial septostomy

    Time frame: Over registry run-time (5.5 years)

  4. Potts shunt

    Time frame: Over registry run-time (5.5 years)

  5. Time to clinical worsening

    Time frame: Over registry run-time (5.5 years)

    Various composites of above parameters

  6. Decline in 6-minute walk test (6MWT)

    Time frame: Over registry run-time (5.5 years)

  7. Type of treatment

    Time frame: Over registry run-time (5.5 years)

    Type of treatment (drug, mono/combination, administration route)

  8. Switch in treatment

    Time frame: Over registry run-time (5.5 years)

  9. Escalation of treatment

    Time frame: Over registry run-time (5.5 years)

  10. Reasons for treatment change

    Time frame: Over registry run-time (5.5 years)

  11. Decline in WHO functional class

    Time frame: Over registry run-time (5.5 years)

  12. Decline in Panama functional class

    Time frame: Over registry run-time (5.5 years)

  13. Worsening of echocardiographic parameters (ECHO)

    Time frame: Over registry run-time (5.5 years)

    Parameters encompass TAPSE and RV/LV dimension ratio

  14. Increase in Brain Natriuretic Peptide (BNP)

    Time frame: Over registry run-time (5.5 years)

  15. Increase in N-terminal-proBNP (NT-proBNP)

    Time frame: Over registry run-time (5.5 years)

Sponsors and collaborators

Lead sponsor

Association for Pediatric Pulmonary Hypertension

Other

Registry information

Acronym: TOPP-2

Important dates

Study start
2015
Primary completion
2022
Study completion
2022
First posted
Nov 20, 2015
Registry last updated
Apr 13, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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