Intramuscular vs. Enteral Penicillin Prophylaxis to Prevent Progression of Latent Rheumatic Heart Disease: A Non-inferiority Randomized Trial
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 1,004
- 试验地点
- 1
- 主要终点
- Progression
研究概览
简要总结
The goal of this clinical trial is to determine if twice daily oral penicillin prophylaxis is non-inferior to monthly IM penicillin prophylaxis in preventing latent Rheumatic Heart Disease Progression in children between the ages of 5-17 years. The main objective is to compare the proportion of children aged 5-17 years with latent RHD receiving oral penicillin prophylaxis who progress to worse valvular disease at 2-years compared to children who receive IM penicillin prophylaxis.
详细描述
GOALIS is a randomized controlled trial developed to provide high quality contemporary evidence on the efficacy of oral penicillin as RHD prophylaxis.
Aim 1: To compare the proportion of children aged 5-17 years with latent RHD receiving oral penicillin prophylaxis who progress to worse valvular disease at 2-years compared to children who receive IM penicillin prophylaxis.
Aim 2: To evaluate the economic equivalence and cost-effectiveness of oral penicillin compared to IM penicillin, after echocardiographic screening for latent RHD detection.
Aim 3: Compare patient-reported outcomes (treatment acceptance, treatment satisfaction, and health-related quality of life) between children receiving oral and IM penicillin prophylaxis.
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
- 5 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Has a new diagnosis of latent RHD detected through primary or secondary school echocardiographic screening.
- •Has agreed to participate in the study via the study's informed consent/assent process.
- •Operational Definition of Latent RHD Borderline RHD or Mild Definite RHD (to include no more than mild regurgitation at the mitral or aortic valve, normal mean mitral and aortic valve gradients, normal bi-ventricular function) according to the 2012 WHF consensus criteria.
排除标准
- •Known history of ARF or RHD
- •Newly diagnosed RHD by echo screening considered to be "missed clinical RHD" as compared to true latent RHD including: > mild pathological valvular regurgitation at the mitral valve or aortic valve, mitral stenosis (mean MV gradient ≥ 4mmHg) (definite B61), aortic stenosis (mean AV gradient ≥ 20mmHg)
- •Structural or functional cardiac defects, other than those consistent with RHD, that were known prior to or detected through echo screening (except patent foramen ovale, small atrial septal defect, small ventricular septal defect, small patent ductus arteriosus)
- •Self-report of prior allergic reaction to penicillin
- •Any known conditions predisposing to thrombocytopenia or hypercoagulability, or other contraindications to intramuscular injection
- •Any known co-morbid conditions (ex. HIV, renal deficiencies, severe malnutrition) that have resulted in prescription of regular antibiotic prophylaxis)
研究组 & 干预措施
Oral Pencillin
Oral phenoxymethyl penicillin (Pen V) prophylaxis 250mg twice daily.
干预措施: phenoxymethyl penicillin (Drug)
IM Penicillin
Intramuscular benzathine benzylpenicillin G (BPG) prophylaxis (600,000 IU for children <30kg, 1.2 million IU for children ≥30kg), every 28 days
干预措施: intramuscular benzathine penicillin G (BPG) prophylaxis (Drug)
结局指标
主要结局
Progression
时间窗: 2 year endpoint
Progression of echocardiographic features of latent RHD to borderline to definite, or definite to mild or definite to moderate/severe
次要结局
- Demonstrate non-inferior costs and cost-effectiveness of oral penicillin compared to IM penicillin(baseline, 6, 12, 18, 24 months post randomization)
- Treatment Satisfaction(baseline, 6, 12, 18, 24 months post randomization)
- Health-Related Quality of Life(baseline, 6, 12, 18, 24 months post randomization)
- Patient-reported outcomes(baseline, 6, 12, 18, 24 months post randomization)
- Obtain cost data associated with the receipt of oral or IM penicillin in the community from a healthcare provider perspective.(2 year endpoint)
- Treatment Acceptance(baseline, 6, 12, 18, 24 months post randomization)