## Protocol Section
### Identification Module
**NCT ID:** NCT06429579

**Brief Title:** Potts-shunt for the Treatment of Pediatric Patients With Severe Pulmonary Hypertension

**Official Title:** Multicenter, Prospective, and Exploratory Study of Potts-shunt for the Treatment of Pediatric Patients With Severe Pulmonary Hypertension

#### Organization Study ID Info
**ID:** NCRC2024001
#### Organization
**Class:** OTHER_GOV
**Full Name:** China National Center for Cardiovascular Diseases
### Status Module
#### Completion Date
**Date:** 2029-04-23

**Type:** ESTIMATED

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-05-28

**Type:** ACTUAL

**Last Update Submit Date:** 2024-05-23

**Overall Status:** RECRUITING

#### Primary Completion Date
**Date:** 2029-04-23

**Type:** ESTIMATED

#### Start Date
**Date:** 2024-04-23

**Type:** ACTUAL

**Status Verified Date:** 2024-04

#### Study First Post Date
**Date:** 2024-05-28
**Type:** ACTUAL
**Study First Submit Date:** 2024-05-05
**Study First Submit QC Date:** 2024-05-23
### Sponsor Collaborators Module

#### Lead Sponsor

**Class:** OTHER_GOV

**Name:** China National Center for Cardiovascular Diseases

#### Responsible Party

**Type:** SPONSOR

### Oversight Module

**Is FDA Regulated Device:** False

**Is FDA Regulated Drug:** False

**Oversight Has DMC:** True

### Description Module

**Brief Summary:** This is a multi-center, perspective, and exploratory study aimed at evaluating the 3-years clinical outcome of Potts-shunt procedure for pediatric patients with severe pulmonary artery hypertension (PAH). The included criteria are as followed: 1)6 months \< age ≤ 18 years; 2) ESC 2022 Group I PAH; 3) Have received standardized drug therapy for at least 6-9 months and still remain at intermediate to high/high-risk status of the criteria of ESC2022; 4) Presenting with significant clinical manifestations (i.e., progressive symptoms/syncope history/growth and development restriction, etc); 5) Informed consent form signed by the patient and their guardian. The excluded criteria are as followed: 1) ESC 2022 Group II-V PAH; 2) Poor right ventricular function: RVEF \< 25% or RVFAC \< 20%; 3) Deteriorated general condition: requiring ICU resuscitation or ECMO assistance; 4) Pulmonary artery pressure/main arterial pressure ratio \< 0.7; 5) Six-minute walk distance \< 150 meters (only applicable to patients aged 8 and above); 6) No significant improvement in RVEF under triple drug therapy. All of the pediatric patients with severe PAH who attend to pediatric cardiac outpatient clinic and meet the designed included criteria and excluded criteria will be enrolled in this study. All of the participants will be divided into two groups (Potts-shunt combined with conventional drug therapy group and only conventional drug therapy group) according to their individual health status (i.e., some contraindications of surgery) and their (or their parents') aspiration for Potts-shunts procedure. Follow-up is designed (eight-times follow-up) at the time of Potts-shunt procedure, post-operative ICU period, one month, three months, six months, one year, two years, and three years after Potts-shunt procedure or the rejection of Potts-shunt procedure. The items of follow-up include state of survival, whether or not have the lung transplantation (LTx), clinical manifestation, laboratory examination, function of right ventricle (detected by echocardiogram and cardiac magnetic resonance imaging), and the pulmonary circulation pressure (detected by right heart catheterization or Swan-Ganz catheterization). Primary outcome is the incidence rates of death or LTx three-years after Potts-shunt. Secondary outcomes are as followed: 1) Number and incidence rate of postoperative complications in patients undergoing Potts-shunt procedure; 2) Three-year WHO cardiac functional and 6-minute walk distance after Potts-shunt procedure; 3) the NT-ProBNP levels three-years after Potts-shunt procedure; 4) Right ventricular function on echocardiography three years after Potts-shunt procedure; 5) Right ventricular function on cardiac magnetic resonance imaging three years after Potts-shunt procedure; 6) Pulmonary circulation pressure measured by right heart catheterization or Swan-Ganz catheterization three years after Potts-shunt procedure; 7) Three-year mortality or LTx incidence rates after only conventional drug therapy.

