Serine and Fenofibrate Study in Patients With MacTel Type 2

Last updated: March 12, 2024
Sponsor: The Lowy Medical Research Institute Limited
Overall Status: Active - Not Recruiting

Phase

2

Condition

N/A

Treatment

Fenofibrate

Serine

Clinical Study ID

NCT04907084
2020-002
  • Ages > 21
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Signed and dated written informed consent obtained from the participant in accordancewith the local regulations;
  2. Males/females 21 years of age or older;
  3. English speaking;
  4. Enrolled in the Natural History Observation and Registry Study (NHOR) and diagnosedwith confirmed MacTel type 2 in at least one eye;
  5. Willing to use contraception, if applicable; and
  6. Willing to comply with study protocol and follow-up visits.

Exclusion

Exclusion Criteria:

  1. Participant is unable to provide informed consent;
  2. Participant is less than 21 years of age;
  3. Participant is currently taking, or has taken within four weeks prior to screening, aserine or glycine supplement;
  4. Participant is currently taking, or has taken within 12 months prior to screening,fibrates including clofibrate, ciprofibrate, bezafibrate, fenofibrate, andgemfibrozil;
  5. Participant is currently taking an anticoagulant, colchicine, cyclosporine, tacrolimusor bile acid binding resins;
  6. Participant has known allergy to fibrates and/or serine;
  7. Participant has a known history of clinically significant myopathy or myalgia relatedto cholesterol-lowering drugs;
  8. Participant has active liver disease and/or elevated liver enzymes*;
  9. Participant has renal dysfunction as evidenced by elevated serum creatinine* and/ orglomerular filtration rate (GFR) less than 90 mL/min;
  10. Participant has thrombocytopenia as evidenced by a platelet count below 100,000 permicroliter, anemia as evidenced by hemoglobin levels below 10 g/dL, or history ofbleeding disorder;
  11. Participant has a history of gallbladder disease or has had a cholecystectomy;
  12. Participant has triglyceride levels greater than 400 mg/dL on treatment, or greaterthan 700 mg/dL on no treatment;
  13. Participant has untreated/uncured Hepatitis C, or a history of Hepatitis B, autoimmunehepatitis, or HIV;
  14. Participant has had any malignancies within the last 5 years (not including basal cellcarcinoma);
  15. Participant has ever been enrolled in a clinical trial involving ciliary neurotrophicfactor (CNTF) treatment;
  16. Participant is currently enrolled in another clinical trial that involves treatment orparticipated in one within the last 30 days;
  17. Participant is pregnant, breastfeeding or planning a pregnancy;
  18. Participant is medically unable to comply with study procedures or follow-up visits;
  19. Participant has, in the opinion of the Investigator, any physical or mental conditionthat would increase the patient's risk of participation in the study or may interferewith the study procedures, evaluations and outcome assessments; or
  20. Patient is unavailable for follow-up visits. *based on reference range for the locallaboratory used

Study Design

Total Participants: 60
Treatment Group(s): 2
Primary Treatment: Fenofibrate
Phase: 2
Study Start date:
April 07, 2022
Estimated Completion Date:
March 31, 2024

Study Description

Additional Procedures include:

  1. Fasting blood work

  2. Collection of microbiome samples

Connect with a study center

  • Moorfields Eye Hospital

    London, ECV2PD
    United Kingdom

    Site Not Available

  • Emory University

    Atlanta, Georgia 30322
    United States

    Site Not Available

  • Kellogg Eye Center, University of Michigan

    Ann Arbor, Michigan 48105
    United States

    Site Not Available

  • Retina Associates of Cleveland

    Cleveland, Ohio 44122
    United States

    Site Not Available

  • Southeastern Retina Associates

    Knoxville, Tennessee 37922
    United States

    Site Not Available

  • Retina Consultants of Texas

    Bellaire, Texas 77401
    United States

    Site Not Available

  • Moran Eye Center, University of Utah

    Salt Lake City, Utah 84132
    United States

    Site Not Available

  • University of Washington

    Seattle, Washington 98104
    United States

    Site Not Available

  • The Eye Institute, Medical College of Wisconsin

    Milwaukee, Wisconsin 53226
    United States

    Site Not Available

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.