Studie serinu a fenofibrátu u pacientů s MacTel typem 2 (SAFE)
Studie fáze 2a vlivu suplementace serinem a léčby fenofibrátem na hladiny deoxysfinganinu v séru u pacientů s makulární teleangiektázií (MacTel) typu 2 (studie SAFE)
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.
Přehled studie
Postavení
Podmínky
Intervence / Léčba
Detailní popis
Additional Procedures include:
- Fasting blood work
- Collection of microbiome samples
Typ studie
Zápis (Aktuální)
Fáze
- Fáze 2
Kontakty a umístění
Studijní místa
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London, Spojené království, ECV2PD
- Moorfields Eye Hospital
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Georgia
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Atlanta, Georgia, Spojené státy, 30322
- Emory University
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Michigan
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Ann Arbor, Michigan, Spojené státy, 48105
- Kellogg Eye Center, University of Michigan
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Ohio
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Cleveland, Ohio, Spojené státy, 44122
- Retina Associates of Cleveland
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Tennessee
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Knoxville, Tennessee, Spojené státy, 37922
- Southeastern Retina Associates
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Texas
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Bellaire, Texas, Spojené státy, 77401
- Retina Consultants of Texas
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Utah
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Salt Lake City, Utah, Spojené státy, 84132
- Moran Eye Center, University of Utah
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Washington
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Seattle, Washington, Spojené státy, 98104
- University of Washington
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Wisconsin
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Milwaukee, Wisconsin, Spojené státy, 53226
- The Eye Institute, Medical College of Wisconsin
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Kritéria účasti
Kritéria způsobilosti
Věk způsobilý ke studiu
Přijímá zdravé dobrovolníky
Pohlaví způsobilá ke studiu
Popis
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
Studijní plán
Jak je studie koncipována?
Detaily designu
- Primární účel: Jiný
- Přidělení: Randomizované
- Intervenční model: Faktorové přiřazení
- Maskování: Žádné (otevřený štítek)
Zbraně a zásahy
Skupina účastníků / Arm |
Intervence / Léčba |
|---|---|
|
Experimentální: 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) |
|
Experimentální: 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) |
|
Experimentální: Fenofibrate 160 mg/day
Fenofibrate 160 mg per day. To be taken once per day for 6 weeks. |
Fenofibrate 160mg pill, taken orally |
|
Experimentální: 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 |
|
Experimentální: 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 |
|
Žádný zásah: No treatment
Control group: no investigational product taken |
Co je měření studie?
Primární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
|---|---|---|
|
Safety Assessment
Časové okno: 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
Časové okno: 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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Sekundární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
|---|---|---|
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Percentage Change in Serum Lipid Levels From Baseline to 6 Weeks
Časové okno: 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
Časové okno: 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
|
Spolupracovníci a vyšetřovatelé
Vyšetřovatelé
- Vrchní vyšetřovatel: Mari A Gantner, PhD, Lowy Medical Research Institute
Publikace a užitečné odkazy
Termíny studijních záznamů
Hlavní termíny studia
Začátek studia (Aktuální)
Primární dokončení (Aktuální)
Dokončení studie (Aktuální)
Termíny zápisu do studia
První předloženo
První předloženo, které splnilo kritéria kontroly kvality
První zveřejněno (Aktuální)
Aktualizace studijních záznamů
Poslední zveřejněná aktualizace (Aktuální)
Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality
Naposledy ověřeno
Více informací
Termíny související s touto studií
Další relevantní podmínky MeSH
- Aminokyseliny, peptidy a proteiny
- Organické chemikálie
- Ethers
- Uhlovodíky
- Uhlovodíky, cyklické
- Kyseliny, acyklické
- Karboxylové kyseliny
- Uhlovodíky, aromatické
- Aminokyseliny
- Fenoly
- Deriváty benzenu
- Butyráty
- Fenyl ethery
- Ketony
- Kyseliny fibrové
- Isobutyrates
- Benzofenony
- Aminokyseliny, neutrální
- Fenofibrát
- Serin
Další identifikační čísla studie
- 2020-002
Plán pro data jednotlivých účastníků (IPD)
Plánujete sdílet data jednotlivých účastníků (IPD)?
Informace o lécích a zařízeních, studijní dokumenty
Studuje lékový produkt regulovaný americkým FDA
Studuje produkt zařízení regulovaný americkým úřadem FDA
produkt vyrobený a vyvážený z USA
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