Tracking Outcomes and Practice in Pediatric Pulmonary Hypertension

NCT02610660 | Completed

• 1 other identifier: TOPP-2
• Study Type: observational
• Target: 388
• Locations: 18 countries
• Sites: 32

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.

Conditions

Hypertension, Pulmonary

Outcome Measures

Primary Outcomes (3)

• Death

  Over registry run-time (5.5 years)

• Transplantation

  Over registry run-time (5.5 years)

• Adverse events

  Over registry run-time (5.5 years)

Secondary Outcomes (15)

• Hospitalisation related to pulmonary arterial hypertension (PAH)

  Over registry run-time (5.5 years)

• Use/initiation of i.v./s.c. prostanoids

  Over registry run-time (5.5 years)

• Atrial septostomy

  Over registry run-time (5.5 years)

• Potts shunt

  Over registry run-time (5.5 years)

• Time to clinical worsening

  Over registry run-time (5.5 years)

Eligibility Criteria

• Age: 3 Months - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• Patients belonging to Group 2 according to updated Nice clinical classification

Sponsors & Collaborators

• Association for Pediatric Pulmonary Hypertension: lead

Study Sites (32)

Stanford Children's Hospital

Palo Alto, California, 94304, United States

Location

University of Colorado Denver School of Medicine, Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

St Louis Children's Hospital

St Louis, Missouri, 63110, United States

Location

University of Nebraska Medical Center

Omaha, Nebraska, 68198, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Vanderbilt University Children's Hospital

Nashville, Tennessee, 37232-9310, United States

Location

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

Primary Children's Hospital

Salt Lake City, Utah, 84113, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

Royal Children's Hospital

Melbourne, 3052, Australia

Location

University of São Paulo

São Paulo, 05403-000, Brazil

Location

The Hospital for Sick Children

Toronto, Ontario, M5G 1XB, Canada

Location

Fu Wai Hospital

Beijing, 100037, China

Location

Hospital Pediátrico La Misericordia

Bogotá, Colombia

Location

Université Paris Descartes

Paris, 75015, France

Location

University Children's Hospital Ulm

Ulm, 89075, Germany

Location

Institute of Cardiology

Budapest, Hungary

Location

Hadassah, Hebrew University Medical Center

Jerusalem, 91120, Israel

Location

University of Padova

Padua, 35127, Italy

Location

Toho Universit Omori Medical Center

Tokyo, 1438541, Japan

Location

Keio University

Tokyo, 160-8582, Japan

Location

National Heart Institute

Mexico City, 14050, Mexico

Location

Beatrix Children's Hospital, University Medical Center Groningen

Groningen, 9700, Netherlands

Location

Children's Memorial

Warsaw, 04-730, Poland

Location

King Fahd Armed Forces Hospital

Jeddah, 23311, Saudi Arabia

Location

The Queen Silvia's Children's Hospital

Gothenburg, 41685, Sweden

Location

Hôpital des Enfants

Geneva, 1211, Switzerland

Location

University Children's Hospital

Zurich, 8032, Switzerland

Location

Related Links

• Webpage of the Association for Pediatric Pulmonary Hypertension

MeSH Terms

Conditions

Hypertension, Pulmonary

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Hypertension; Vascular Diseases; Cardiovascular Diseases

Study Officials

• Dunbar Ivy, Prof

  Association for Pediatric Pulmonary Hypertension

  STUDY CHAIR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 18 Months
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 10, 2015
• First Posted: November 20, 2015
• Study Start: August 1, 2015
• Primary Completion: December 31, 2022
• Study Completion: December 31, 2022
• Last Updated: April 13, 2023

Record last verified: 2023-04

Locations

US (13); BR (1); HU (1); FR (1); SA (1); PL (1); SE (1); JP (2); CA (1); CO (1); ME (1); AU (1); CN (1); DE (1); IL (1); CH (2); IT (1); NL (1)