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NCT Number: NCT07146048

A Non-Inferiority Trial of Stopping Penicillin in Early Rheumatic Heart Disease: GOAL-Stop

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).

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Key information

Conditions

Rheumatic Heart Disease Bacterial Infections Bacterial Infections and Mycoses Cardiovascular Diseases Gram-Positive Bacterial Infections Heart Diseases Infections Rheumatic Fever Streptococcal Infections

Age range

5 year–20 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Secondary Prophylaxis

Drug

Continue standard of care with continued secondary antibiotic prophylaxis

Primary outcomes

  1. Presence of echocardiographic progression of RHD at two years

    Time frame: 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 outcomes

  1. Non-inferiority among sub-groups

    Time frame: 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

Other outcomes

  1. Exploratory sub-analysis

    Time frame: enrollment to the end of 2 years of participation

    Number of echocardiographic progression outcomes based on the initial type of prophylaxis during the GOALIE Trial, oral prophylaxis vs. intramuscular prophylaxis.

Study contacts

Contact information is provided by the study sponsor or research team.

Ndate Fall, PhD

CONTACT

[email protected]

15135171327

Sponsors and collaborators

Lead sponsor

Children's Hospital Medical Center, Cincinnati

Other

Collaborators

  • Open Philanthropy
  • Thrasher Research Fund
  • Uganda Heart Institute

Registry information

Acronym: GOAL-Stop

Important dates

Study start
2026
Primary completion
2029
Study completion
2029
First posted
Aug 28, 2025
Registry last updated
Jun 22, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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