This is a Phase 2a study of the effect of serine supplementation and fenofibrate treatment on serum deoxysphingolipid levels in patients with macular telangiectasia type 2 (MacTel). This study involves six arms. Participants will be randomly assigned to one of the following treatment groups: serine 200 mg/kg/day, serine 400 mg/kg/day, fenofibrate 160 mg/day, both serine 200 mg/kg/day and fenofibrate 160 mg/day, both serine 400 mg/kg/day and fenofibrate 160 mg/day, or no treatment (control group). Serum deoxysphingolipid levels will be used as the primary outcome, and safety will be evaluated. The participants will be followed for 10 weeks, with visits at Screening, Week 0, 3, 6 and 10.
Additional Procedures include: 1. Fasting blood work 2. Collection of microbiome samples
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
OTHER
Masking
NONE
Enrollment
89
Powdered serine supplement (Dosed out individually per participant. Participant to mix with water and ingest orally)
Fenofibrate 160mg pill, taken orally
Emory University
Atlanta, Georgia, United States
Kellogg Eye Center, University of Michigan
Ann Arbor, Michigan, United States
Retina Associates of Cleveland
Cleveland, Ohio, United States
Southeastern Retina Associates
Knoxville, Tennessee, United States
Retina Consultants of Texas
Bellaire, Texas, United States
Moran Eye Center, University of Utah
Salt Lake City, Utah, United States
University of Washington
Seattle, Washington, United States
The Eye Institute, Medical College of Wisconsin
Milwaukee, Wisconsin, United States
Moorfields Eye Hospital
London, United Kingdom
Safety Assessment
Safety assessment measured via examined AEs and SAEs (during study visits) and via participant self-reported AEs and SAEs that occurred between visits
Time frame: Reported over the10 weeks from baseline to exit visit
Percentage Change of Serum Deoxysphingolipid Levels From Baseline to 6 Weeks
Percentage change in the sum of 7 different deoxy-dihydroceramides species detected. Negative values mean these metabolites decreased during the treatment period. Deoxy-sphingolipids were measured in serum using LC-MS.
Time frame: Blood draw at baseline and 6 weeks on product
Percentage Change in Serum Lipid Levels From Baseline to 6 Weeks
Percent changes in lipid levels measured between baseline and 6 weeks. These lipid levels were obtained from the local clinical lab at each site as part of a lipid panel. Negative values mean these metabolites decreased during the treatment period.
Time frame: blood draws from baseline and week 6 on product
Percentage Change in Serum Serine and Glycine Levels From Baseline to 6 Weeks
Percentage change in fasting levels of the two amino acids serine and glycine. Serine supplementation is part of the treatment and glycine can be produced from serine by different cells in the body. Serine and glycine levels were measured from serum using LC-MS.
Time frame: blood draws from baseline and week 6 on product
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.