Clinical Trial: Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational [Patient Registry]

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.


Detailed Summary:
Sponsor: Association for Pediatric Pulmonary Hypertension

Current Primary Outcome:

  • Death [ Time Frame: Min. 3 years ]
  • Transplantation [ Time Frame: Min. 3 years ]
  • Adverse events [ Time Frame: Min. 3 years ]


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 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) [ 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 ]
  • Decline in WHO functional class [ Time Frame: Min. 3 years ]
  • Decline in Panama functional class [ Time Frame: Min. 3 years ]
  • Worsening of echocardiographic parameters (ECHO) [ Time Frame: Min. 3 years ]
    Parameters encompass TAPSE and RV/LV dimension ratio
  • Increase in Brain Natriuretic Peptide (BNP) [ Time Frame: Min. 3 years ]
  • Increase in N-terminal-proBNP (NT-proBNP) [ Time Frame: Min. 3 years ]


Original Secondary Outcome:

  • 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 ]


Information By: Association for Pediatric Pulmonary Hypertension

Dates:
Date Received: November 10, 2015
Date Started: August 2015
Date Completion: December 2020
Last Updated: April 28, 2017
Last Verified: April 2017