A Method Characterization Study to Evaluate Thermoregulatory Sweat Production in Healthy Volunteers and Patients With ROSAH Syndrome)
A Method Characterization Study to Evaluate Thermoregulatory Sweat Production in Healthy Volunteers and Patients With ROSAH Syndrome
Panoramica dello studio
Stato
Stato
Condizioni
Condizioni
Intervento / Trattamento
Intervento / Trattamento
Descrizione dettagliata
Tipo di studio
Tipo di studio
Iscrizione (Stimato)
Iscrizione
Fase
Fase
- Non applicabile
Criteri di partecipazione
Criteri di ammissibilità
Criteri di ammissibilità
Età idonea allo studio
- Bambino
- Adulto
Accetta volontari sani
Descrizione
Inclusion Criteria:
- Male or female participants 12 to 65 years of age at screening.
- Adolescent participants must be Tanner stage IV or V or have reached an equivalent level of sexual maturity.
- For the healthy-volunteer cohort, the participant must be considered healthy based on medical history and investigator assessment.
- For the ROSAH syndrome cohort, the participant must have a genetically and clinically confirmed diagnosis of ROSAH syndrome and must be considered clinically stable by the investigator.
- Able and willing to complete three controlled sauna heat-exposure tests.
- Able to remain seated in a temperature-controlled sauna chamber for up to 30 minutes.
- Willing to wear only underwear during testing; female participants may wear a bra.
- Willing to have hair at testing sites trimmed or shaved if necessary.
- Able to drink 300 mL of water before each test.
- Able and willing to comply with study requirements, instructions, and protocol restrictions and likely to complete the study as planned.
- Able to provide written informed consent.
- For minors, written informed consent must be provided by a parent or legal representative, and written assent must be provided by the participant.
Exclusion Criteria:
- Baseline body temperature greater than 37.5°C on the day of testing.
- Fever, acute infection, or acute illness within 7 days before testing.
- History of heat stroke, severe heat intolerance, heat-associated syncope, or severe dehydration.
- Clinically significant cardiovascular disease, uncontrolled hypertension, cardiac arrhythmia, or unstable respiratory disease.
- Severe anemia or unstable cytopenia, in the investigator's judgment.
- Active skin disease, wound, dermatitis, or skin infection at a filter paper placement site.
- Pregnant or breastfeeding.
- Current use of medication that may substantially affect sweating or thermoregulation unless approved by the investigator, including anticholinergic agents, systemic beta blockers, diuretics, selected antihistamines, or medications that reduce heat tolerance.
- Use of depilatory cream, waxing, an epilator, or another chemical or mechanical hair-removal method other than shaving from 7 days before the first sweat test through the final sweat test.
- Laser, intense pulsed light, or other energy-based hair removal within 3 months before screening.
- Unable to comply with clothing, privacy, or testing requirements.
- Any other condition that, in the investigator's judgment, would create an unacceptable risk or interfere with interpretation of the study results.
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Altro
- Assegnazione: N / A
- Modello interventistico: Assegnazione di gruppo singolo
- Mascheramento: Nessuno (etichetta aperta)
Numero di armi
Armi e interventi
Gruppo di partecipanti / ArmGruppo di partecipanti / Arm |
Intervento / TrattamentoIntervento / Trattamento |
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Sperimentale: Controlled Heat Exposure and Gravimetric Sweat Assessment
Approximately 20 healthy volunteers and up to 10 participants with genetically confirmed ROSAH syndrome will undergo three standardized passive heat-exposure sessions in a temperature-controlled sauna chamber.
Each session will last approximately 30 minutes, will be separated by at least 1 day, and all three sessions will be completed within 7 days.
Preweighed filter papers will be placed at the chest, back, axilla, forehead, and forearm and reweighed after exposure to quantify total and regional sweat production.
Physiologic and environmental parameters will be monitored throughout the procedure.
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Participants will drink 300 mL of water before testing and will sit in a temperature-controlled sauna chamber for approximately 30 minutes.
The mean internal exposure temperature will be maintained above 42°C and below 44°C, with an adjustable nominal operating set point of approximately 45°C.
Preweighed filter papers will be attached to prespecified anatomical sites and will be removed and reweighed immediately after exposure using a calibrated analytical balance.
