UK mitochondrial disease trials and research studi… Page 2
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Fighting mito,
finding hope.

UK trials and research studies

Current UK clinical trials and research studies in mitochondrial disease

When it comes to mitochondrial disease, The Lily Foundation form a vital connection between families affected by the condition and the scientific community. We like to think of ourselves as the bridge between clinical insights and the personal experiences of patients.

How does The Lily Foundation support research participation?

Through strong partnerships with researchers worldwide, we’re able to provide essential input on study design, research priorities and patient needs. Our relationships with these teams enable Lily patients and families to participate in cutting-edge studies that contribute to advancing treatments and moving closer to a cure.

Are there any UK-based providers offering mitochondrial disease clinical trials?

We’ve gathered together below details of all current mitochondrial disease research studies in the UK, including eligibility criteria and contact information. This resource helps patients and families find studies they may qualify for and feel better informed about participation options. We would always recommend discussing any research participation with your doctor before taking part to ensure you understand any risks and potential benefits.

Why should I participate in mitochondrial disease research?

By joining a research study, patients can play a crucial role in advancing our understanding of mitochondrial disease. Every participant contributes valuable data that brings us one step closer to effective treatments and, ultimately, a cure.

Mito ataxia study

This study aims to measure and track cerebellar ataxia in patients with mitochondrial disease.

Study status:
Currently recruiting
Key criteria:
Patient participants must be between the ages of 16 and 70 years, with a genetically proven diagnosis of mitochondrial disease
Study site:
Newcastle
Study sponsor:
Newcastle upon Tyne NHS Foundation Trust (NuTH)

GYM – Generate Your Muscle study

This study will investigate whether stem cell therapy can potentially be used to reduce the levels of m.3243A>G mutation in the muscles in mitochondrial disease patients.

Study status:
Currently recruiting in the Netherlands
Key criteria:
Patient participants must be between the ages of 18 to 65 years of age, with a confirmed diagnosis of Primary Mitochondrial Disease caused by the 3243A>G mutation in mitochondrial DNA
Study site:
Maastricht, the Netherlands
Study sponsor:
Maastricht UMC, Netherlands