NCT06060197|Completed
MCT8 Deficiency Caregiver Study
Caregiver-reported Economic Burden and Quality of Life Impact of Monocarboxylate Transporter 8 (MCT8) Deficiency
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Caregiver-reported Economic Burden and Quality of Life Impact of Monocarboxylate Transporter 8 (MCT8) Deficiency
Sponsor study identifier
observational
21
1 country
1
Study Start
August 23, 2022
Actual (reported)Primary Completion
April 12, 2024
Actual (reported)Study Completion
April 12, 2024
Actual (reported)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.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
April 12, 2024
Actual (reported)Click on a node to explore related trials.
Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
August 23, 2022
Actual (reported)August 11, 2023
Actual (reported)September 29, 2023
Actual (reported)April 12, 2024
Actual (reported)April 12, 2024
Actual (reported)Quantify the economic burden of caring for a patient with MCT8 deficiency using bespoke questions
Economic burden will be assessed by using bespoke questions on the time spent providing care and impact on caregiver's family and work circumstances.
once, at enrolment
Quantify the cost of caring for a patient with MCT8 deficiency using bespoke questions
Costs will be assessed by using bespoke questions on the caregiver's employment income, costs of travel/transport, contributions to the costs of treatments and other costs of care.
once, at enrolment
Quantify the health-related quality of life (HRQoL) burden of caring for a patient with MCT8 deficiency using the EQ-5D-5L
The impact on the caregiver's wellbeing will be assessed using the EQ-5D-5L, which comprises two parts. The descriptive system comprises 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression), each with 5 levels (1 - no problems, 2 - slight problems, 3 - moderate problems, 4 - severe problems, and 5 - extreme problems). The digits for 5 dimensions can be combined in a 5-digit number describing the respondent's health state. The EQ Visual Analogue Scale (EQ-VAS) records the respondent's self-rated health on a vertical scale with endpoints labelled 100, representing 'the best health you can imagine' and 0, 'the worst health you can imagine'. Higher scores represent better self-perceived health.
Describe the social and demographic characteristics of patients and caregivers
once, at enrolment
Investigate the experiences of caregivers providing care and support to patients affected by MCT8 deficiency
once, at enrolment
Provide additional evidence on the HRQoL of patients with MCT8 deficiency across health states using the EQ-5D-5L proxy version 1
once, at enrolment
Adults who are the main caregiver to a person diagnosed with MCT8 deficiency or Allan-Herndon-Dudley syndrome and regularly provide a significant amount of help to them.
Vitaccess Ltd
Oxford, Oxfordshire, OX4 4GA, United Kingdom
Mark Larkin, PhD
Vitaccess Limited
21 participants (actual enrollment reported by the registry).
First Submitted
August 11, 2023
First Posted
September 29, 2023
Study Start
August 23, 2022
Actual (reported)Register for Patients With Thyroid Hormone Resistance.
once, at enrolment
Quantify the health-related quality of life (HRQoL) burden of caring for a patient with MCT8 deficiency using the PedsQL
The impact on the caregiver's family will be assessed using the Pedriatric Quality of Life Inventory (PedsQL™) Family Impact Module. The PedsQL™ has 36 items broken down into 7 subdomains of physical, emotional, social, and cognitive functioning, communication, and worry as well as family daily activities and family relationships. A 5-point response scale is utilized (0 = never a problem; 4 = always a problem). Items are reverse-scored and linearly transformed to a 0-100 scale (0 = 100, 1 = 75, 2 = 50, 3 = 25, 4 = 0), so that higher scores indicate better functioning (less negative impact).
once, at enrolment
Contact the study team to confirm eligibility.
Primary Completion
April 12, 2024
Actual (reported)Study Completion
April 12, 2024
Actual (reported)Last Updated
April 25, 2024
Record last verified: 2024-04
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Source: ClinicalTrials.gov.
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