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

NCT ID: NCT05693545

Last Updated: 2024-12-12

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

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

1004 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-09-01

Study Completion Date

2027-09-30

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Rheumatic Heart Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

SINGLE

Outcome Assessors

Study Groups

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Oral Pencillin

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

Group Type EXPERIMENTAL

phenoxymethyl penicillin

Intervention Type DRUG

Prophylaxis

IM Penicillin

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

Group Type ACTIVE_COMPARATOR

intramuscular benzathine penicillin G (BPG) prophylaxis

Intervention Type DRUG

Prophylaxis

Interventions

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phenoxymethyl penicillin

Prophylaxis

Intervention Type DRUG

intramuscular benzathine penicillin G (BPG) prophylaxis

Prophylaxis

Intervention Type DRUG

Other Intervention Names

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Pen V benzathine penicillin G, BPG, penicillin prophylaxis

Eligibility Criteria

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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)
Minimum Eligible Age

5 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Uganda Heart Institute

OTHER

Sponsor Role collaborator

Children's Hospital Medical Center, Cincinnati

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Andrea Z Beaton

Role: PRINCIPAL_INVESTIGATOR

Cincinnati Chidren's hospital

Locations

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Uganda Heart Institute

Kampala, , Uganda

Site Status RECRUITING

Countries

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Uganda

Central Contacts

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Ndate Fall, MS RN

Role: CONTACT

Phone: 15135171327

Email: [email protected]

Mary Banks

Role: CONTACT

Email: [email protected]

Facility Contacts

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Emmy H Okello

Role: primary

References

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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.

Reference Type DERIVED
PMID: 38797460 (View on PubMed)

Other Identifiers

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2022-0892

Identifier Type: -

Identifier Source: org_study_id