Macular Telangiectasia Type 2Phase 2SAFENCT04907084
Serine and Fenofibrate Study in Patients With MacTel Type 2
A Phase 2 study testing Serine and Fenofibrate in people with Macular Telangiectasia Type 2, run by The Lowy Medical Research Institute Limited at 9 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 21 years and older
- Conditions the study is for
- Macular Telangiectasia Type 2
- What is being tested?
- Serine and Fenofibrate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 89 participants
- Where
- 9 sitesin 2 countries
- Study dates
- 7 Apr 2022to 30 Sep 2024
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 21 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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;
Show all 20Show fewer
- 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
Trial locations and contacts
9 sites in 2 countries.
9 sites in 2 countries
United States8 sites
Kellogg Eye Center, University of Michigan
Ann Arbor, Michigan, 48105
Emory University
Atlanta, Georgia, 30322
Retina Consultants of Texas
Bellaire, Texas, 77401
Retina Associates of Cleveland
Cleveland, Ohio, 44122
Southeastern Retina Associates
Knoxville, Tennessee, 37922
The Eye Institute, Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Moran Eye Center, University of Utah
Salt Lake City, Utah, 84132
University of Washington
Seattle, Washington, 98104
About this study
Official title: Phase 2a Study of the Effect of Serine Supplementation and Fenofibrate Treatment on Serum Deoxysphinganine Levels in Patients With Macular Telangiectasia (MacTel) Type 2 (SAFE Study)
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.
Read the detailed description
Additional Procedures include:
- Fasting blood work
- Collection of microbiome samples
- Conditions
- Macular Telangiectasia Type 2
- Being tested
- Serine, Fenofibrate
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SerineDietary supplement
Powdered serine supplement (Dosed out individually per participant. Participant to mix with water and ingest orally)
FenofibrateDrug
Fenofibrate 160mg pill, taken orally
| Study group | Role | What they receive |
|---|---|---|
| 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)). Receives: 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)). Receives: Dietary Supplement: Serine |
| Fenofibrate 160 mg/day | Experimental | Fenofibrate 160 mg per day. To be taken once per day for 6 weeks. Receives: 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. Receives: 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. Receives: Dietary Supplement: Serine; Drug: Fenofibrate |
| No treatment | No intervention | Control group: no investigational product taken |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Reported over the10 weeks from baseline to exit visit
Percentage Change of Serum Deoxysphingolipid Levels From Baseline to 6 Weeks
Percentage change in the sum of 7 different deoxy-dihydroceramides species detected. Negative values mean these metabolites decreased during the treatment period. Deoxy-sphingolipids were measured in serum using LC-MS.
Measured: Blood draw at baseline and 6 weeks on product
Secondary outcomes (2)
Percentage Change in Serum Lipid Levels From Baseline to 6 Weeks
Percent changes in lipid levels measured between baseline and 6 weeks. These lipid levels were obtained from the local clinical lab at each site as part of a lipid panel. Negative values mean these metabolites decreased during the treatment period.
Measured: blood draws from baseline and week 6 on product
Percentage Change in Serum Serine and Glycine Levels From Baseline to 6 Weeks
Percentage change in fasting levels of the two amino acids serine and glycine. Serine supplementation is part of the treatment and glycine can be produced from serine by different cells in the body. Serine and glycine levels were measured from serum using LC-MS.
Measured: blood draws from baseline and week 6 on product
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 May 2021
- Study start7 Apr 2022
- Primary completion30 Sep 2024
- Study completion30 Sep 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Macular Telangiectasia Type 2Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Factorial
- Model description
- 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).
- Masking
- None (open label)
- Enrolment
- 89 participants (actual)
Sponsor and investigators
- Lead sponsor
- The Lowy Medical Research Institute Limited
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Mari A Gantner, PhD, Principal investigator, Lowy Medical Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04907084
- Sponsor's protocol ID
- 2020-002
- Acronym
- SAFE
- Status last verified
- Aug 2026
- First submitted
- 25 May 2021
- Last updated
- 21 Sep 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Jung M, Bucher F. [Type 2 Macular Telangiectasia: Pathogenesis-The Interplay Between Neurodegeneration and Metabolic Changes]. Klin Monbl Augenheilkd. 2026 Aug 27. doi: 10.1055/a-2936-0118. Online ahead of print. German.
