Thiamine DeficiencyNCT05390086
Further Development and Assessment of Tools to Measure Risk Factors for and Treatment of Thiamine Deficiency Disorders
An observational study of Thiamine Deficiency, run by University of California, Davis at 2 sites in Laos.
Trial summary
Thiamine deficiency, including the most severe form infantile beriberi, is a public health concern across South and Southeast Asia, where monotonous diets rely on thiamine-poor white rice. Read more about this study
- Who is it for?
- Ages 21 days to 18 months
- Conditions the study is for
- Thiamine Deficiency
- How many people
- 42 participants
- Where
- 2 sitesLaos
- Study dates
- 15 Jun 2022to 26 Aug 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 21 days to 18 months
- All sexes
- Child
Study population: Infants and young children in the target age range (21 days - <18 months) who are admitted to the collaborating hospitals with at least one of the signs/symptoms suggestive of TDD will be eligible for participation. Mothers of all participating children will be invited to participate in the study.
Who may be able to join
- Infants and young children aged 21 days - <18 months presenting to the collaborating hospitals and presenting with at least one of the following signs and symptoms consistent with TDD:
- Enlarged liver (liver edge >2 cm below the right costal margin)
- Edema
- Tachypnea (respiratory rate >60/min for 3-8 weeks; >50/min for 2-11 months; >40/min for 12-18 months)
- Tachycardia (heart rate >160/min for <12 months; >120/min for 12-18 months)
- Oxygen saturation <92%
Show all 17Show fewer
- Difficulty breathing (chest indrawing or nasal flaring)
- Refusal to breastfeed or of infant formula or food for >24 hours
- Repetitive or recurrent vomiting with no obvious other cause (>3 times in past 24 hours)
- Persistent crying not relieved by soothing or feeding with no obvious other cause
- Hoarse voice/cry or loss of voice
- Nystagmus or other unusual eye movements
- Muscle twitching
- Loss of consciousness
- Convulsion
- Opisthotonos/abnormal posturing
- Acute/flaccid paralysis
Who cannot join
- Infants and young children who do not meet any of the TDD-like signs and symptoms will not be eligible for study participation.
Trial locations and contacts
2 sites in 1 country.
Laos2 sites
Lao Friends Hospital for Children
Luang Prabang
Children's Hospital
Vientiane
About this study
Official title: Further Development and Assessment of Tools to Measure Risk Factors for and Treatment of Thiamine Deficiency Disorders: a Comparison of Rural and Urban Contexts in Lao PDR
Thiamine deficiency, including the most severe form infantile beriberi, is a public health concern across South and Southeast Asia, where monotonous diets rely on thiamine-poor white rice. Food insecurity, food preparation and cooking practices, anti-thiamine compound consumption and culturally determined postpartum food restrictions precipitate thiamine deficiency in these high-risk regions. The risk of thiamine deficiency is highest in the first year of life, especially among exclusively or predominantly breastfed infants of mothers who are thiamine deficient themselves, as thiamine content of breastmilk is related to maternal thiamine status.
However, diagnosis of infantile thiamine deficiency is challenging due to the highly variable, non-specific clinical manifestations, referred to as thiamine deficiency disorders (TDD), that often overlap with other conditions, resulting in misdiagnosis and missed treatment opportunities, which can be fatal or have irreversible consequences. Considering that breastfed infants are at highest risk, a large proportion of infant deaths could be avoided if: 1) infants with TDD were immediately treated with thiamine when medically indicated and, importantly; 2) thiamine deficiency was prevented by improving thiamine status among breastfeeding women. The latter is important given emerging evidence of long-term neurocognitive deficits of severe and even subclinical thiamine deficiency.
In light of these diagnostic uncertainties, it was recognised that a case definition for thiamine responsive disorders (TRD) would help to better identify infants with TDD who would benefit from timely thiamine treatment. This study will test the usefulness of a recently developed case definition for TRD and practical tool in different contexts in Lao PDR where TDD have been reported. Secondly, data on diet, maternal and household risk factors for TDD in different contexts will be used to propose a community risk factor screening tool to better identify populations at risk of thiamine deficiency and help advocate for and guide planning of preventive programmes.
