Ignite Creation Date:
2024-05-06 @ 10:45 AM
Last Modification Date:
2024-10-26 @ 12:35 PM
Study NCT ID:
NCT03346525
Status:
UNKNOWN
Last Update Posted:
2019-10-01
First Post:
2017-11-15
Brief Title:
Determining the Impact of Penicillin in Latent RHD The GOAL Trial
Sponsor:
Childrens National Research Institute
Organization:
Childrens National Research Institute
Study Overview
Official Title:
The GOAL GwokO Adunu pa Lutino Trial Determining the Impact of Penicillin on Latent Rheumatic Heart Disease A Randomized Controlled Trial in Uganda
Status:
UNKNOWN
Status Verified Date:
2019-09
Last Known Status:
ACTIVE_NOT_RECRUITING
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
GOAL
Brief Summary:
Rheumatic heart disease RHD affects at least 329 million people mostly children living in low-resource settings Long-term intramuscular benzathine penicillin G BPG prophylaxis is proven to prevent progression of chronic valve changes in patients with established rheumatic heart disease RHD and to allow regression of valve changes in patients with a history of acute rheumatic fever ARF with mild RHD However in low-resource settings ARF is an elusive diagnosis and most patients 85 are diagnosed only when RHD is severe and irreversible medications ineffective and surgical intervention is expensive andor unavailable
Identification of latent RHD might be an opportunity to substantially reduce RHD morbidity and mortality However detection of latent RHD is only important if outcomes are improved The appropriate management of children with latent RHD is unknown and no formal recommendations exist While some clinicians prescribe penicillin prophylaxis for children with latent RHD clinical equipoise exists regarding the best practice
To fill this gap the investigators propose a randomized controlled trial in children with latent RHD to evaluate the efficacy of BPG prophylaxis compared to no prophylaxis Our primary outcome measure is progression of valvular changes on echocardiogram at 2 years A sample size of 916 children is needed to detect a 50 reduction of progression expected range 75-125 progression in BPG-arm vs 15-25 progression in control-arm with 90 power
AIM 1 To compare the proportion of children aged 5-17 years with latent RHD receiving BPG prophylaxis who progress to worse valvular disease at 2-years compared to children not receiving BPG prophylaxis
Hypothesis 1 Prophylaxis with BPG will result in fewer children with latent RHD showing progression of echocardiographic valve changes at 2 years compared to children with latent RHD not receiving BPG prophylaxis The investigators expect at least a 50 relative reduction in progression in the BPG arm range 15-25 control arm vs 75-125 BPG-arm
AIM 2 To compare the proportion of children aged 5-17 years with latent RHD receiving BPG prophylaxis who regress to improved valvular disease at 2-years compared to children not receiving BPG prophylaxis
Hypothesis 2 Prophylaxis with BPG will result in more children with latent RHD showing regression of echocardiographic valve changes by 2 years compared to children with latent RHD not receiving BPG prophylaxis The investigators expect at least a 50 relative increase in regression in the BPG arm range 10-20 control arm vs 20-40 BPG arm
This study is highly significant because it will establish if BPG prophylaxis improves outcomes for children with latent RHD Feasibility will be ensured through the experience resources community support and accessible patient population of our investigational team The results of our study will have high impact immediately informing international policy on the standard of care for children diagnosed with latent RHD and shaping over 2-3 years practical and scalable programs that could substantially decrease the global burden of RHD
Identification of latent RHD might be an opportunity to substantially reduce RHD morbidity and mortality However detection of latent RHD is only important if outcomes are improved The appropriate management of children with latent RHD is unknown and no formal recommendations exist While some clinicians prescribe penicillin prophylaxis for children with latent RHD clinical equipoise exists regarding the best practice
To fill this gap the investigators propose a randomized controlled trial in children with latent RHD to evaluate the efficacy of BPG prophylaxis compared to no prophylaxis Our primary outcome measure is progression of valvular changes on echocardiogram at 2 years A sample size of 916 children is needed to detect a 50 reduction of progression expected range 75-125 progression in BPG-arm vs 15-25 progression in control-arm with 90 power
AIM 1 To compare the proportion of children aged 5-17 years with latent RHD receiving BPG prophylaxis who progress to worse valvular disease at 2-years compared to children not receiving BPG prophylaxis
Hypothesis 1 Prophylaxis with BPG will result in fewer children with latent RHD showing progression of echocardiographic valve changes at 2 years compared to children with latent RHD not receiving BPG prophylaxis The investigators expect at least a 50 relative reduction in progression in the BPG arm range 15-25 control arm vs 75-125 BPG-arm
AIM 2 To compare the proportion of children aged 5-17 years with latent RHD receiving BPG prophylaxis who regress to improved valvular disease at 2-years compared to children not receiving BPG prophylaxis
Hypothesis 2 Prophylaxis with BPG will result in more children with latent RHD showing regression of echocardiographic valve changes by 2 years compared to children with latent RHD not receiving BPG prophylaxis The investigators expect at least a 50 relative increase in regression in the BPG arm range 10-20 control arm vs 20-40 BPG arm
This study is highly significant because it will establish if BPG prophylaxis improves outcomes for children with latent RHD Feasibility will be ensured through the experience resources community support and accessible patient population of our investigational team The results of our study will have high impact immediately informing international policy on the standard of care for children diagnosed with latent RHD and shaping over 2-3 years practical and scalable programs that could substantially decrease the global burden of RHD
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| EW Al Thrasher Award | OTHER | Thrasher Research Fund | None |