Thiamine Responsive Disorders (TRD) Among Infants in Lao PDR
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|ClinicalTrials.gov Identifier: NCT03626337|
Recruitment Status : Recruiting
First Posted : August 13, 2018
Last Update Posted : June 26, 2019
|Condition or disease||Intervention/treatment|
|Thiamine Deficiency||Drug: Thiamine 100 MG/ML|
The study will address the following questions:
- Among infants with symptoms consistent with thiamine deficiency disorder (TDD), which clinical symptoms and risk factors distinguish those who respond positively to thiamine administration from those who do not respond?
- Among young infants, which biomarkers of thiamine status and cut-offs of these biomarkers are associated with thiamine responsive disorder?
To answer the first question, the investigators will develop and validate clinical prediction models that will use clinical signs and risk factors that can be reliably measured early in patients diagnosed with suspected thiamine deficiency to predict which patients would respond favorably to thiamine supplementation. Such validated clinical prediction rules would be of value in making thiamine treatment decisions and in identifying patient characteristics with prognostic significance. To answer the second question, investigators will contrast TRD responders to hospitalized non-TRD infants and to a community-based control group, to compare the distribution of patient characteristics, other risk factors (exposures) and biomarkers of thiamine status. The investigators will also develop multiple logistic regression models that use selected patient characteristics and exposures. These results would identify characteristics that may more clearly distinguish individuals that have TDD-like symptoms that would respond favorably to thiamine supplementation from individuals in the same community who do not have TDD-like symptoms. These characteristics may be of value in targeting thiamine screening and supplementation efforts in low-resource settings.
Infants in the target age range, who are admitted to the participating hospital, will be screened by hospital 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 infants who would respond clinically to thiamine administration to correct the deficiency. If a child is in the target age range (21 days to <12 months) meets any one of the inclusion criteria, parental consent will be obtained and infants 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, namely 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 infants and infants 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 infants's mothers to assess maternal thiamine status, and among breast feeding mothers a breastmilk sample for assessment of thiamine concentration.
|Study Type :||Observational|
|Estimated Enrollment :||927 participants|
|Official Title:||A Study to Establish a Case Definition of Thiamine Responsive Disorders (TRD) Among Infants in Lao PDR|
|Actual Study Start Date :||June 17, 2019|
|Estimated Primary Completion Date :||June 16, 2020|
|Estimated Study Completion Date :||June 16, 2020|
100 mg thiamine provided via intramuscular and/or intravenous injection (Thiamine 100 MG/ML) daily for 3 days
Drug: Thiamine 100 MG/ML
100 mg thiamine provided as intramuscular injection
Other Name: Standard of care
Sex-, age- and regionally matched control group
- Thiamine Responsive Disorder (TRD) [ Time Frame: 48-72 hours ]Diagnosis of TRD will be determined based on improvements of initially abnormal physical findings such as hepatomegaly, heart rate, and respiratory rate, and resolution of echocardiographic findings of enlarged and poorly functioning ventricles
- Biomarkers of whole blood thiamine diphosphate (ThDP) and erythrocyte transketolase activity coefficient (ETKac) [ Time Frame: Baseline ]Associations between ThDP and ETKac with TRD will be determined and appropriate cut-offs of these biomarkers suggesting TRD will be proposed
Biospecimen Retention: Samples Without DNA
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03626337
|Contact: Sonja Y Hess, PhD||530 752 email@example.com|
|Lao People's Democratic Republic|
|Lao Friends Hospital for Children||Recruiting|
|Luang Prabang, Lao People's Democratic Republic|
|Contact: Indi Trehan, MD firstname.lastname@example.org|
|Principal Investigator:||Sengchanh Kounnavong, MD, PhD||Lao Tropical and Public Health Institute|