Clinical trial NCT06060197: MCT8 Deficiency Caregiver Study
Official title: Caregiver-reported Economic Burden and Quality of Life Impact of Monocarboxylate Transporter 8 (MCT8) Deficiency
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Monocarboxylate Transporter 8 (MCT8) Deficiency
Allan-Herndon-Dudley Syndrome
Keywords: MCT8 deficiency
caregiver
economic burden
HRQoL
EQ-5D-5L
PROs
Lead sponsor: Rare Thyroid Therapeutics International AB
Sponsor class: INDUSTRY
Collaborators: name: Vitaccess Ltd
First posted: 2023-09-29
Enrollment: 21
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: ECOLOGIC_OR_COMMUNITY
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: 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.

Brief summary: 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.

Detailed description: 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.

Eligibility criteria: 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

Exclusion Criteria:

* Paid caregivers to a person diagnosed with MCT8 deficiency

Primary outcomes:
measure: Quantify the economic burden of caring for a patient with MCT8 deficiency using bespoke questions
timeFrame: once, at enrolment
measure: Quantify the cost of caring for a patient with MCT8 deficiency using bespoke questions
timeFrame: once, at enrolment
measure: Quantify the health-related quality of life (HRQoL) burden of caring for a patient with MCT8 deficiency using the EQ-5D-5L
timeFrame: once, at enrolment
measure: Quantify the health-related quality of life (HRQoL) burden of caring for a patient with MCT8 deficiency using the PedsQL
timeFrame: once, at enrolment
Secondary outcomes:
measure: Describe the social and demographic characteristics of patients and caregivers
timeFrame: once, at enrolment
measure: Investigate the experiences of caregivers providing care and support to patients affected by MCT8 deficiency
timeFrame: once, at enrolment
measure: Provide additional evidence on the HRQoL of patients with MCT8 deficiency across health states using the EQ-5D-5L proxy version 1
timeFrame: once, at enrolment
Other outcomes:
Locations:
city: Oxford
country: United Kingdom
facility: Vitaccess Ltd
state: Oxfordshire
Study officials:
affiliation: Vitaccess Limited
name: Mark Larkin, PhD
role: PRINCIPAL_INVESTIGATOR
References: