A Non-Inferiority Trial of Stopping Penicillin in Early Rheumatic Heart Disease: GOAL-Stop
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 922
- 试验地点
- 1
- 主要终点
- Presence of echocardiographic progression of RHD at two years
研究概览
简要总结
GOAL-Stop is a randomized, controlled, non-inferiority trial designed to evaluate whether discontinuing secondary antibiotic prophylaxis (SAP) is non-inferior to continuing SAP in preventing progression of rheumatic heart disease (RHD) among children and adolescents. The trial will enroll participants aged 5-20 years with previously diagnosed mild RHD who have received at least 2 years of SAP and who demonstrate either echocardiographic normalization or stability (persistent mild RHD). Participants will be randomized to either continue SAP or discontinue SAP for 2 years. The primary outcome is echocardiographic progression of RHD at 2 years, assessed by blinded adjudicators using the 2023 World Heart Federation criteria. Subgroup analyses will evaluate outcomes in participants with echocardiographic normalization versus stable mild RHD, and an exploratory analysis will assess whether outcomes differ by prior prophylaxis route (oral vs. intramuscular).
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)
盲法说明
Cardiologists who make up the adjudication panel.
入排标准
- 年龄范围
- 5 Years 至 20 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participated in GOALIE
- •Concluded GOALIE with either echocardiographic normalization or stable mild RHD
- •Between ages 5-20 years at time of enrollment
排除标准
- •RHD Stage C/D at GOALIE end of study echocardiogram
- •Relocation to extremely distant residence or school
- •Clinically documented penicillin allergy
研究组 & 干预措施
Continue prophylaxis
The control arm will receive intramuscular benzathine benzylpenicillin G (BPG) prophylaxis (600,000 IU for children <30kg, 1.2 million IU for children ≥30kg), every 28 days for a 2-year period. The standard of care for RHD is BPG injections which is why the control arm is continuation of monthly injections.
干预措施: Secondary Prophylaxis (Drug)
Stop prophylaxis
The intervention arm will not receive prophylaxis.
结局指标
主要结局
Presence of echocardiographic progression of RHD at two years
时间窗: Enrollment to the end of treatment at 2 years
The Primary Outcome will be the presence of echocardiographic progression of RHD at two years, defined by the 2023 World Heart Federation Criteria (none, mild RHD (stage A/B), or moderate/severe RHD (stage C/D).
次要结局
- Non-inferiority among sub-groups(Enrollment to the end of 2 years participation)