A Long-term Follow up Study for Patients Who Participated in the GOAL Trial (GOAL-Post)

Sponsor

Children's Hospital Medical Center, Cincinnati (Other)

Overall Status

Recruiting

CT.gov ID

NCT05211024

Collaborator

Uganda Heart Institute, Kampala, Uganda (Other), Murdoch Children's Research Institute, Melbourne Australia (Other)

1,102

Enrollment

Location

28.1

Anticipated Duration (Months)

39.2

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

GOAL-Post is comprised of two studies that will help refine recommendations for the duration of secondary prophylaxis for children diagnosed with latent RHD, which is currently not known

Condition or Disease	Intervention/Treatment	Phase
Rheumatic Heart Disease	Drug: Penicillin G Benzathine

Detailed Description

GOAL-Post is comprised of two studies that will help refine recommendations for the duration of secondary prophylaxis for children diagnosed with latent RHD, which is currently not known.

Aim 1: Compare the two-year risk of developing RHD (borderline or definite) between children and adolescents who completed the GOAL Trial with a normal echocardiogram (prior diagnosis of latent RHD) and age/sex/and geographically matched controls with repeated normal echocardiograms (normal in both the original GOAL screening in 2017/2018 and in the planned GOAL-Post screening in 2021).

Aim 2: Determine the five-year rate of RHD progression and regression among children with persistent latent RHD who receive secondary antibiotic prophylaxis (medium-term impact of prophylaxis). Five years includes time from initial GOAL enrollment to the end of GOAL-Post.

Aim 3: Create a RHD biobank that will support further research on RHD genetic susceptibility and pathophysiology

Study Design

Study Type:

Observational

Anticipated Enrollment :

1102 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

A Follow-up Study for Patients Who Participated in the GOAL Trial (GOAL-Post)

Actual Study Start Date :

Jan 28, 2022

Anticipated Primary Completion Date :

Feb 1, 2024

Anticipated Study Completion Date :

Jun 1, 2024

Arms and Interventions

Arm	Intervention/Treatment
Aim 1: Group A Children and adolescent who are prior GOAL participants who have a normal echocardiogram at the 2 year endpoint and are no receiving secondary antibiotic prophylaxis
Aim 1 Group B Children and adolescent who have a normal echocardiogram at the start of the study
Aim 2 Children and adolescent who are prior GOAL participants who have persistent latent RHD on echocardiogram at the 2 year endpoint.	Drug: Penicillin G Benzathine Participants in Aim 2 are receiving 28 days interval BPG intramuscular injection as part of standard of care

Arm

Intervention/Treatment

Aim 1: Group A

Children and adolescent who are prior GOAL participants who have a normal echocardiogram at the 2 year endpoint and are no receiving secondary antibiotic prophylaxis

Aim 1 Group B

Children and adolescent who have a normal echocardiogram at the start of the study

Aim 2

Children and adolescent who are prior GOAL participants who have persistent latent RHD on echocardiogram at the 2 year endpoint.

Drug: Penicillin G Benzathine

Participants in Aim 2 are receiving 28 days interval BPG intramuscular injection as part of standard of care

Outcome Measures

Primary Outcome Measures

Progression [2 year endpoint]
Progression of echocardiographic features of latent RHD to borderline to definite, or definite to mild or definitive to moderate/severe
Regression [2 year endpoint]
Regression of echocardiographic features of latent RHD

Eligibility Criteria

Criteria

Ages Eligible for Study:

5 Years to 17 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Yes

Inclusion Criteria:

Aim 1: Group A: Children and adolescents will be eligible for Aim 1 if they (1) are a prior GOAL participant deemed by the adjudication panel to have a normal echocardiogram at the 2-year endpoint, (2) are not receiving secondary antibiotic prophylaxis, and (3) agree to participate in the study via the study's informed consent/assent process.

Group B: Children and adolescents will be eligible for Aim 1 if they (1) have a normal echocardiogram at the start of the study, (2) Meet the age/sex/geographic match requirement (from former GOAL participants), and (3) have agreed to participate in the study via the study's informed consent/assent process.

Aim 2: Children and adolescents will be eligible for Aim 2 if they (1) are a prior GOAL participant deemed by the adjudication panel to have persistent latent RHD on echocardiogram at the 2-year endpoint, (2) have been prescribed secondary antibiotic prophylaxis, and (3) agree to participate in the study via the study's informed consent/assent process.

Aim 3: Children and adolescents will be eligible for Aim 3 if they are prior GOAL participants deemed by the adjudication panel to (1) have a normal echocardiogram at the 2-year endpoint (Aim 1 participants), (2) deemed by the adjudication panel to have persistent latent RHD at the 2-year endpoint (Aim 2 participants), or (3) deemed by the adjudication panel to have moderate/severe RHD at the 2-year endpoint. Aim 3 will include a separate consent/assent and participants will be able to participate in each aim independently.

Exclusion Criteria:

Aim 1: Group A: Residence or school is no longer in Gulu District or one of the surrounding districts.

Group B: Known history of ARF or RHD, or evidence of RHD on baseline echocardiogram or 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).

Aim 2 + 3: Residence or school is no longer in Gulu District or one of the surrounding districts.