An observational study in Thiamine Deficiency, sponsored by University of California, Davis. Completed at 1 site in Lao People's Democratic Republic. Open to participants aged 21 Days to 17 Months, including healthy volunteers. Per ClinicalTrials.gov, last updated 2022-08-15.
Sponsored by University of California, Davis · Observational
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.
The study's 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 infants's mothers to assess maternal thiamine status, and among breast feeding mothers a breastmilk sample for assessment of thiamine concentration.
18 studies on the registry are indexed under Thiamine Deficiency; 1 is open to participants now.
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Hospitalized children:
Infants and children in the target age range (21 days to \<18 months), who are admitted to the participating hospitals, will be screened by hospital staff to determine the presence of at least one of the inclusion criteria
Community-based cohort:
A sex-, age- and regionally-similar comparison group will be enrolled based on the characteristics of the hospitalized group.
Mothers of all participating infants will be invited to the study.
Inclusion criteria of hospital-based children:
Exclusion criteria of hospital-based children:
Inclusion criteria of community-based children, who will be frequency-matched based on sex, age and residence to hospital-based participants :
Exclusion criteria of community-based children:
Inclusion criteria of participants' mothers:
Exclusion criteria of participants' mothers:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
100 mg thiamine provided via intramuscular and/or intravenous injection (Thiamine 100 MG/ML) daily for 3 days
Drug: Thiamine 100 MG/ML
Sex-, age- and regionally matched comparison group
Drug page: Thiamine
100 mg thiamine provided as intramuscular injection
Also known as: Standard of care
Thiamine Responsive Disorder (TRD)
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
Time frame: 48-72 hours
Biomarkers of whole blood thiamine diphosphate (ThDP) and erythrocyte transketolase activity coefficient (ETKac)
Associations between ThDP and ETKac with TRD will be determined and appropriate cut-offs of these biomarkers suggesting TRD will be proposed
Time frame: Baseline
Hemoglobin and biomarkers of other micronutrients (ferritin, transferrin receptor, retinol binding protein, erythrocyte glutathione reductase activation coefficient (EGRAC)), and inflammation (CRP, AGP)
Nutrition and health status will be assessed to explore potential risk factors for TRD
Time frame: Baseline
Plan to share: Yes — The complete de-identified dataset will be made publically available. Associated data dictionaries will be made available along with the datasets.
Supporting information: Study protocol, Statistical analysis plan, Informed consent form
Time frame: Within 3 years after completion of data collection
Ran from Jun 2019 to primary completion in Feb 2021, 20 months.
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This study is completed, as verified in Aug 2022. You cannot join it, but the record below documents what was studied.
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