MacTel Type 2 환자의 세린 및 페노피브레이트 연구 (SAFE)
황반 모세혈관확장증(MacTel) 2형 환자의 혈청 데옥시스핑가닌 수치에 대한 세린 보충 및 페노피브레이트 치료의 효과에 대한 2a상 연구(SAFE 연구)
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.
연구 개요
상세 설명
Additional Procedures include:
- Fasting blood work
- Collection of microbiome samples
연구 유형
등록 (실제)
단계
- 2 단계
연락처 및 위치
연구 장소
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Georgia
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Atlanta, Georgia, 미국, 30322
- Emory University
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Michigan
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Ann Arbor, Michigan, 미국, 48105
- Kellogg Eye Center, University of Michigan
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Ohio
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Cleveland, Ohio, 미국, 44122
- Retina Associates of Cleveland
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Tennessee
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Knoxville, Tennessee, 미국, 37922
- Southeastern Retina Associates
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Texas
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Bellaire, Texas, 미국, 77401
- Retina Consultants of Texas
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Utah
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Salt Lake City, Utah, 미국, 84132
- Moran Eye Center, University of Utah
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Washington
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Seattle, Washington, 미국, 98104
- University of Washington
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Wisconsin
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Milwaukee, Wisconsin, 미국, 53226
- The Eye Institute, Medical College of Wisconsin
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London, 영국, ECV2PD
- Moorfields Eye Hospital
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참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
설명
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
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 다른
- 할당: 무작위
- 중재 모델: 요인 할당
- 마스킹: 없음(오픈 라벨)
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
|---|---|
|
실험적: Serine 200 mg/kg/day
Serine 200 mg/kg per day. To be taken once per day for 6 weeks. Exact dose dependent on participant's weight (in kg)). |
Powdered serine supplement (Dosed out individually per participant. Participant to mix with water and ingest orally) |
|
실험적: Serine 400 mg/kg/day
Serine 400 mg/kg per day. To be taken once per day for 6 weeks. Exact dose dependent on participant's weight (in kg)). |
Powdered serine supplement (Dosed out individually per participant. Participant to mix with water and ingest orally) |
|
실험적: Fenofibrate 160 mg/day
Fenofibrate 160 mg per day. To be taken once per day for 6 weeks. |
Fenofibrate 160mg pill, taken orally |
|
실험적: Serine 200 mg/kg/day and Fenofibrate 160 mg/day
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. |
Powdered serine supplement (Dosed out individually per participant. Participant to mix with water and ingest orally) Fenofibrate 160mg pill, taken orally |
|
실험적: Serine 400 mg/kg/day and Fenofibrate 160 mg/day
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. |
Powdered serine supplement (Dosed out individually per participant. Participant to mix with water and ingest orally) Fenofibrate 160mg pill, taken orally |
|
간섭 없음: No treatment
Control group: no investigational product taken |
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
|
Safety Assessment
기간: Reported over the10 weeks from baseline to exit visit
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Safety assessment measured via examined AEs and SAEs (during study visits) and via participant self-reported AEs and SAEs that occurred between visits |
Reported over the10 weeks from baseline to exit visit
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Percentage Change of Serum Deoxysphingolipid Levels From Baseline to 6 Weeks
기간: Blood draw at baseline and 6 weeks on product
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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. |
Blood draw at baseline and 6 weeks on product
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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Percentage Change in Serum Lipid Levels From Baseline to 6 Weeks
기간: blood draws from baseline and week 6 on product
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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. |
blood draws from baseline and week 6 on product
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Percentage Change in Serum Serine and Glycine Levels From Baseline to 6 Weeks
기간: blood draws from baseline and week 6 on product
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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. |
blood draws from baseline and week 6 on product
|
공동 작업자 및 조사자
수사관
- 수석 연구원: Mari A Gantner, PhD, Lowy Medical Research Institute
간행물 및 유용한 링크
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
기본 완료 (실제)
연구 완료 (실제)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
추가 관련 MeSH 약관
기타 연구 ID 번호
- 2020-002
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
미국에서 제조되어 미국에서 수출되는 제품
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