MacTel SAFE Study
March 6, 2024
The following information was sent out via email to people who registered in the LMRI Contact Database.
LMRI is conducting the SAFE study (Phase 2a Study of the Effect of Serine Supplementation and Fenofibrate Treatment on Serum Deoxysphinganine Levels in Patients with Macular Telangiectasia (MacTel) Type 2). The study aims to determine the effect of serine and/or fenofibrate on circulating metabolite and lipid levels in patient blood.
The basis of this study comes from a discovery made by the LMRI network of researchers. By analyzing the blood of a subset of MacTel Project Registry participants, this group of scientists and clinicians discovered that people with MacTel generally have lower levels of serine in their blood than people who do not have MacTel. Serine is an important amino acid in the body and when its levels are low multiple other metabolites are impacted. One of its many functions is to generate a type of lipid (fat), which are necessary for cell function. When serine levels are low, the body makes an alternate lipid which can be toxic to nerve cells, including photoreceptors (the light sensing cells in the eye).
The SAFE study is testing two approaches to reduce the consequences of low serine levels: one is consuming extra serine, via supplementation; the other is to restore altered lipid levels by consuming fenofibrate, a medication used to lower cholesterol and triglycerides in the blood.
The SAFE Study was conducted at eight clinical sites in the US and one in London, UK. The clinics screened 143articipants; of those, 90 were eligible. This study involved six arms with participants randomly assigned to one of the following groups: low dose serine, high dose serine, fenofibrate, low dose serine plus fenofibrate, high dose serine and fenofibrate, and no treatment.
The study is now closed to enrollment and the last participants will be seen at the end of March 2024.
We are now in the process of analyzing all the data and specimens. The results will determine the direction of further research with serine and/or fenofibrate. Until further research is done, we do not endorse the use of these substances for the treatment of MacTel. The results of the SAFE study will be shared with you when they are available.
A huge thank you to all who participated in this study. To those who could not be enrolled due to our conservative enrollment eligibility criteria, please know that the information obtained at the screening visit will further our understanding of macular telangiectasia type 2.
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