MCT8 Deficiency Caregiver Study

Rare Thyroid Therapeutics

Status: Completed

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

Study type: Observational

Funder types: Industry

Identifiers

NCT06060197

4253-09-2021

Details and patient eligibility

About

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.

Full 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.

Enrollment: 21 patients

Sex: All

Ages: 18+ years old

Volunteers: Accepts Healthy Volunteers

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