Read the detailed description
The study's primary objective is to determine the usefulness of a TRD case definition and practical tool in identifying infants and young children that would benefit from thiamine treatment in different contexts in Lao PDR, specifically urban Vientiane and rural Luang Prabang. Clinical observers based at the participating hospitals within each country/region will record the presenting signs and symptoms of all children within the target age range (21 days - <18 months) over a one month period. For children who meet the eligibility criteria and informed consent is obtained, information will be collected on duration of hospital stay, received medical treatments, final physician diagnosis and risk factor questionnaires (see below). This data will be used to determine the sensitivity and specificity of the practical TRD case definition tool to accurately identify children who would benefit from thiamine treatment and reduce treatment uncertainties
The secondary objective is to explore risk factors associated with TDD/TRD among infants and young children and their mothers. This will help to identify potential metrics that could be included in a community risk factor screening tool to identify population groups at risk of thiamine deficiency so appropriate preventive interventions can be implemented. Data will be collected on diet and household risk factors for thiamine deficiency, including infant and young child feeding practices, maternal dietary practices, maternal health status and household socioeconomic status and food security.
- Conditions
- Thiamine Deficiency
- Being tested
- Standard of care
- Medical subject headings
- Thiamine Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Standard of careOther
The study is being implemented among children who are seeking care at collaborating hospitals for signs and symptoms suggestive of TDD. All children will be treated by the hospital physicians following the usual hospital procedures. The study will not interfere in any with the administration of required treatments and interventions.
What is being measured
The primary outcome is the main question the study is designed to answer.
Thiamine responsive disorder (TRD)
Presenting signs and symptoms compatible with the thiamine responsive disorders (TRD) case definition and physician diagnosis of beriberi
Measured: From hospital admission to hospital discharge
Secondary outcomes (1)
Risk factors for TDD/TRD
Risk factors related to TDD, TRD and physician diagnosed beriberi
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 May 2022
- Study start15 Jun 2022
- Primary completion26 Aug 2022
- Study completion26 Aug 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thiamine DeficiencyStudies of the same condition, open and under way.
- More trials run by University of California, DavisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Other
- Enrolment
- 42 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, Davis
- Sponsor type
- University, hospital or other
- Collaborators
- Lao Tropical and Public Health Institute
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05390086
- Sponsor's protocol ID
- 1867468
- Status last verified
- Apr 2025
- First submitted
- 16 May 2022
- Last updated
- 23 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: The complete de-identified dataset will be made publicly available.
Study at a glance
From the registry record, last updated by the sponsor on 23 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Further Development and Assessment of Tools to Measure Risk Factors for and Treatment of Thiamine Deficiency Disorders: a Comparison of Rural and Urban Contexts in Lao PDR
- ClinicalTrials.gov ID
- NCT05390086
- Acronym / sponsor ID
- 1867468
- Conditions
- Thiamine Deficiency
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 42 participants (actual)
- Lead sponsor
- University of California, DavisUniversity, hospital or other
- Collaborators
- Lao Tropical and Public Health Institute
- Study start
- 15 Jun 2022 (actual)
- Primary completion
- 26 Aug 2022 (actual)
- Study completion
- 26 Aug 2022 (actual)
- First posted
- 25 May 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Thiamine deficiency, including the most severe form infantile beriberi, is a public health concern across South and Southeast Asia, where monotonous diets rely on thiamine-poor white rice. Food insecurity, food preparation and cooking practices, anti-thiamine compound consumption and culturally determined postpartum food restrictions precipitate thiamine deficiency in these high-risk regions. The risk of thiamine deficiency is highest in the first year of life, especially among exclusively or predominantly breastfed infants of mothers who are thiamine deficient themselves, as thiamine content of breastmilk is related to maternal thiamine status.