### Conditions Module

**Conditions:**

- Pulmonary Artery Hypertension

### Design Module

#### Design Info

**Allocation:** NON_RANDOMIZED

**Intervention Model:** PARALLEL

##### Masking Info

**Masking:** NONE

**Primary Purpose:** TREATMENT

#### Enrollment Info

**Count:** 120

**Type:** ESTIMATED

**Phases:**

- NA

**Study Type:** INTERVENTIONAL

### Arms Interventions Module

#### Arm Group 1

**Description:** The comprehensive therapy included Potts-shunt procedure and conventional drug therapy (i.e., Prostacyclin analogues, PDE-5 inhibitors, and Endothelin receptor antagonists)

**Intervention Names:**

- Procedure: Comprehensive therapy combined Potts-shunt procedure and conventional drug therapy

**Label:** Potts-shunt combined with conventional drug therapy group

**Type:** EXPERIMENTAL

#### Arm Group 2

**Description:** Only admitted with conventional drug therapy (i.e., Prostacyclin analogues, PDE-5 inhibitors, and Endothelin receptor antagonists)

**Intervention Names:**

- Drug: Only conventional drug therapy

**Label:** Only conventional drug therapy group

**Type:** ACTIVE_COMPARATOR

### Interventions

#### Intervention 1

**Arm Group Labels:**

- Potts-shunt combined with conventional drug therapy group

**Description:** Potts-shunt procedure is the surgery that connect the descending aorta and the left/main pulmonary artery by direct anastomosis, a Gore-tex tube or a Gore-tex tube with flap.

**Name:** Comprehensive therapy combined Potts-shunt procedure and conventional drug therapy

**Type:** PROCEDURE

#### Intervention 2

**Arm Group Labels:**

- Only conventional drug therapy group

**Description:** Only conventional drug therapy (i.e., Prostacyclin analogues, PDE-5 inhibitors, and Endothelin receptor antagonists)

**Name:** Only conventional drug therapy

**Type:** DRUG

### Outcomes Module

#### Primary Outcomes

**Description:** The incidence rates of death or lung transplantation 3 years after Potts-shunt, accessed by in-patient, out-patient, or telephone follow-up

**Measure:** Mortality or Lung transplantation

**Time Frame:** From the date of enrollment until the date of dear from any cause or lung transplantation, which ever came first, accessed up to 3 years

#### Secondary Outcomes

**Description:** Postoperative complications include hemoptysis, syncope, pulmonary hypertension crisis, low blood oxygen in the lower extremities etc. accessed by pediatric ICU doctors.

**Measure:** Number and incidence rate of postoperative complications in patients undergoing Potts-shunt procedure

**Time Frame:** From the date of enrollment to the date of any postoperative complications, whichever came first, accessed up to 3 years

**Description:** WHO-FC was designed to measure the severity of pulmonary hypertension based on symptoms of dyspnea, fatigue, chest pain, and related syncope, accessed by the pediatric doctors.

**Measure:** World Health Organization functional class (WHO-FC) after Potts-shunt procedure

**Time Frame:** From the date of enrollment to the date of every view-point, assessed up to 3 years

**Description:** NT-ProBNP (pg/ml) accessed by laboratory test

**Measure:** NT-ProBNP levels after Potts-shunt procedure

**Time Frame:** From the date of enrollment to the date of every view-point, assessed up to 3 years

**Description:** The RVEF (%) accessed by cardiac MRI

**Measure:** Right ventricular function on cardiac magnetic resonance imaging after Potts-shunt procedure

**Time Frame:** From the date of enrollment to the date of every view-point, assessed up to 3 years

**Description:** The RVFAC (%) accessed by echocardiography

**Measure:** Right ventricular function on echocardiography after Potts-shunt procedure

**Time Frame:** From the date of enrollment to the date of every view-point, assessed up to 3 years

**Description:** SPAP, DPAP, MPAP (mmHg) etc. accessed by right heart catheterization or Swan-Ganz catheterization

**Measure:** Pulmonary circulation pressure measured by right heart catheterization or Swan-Ganz catheterization after Potts-shunt procedure

**Time Frame:** From the date of enrollment to the date of every view-point, assessed up to 3 years

**Description:** The incidence rates of death or lung transplantation 3 years after enrollment

**Measure:** Mortality or lung transplantation incidence rates after only conventional drug therapy

**Time Frame:** From the date of enrollment until the date of dear from any cause or lung transplantation, which ever came first, accessed up to 3 years

**Description:** The 6-minutes distance (meter) was designed to measure activity tolerance, accessed by pediatric doctors or parents of participants.

