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Serine and Fenofibrate Study in Patients With MacTel Type 2 (SAFE)

Primary Purpose

Macular Telangiectasia Type 2

Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Serine
Fenofibrate
Sponsored by
The Lowy Medical Research Institute Limited
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional other trial for Macular Telangiectasia Type 2

Eligibility Criteria

21 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Signed and dated written informed consent obtained from the participant in accordance with 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 diagnosed with 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 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, a serine or glycine supplement;
  4. Participant is currently taking, or has taken within 12 months prior to screening, fibrates including clofibrate, ciprofibrate, bezafibrate, fenofibrate, and gemfibrozil;
  5. Participant is currently taking an anticoagulant, colchicine, cyclosporine, tacrolimus or 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 related to 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/ or glomerular filtration rate (GFR) less than 90 mL/min;
  10. 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;
  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 greater than 700 mg/dL on no treatment;
  13. Participant has untreated/uncured Hepatitis C, or a history of Hepatitis B, autoimmune hepatitis, or HIV;
  14. Participant has had any malignancies within the last 5 years (not including basal cell carcinoma);
  15. Participant has ever been enrolled in a clinical trial involving ciliary neurotrophic factor (CNTF) treatment;
  16. Participant is currently enrolled in another clinical trial that involves treatment or participated 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 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
  20. Patient is unavailable for follow-up visits. *based on reference range for the local laboratory used

Sites / Locations

  • Emory UniversityRecruiting
  • Kellogg Eye Center, University of MichiganRecruiting
  • Retina Associates of ClevelandRecruiting
  • Southeastern Retina AssociatesRecruiting
  • Retina Consultants of TexasRecruiting
  • Moran Eye Center, University of UtahRecruiting
  • University of WashingtonRecruiting
  • The Eye Institute, Medical College of WisconsinRecruiting
  • Moorfields Eye HospitalRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm 6

Arm Type

Experimental

Experimental

Experimental

Experimental

Experimental

No Intervention

Arm Label

Serine 200 mg/kg/day

Serine 400 mg/kg/day

Fenofibrate 160 mg/day

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

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

No treatment

Arm Description

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 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 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 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.

Control group: no investigational product taken

Outcomes

Primary Outcome Measures

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
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

Secondary Outcome Measures

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
Amino Acid Levels
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

Full Information

First Posted
May 25, 2021
Last Updated
March 22, 2023
Sponsor
The Lowy Medical Research Institute Limited
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1. Study Identification

Unique Protocol Identification Number
NCT04907084
Brief Title
Serine and Fenofibrate Study in Patients With MacTel Type 2
Acronym
SAFE
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)
Study Type
Interventional

2. Study Status

Record Verification Date
March 2023
Overall Recruitment Status
Recruiting
Study Start Date
April 7, 2022 (Actual)
Primary Completion Date
June 2023 (Anticipated)
Study Completion Date
December 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
The Lowy Medical Research Institute Limited

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
Yes
Data Monitoring Committee
No

5. Study Description

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: Fasting blood work Collection of microbiome samples

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Macular Telangiectasia Type 2

