The Effect of Glutamine on Reducing Pulmonary Arterial Pressure in Non-transfusion-dependent Thalassemia Patients: a Single Blind Randomized Clinical Trial
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 8
- 试验地点
- 1
- 主要终点
- Change in Pulmonary Artery Pressure (PAP)
研究概览
简要总结
The goal of this clinical trial is to learn whether L-glutamine can help lower pulmonary artery pressure in adults with non-transfusion-dependent thalassemia (NTDT).
The main questions it aims to answer are:
Does L-glutamine reduce pulmonary artery pressure after 60 days of treatment?
Is the effect of L-glutamine different from standard care alone?
Researchers will compare two groups:
Intervention group: Participants receive oral L-glutamine in addition to their standard treatment.
Control group: Participants continue with standard treatment only.
Participants will:
Take either L-glutamine (by mouth) or standard care for 60 days. Undergo echocardiography at the beginning and end of the study to measure pulmonary artery pressure.
Attend follow-up visits to monitor safety, adherence, and possible side effects.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Single (Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adults (≥18 years), any sex.
- •Diagnosis of non-transfusion-dependent β-thalassemia (NTDT).
- •Pulmonary artery pressure (PAP) > 35 mmHg estimated by Doppler echocardiography at screening.
- •Able and willing to provide written informed consent.
- •On a stable standard-of-care regimen (e.g., chelation and/or hydroxyurea) per treating physician judgment.
排除标准
- •Age <18 years.
- •Refusal or inability to provide informed consent.
- •Hepatic dysfunction: ALT >3× upper limit of normal.
- •Renal dysfunction: serum creatinine >2× upper limit of normal.
- •Known hypersensitivity to L-glutamine.
- •Pregnancy or breastfeeding.
- •Use of amino-acid/protein supplements within the past 3 months.
- •History of other cardiac diseases associated with pulmonary hypertension (per investigator assessment).
研究组 & 干预措施
L-Glutamine + Standard Care
Participants in this arm will receive oral L-glutamine at a dose of 0.1 g/kg/day for 60 days, in addition to their routine standard care for non-transfusion-dependent thalassemia
干预措施: L-glutamine (Drug)
结局指标
主要结局
Change in Pulmonary Artery Pressure (PAP)
时间窗: Baseline to 60 days
Pulmonary artery pressure (PAP) will be measured by Doppler echocardiography at baseline and at 60 days. The primary endpoint is the change in PAP (ΔPAP = Follow-up PAP - Baseline PAP) to assess whether L-glutamine reduces pulmonary artery pressure compared with standard care alone.
次要结局
- Clinical Response Rate(60 days)
- Safety and Tolerability of L-Glutamine(Baseline to 60 days)
- Iron Load(Baseline to 60 days)
研究者
Rozita Gorzin
Pediatrician
Mazandaran University of Medical Sciences