**Measure:** The 6-minutes distance after Potts-shunt procedure

**Time Frame:** From the date of enrollment to the date of every view-point, assessed up to 3 years

### Eligibility Module

**Eligibility Criteria:** Inclusion Criteria:

1. 6 months \&lt; age ≤ 18 years;
2. ESC 2022 Group I PAH;
3. Have received standardized drug therapy for at least 6-9 months and still remain at intermediate to high/high-risk status of the criteria of ESC2022;
4. Presenting with significant clinical manifestations (i.e., progressive symptoms/syncope history/growth and development restriction, etc);
5. Informed consent form signed by the patient and their guardian.

Exclusion Criteria:

1. ESC 2022 Group II-V PAH;
2. Poor right ventricular function: RVEF \&lt; 25% or RVFAC \&lt; 20%;
3. Deteriorated general condition: requiring ICU resuscitation or ECMO assistance;
4. Pulmonary artery pressure/main arterial pressure ratio \&lt; 0.7;
5. Six-minute walk distance \&lt; 150 meters (only applicable to patients aged 8 and above);
6. No significant improvement in RVEF under triple drug therapy.

**Maximum Age:** 18 Years

**Minimum Age:** 6 Months

**Sex:** ALL

**Standard Ages:**

- CHILD

- ADULT

### Contacts Locations Module

#### Central Contacts

**Contact 1:**

**Email:** drlishoujunfw@163.com

**Name:** Shoujun Li, MD

**Phone:** +8613501071589

**Role:** CONTACT

#### Locations

**Location 1:**

**City:** Beijing

**Contacts:**

***Contact 1:***

- **Email:** drlishoujunfw@163.com

- **Name:** Shoujun Li, MD

- **Phone:** +8613501071589

- **Role:** CONTACT

**Country:** China

**Facility:** Pediatric cardic surgical center, Fuwai Hospital

**State:** Beijing

**Status:** RECRUITING

**Zip:** 100037

### IPD Sharing Statement Module

**IPD Sharing:** NO

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000014652

- Term: Vascular Diseases

- ID: D000002318

- Term: Cardiovascular Diseases

- ID: D000008171

- Term: Lung Diseases

- ID: D000012140

- Term: Respiratory Tract Diseases

### Condition Browse Module - Browse Branches

- Abbrev: BC14

- Name: Heart and Blood Diseases

- Abbrev: All

- Name: All Conditions

- Abbrev: BC08

- Name: Respiratory Tract (Lung and Bronchial) Diseases

- Abbrev: Rare

- Name: Rare Diseases

### Condition Browse Module - Browse Leaves

- ID: M10024

- Name: Hypertension

- Relevance: HIGH

- As Found: Hypertension

- ID: M2261

- Name: Pulmonary Arterial Hypertension

- Relevance: HIGH

- As Found: Pulmonary Artery Hypertension

- ID: M10027

- Name: Hypertension, Pulmonary

- Relevance: HIGH

- As Found: Pulmonary Hypertension

- ID: M17400

- Name: Vascular Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M11168

- Name: Lung Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M14977

- Name: Respiratory Tract Diseases

- Relevance: LOW

- As Found: Unknown

- ID: T4807

- Name: Pulmonary Arterial Hypertension

- Relevance: HIGH

- As Found: Pulmonary Artery Hypertension

### Condition Browse Module - Meshes

- ID: D000006976

- Term: Hypertension, Pulmonary

- ID: D000081029

- Term: Pulmonary Arterial Hypertension

- ID: D000006973

- Term: Hypertension

### Intervention Browse Module - Browse Branches

- Abbrev: All

- Name: All Drugs and Chemicals

- Abbrev: AnAg

- Name: Antihypertensive Agents

- Abbrev: PlAggInh

- Name: Platelet Aggregation Inhibitors

- Abbrev: VaDiAg

- Name: Vasodilator Agents

### Intervention Browse Module - Browse Leaves

- ID: M30466

- Name: Endothelin Receptor Antagonists

- Relevance: LOW

- As Found: Unknown

- ID: M29332

- Name: Phosphodiesterase 5 Inhibitors

- Relevance: LOW

- As Found: Unknown

- ID: M14328

- Name: Epoprostenol

- Relevance: LOW

- As Found: Unknown

- ID: M262431

- Name: Tezosentan

- Relevance: LOW

- As Found: Unknown

### Misc Info Module

- Version Holder: 2024-05-31
