Ignite Creation Date:
2024-05-06 @ 11:53 AM
Last Modification Date:
2024-10-26 @ 12:51 PM
Study NCT ID:
NCT03626337
Status:
COMPLETED
Last Update Posted:
2022-08-15
First Post:
2018-07-17
Brief Title:
Thiamine Responsive Disorders TRD Among Infants in Lao PDR
Sponsor:
University of California Davis
Organization:
University of California Davis
Study Overview
Official Title:
A Study to Establish a Case Definition of Thiamine Responsive Disorders TRD Among Infants in Lao PDR
Status:
COMPLETED
Status Verified Date:
2022-08
Last Known Status:
None
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:
None
Brief Summary:
A hospital- and community-based study in Luang Prabang Lao PDR which will include a group of hospitalized children 21 days to 18 months of age who are diagnosed with symptoms compatible with thiamine deficiency disorder TDD Based on the infants response to thiamine administration children will be defined as either thiamine responsive disorder TRD cases or non-responders A community-based comparison group of infants in the same age range will be included in the study to serve as a control group for identification of potential risk factors
Detailed Description:
The studys primary objective is the development of a case definition for TRD among infants and young children with symptoms consistent with TDD with the case definition being based on those clinical symptoms and other predictors that we find are better able to distinguish those who respond positively to thiamine administration from those who do not respond As diagnostic tools available to treating physicians may differ by setting we will repeat analyses after excluding from the set of candidate predictors those that are derived from assessments only available in higher-resource settings such as laboratory biomarkers and ultrasonography
A secondary objective is to fill the knowledge gap surrounding biomarkers of thiamine status in at-risk populations Biomarker cut-offs for TRD will be developed in the hospital cohort and distributions will be characterised in both the hospital and community cohorts The performance of our proposed cut-off will be compared to the performance of existing literature cut-offs
Additionally all identified predictors and biomarker cut-offs will be compared across the community cohort non-TRD hospital cohort and TRD hospital cohort to assess the prevalence of risk factors among apparently healthy infants and young children and assess the usefulness of the TRD case definition in various settings
Infants and children in the target age range who are seeking care at the collaborating hospital will be screened by study staff to determine the presence of any of the inclusion criteria The list of inclusion criteria has been developed based on a broad range of TDD-compatible symptoms to reduce the risk of potentially missing children who would respond clinically to thiamine administration to correct the deficiency If a child is in the target age range 21 days to 18 months meets any one of the inclusion criteria parental consent will be obtained and children will be referred to a study physician for a detailed physical exam An echocardiogram and cranial ultrasound will be performed to explore the complete range of TDD complications A venous blood sample will be obtained by venipuncture for assessment of indicators of thiamine status The data collection will follow a structured timeline after the first thiamine dose has been administered In particular the thiamine administration will be defined as hour zero and the above described physical exam will be repeated 4 8 12 24 36 48 and 72 hours after the initial thiamine administration
Blood samples will be analyzed for whole blood thiamine diphosphate ThDP and erythrocyte transketolase activity coefficient ETKac inflammation and cardiac biomarkers and for a complete blood count CBC The purpose of determining these indicators is to better describe the TRD cases and explore differences between TRD cases non-TRD children and children in the community with the ultimate goal that these indicators may be useful for screening in the future Moreover to determine the association between TRD and maternal thiamine status as a potential risk factor investigators will collect a blood sample from infantss mothers to assess maternal thiamine status and among breast feeding mothers a breastmilk sample for assessment of thiamine concentration
A secondary objective is to fill the knowledge gap surrounding biomarkers of thiamine status in at-risk populations Biomarker cut-offs for TRD will be developed in the hospital cohort and distributions will be characterised in both the hospital and community cohorts The performance of our proposed cut-off will be compared to the performance of existing literature cut-offs
Additionally all identified predictors and biomarker cut-offs will be compared across the community cohort non-TRD hospital cohort and TRD hospital cohort to assess the prevalence of risk factors among apparently healthy infants and young children and assess the usefulness of the TRD case definition in various settings
Infants and children in the target age range who are seeking care at the collaborating hospital will be screened by study staff to determine the presence of any of the inclusion criteria The list of inclusion criteria has been developed based on a broad range of TDD-compatible symptoms to reduce the risk of potentially missing children who would respond clinically to thiamine administration to correct the deficiency If a child is in the target age range 21 days to 18 months meets any one of the inclusion criteria parental consent will be obtained and children will be referred to a study physician for a detailed physical exam An echocardiogram and cranial ultrasound will be performed to explore the complete range of TDD complications A venous blood sample will be obtained by venipuncture for assessment of indicators of thiamine status The data collection will follow a structured timeline after the first thiamine dose has been administered In particular the thiamine administration will be defined as hour zero and the above described physical exam will be repeated 4 8 12 24 36 48 and 72 hours after the initial thiamine administration
Blood samples will be analyzed for whole blood thiamine diphosphate ThDP and erythrocyte transketolase activity coefficient ETKac inflammation and cardiac biomarkers and for a complete blood count CBC The purpose of determining these indicators is to better describe the TRD cases and explore differences between TRD cases non-TRD children and children in the community with the ultimate goal that these indicators may be useful for screening in the future Moreover to determine the association between TRD and maternal thiamine status as a potential risk factor investigators will collect a blood sample from infantss mothers to assess maternal thiamine status and among breast feeding mothers a breastmilk sample for assessment of thiamine concentration
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?:
None
Is an FDA AA801 Violation?:
None