GOALIE: Intramuscular vs. Enteral Penicillin Prophylaxis to Prevent Progression of Latent RHD Trial

Cincinnati Children's Hospital Medical Center

Status and phase: Enrolling; Phase 2

Conditions: Rheumatic Heart Disease

Treatments: Drug: phenoxymethyl penicillin; Drug: intramuscular benzathine penicillin G (BPG) prophylaxis

Study type: Interventional

Funder types: Other

Identifiers

NCT05693545

2022-0892

Details and patient eligibility

About

The goal of this clinical trial is to determine if twice daily oral penicillin prophylaxis is non-inferior to monthly IM penicillin prophylaxis in preventing latent Rheumatic Heart Disease Progression in children between the ages of 5-17 years. The main objective is to compare the proportion of children aged 5-17 years with latent RHD receiving oral penicillin prophylaxis who progress to worse valvular disease at 2-years compared to children who receive IM penicillin prophylaxis.

Full description

GOALIS is a randomized controlled trial developed to provide high quality contemporary evidence on the efficacy of oral penicillin as RHD prophylaxis.

Aim 1: To compare the proportion of children aged 5-17 years with latent RHD receiving oral penicillin prophylaxis who progress to worse valvular disease at 2-years compared to children who receive IM penicillin prophylaxis.

Aim 2: To evaluate the economic equivalence and cost-effectiveness of oral penicillin compared to IM penicillin, after echocardiographic screening for latent RHD detection.

Aim 3: Compare patient-reported outcomes (treatment acceptance, treatment satisfaction, and health-related quality of life) between children receiving oral and IM penicillin prophylaxis.

Enrollment: 1,004 estimated patients

Sex: All

Ages: 5 to 17 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Has a new diagnosis of latent RHD detected through primary or secondary school echocardiographic screening.
• Has agreed to participate in the study via the study's informed consent/assent process.

Operational Definition of Latent RHD Borderline RHD or Mild Definite RHD (to include no more than mild regurgitation at the mitral or aortic valve, normal mean mitral and aortic valve gradients, normal bi-ventricular function) according to the 2012 WHF consensus criteria.

Exclusion criteria

• Known history of ARF or RHD
• Newly diagnosed RHD by echo screening considered to be "missed clinical RHD" as compared to true latent RHD including: > mild pathological valvular regurgitation at the mitral valve or aortic valve, mitral stenosis (mean MV gradient ≥ 4mmHg) (definite B61), aortic stenosis (mean AV gradient ≥ 20mmHg)
• Structural or functional cardiac defects, other than those consistent with RHD, that were known prior to or detected through echo screening (except patent foramen ovale, small atrial septal defect, small ventricular septal defect, small patent ductus arteriosus)
• Self-report of prior allergic reaction to penicillin
• Any known conditions predisposing to thrombocytopenia or hypercoagulability, or other contraindications to intramuscular injection
• Any known co-morbid conditions (ex. HIV, renal deficiencies, severe malnutrition) that have resulted in prescription of regular antibiotic prophylaxis)

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

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1,004 participants in 2 patient groups

Oral Pencillin

Experimental group

Description:

Oral phenoxymethyl penicillin (Pen V) prophylaxis 250mg twice daily.

Treatment:

Drug: phenoxymethyl penicillin

IM Penicillin

Active Comparator group

Description:

Intramuscular benzathine benzylpenicillin G (BPG) prophylaxis (600,000 IU for children \<30kg, 1.2 million IU for children ≥30kg), every 28 days

Treatment:

Drug: intramuscular benzathine penicillin G (BPG) prophylaxis