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

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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

Tracking Information
First Submitted Date November 10, 2015
First Posted Date November 20, 2015
Last Update Posted Date September 6, 2019
Study Start Date August 2015
Estimated Primary Completion Date December 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: July 27, 2018)
  • Death [ Time Frame: Over registry run-time (5.5 years) ]
  • Transplantation [ Time Frame: Over registry run-time (5.5 years) ]
  • Adverse events [ Time Frame: Over registry run-time (5.5 years) ]
Original Primary Outcome Measures
 (submitted: November 19, 2015)
  • Death [ Time Frame: Min. 3 years ]
  • Transplantation [ Time Frame: Min. 3 years ]
  • Adverse events [ Time Frame: Min. 3 years ]
Change History
Current Secondary Outcome Measures
 (submitted: July 27, 2018)
  • Hospitalisation related to pulmonary arterial hypertension (PAH) [ Time Frame: Over registry run-time (5.5 years) ]
  • Use/initiation of i.v./s.c. prostanoids [ Time Frame: Over registry run-time (5.5 years) ]
  • Atrial septostomy [ Time Frame: Over registry run-time (5.5 years) ]
  • Potts shunt [ Time Frame: Over registry run-time (5.5 years) ]
  • Time to clinical worsening [ Time Frame: Over registry run-time (5.5 years) ]
    Various composites of above parameters
  • Decline in 6-minute walk test (6MWT) [ Time Frame: Over registry run-time (5.5 years) ]
  • Type of treatment [ Time Frame: Over registry run-time (5.5 years) ]
    Type of treatment (drug, mono/combination, administration route)
  • Switch in treatment [ Time Frame: Over registry run-time (5.5 years) ]
  • Escalation of treatment [ Time Frame: Over registry run-time (5.5 years) ]
  • Reasons for treatment change [ Time Frame: Over registry run-time (5.5 years) ]
  • Decline in WHO functional class [ Time Frame: Over registry run-time (5.5 years) ]
  • Decline in Panama functional class [ Time Frame: Over registry run-time (5.5 years) ]
  • Worsening of echocardiographic parameters (ECHO) [ Time Frame: Over registry run-time (5.5 years) ]
    Parameters encompass TAPSE and RV/LV dimension ratio
  • Increase in Brain Natriuretic Peptide (BNP) [ Time Frame: Over registry run-time (5.5 years) ]
  • Increase in N-terminal-proBNP (NT-proBNP) [ Time Frame: Over registry run-time (5.5 years) ]
Original Secondary Outcome Measures
 (submitted: November 19, 2015)
  • Hospitalisation related to pulmonary arterial hypertension (PAH) [ Time Frame: Min. 3 years ]
  • Use/initiation of i.v./s.c. prostanoids [ Time Frame: Min. 3 years ]
  • Atrial septostomy [ Time Frame: Min. 3 years ]
  • Potts shunt [ Time Frame: Min. 3 years ]
  • Time to clinical worsening [ Time Frame: Min. 3 years ]
    Various composites of above parameters
  • Decline in 6-minute walk test (6MWT), functional class, worsening of ECHO, increase in Brain Natriuretic Peptide (BNP)/N-terminal-proBNP (NT-proBNP) [ Time Frame: Min. 3 years ]
  • Type of treatment [ Time Frame: Min. 3 years ]
    Type of treatment (drug, mono/combination, administration route)
  • Switch in treatment [ Time Frame: Min. 3 years ]
  • Escalation of treatment [ Time Frame: Min. 3 years ]
  • Reasons for treatment change [ Time Frame: Min. 3 years ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension
Official Title Tracking Outcomes and Practice in 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.

Detailed Description Not Provided
Study Type Observational [Patient Registry]
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration 1 Day
Biospecimen Not Provided
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.
Condition Hypertension, Pulmonary
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: July 27, 2018)
440
Original Estimated Enrollment
 (submitted: November 19, 2015)
200
Estimated Study Completion Date December 2021
Estimated Primary Completion Date December 2021   (Final data collection date for primary outcome measure)
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
Sex/Gender
Sexes Eligible for Study: All
Ages 3 Months to 18 Years   (Child, Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Fabrizio Canonaco, Dr fabrizio.canonaco@peph-association.org
Contact: Simone Lerch, Dr simone.lerch@peph-association.org
Listed Location Countries Australia,   Brazil,   Canada,   China,   Colombia,   France,   Germany,   Hungary,   Israel,   Italy,   Japan,   Mexico,   Netherlands,   Poland,   Saudi Arabia,   Sweden,   Switzerland,   United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT02610660
Other Study ID Numbers TOPP-2
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Association for Pediatric Pulmonary Hypertension
Study Sponsor Association for Pediatric Pulmonary Hypertension
Collaborators Not Provided
Investigators
Study Chair: Dunbar Ivy, Prof Association for Pediatric Pulmonary Hypertension
PRS Account Association for Pediatric Pulmonary Hypertension
Verification Date September 2019