Serine and Fenofibrate Study in Patients With MacTel Type 2 (NCT04907084) | Clinical Trial Compass
Active — Not RecruitingPhase 2
Serine and Fenofibrate Study in Patients With MacTel Type 2
United States, United Kingdom60 participantsStarted 2022-04-07
Plain-language summary
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.
Who can participate
Age range
21 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Signed and dated written informed consent obtained from the participant in accordance with the local regulations;
. Males/females 21 years of age or older;
. English speaking;
. Enrolled in the Natural History Observation and Registry Study (NHOR) and diagnosed with confirmed MacTel type 2 in at least one eye;
. Willing to use contraception, if applicable; and
. Willing to comply with study protocol and follow-up visits.
Exclusion criteria
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Serum Deosxysphingolipid Levels
Timeframe: blood draws from week 3, 6, and 10
2
Safety Assessment
Timeframe: Assessment at each study visit (reviewed at week 3, week 6, and week 10 of study)
Trial details
NCT IDNCT04907084
SponsorThe Lowy Medical Research Institute Limited
. Participant is unable to provide informed consent;
. Participant is less than 21 years of age;
. Participant is currently taking, or has taken within four weeks prior to screening, a serine or glycine supplement;
. Participant is currently taking, or has taken within 12 months prior to screening, fibrates including clofibrate, ciprofibrate, bezafibrate, fenofibrate, and gemfibrozil;
. Participant is currently taking an anticoagulant, colchicine, cyclosporine, tacrolimus or bile acid binding resins;
. Participant has known allergy to fibrates and/or serine;
. Participant has a known history of clinically significant myopathy or myalgia related to cholesterol-lowering drugs;
. Participant has active liver disease and/or elevated liver enzymes\*;