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

NCT ID: NCT07146048

Last Updated: 2025-12-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NOT_YET_RECRUITING

Clinical Phase

PHASE3

Total Enrollment

922 participants

Study Classification

INTERVENTIONAL

Study Start Date

2026-02-28

Study Completion Date

2029-12-31

Brief Summary

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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).

Detailed Description

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Conditions

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Rheumatic Heart Disease

Keywords

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Rheumatic heart disease global health secondary prophylaxis children

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Outcome Assessors
Cardiologists who make up the adjudication panel.

Study Groups

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Stop prophylaxis

The intervention arm will not receive prophylaxis.

Group Type NO_INTERVENTION

No interventions assigned to this group

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.

Group Type ACTIVE_COMPARATOR

Secondary Prophylaxis

Intervention Type DRUG

Continue standard of care with continued secondary antibiotic prophylaxis

Interventions

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Secondary Prophylaxis

Continue standard of care with continued secondary antibiotic prophylaxis

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

5 Years

Maximum Eligible Age

20 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Uganda Heart Institute

OTHER

Sponsor Role collaborator

Open Philanthropy

OTHER

Sponsor Role collaborator

Thrasher Research Fund

OTHER

Sponsor Role collaborator

Children's Hospital Medical Center, Cincinnati

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Related Links

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https://www.rrcuganda.org/

Related Info

Other Identifiers

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2025-0604

Identifier Type: -

Identifier Source: org_study_id