TOPP-2: Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension

• Sponsor: Association for Pediatric Pulmonary Hypertension (Other)
• Overall Status: Active, not recruiting
• CT.gov ID: NCT02610660
• Collaborator: (none)
• Enrollment: 388
• Locations: 32
• Duration (Months): 88
• Patients Per Site: 12.1
• Patients Per Site Per Month: 0.1

Study Details

Study Description

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.

All enrolled patients will have a follow-up period of 18 months.

Condition or Disease	Intervention/Treatment	Phase
Hypertension, Pulmonary

Study Design

• Study Type: Observational [Patient Registry]
• Actual Enrollment: 388 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension
• Study Start Date: Aug 1, 2015
• Anticipated Primary Completion Date: Dec 1, 2022
• Anticipated Study Completion Date: Dec 1, 2022

Outcome Measures

Primary Outcome Measures

1. Death [Over registry run-time (5.5 years)]

2. Transplantation [Over registry run-time (5.5 years)]

3. Adverse events [Over registry run-time (5.5 years)]

Secondary Outcome Measures

1. Hospitalisation related to pulmonary arterial hypertension (PAH) [Over registry run-time (5.5 years)]

2. Use/initiation of i.v./s.c. prostanoids [Over registry run-time (5.5 years)]

3. Atrial septostomy [Over registry run-time (5.5 years)]

4. Potts shunt [Over registry run-time (5.5 years)]

5. Time to clinical worsening [Over registry run-time (5.5 years)]

   Various composites of above parameters

6. Decline in 6-minute walk test (6MWT) [Over registry run-time (5.5 years)]

7. Type of treatment [Over registry run-time (5.5 years)]

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

8. Switch in treatment [Over registry run-time (5.5 years)]

9. Escalation of treatment [Over registry run-time (5.5 years)]

10. Reasons for treatment change [Over registry run-time (5.5 years)]

11. Decline in WHO functional class [Over registry run-time (5.5 years)]

12. Decline in Panama functional class [Over registry run-time (5.5 years)]

13. Worsening of echocardiographic parameters (ECHO) [Over registry run-time (5.5 years)]

    Parameters encompass TAPSE and RV/LV dimension ratio

14. Increase in Brain Natriuretic Peptide (BNP) [Over registry run-time (5.5 years)]

15. Increase in N-terminal-proBNP (NT-proBNP) [Over registry run-time (5.5 years)]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 3 Months to 18 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Stanford Children's Hospital	Palo Alto	California	United States	94304
2	University of Colorado Denver School of Medicine, Children's Hospital Colorado	Aurora	Colorado	United States	80045
3	Children's National Medical Center	Washington	District of Columbia	United States	20010
4	Emory University	Atlanta	Georgia	United States	30322
5	Boston Children's Hospital	Boston	Massachusetts	United States	02115
6	St Louis Children's Hospital	Saint Louis	Missouri	United States	63110
7	University of Nebraska Medical Center	Omaha	Nebraska	United States	68198
8	Columbia University Medical Center	New York	New York	United States	10032
9	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio	United States	45229
10	Vanderbilt University Children's Hospital	Nashville	Tennessee	United States	37232-9310
11	Texas Children's Hospital	Houston	Texas	United States	77030
12	Primary Children's Hospital	Salt Lake City	Utah	United States	84113
13	Seattle Children's Hospital	Seattle	Washington	United States	98105
14	Royal Children's Hospital	Melbourne		Australia	3052
15	University of São Paulo	São Paulo		Brazil	05403-000
16	The Hospital for Sick Children	Toronto	Ontario	Canada	M5G 1XB
17	Fu Wai Hospital	Beijing		China	100037
18	Hospital Pediátrico La Misericordia	Bogota		Colombia
19	Université Paris Descartes	Paris		France	75015
20	University Children's Hospital Ulm	Ulm		Germany	89075
21	Institute of Cardiology	Budapest		Hungary
22	Hadassah, Hebrew University Medical Center	Jerusalem		Israel	91120
23	University of Padova	Padova		Italy	35127
24	Toho Universit Omori Medical Center	Tokyo		Japan	1438541
25	Keio University	Tokyo		Japan	160-8582
26	National Heart Institute	Mexico City		Mexico	14050
27	Beatrix Children's Hospital, University Medical Center Groningen	Groningen		Netherlands	9700
28	Children's Memorial	Warsaw		Poland	04-730
29	King Fahd Armed Forces Hospital	Jeddah		Saudi Arabia	23311
30	The Queen Silvia's Children's Hospital	Gothenburg		Sweden	41685
31	Hôpital des Enfants	Geneva		Switzerland	1211
32	University Children's Hospital	Zurich		Switzerland	8032

Sponsors and Collaborators

• Association for Pediatric Pulmonary Hypertension

Investigators

• Study Chair: Dunbar Ivy, Prof, Association for Pediatric Pulmonary Hypertension

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• Webpage of the Association for Pediatric Pulmonary Hypertension

Publications

None provided.

• Responsible Party: Association for Pediatric Pulmonary Hypertension
• ClinicalTrials.gov Identifier: NCT02610660
• Other Study ID Numbers: TOPP-2
• First Posted: Nov 20, 2015
• Last Update Posted: Aug 3, 2021
• Last Verified: Aug 1, 2021

• Additional relevant MeSH terms: Hypertension, Pulmonary; Hypertension

Study Results

No Results Posted as of Aug 3, 2021