Serine and Fenofibrate Study in Patients With MacTel Type 2

NCT04907084 | Active Not Recruiting Phase 2 FDA Drug

• 1 other identifier: 2020-002
• Study Type: interventional
• Target: 60
• Locations: 2 countries
• Sites: 9

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.

Conditions

Macular Telangiectasia Type 2

Outcome Measures

Primary Outcomes (2)

• Serum Deosxysphingolipid Levels

  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

  blood draws from week 3, 6, and 10

• 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

  Assessment at each study visit (reviewed at week 3, week 6, and week 10 of study)

Secondary Outcomes (2)

• Lipid Levels

  blood draws from week 3, 6, and 10

• Amino Acid Levels

  blood draws from week 3, 6, and 10

Study Arms (6)

Serine 200 mg/kg/day

EXPERIMENTAL

Serine 200 mg/kg per day. To be taken once per day for 6 weeks. Exact dose dependent on participant's weight (in kg)).

Dietary Supplement: Serine

Serine 400 mg/kg/day

EXPERIMENTAL

Serine 400 mg/kg per day. To be taken once per day for 6 weeks. Exact dose dependent on participant's weight (in kg)).

Dietary Supplement: Serine

Fenofibrate 160 mg/day

EXPERIMENTAL

Fenofibrate 160 mg per day. To be taken once per day for 6 weeks.

Drug: Fenofibrate

Serine 200 mg/kg/day and Fenofibrate 160 mg/day

EXPERIMENTAL

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.

Dietary Supplement: Serine; Drug: Fenofibrate

Serine 400 mg/kg/day and Fenofibrate 160 mg/day

EXPERIMENTAL

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.

Dietary Supplement: Serine; Drug: Fenofibrate

No treatment

NO INTERVENTION

Control group: no investigational product taken

Interventions

Serine DIETARY_SUPPLEMENT

Powdered serine supplement (Dosed out individually per participant. Participant to mix with water and ingest orally)

Serine 200 mg/kg/day; Serine 200 mg/kg/day and Fenofibrate 160 mg/day; Serine 400 mg/kg/day; Serine 400 mg/kg/day and Fenofibrate 160 mg/day

Fenofibrate DRUG

Fenofibrate 160mg pill, taken orally

Fenofibrate 160 mg/day; Serine 200 mg/kg/day and Fenofibrate 160 mg/day; Serine 400 mg/kg/day and Fenofibrate 160 mg/day

Eligibility Criteria

• Age: 21 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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;

Sponsors & Collaborators

• The Lowy Medical Research Institute Limited: lead

Study Sites (9)

Emory University

Atlanta, Georgia, 30322, United States

Location

Kellogg Eye Center, University of Michigan

Ann Arbor, Michigan, 48105, United States

Location

Retina Associates of Cleveland

Cleveland, Ohio, 44122, United States

Location

Southeastern Retina Associates

Knoxville, Tennessee, 37922, United States

Location

Retina Consultants of Texas

Bellaire, Texas, 77401, United States

Location

Moran Eye Center, University of Utah

Salt Lake City, Utah, 84132, United States

Location

University of Washington

Seattle, Washington, 98104, United States

Location

The Eye Institute, Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Moorfields Eye Hospital

London, ECV2PD, United Kingdom

Location

MeSH Terms

Interventions

Serine; Fenofibrate

Intervention Hierarchy (Ancestors)

Amino Acids, Neutral; Amino Acids; Amino Acids, Peptides, and Proteins; Fibric Acids; Isobutyrates; Butyrates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Phenyl Ethers; Ethers; Benzophenones; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Phenols; Ketones

Study Officials

• Mari A Gantner, PhD

  Lowy Medical Research Institute

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Masking Details: This is an open-label study. Participants will be told if they are taking serine and/or fenofibrate, or if they are participating as a control (no treatment).
• Purpose: OTHER
• Intervention Model: FACTORIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: The randomization model will stratify patients by diabetes status, ensuring equal treatment of diabetics / non-diabetics in the study. A permuted block design will be used to randomize participants in a 1:1:1:1:1:1 ratio to either 1) serine 200 mg/kg/day, 2) serine 400 mg/kg/day, 3) fenofibrate 200 mg/day, 4) serine 200 mg/kg/day AND fenofibrate 200 mg/day, 5) serine 400 mg/kg/day AND fenofibrate 200 mg/day, or 6) no treatment (control group).

Study Record Dates

• First Submitted: May 25, 2021
• First Posted: May 28, 2021
• Study Start: April 7, 2022
• Primary Completion: March 1, 2024
• Study Completion: March 1, 2024
• Last Updated: March 13, 2024

Record last verified: 2024-03

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1); US (8)