The Leigh Syndrome International Consortium is funding a global Natural History Study of Leigh syndrome. Its purpose is to rapidly build a valuable research data set to facilitate clinical trials on Leigh syndrome. This study will also build infrastructure to collect, analyse and share data among consortium members for future international collaboration.
What is a Natural History Study?
A Natural History Study observes a group of people over time to understand how a specific health condition or disease impacts them without intervention. Researchers typically collect a wide range of data as they track the condition to develop a holistic understanding of its progression. These findings provide a baseline and can then be used as a comparison point against which to measure the success of future treatment trials.
What Information Will This Study Collect?
The Natural History Study will initially track at least 150 people with Leigh syndrome across several global sites over three years. During this time, researchers will collect objective and subjective assessments of symptom involvement. This will include objective clinician assessments, subjective patient or parent-reported outcomes, and patient medical history.
This study’s primary outcome is to understand and quantify the natural history of Leigh syndrome which is expected to increase clinical trial readiness for Leigh syndrome.
The secondary outcome is to form a foundation for future international studies and demonstrate the benefits of intensive collaboration.