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

NCT05693545 | Active Not Recruiting Phase 2 DMC

• 1 other identifier: 2022-0892
• Study Type: interventional
• Target: 1,004
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Rheumatic Heart Disease

Outcome Measures

Primary Outcomes (1)

• Progression

  Progression of echocardiographic features of latent RHD to borderline to definite, or definite to mild or definite to moderate/severe

  2 year endpoint

Secondary Outcomes (6)

• Demonstrate non-inferior costs and cost-effectiveness of oral penicillin compared to IM penicillin

  baseline, 6, 12, 18, 24 months post randomization

• Obtain cost data associated with the receipt of oral or IM penicillin in the community from a healthcare provider perspective.

  2 year endpoint

• Treatment Acceptance

  baseline, 6, 12, 18, 24 months post randomization

• Treatment Satisfaction

  baseline, 6, 12, 18, 24 months post randomization

• Health-Related Quality of Life

  baseline, 6, 12, 18, 24 months post randomization

Study Arms (2)

Oral Pencillin

EXPERIMENTAL

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

Drug: phenoxymethyl penicillin

IM Penicillin

ACTIVE COMPARATOR

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

Drug: intramuscular benzathine penicillin G (BPG) prophylaxis

Interventions

phenoxymethyl penicillin DRUG

Prophylaxis

Also known as: Pen V

Oral Pencillin

intramuscular benzathine penicillin G (BPG) prophylaxis DRUG

Prophylaxis

Also known as: benzathine penicillin G, BPG, penicillin prophylaxis

IM Penicillin

Eligibility Criteria

• Age: 5 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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)

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead
• Uganda Heart Institute: collaborator

Study Sites (1)

Uganda Heart Institute

Kampala, Uganda, Uganda

Location

Related Publications (1)

• Rwebembera J, Ndagire E, Carvalho N, Webel AR, Sable C, Okello E, Sarnacki R, Spaziani AM, Mucunguzi A, Engelman D, Grobler A, Steer A, Beaton A. Intramuscular versus enteral penicillin prophylaxis to prevent progression of rheumatic heart disease: Study protocol for a noninferiority randomized trial (the GOALIE trial). Am Heart J. 2024 Sep;275:74-85. doi: 10.1016/j.ahj.2024.05.012. Epub 2024 May 24.

  PMID: 38797460 DERIVED

MeSH Terms

Conditions

Rheumatic Heart Disease

Interventions

Penicillin V; Penicillin G

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Penicillins; beta-Lactams; Lactams; Amides; Organic Chemicals; Sulfur Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds

Study Officials

• Andrea Z Beaton

  Cincinnati Chidren's hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 4, 2023
• First Posted: January 23, 2023
• Study Start: September 1, 2023
• Primary Completion (Estimated): March 31, 2027
• Study Completion (Estimated): September 30, 2027
• Last Updated: March 17, 2026

Record last verified: 2026-03

Locations

UG (1)