MCT8 Deficiency Caregiver Study

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

21 participants

Study Classification

OBSERVATIONAL

Study Start Date

2022-08-23

Study Completion Date

2024-04-12

Brief Summary

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Caregivers face many responsibilities outside of their role as a friend or parent, which can lead to emotional, financial, social, and professional challenges. To better understand the impact of MCT8 deficiency on caregivers, Egetis Therapeutics are conducting an online survey for adult caregivers of persons living with the MCT8 deficiency.

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Detailed Description

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Understanding the impact of MCT8 deficiency on caregivers and patients is key to facilitating evidence-based policy interventions and identifying better treatments and treatment practices that take full account of the actual environment in which care is delivered.

This study will address the gap in research about the wider impact of MCT8 deficiency by exploring the caregiver-reported economic burden and health-related quality of life (HRQoL) impact of the disease. Adult caregivers will report HRQoL outcomes for themselves and the person with MCT8 deficiency that they care for, as affected patients have limited cognitive function and communication abilities and cannot report the information themselves.

The results from this study will provide insight into the economic and HRQoL impact of MCT8 deficiency, as reported by caregivers, in the UK, US, Canada, France, Netherlands, Spain, Australia, Italy, and Germany.

Conditions

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Monocarboxylate Transporter 8 (MCT8) Deficiency Allan-Herndon-Dudley Syndrome

Study Design

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Observational Model Type

ECOLOGIC_OR_COMMUNITY

Study Time Perspective

CROSS_SECTIONAL

Eligibility Criteria

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Inclusion Criteria

* 18 years or older
* Resident in the UK, US, Canada (except Newfoundland \& Labrador and Alberta), France, Netherlands, Spain, Australia, Italy, and Germany
* Main caregiver to a person diagnosed with MCT8 deficiency