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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.
Additional Procedures include:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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)). |
|
| 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)). |
|
| Fenofibrate 160 mg/day | Experimental | Fenofibrate 160 mg per day. To be taken once per day for 6 weeks. |
|
| 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. |
|
| 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. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Serine | Dietary Supplement | Powdered serine supplement (Dosed out individually per participant. Participant to mix with water and ingest orally) |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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) |
| Measure | Description | Time Frame |
|---|---|---|
| Lipid Levels | 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 | blood draws from week 3, 6, and 10 |
| Amino Acid Levels |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Mari A Gantner, PhD | Lowy Medical Research Institute | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Emory University | Atlanta | Georgia | 30322 | United States | ||
| Kellogg Eye Center, University of Michigan |
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| Type | Date | Date Unknown |
|---|---|---|
| Release | Apr 6, 2026 | |
| Reset | Apr 24, 2026 |
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| Release Date | Unrelease Date | Unrelease Date Unknown | Reset Date | MCP Release Number |
|---|---|---|---|---|
| Apr 6, 2026 | Apr 24, 2026 | |||
| May 12, 2026 |
| ID | Term |
|---|---|
| D012694 | Serine |
| D011345 | Fenofibrate |
| ID | Term |
|---|---|
| D021542 | Amino Acids, Neutral |
| D000596 | Amino Acids |
| D000602 | Amino Acids, Peptides, and Proteins |
| D058607 | Fibric Acids |
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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).
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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).
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|
| No treatment | No Intervention | Control group: no investigational product taken |
| Fenofibrate | Drug | Fenofibrate 160mg pill, taken orally |
|
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
| blood draws from week 3, 6, and 10 |
| Ann Arbor |
| Michigan |
| 48105 |
| United States |
| Retina Associates of Cleveland | Cleveland | Ohio | 44122 | United States |
| Southeastern Retina Associates | Knoxville | Tennessee | 37922 | United States |
| Retina Consultants of Texas | Bellaire | Texas | 77401 | United States |
| Moran Eye Center, University of Utah | Salt Lake City | Utah | 84132 | United States |
| University of Washington | Seattle | Washington | 98104 | United States |
| The Eye Institute, Medical College of Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
| Moorfields Eye Hospital | London | ECV2PD | United Kingdom |
| Jun 5, 2026 |
| 10 |
| D058610 |
| Isobutyrates |
| D002087 | Butyrates |
| D000144 | Acids, Acyclic |
| D002264 | Carboxylic Acids |
| D009930 | Organic Chemicals |
| D010647 | Phenyl Ethers |
| D004987 | Ethers |
| D001577 | Benzophenones |
| D001555 | Benzene Derivatives |
| D006841 | Hydrocarbons, Aromatic |
| D006844 | Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D010636 | Phenols |
| D007659 | Ketones |