However, diagnosis of infantile thiamine deficiency is challenging due to the highly variable, non-specific clinical manifestations, referred to as thiamine deficiency disorders (TDD), that often overlap with other conditions, resulting in misdiagnosis and missed treatment opportunities, which can be fatal or have irreversible consequences. Considering that breastfed infants are at highest risk, a large proportion of infant deaths could be avoided if: 1) infants with TDD were immediately treated with thiamine when medically indicated and, importantly; 2) thiamine deficiency was prevented by improving thiamine status among breastfeeding women. The latter is important given emerging evidence of long-term neurocognitive deficits of severe and even subclinical thiamine deficiency.
In light of these diagnostic uncertainties, it was recognised that a case definition for thiamine responsive disorders (TRD) would help to better identify infants with TDD who would benefit from timely thiamine treatment. This study will test the usefulness of a recently developed case definition for TRD and practical tool in different contexts in Lao PDR where TDD have been reported. Secondly, data on diet, maternal and household risk factors for TDD in different contexts will be used to propose a community risk factor screening tool to better identify populations at risk of thiamine deficiency and help advocate for and guide planning of preventive programmes.
Detailed description
The study's primary objective is to determine the usefulness of a TRD case definition and practical tool in identifying infants and young children that would benefit from thiamine treatment in different contexts in Lao PDR, specifically urban Vientiane and rural Luang Prabang. Clinical observers based at the participating hospitals within each country/region will record the presenting signs and symptoms of all children within the target age range (21 days - <18 months) over a one month period. For children who meet the eligibility criteria and informed consent is obtained, information will be collected on duration of hospital stay, received medical treatments, final physician diagnosis and risk factor questionnaires (see below). This data will be used to determine the sensitivity and specificity of the practical TRD case definition tool to accurately identify children who would benefit from thiamine treatment and reduce treatment uncertainties
The secondary objective is to explore risk factors associated with TDD/TRD among infants and young children and their mothers. This will help to identify potential metrics that could be included in a community risk factor screening tool to identify population groups at risk of thiamine deficiency so appropriate preventive interventions can be implemented. Data will be collected on diet and household risk factors for thiamine deficiency, including infant and young child feeding practices, maternal dietary practices, maternal health status and household socioeconomic status and food security.
Study design
- Observational model
- Case only
- Time perspective
- Other
Who may qualify
- Ages
- 21 days to 18 months
- Sex
- All
- Age groups
- Child
- Study population
- Infants and young children in the target age range (21 days - <18 months) who are admitted to the collaborating hospitals with at least one of the signs/symptoms suggestive of TDD will be eligible for participation. Mothers of all participating children will be invited to participate in the study.
Key inclusion criteria (17 listed)
- Infants and young children aged 21 days - <18 months presenting to the collaborating hospitals and presenting with at least one of the following signs and symptoms consistent with TDD:
Key exclusion criteria (1 listed)
- Infants and young children who do not meet any of the TDD-like signs and symptoms will not be eligible for study participation.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Standard of careThe study is being implemented among children who are seeking care at collaborating hospitals for signs and symptoms suggestive of TDD. All children will be treated by the hospital physicians following the usual hospital procedures. The study will not interfere in any with the administration of required treatments and interventions. | Other | – |
Primary outcome
Thiamine responsive disorder (TRD)
Time frame: From hospital admission to hospital discharge
Presenting signs and symptoms compatible with the thiamine responsive disorders (TRD) case definition and physician diagnosis of beriberi
1 secondary outcome
Risk factors for TDD/TRD
Time frame: Baseline
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Apr 2025.
- Individual participant dataSharing planned: Yes. The complete de-identified dataset will be made publicly available.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Thiamine Deficiency, run by University of California, Davis at 2 sites in Laos.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Laos. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Thiamine responsive disorder (TRD) (time frame: From hospital admission to hospital discharge). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Apr 2025.