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Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension (TOPP-2)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02610660
Recruitment Status : Recruiting
First Posted : November 20, 2015
Last Update Posted : September 6, 2019
Sponsor:
Information provided by (Responsible Party):
Association for Pediatric Pulmonary Hypertension

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.

At least 200 of the enrolled patients will have a follow-up period of 3 years.


Condition or disease
Hypertension, Pulmonary

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Study Type : Observational [Patient Registry]
Estimated Enrollment : 440 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 1 Day
Official Title: Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension
Study Start Date : August 2015
Estimated Primary Completion Date : December 2021
Estimated Study Completion Date : December 2021

Resource links provided by the National Library of Medicine





Primary Outcome Measures :
  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 Outcome Measures :
  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) ]


Information from the National Library of Medicine

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Ages Eligible for Study:   3 Months to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02610660


Contacts
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Contact: Fabrizio Canonaco, Dr fabrizio.canonaco@peph-association.org
Contact: Simone Lerch, Dr simone.lerch@peph-association.org

Locations
Show Show 32 study locations
Sponsors and Collaborators
Association for Pediatric Pulmonary Hypertension
Investigators
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Study Chair: Dunbar Ivy, Prof Association for Pediatric Pulmonary Hypertension

Additional Information:
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Responsible Party: Association for Pediatric Pulmonary Hypertension
ClinicalTrials.gov Identifier: NCT02610660    
Other Study ID Numbers: TOPP-2
First Posted: November 20, 2015    Key Record Dates
Last Update Posted: September 6, 2019
Last Verified: September 2019
Additional relevant MeSH terms:
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Hypertension, Pulmonary
Hypertension
Vascular Diseases
Cardiovascular Diseases
Lung Diseases
Respiratory Tract Diseases