Hypertension, PulmonaryTOPP-2NCT02610660
Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension
An observational study of Hypertension, Pulmonary, run by Association for Pediatric Pulmonary Hypertension at 32 sites in 18 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 months to 18 years
- Conditions the study is for
- Hypertension, Pulmonary
- How many people
- 388 participants
- Where
- 32 sitesin 18 countries
- Study dates
- Aug 2015to 31 Dec 2022
- How long will I take part?
- 18 Months
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
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.
Who may be able to join
- 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
Show all 13Show fewer
- 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.
Who cannot join
- Patients belonging to Group 2 according to updated Nice clinical classification
Trial locations and contacts
32 sites in 18 countries.
32 sites in 18 countries
United States13 sites
Emory University
Atlanta, Georgia, 30322
University of Colorado Denver School of Medicine, Children's Hospital Colorado
Aurora, Colorado, 80045
Boston Children's Hospital
Boston, Massachusetts, 02115
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Texas Children's Hospital
Houston, Texas, 77030
Vanderbilt University Children's Hospital
Nashville, Tennessee, 37232-9310
Columbia University Medical Center
New York, New York, 10032
University of Nebraska Medical Center
Omaha, Nebraska, 68198
Stanford Children's Hospital
Palo Alto, California, 94304
Primary Children's Hospital
Salt Lake City, Utah, 84113
Seattle Children's Hospital
Seattle, Washington, 98105
St Louis Children's Hospital
St Louis, Missouri, 63110
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Japan2 sites
Toho Universit Omori Medical Center
Tokyo, 1438541
Keio University
Tokyo, 160-8582
Switzerland2 sites
Hôpital des Enfants
Geneva, 1211
University Children's Hospital
Zurich, 8032
Netherlands1 site
Beatrix Children's Hospital, University Medical Center Groningen
Groningen, 9700
About this study
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.
Read more
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.
- Conditions
- Hypertension, Pulmonary
- Medical subject headings
- Hypertension, Pulmonary
What is being measured
The primary outcome is the main question the study is designed to answer.
Death
Measured: Over registry run-time (5.5 years)
Transplantation
Measured: Over registry run-time (5.5 years)
Adverse events
Measured: Over registry run-time (5.5 years)
Secondary outcomes (15)
Hospitalisation related to pulmonary arterial hypertension (PAH)
Measured: Over registry run-time (5.5 years)
Use/initiation of i.v./s.c. prostanoids
Measured: Over registry run-time (5.5 years)
Atrial septostomy
Measured: Over registry run-time (5.5 years)
Potts shunt
Measured: Over registry run-time (5.5 years)
Time to clinical worsening
Various composites of above parameters
Measured: Over registry run-time (5.5 years)
Decline in 6-minute walk test (6MWT)
Measured: Over registry run-time (5.5 years)
Type of treatment
Type of treatment (drug, mono/combination, administration route)
Measured: Over registry run-time (5.5 years)
Switch in treatment
Measured: Over registry run-time (5.5 years)
Escalation of treatment
Measured: Over registry run-time (5.5 years)
Reasons for treatment change
Measured: Over registry run-time (5.5 years)
Decline in WHO functional class
Measured: Over registry run-time (5.5 years)
Decline in Panama functional class
Measured: Over registry run-time (5.5 years)
Worsening of echocardiographic parameters (ECHO)
Parameters encompass TAPSE and RV/LV dimension ratio
Measured: Over registry run-time (5.5 years)
Increase in Brain Natriuretic Peptide (BNP)
Measured: Over registry run-time (5.5 years)
Increase in N-terminal-proBNP (NT-proBNP)
Measured: Over registry run-time (5.5 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Nov 2015 (estimated)
- Study startAug 2015
- Primary completion31 Dec 2022
- Study completion31 Dec 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hypertension, PulmonaryStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 388 participants (actual)
- Target duration
- 18 Months
Sponsor and investigators
- Lead sponsor
- Association for Pediatric Pulmonary Hypertension
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Dunbar Ivy, Prof, Study chair, Association for Pediatric Pulmonary Hypertension
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02610660
- Sponsor's protocol ID
- TOPP-2
- Acronym
- TOPP-2
- Status last verified
- Apr 2023
- First submitted
- 10 Nov 2015
- Last updated
- 13 Apr 2023
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension
- ClinicalTrials.gov ID
- NCT02610660
- Acronym / sponsor ID
- TOPP-2 / TOPP-2
- Conditions
- Hypertension, Pulmonary
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 388 participants (actual)
- Lead sponsor
- Association for Pediatric Pulmonary HypertensionUniversity, hospital or other
- Study start
- Aug 2015
- Primary completion
- 31 Dec 2022 (actual)
- Study completion
- 31 Dec 2022 (actual)
- First posted
- 20 Nov 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 18 Months
Who may qualify
- Ages
- 3 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- 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.
Key inclusion criteria (13 listed)
- 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
- 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
Key exclusion criteria (1 listed)
- Patients belonging to Group 2 according to updated Nice clinical classification
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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)
15 secondary outcomes
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)
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)
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)
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)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Apr 2023.
- Webpage of the Association for Pediatric Pulmonary HypertensionLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Dunbar Ivy, Prof study chair
Association for Pediatric Pulmonary Hypertension
32 sites in 18 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hypertension, Pulmonary, run by Association for Pediatric Pulmonary Hypertension at 32 sites in 18 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 13 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 32 sites in United States, Japan, Switzerland, Australia, Brazil and 13 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Death (time frame: Over registry run-time (5.5 years)). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Apr 2023.