Body temperature, heart rate, symptoms, sauna temperature, and relative humidity will be monitored.
The procedure will be stopped early if prespecified safety or intolerance criteria are met.
The procedure will be repeated three times, with sessions at least 1 day apart and completed within 7 days.
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Cosa sta misurando lo studio?
Misure di risultato primarie
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
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Total Gravimetric Sweat Production (TGSP)
Lasso di tempo: During each of three approximately 30-minute heat-exposure sessions, with sessions at least 1 day apart and all three sessions completed within 7 days.
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TGSP is defined as the sum of sweat mass collected from all prespecified and evaluable anatomical sites during a standardized heat-exposure session.
For each site, sweat mass is calculated as the post-exposure filter paper weight minus the pre-exposure filter paper weight.
TGSP will be reported in milligrams and may also be standardized by filter paper area according to the statistical analysis plan.
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During each of three approximately 30-minute heat-exposure sessions, with sessions at least 1 day apart and all three sessions completed within 7 days.
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Misure di risultato secondarie
Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Regional Gravimetric Sweat Production by Anatomical Site
Lasso di tempo: During each of three approximately 30-minute heat-exposure sessions, with sessions at least 1 day apart and completed within 7 days.
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Sweat mass at the chest, back, axilla, forehead, and forearm will be calculated separately as the post-exposure filter paper weight minus the pre-exposure filter paper weight.
Results will be reported in milligrams and may also be standardized by filter paper area.
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During each of three approximately 30-minute heat-exposure sessions, with sessions at least 1 day apart and completed within 7 days.
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Test-Retest Reliability of Total Gravimetric Sweat Production
Lasso di tempo: Across the three heat-exposure sessions completed within 7 days.
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Short-term test-retest reliability of TGSP across the three heat-exposure sessions will be assessed using the intraclass correlation coefficient and graphical repeated-measures assessments.
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Across the three heat-exposure sessions completed within 7 days.
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Within-Participant Variability of Total Gravimetric Sweat Production
Lasso di tempo: Across the three heat-exposure sessions completed within 7 days.
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Within-participant variability in TGSP across the three sessions will be summarized using within-participant standard deviation and coefficient of variation.
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Across the three heat-exposure sessions completed within 7 days.
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Test-Retest Reliability and Variability of Regional Sweat Production
Lasso di tempo: Across the three heat-exposure sessions completed within 7 days.
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Test-Retest Reliability and Variability of Regional Sweat Production
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Across the three heat-exposure sessions completed within 7 days.
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Total and Regional Sweat Production by Clinical Group, Sex, and Age Category
Lasso di tempo: During the three heat-exposure sessions completed within 7 days.
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TGSP and regional sweat production will be summarized descriptively by clinical group (healthy volunteers and participants with ROSAH syndrome), sex, and age category (adolescents and adults).
Comparisons are exploratory and are not powered for confirmatory hypothesis testing.
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During the three heat-exposure sessions completed within 7 days.
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Sauna Chamber Temperature Stability
Lasso di tempo: Throughout each approximately 30-minute heat-exposure session during the three sessions completed within 7 days.
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Sauna chamber temperature will be recorded continuously or at protocol-defined frequent intervals.
Stability will be summarized using descriptive statistics and the extent of time within the protocol-defined operating range.
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Throughout each approximately 30-minute heat-exposure session during the three sessions completed within 7 days.
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Sauna Chamber Relative Humidity Stability
Lasso di tempo: Throughout each approximately 30-minute heat-exposure session during the three sessions completed within 7 days.
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Sauna chamber relative humidity will be recorded continuously or at protocol-defined frequent intervals and summarized descriptively to characterize environmental consistency across sessions.
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Throughout each approximately 30-minute heat-exposure session during the three sessions completed within 7 days.
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Change in Body Temperature During Controlled Heat Exposure
Lasso di tempo: Before, during, immediately after, and during recovery from each of the three heat-exposure sessions completed within 7 days.
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Body temperature will be measured before sauna entry, at least every 5 minutes during exposure if not continuously monitored, at the end of exposure, and during recovery when clinically indicated.
Change from the pre-exposure value and maximum observed temperature will be summarized.
