Rheumatic Heart Disease
Conditions
Keywords
Rheumatic heart disease, global health, secondary prophylaxis, children
Brief summary
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).
Interventions
DRUGSecondary Prophylaxis
Continue standard of care with continued secondary antibiotic prophylaxis
Sponsors
Children's Hospital Medical Center, Cincinnati
Uganda Heart Institute
Open Philanthropy
Thrasher Research Fund
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Outcomes Assessor)
Masking description
Cardiologists who make up the adjudication panel.
Eligibility
Sex/Gender
ALL
Age
5 Years to 20 Years
Healthy volunteers
No
Inclusion criteria
* Participated in GOALIE * Concluded GOALIE with either echocardiographic normalization or stable mild RHD * Between ages 5-20 years at time of enrollment
Exclusion criteria
* RHD Stage C/D at GOALIE end of study echocardiogram * Relocation to extremely distant residence or school * Clinically documented penicillin allergy
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Presence of echocardiographic progression of RHD at two years | 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). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Non-inferiority among sub-groups | Enrollment to the end of 2 years participation | Number of participants who show echocardiographic progression (2023 World Heart Federation Criteria) between those with echocardiographic normalization and those with stabilized mild RHD |
Countries
Uganda
Contacts
CONTACTNdate Fall, PhD
PRINCIPAL_INVESTIGATORAndrea Z Beaton, Dr
Children's Hospital Medical Center, Cincinnati
Outcome results
None listed