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).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
922
Continue standard of care with continued secondary antibiotic prophylaxis
Presence of echocardiographic progression of RHD at two 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).
Time frame: Enrollment to the end of treatment at 2 years
Non-inferiority among sub-groups
Number of participants who show echocardiographic progression (2023 World Heart Federation Criteria) between those with echocardiographic normalization and those with stabilized mild RHD
Time frame: Enrollment to the end of 2 years participation
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