7. Study Design

Primary Purpose
Other
Study Phase
Phase 2
Interventional Study Model
Factorial Assignment
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)
Masking Description
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).
Allocation
Randomized
Enrollment
60 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Serine 200 mg/kg/day
Arm Type
Experimental
Arm Description
Serine 200 mg/kg per day. To be taken once per day for 6 weeks. Exact dose dependent on participant's weight (in kg)).
Arm Title
Serine 400 mg/kg/day
Arm Type
Experimental
Arm Description
Serine 400 mg/kg per day. To be taken once per day for 6 weeks. Exact dose dependent on participant's weight (in kg)).
Arm Title
Fenofibrate 160 mg/day
Arm Type
Experimental
Arm Description
Fenofibrate 160 mg per day. To be taken once per day for 6 weeks.
Arm Title
Serine 200 mg/kg/day and Fenofibrate 160 mg/day
Arm Type
Experimental
Arm Description
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.
Arm Title
Serine 400 mg/kg/day and Fenofibrate 160 mg/day
Arm Type
Experimental
Arm Description
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.
Arm Title
No treatment
Arm Type
No Intervention
Arm Description
Control group: no investigational product taken
Intervention Type
Dietary Supplement
Intervention Name(s)
Serine
Intervention Description
Powdered serine supplement (Dosed out individually per participant. Participant to mix with water and ingest orally)
Intervention Type
Drug
Intervention Name(s)
Fenofibrate
Intervention Description
Fenofibrate 160mg pill, taken orally
Primary Outcome Measure Information:
Title
Serum Deosxysphingolipid Levels
Description
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
Time Frame
blood draws from week 3, 6, and 10
Title
Safety Assessment
Description
Safety assessment measured via examined AEs and SAEs (during study visits) and via participant self-reported AEs and SAEs that occurred between visits
Time Frame
Assessment at each study visit (reviewed at week 3, week 6, and week 10 of study)
Secondary Outcome Measure Information:
Title
Lipid Levels
Description
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
Time Frame
blood draws from week 3, 6, and 10
Title
Amino Acid Levels
Description
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
Time Frame
blood draws from week 3, 6, and 10

10. Eligibility

Sex
All
Minimum Age & Unit of Time
21 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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. Exclusion Criteria: 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Jennifer Trombley, RN MSN DAOM CCRC
Phone
858-249-7109
Email
jtrombley@lmri.net
First Name & Middle Initial & Last Name or Official Title & Degree
Katie Nardo, MPH
Phone
858-249-7113
Email
knardo@lmri.net
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Mari A Gantner, PhD
Organizational Affiliation
Lowy Medical Research Institute
Official's Role
Principal Investigator
Facility Information:
Facility Name
Emory University
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Donna Leef
Phone
404-778-4134
Email
dleef@emory.edu
First Name & Middle Initial & Last Name & Degree
Jiong Yan, MD
Facility Name
Kellogg Eye Center, University of Michigan
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48105
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Pamela Campbell
Email
pamtitus@med.umich.edu
First Name & Middle Initial & Last Name & Degree
Grant Comer, MD
Facility Name
Retina Associates of Cleveland
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44122
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Dianne Himmelman, RN
Phone
216-831-5700
Ext
253
Email
dhimmelman@retina-assoc.com
First Name & Middle Initial & Last Name & Degree
Lawrence Singerman, MD
Facility Name
Southeastern Retina Associates
City
Knoxville
State/Province
Tennessee
ZIP/Postal Code
37922
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kristina Oliver
Email
koliver@seretina.com
First Name & Middle Initial & Last Name & Degree
Scott Barb, MD
Facility Name
Retina Consultants of Texas
City
Bellaire
State/Province
Texas
ZIP/Postal Code
77401
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Sarah Weidlein
Phone
832-200-3905
Email
sarah.weidlein@retinaconsultantstexas.com
First Name & Middle Initial & Last Name & Degree
Charles Wykoff, MD
Facility Name
Moran Eye Center, University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Barbara Hart
Email
barbara.hart@hsc.utah.edu
First Name & Middle Initial & Last Name & Degree
Paul Bernstein, MD
Facility Name
University of Washington
City
Seattle
State/Province
Washington
ZIP/Postal Code
98104
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Simona Vuletic
Email
simona@uw.edu
First Name & Middle Initial & Last Name & Degree
Lisa Olmos de Koo, MD
Facility Name
The Eye Institute, Medical College of Wisconsin
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
David Weinberg, MD
Phone
414-955-2026
Email
dweinber@mcw.edu
First Name & Middle Initial & Last Name & Degree
David Weinberg, MD
Facility Name
Moorfields Eye Hospital
City
London
ZIP/Postal Code
ECV2PD
Country
United Kingdom
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Abraham Olvera, MD
Email
aolvera@nhs.net
First Name & Middle Initial & Last Name & Degree
Cathy Egan, MD

12. IPD Sharing Statement

Plan to Share IPD
No

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Serine and Fenofibrate Study in Patients With MacTel Type 2

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