PubMed 42660511
Study at a glance
From the registry record, last updated by the sponsor on 21 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2a Study of the Effect of Serine Supplementation and Fenofibrate Treatment on Serum Deoxysphinganine Levels in Patients With Macular Telangiectasia (MacTel) Type 2 (SAFE Study)
- ClinicalTrials.gov ID
- NCT04907084
- Acronym / sponsor ID
- SAFE / 2020-002
- Conditions
- Macular Telangiectasia Type 2
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 89 participants (actual)
- Lead sponsor
- The Lowy Medical Research Institute LimitedUniversity, hospital or other
- Study start
- 7 Apr 2022 (actual)
- Primary completion
- 30 Sep 2024 (actual)
- Study completion
- 30 Sep 2024 (actual)
- First posted
- 28 May 2021 (actual)
- Results
- Posted 21 Sep 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- Fasting blood work
- Collection of microbiome samples
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Factorial
- Masking
- None (open label)
- Number of arms
- 6
- Model description
- 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).
| Arm | Type | Receives |
|---|---|---|
| Serine 200 mg/kg/day | Experimental | Dietary Supplement: SerineSerine 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 | Dietary Supplement: SerineSerine 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 | Drug: FenofibrateFenofibrate 160 mg per day. To be taken once per day for 6 weeks. |
| Serine 200 mg/kg/day and Fenofibrate 160 mg/day | Experimental | Dietary Supplement: Serine, Drug: FenofibrateSerine 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 | Dietary Supplement: Serine, Drug: FenofibrateSerine 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. |
| No treatment | No intervention | –Control group: no investigational product taken |
Who may qualify
- Ages
- 21 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (20 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SerinePowdered serine supplement (Dosed out individually per participant. Participant to mix with water and ingest orally) | Dietary supplement | 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 |
| FenofibrateFenofibrate 160mg pill, taken orally | Drug | Fenofibrate 160 mg/day, Serine 200 mg/kg/day and Fenofibrate 160 mg/day, Serine 400 mg/kg/day and Fenofibrate 160 mg/day |
Primary outcome
Safety Assessment
Time frame: Reported over the10 weeks from baseline to exit visit
Safety assessment measured via examined AEs and SAEs (during study visits) and via participant self-reported AEs and SAEs that occurred between visits
Percentage Change of Serum Deoxysphingolipid Levels From Baseline to 6 Weeks
Time frame: Blood draw at baseline and 6 weeks on product
Percentage change in the sum of 7 different deoxy-dihydroceramides species detected. Negative values mean these metabolites decreased during the treatment period. Deoxy-sphingolipids were measured in serum using LC-MS.
2 secondary outcomes
Percentage Change in Serum Lipid Levels From Baseline to 6 Weeks
Time frame: blood draws from baseline and week 6 on product
Percentage Change in Serum Serine and Glycine Levels From Baseline to 6 Weeks
Time frame: blood draws from baseline and week 6 on product
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Sep 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 42660511Jung M, Bucher F. [Type 2 Macular Telangiectasia: Pathogenesis-The Interplay Between Neurodegeneration and Metabolic Changes]. Klin Monbl Augenheilkd. 2026 Aug 27. doi: 10.1055/a-2936-0118. Online ahead of print. German.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mari A Gantner, PhD principal investigator
Lowy Medical Research Institute
9 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Serine and Fenofibrate in people with Macular Telangiectasia Type 2, run by The Lowy Medical Research Institute Limited at 9 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 21 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 21 Sep 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety Assessment (time frame: Reported over the10 weeks from baseline to exit visit). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04907084, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Sep 2026.