A Prospective Single-arm Exploratory Study on the Efficacy and Safety of Treprostinil Injection in the Treatment of Patients With Intermediate-risk Pulmonary Arterial Hypertension
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 32
- 主要终点
- Change From Baseline in Right Ventricular Stroke Volume (RVSV)
研究概览
简要总结
Pulmonary Arterial Hypertension (PAH) is a serious condition that puts strain on the right side of the heart. While oral medications can help, many patients with intermediate-risk PAH may not see enough improvement, and their heart function can continue to decline.
This study aims to find out if adding an injectable medication, Treprostinil, to a patient's current oral PAH therapy can improve heart function and overall health. This is a single-arm study, which means all participants will receive the study treatment.
The main goal is to measure the change in the amount of blood the right side of the heart pumps with each beat (Right Ventricular Stroke Volume, or RVSV) after 3 months of treatment. This will be measured using a specialized heart scan called Cardiac Magnetic Resonance Imaging (CMR). Researchers will also assess changes in exercise ability (with a 6-minute walk test), blood markers, and patient symptoms.
Participants will be in the main part of the study for 3 months, with follow-up for a total of 24 months to monitor the long-term effects and safety of the treatment.
详细描述
Background and Rationale:
Pulmonary Arterial Hypertension (PAH) is a progressive vasculopathy leading to increased pulmonary vascular resistance, right ventricular (RV) failure, and mortality. While guidelines recommend initial combination oral therapy, a substantial number of patients, particularly those at intermediate-risk, do not achieve a low-risk status. Subclinical RV deterioration can occur even in seemingly stable patients, suggesting that more intensive therapeutic strategies may be warranted to improve long-term outcomes.
Parenteral prostacyclin analogues, like Treprostinil, are potent therapies recommended for high-risk PAH. However, there is a lack of prospective data on their efficacy in inducing positive RV remodeling and improving clinical outcomes when initiated in patients with intermediate-risk PAH who are already on background oral therapies. This study aims to address this evidence gap.
Study Design and Objectives:
This is a prospective, single-center, single-arm, open-label, exploratory study designed to evaluate the efficacy and safety of adding subcutaneous Treprostinil to standard-of-care oral therapy for patients with intermediate-risk PAH.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed the informed consent form.
- •Age between 18 and 75 years, inclusive.
- •Diagnosed with Pulmonary Arterial Hypertension (PAH, WHO Group 1) of the following etiologies:Idiopathic;Heritable;Drug or toxin-induced (excluding amphetamine-induced PAH);Associated with repaired congenital systemic-to-pulmonary shunts (repair performed ≥1 year prior);Associated with connective tissue disease;Associated with HIV infection;
- •Has undergone a right heart catheterization (RHC) within 6 months of the baseline visit.
- •Baseline RHC demonstrates the following hemodynamics:Mean pulmonary arterial pressure (mPAP) > 20 mmHg;Pulmonary artery wedge pressure (PAWP) ≤ 15 mmHg;Pulmonary vascular resistance (PVR) > 2 Wood units;
- •WHO Functional Class II, III, or IV.
- •Risk stratification according to 2022 ESC/ERS guidelines is either intermediate-risk at initial diagnosis or low- to intermediate-risk during follow-up.
- •Patient is on a stable dose of an endothelin receptor antagonist (ERA) and/or a phosphodiesterase-5 inhibitor (PDE-5i) or a soluble guanylate cyclase (sGC) stimulator for at least 30 days prior to baseline; OR, if treatment-naïve, is willing to initiate one of these therapies in addition to the study drug.
排除标准
- •Age < 18 years.
- •Diagnosis of Pulmonary Hypertension belonging to WHO Groups 2, 3, 4, or
- •Patients undergoing dialysis.
- •Severe hepatic impairment (Child-Pugh Class C).
- •Use of any prostacyclin-pathway drugs (epoprostenol, treprostinil, iloprost, beraprost, or selexipag) within 30 days prior to the baseline visit.
- •Presence of implanted metallic devices that would interfere with Cardiac - - - Magnetic Resonance (CMR) imaging, such as defibrillators, neurostimulators, cochlear implants, permanent infusion devices, implanted pumps, or certain plates/screws.
- •In the investigator's judgment, the patient is unable to complete or comply with study requirements.
- •Refusal to sign the informed consent form or refusal to participate in follow-up visits.
- •Participation in any other clinical trial involving an investigational drug, device, or procedure within 30 days prior to the baseline visit.
研究组 & 干预措施
Treatment Group
Treatment is added to the participant's stable background regimen of standard-of-care oral PAH therapies (e.g., endothelin receptor antagonists, phosphodiesterase-5 inhibitors, and/or soluble guanylate cyclase stimulators).
Treprostinil is initiated at a dose of 1.25 ng/kg/min. The dose is then titrated upwards based on individual patient tolerability and clinical response. The planned titration schedule is an increase of 1.25 ng/kg/min weekly for the first four weeks of treatment, followed by weekly increases of 2.5 ng/kg/min thereafter, as tolerated.
干预措施: Treprostinil (Drug)
结局指标
主要结局
Change From Baseline in Right Ventricular Stroke Volume (RVSV)
时间窗: 3 months
Change in Right Ventricular Stroke Volume (RVSV) from baseline, as measured by Cardiac Magnetic Resonance (CMR). RVSV is the volume of blood pumped from the right ventricle of the heart during one contraction.
次要结局
未报告次要终点
研究者
Junbo Ge
Director of Cardiology
Shanghai Zhongshan Hospital