Serine and Fenofibrate Study in Patients With MacTel Type 2
- Conditions
- Macular Telangiectasia Type 2
- Interventions
- Dietary Supplement: Serine
- Registration Number
- NCT04907084
- Lead Sponsor
- The Lowy Medical Research Institute Limited
- Brief 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.
- Detailed Description
Additional Procedures include:
1. Fasting blood work
2. Collection of microbiome samples
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 60
- 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.
- 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*;
- Participant has renal dysfunction as evidenced by elevated serum creatinine* and/ or glomerular filtration rate (GFR) less than 90 mL/min;
- Participant has thrombocytopenia as evidenced by a platelet count below 100,000 per microliter, anemia as evidenced by hemoglobin levels below 10 g/dL, or history of bleeding disorder;
- Participant has a history of gallbladder disease or has had a cholecystectomy;
- Participant has triglyceride levels greater than 400 mg/dL on treatment, or greater than 700 mg/dL on no treatment;
- Participant has untreated/uncured Hepatitis C, or a history of Hepatitis B, autoimmune hepatitis, or HIV;
- Participant has had any malignancies within the last 5 years (not including basal cell carcinoma);
- Participant has ever been enrolled in a clinical trial involving ciliary neurotrophic factor (CNTF) treatment;
- Participant is currently enrolled in another clinical trial that involves treatment or participated in one within the last 30 days;
- Participant is pregnant, breastfeeding or planning a pregnancy;
- Participant is medically unable to comply with study procedures or follow-up visits;
- Participant has, in the opinion of the Investigator, any physical or mental condition that would increase the patient's risk of participation in the study or may interfere with the study procedures, evaluations and outcome assessments; or
- Patient is unavailable for follow-up visits. *based on reference range for the local laboratory used
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- FACTORIAL
- Arm && Interventions
Group Intervention Description Serine 400 mg/kg/day Serine Serine 400 mg/kg per day. To be taken once per day for 6 weeks. Exact dose dependent on participant's weight (in kg)). Serine 200 mg/kg/day and Fenofibrate 160 mg/day Serine Serine 200 mg/kg per day. To be taken once per day for 6 weeks. Exact dose dependent on participant's weight (in kg)). Fenofibrate 160 mg per day. To be taken once per day for 6 weeks. Serine 200 mg/kg/day Serine Serine 200 mg/kg per day. To be taken once per day for 6 weeks. Exact dose dependent on participant's weight (in kg)). Serine 400 mg/kg/day and Fenofibrate 160 mg/day Serine Serine 400 mg/kg per day. To be taken once per day for 6 weeks. Exact dose dependent on participant's weight (in kg)). Fenofibrate 160 mg per day. To be taken once per day for 6 weeks. Serine 200 mg/kg/day and Fenofibrate 160 mg/day Fenofibrate Serine 200 mg/kg per day. To be taken once per day for 6 weeks. Exact dose dependent on participant's weight (in kg)). Fenofibrate 160 mg per day. To be taken once per day for 6 weeks. Serine 400 mg/kg/day and Fenofibrate 160 mg/day Fenofibrate Serine 400 mg/kg per day. To be taken once per day for 6 weeks. Exact dose dependent on participant's weight (in kg)). Fenofibrate 160 mg per day. To be taken once per day for 6 weeks. Fenofibrate 160 mg/day Fenofibrate Fenofibrate 160 mg per day. To be taken once per day for 6 weeks.
- Primary Outcome Measures
Name Time Method Serum Deosxysphingolipid Levels blood draws from week 3, 6, and 10 Any changes in serum deoxysphingolipid levels measured (determined via serum sample) at 3 weeks, 6 weeks, and/or 10 weeks when compared with baseline levels measured in week 0
Safety Assessment Assessment at each study visit (reviewed at week 3, week 6, and week 10 of study) Safety assessment measured via examined AEs and SAEs (during study visits) and via participant self-reported AEs and SAEs that occurred between visits
- Secondary Outcome Measures
Name Time Method Amino Acid Levels blood draws from week 3, 6, and 10 Any changes in amino acid levels measured (determined via blood tests) at 3 weeks, 6 weeks, and/or 10 weeks when compared with baseline levels measured at screening
Lipid Levels blood draws from week 3, 6, and 10 Any changes in lipid levels measured (determined via blood tests) at 3 weeks, 6 weeks, and/or 10 weeks when compared with baseline levels measured at screening
Trial Locations
- Locations (9)
The Eye Institute, Medical College of Wisconsin
๐บ๐ธMilwaukee, Wisconsin, United States
University of Washington
๐บ๐ธSeattle, Washington, United States
Emory University
๐บ๐ธAtlanta, Georgia, United States
Kellogg Eye Center, University of Michigan
๐บ๐ธAnn Arbor, Michigan, United States
Retina Associates of Cleveland
๐บ๐ธCleveland, Ohio, United States
Southeastern Retina Associates
๐บ๐ธKnoxville, Tennessee, United States
Retina Consultants of Texas
๐บ๐ธBellaire, Texas, United States
Moran Eye Center, University of Utah
๐บ๐ธSalt Lake City, Utah, United States
Moorfields Eye Hospital
๐ฌ๐งLondon, United Kingdom