Phase 3 Study of Serine Supplementation to Protect Vision in MacTel
The Lowy Medical Research Institute Limited
Summary
The goal of this clinical trial is to look at the efficacy and safety of giving oral serine (an amino acid) on the progression of structural and functional changes of the retina in people with MacTel type 2. The main questions it aims to answer are: * Does serine slow the progression of MacTel? * Is long-term serine supplementation safe in people with MacTel? Researchers will compare serine to a placebo (a look-alike substance that contains no drug) to see if serine works to slow the progression of MacTel. Participants will: * Take serine or a placebo twice a day for 24 months * Visit the clinic once every 6 months for eye exam, eye imaging and blood tests * Keep a diary of their symptoms, missed doses, and changes in medications
Description
Macular telangiectasia type 2 (MacTel) is a bilateral, slowly progressive retinal neurodegenerative disease characterized by photoreceptor loss, vascular abnormalities, and gradual decline in visual function. Alterations in serine metabolism and the accumulation of toxic deoxysphingolipids have been implicated in the pathophysiology of MacTel. Oral serine supplementation has been proposed as a potential approach to modify this metabolic pathway and reduce the formation of potentially neurotoxic metabolites. This study is a randomized, double-masked, placebo-controlled, parallel-group clinica…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria * Be able to read, comprehend, and agree to conditions as explained in the informed consent document and provide written informed consent; * Be at least 18 years of age; * Enrolled or enrolling in the Natural History Observation and Registry Study (NHOR) and confirmed with MacTel type 2 by the Reading Center in at least one eye\*; * Participant has clear ocular media (both eyes) for sufficient image quality; * Participant must have steady fixation in the foveal or parafoveal area; * Female participants of childbearing potential must agree to use a highly effective method of…
Interventions
- DrugL-serine
L-serine powder administered twice daily
- OtherPlacebo
Placebo powder administered twice daily
Locations (12)
- Hamilton Glaucoma Center- UCSDLa Jolla, California
- University of Michigan, Kellogg Eye CenterAnn Arbor, Michigan
- Retina Associates Of Cleveland, Inc.Cleveland, Ohio
- University of Utah Health Care, Moran Eye CenterSalt Lake City, Utah
- Save Sight InstituteSydney, New South Wales
- Cerulea- RVEEHMelbourne, Victoria