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Before, during, immediately after, and during recovery from each of the three heat-exposure sessions completed within 7 days.
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Heat-Exposure Sessions Terminated Early Due to Body Temperature of 38.0°C or Higher or Participant Intolerance
Lasso di tempo: During the three heat-exposure sessions completed within 7 days.
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The number and percentage of planned heat-exposure sessions that are terminated early because body temperature reaches or exceeds 38.0°C, the participant requests discontinuation, or the investigator identifies clinically significant intolerance will be summarized.
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During the three heat-exposure sessions completed within 7 days.
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Incidence, Type, and Severity of Adverse Events and Heat-Related Intolerance Events
Lasso di tempo: From initiation of the first study procedure through the follow-up assessment, up to approximately 17 days per participant.
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Adverse events and procedure-related intolerance events will be collected and summarized by event type, severity, seriousness, and relationship to study procedures.
Events of interest include heat intolerance, dizziness, presyncope or syncope, headache, nausea or vomiting, fatigue, palpitations, skin irritation, and clinically significant body-temperature elevation.
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From initiation of the first study procedure through the follow-up assessment, up to approximately 17 days per participant.
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Anhidrosis/Hypohidrosis Impact Questionnaire Total Score
Lasso di tempo: Before the first heat-exposure session and 8 to 10 days after the final heat-exposure session.
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The questionnaire contains six scored items covering heat intolerance, frequency of overheating, activity limitation, use of cooling measures, perceived ability to sweat, and overheating-related symptoms.
Each item is scored from 0 to 4; the total score ranges from 0 to 24, with higher scores indicating greater heat intolerance or impact of impaired sweating.
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Before the first heat-exposure session and 8 to 10 days after the final heat-exposure session.
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Change in Anhidrosis/Hypohidrosis Impact Questionnaire Total Score
Lasso di tempo: From before the first heat-exposure session to 8 to 10 days after the final heat-exposure session.
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Change in questionnaire total score will be calculated as the follow-up total score minus the baseline total score.
Item-level changes may also be summarized descriptively.
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From before the first heat-exposure session to 8 to 10 days after the final heat-exposure session.
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Test-Retest Reliability of the Anhidrosis/Hypohidrosis Impact Questionnaire
Lasso di tempo: Between the baseline assessment before the first heat-exposure session and the follow-up assessment 8 to 10 days after the final heat-exposure session.
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Short-term stability and test-retest reliability of questionnaire total scores will be evaluated using paired descriptive summaries, correlations, intraclass correlation coefficients, and graphical assessments, as appropriate.
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Between the baseline assessment before the first heat-exposure session and the follow-up assessment 8 to 10 days after the final heat-exposure session.
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Altre misure di risultato
Altre misure di risultato
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Spearman Correlation Between Baseline Anhidrosis/Hypohidrosis Impact Questionnaire Total Score and Mean Total Gravimetric Sweat Production (TGSP)
Lasso di tempo: Baseline questionnaire assessment before the first heat-exposure session and TGSP measurements from the three heat-exposure sessions completed within 7 days.
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Spearman correlation will be used to assess the relationship between the Anhidrosis/Hypohidrosis Impact Questionnaire total score and Total Gravimetric Sweat Production (TGSP).
The questionnaire includes 6 scored items, each rated from 0 to 4, yielding a total score of 0 to 24 points; higher scores indicate greater heat intolerance or impact of impaired sweating.
TGSP is measured using a gravimetric filter-paper method and is defined as the sum of post-exposure minus pre-exposure filter-paper weights across all evaluable anatomical sites, expressed in milligrams (mg).
The outcome will be reported as Spearman's rho, ranging from -1 to +1.
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Baseline questionnaire assessment before the first heat-exposure session and TGSP measurements from the three heat-exposure sessions completed within 7 days.
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Patient-Reported Change in Heat Tolerance
Lasso di tempo: At the follow-up assessment 8 to 10 days after the final heat-exposure session.
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The change-impression item will be summarized by response category and may be encoded from -3 (very much worse) to +3 (very much improved).
In this nontherapeutic study, the item should refer to change since the previous questionnaire assessment rather than change since starting treatment.
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At the follow-up assessment 8 to 10 days after the final heat-exposure session.
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