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NCT Number: NCT06196801

Efficacy of Triple-Combination Therapy in Severe PAH-CHD

Congenital heart disease (CHD) is a leading cause of pulmonary arterial hypertension (PAH) worldwide. Treatment for PAH associated with CHD (PAH-CHD) depends on the defect's type, size, and hemodynamic impact. For those with CHD correction indications, early defect repair or interventional closure is crucial to prevent irreversible pulmonary vascular remodeling due to prolonged exposure to a left-to-right shunt.

Current guidelines recommend triple-combination therapy, including phosphodiesterase 5 inhibitors, endothelin receptor antagonist, and parenteral prostacyclin, for patients with intermediate-high or high risk. Recent studies suggest that patients with PAH-CHD and borderline hemodynamics might regain eligibility for surgery after targeted vasodilatory treatment. Consequently, early initiation of triple-combination therapy may be critical for severe PAH-CHD patients to restore their surgical or interventional closure eligibility. Therefore, we conducted this prospective study to assess the effectiveness of triple-combination therapy in severe PAH-CHD cases.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Congenital Heart Disease Cardiovascular Abnormalities Cardiovascular Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Heart Defects, Congenital Heart Diseases Hypertension, Pulmonary Lung Diseases Pulmonary Arterial Hypertension Respiratory Tract Diseases

Age range

14 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Guangdong Cardiovascular Institute, Guangdong Provincial People's Hospital (Guangdong Academy of Medical Sciences), Southern Medical University

Guangzhou, Guangdong, 510080, China

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Subject is diagnosed with congenital heart diseases (CHD) and associated pulmonary arterial hypertension (PAH)
  • Subject is under borderline hemodynamics status and ineligible for congenital heart diseases closure, confirmed by RHC: Qp/Qs：< 1.5, Rp/Rs > 0.3 and PVR > 5 Wood units
  • Subject signs an informed Consent Form and is willing to participate in follow-up visits

Exclusion criteria

  • Subject is diagnosed with other etiology of pulmonary arterial hypertension, e.g. left heart diseases associated pulmonary arterial hypertension.
  • Subject is diagnosed with other types of PAH-CHD, e.g. Eisenmenger syndrome, PAH with small/coincidentalb defects
  • Subject had cerebral hemorrhage, bleeding events in other organs within 3 months or was in high risk of bleeding.
  • Subject is diagnosed of hepatic insufficiency: ALT or AST>3×ULN at the screening visit.
  • Subject is diagnosed of moderate to severe renal insufficiency: eGFR<30ml/min/1.73m2 at the screening visit.
  • Subject has uncontrolled arrhythmia with clinical significance within 90 days.
  • Subject is diagnosed of the following diseases or received following medical interventions with 90 days: unstable angina, severe coronary atherosclerosis or myocardial infarction, cerebrovascular disease, deep vein thrombosis, pulmonary embolism, percutaneous coronary intervention, coronary artery bypass grafting, carotid artery intervention, peripheral artery intervention.
  • Subject is diagnosed of malignant tumor (exception: tumors that have been cured and have not recurred in the last 5 years, basal cell and squamous cell skin cancers that have been completely resected, and cancers of any type in situ that have been completely resected)
  • Subject cannot follow the study procedure due to other acute or chronic diseases.
  • Subject is under other RCT.
  • Subject has a life expectancy <1 year.
  • Subject cannot follow the study procedure due to other reasons in the opinion of the investigators. (alcoholic, drug abuse, lack of compliance)

Treatment and study plan

Triple-combination therapy

Drug

phosphodiesterase 5 inhibitors, endothelin receptor antagonist, and parenteral prostacyclin

Primary outcomes

  1. Pulmonary hemodynamics

    Time frame: 6 months,12 months

    comparison of hemodynamics at baseline, before and after surgery/intervention, assessed by RHC

Secondary outcomes

  1. Pulmonary hemodynamics

    Time frame: perioperative period

  2. Vasodilators average usage and compliance

    Time frame: 6 months, 12 months

  3. Surgery success rate and adverse event

    Time frame: surgery perioperative period

  4. Other monitoring data

    Time frame: surgery perioperative period

  5. Other examination results

    Time frame: surgery perioperative period

Sponsors and collaborators

Lead sponsor

Guangdong Provincial People's Hospital

Other

Registry information

Official study title

Efficacy of Triple-Combination Therapy in Severe Pulmonary Arterial Hypertension Associated Congenital Heart Diseases

Important dates

Study start
2022
Primary completion
2025
Study completion
2025
First posted
Jan 9, 2024
Registry last updated
Mar 12, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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