Studio di serina e fenofibrato in pazienti con MacTel di tipo 2 (SAFE)
Studio di fase 2a sull'effetto della supplementazione di serina e del trattamento con fenofibrato sui livelli sierici di desossisfinganina in pazienti con telangiectasia maculare (MacTel) di tipo 2 (studio 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.
Panoramica dello studio
Stato
Condizioni
Intervento / Trattamento
Descrizione dettagliata
Additional Procedures include:
- Fasting blood work
- Collection of microbiome samples
Tipo di studio
Iscrizione (Effettivo)
Fase
- Fase 2
Contatti e Sedi
Luoghi di studio
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-
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London, Regno Unito, ECV2PD
- Moorfields Eye Hospital
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-
-
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Georgia
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Atlanta, Georgia, Stati Uniti, 30322
- Emory University
-
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Michigan
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Ann Arbor, Michigan, Stati Uniti, 48105
- Kellogg Eye Center, University of Michigan
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Ohio
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Cleveland, Ohio, Stati Uniti, 44122
- Retina Associates of Cleveland
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Tennessee
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Knoxville, Tennessee, Stati Uniti, 37922
- Southeastern Retina Associates
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Texas
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Bellaire, Texas, Stati Uniti, 77401
- Retina Consultants of Texas
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Utah
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Salt Lake City, Utah, Stati Uniti, 84132
- Moran Eye Center, University of Utah
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Washington
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Seattle, Washington, Stati Uniti, 98104
- University of Washington
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Wisconsin
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Milwaukee, Wisconsin, Stati Uniti, 53226
- The Eye Institute, Medical College of Wisconsin
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-
Criteri di partecipazione
Criteri di ammissibilità
Età idonea allo studio
Accetta volontari sani
Sessi ammissibili allo studio
Descrizione
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
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Altro
- Assegnazione: Randomizzato
- Modello interventistico: Assegnazione fattoriale
- Mascheramento: Nessuno (etichetta aperta)
Armi e interventi
Gruppo di partecipanti / Arm |
Intervento / Trattamento |
|---|---|
|
Sperimentale: 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) |
|
Sperimentale: 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) |
|
Sperimentale: Fenofibrate 160 mg/day
Fenofibrate 160 mg per day. To be taken once per day for 6 weeks. |
Fenofibrate 160mg pill, taken orally |
|
Sperimentale: 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 |
|
Sperimentale: 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 |
|
Nessun intervento: No treatment
Control group: no investigational product taken |
Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Safety Assessment
Lasso di tempo: 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 |
Reported over the10 weeks from baseline to exit visit
|
|
Percentage Change of Serum Deoxysphingolipid Levels From Baseline to 6 Weeks
Lasso di tempo: 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. |
Blood draw at baseline and 6 weeks on product
|
Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Percentage Change in Serum Lipid Levels From Baseline to 6 Weeks
Lasso di tempo: blood draws from baseline and week 6 on product
|
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
|
|
Percentage Change in Serum Serine and Glycine Levels From Baseline to 6 Weeks
Lasso di tempo: blood draws from baseline and week 6 on product
|
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
|
Collaboratori e investigatori
Investigatori
- Investigatore principale: Mari A Gantner, PhD, Lowy Medical Research Institute
Pubblicazioni e link utili
Studiare le date dei record
Studia le date principali
Inizio studio (Effettivo)
Completamento primario (Effettivo)
Completamento dello studio (Effettivo)
Date di iscrizione allo studio
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Effettivo)
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo verificato
Maggiori informazioni
Termini relativi a questo studio
Termini MeSH pertinenti aggiuntivi
- Aminoacidi, peptidi e proteine
- Prodotti chimici organici
- ETHETER
- Idrocarburi
- Idrocarburi, ciclici
- Acidi, aciclici
- Acidi carbossilici
- Idrocarburi, aromatici
- Aminoacidi
- Fenoli
- Derivati di benzene
- Butirati
- Fenil eteri
- Chetoni
- Acidi fibrici
- Isobutirati
- Benzofenoni
- Aminoacidi, neutri
- Fenofibrato
- Serina
Altri numeri di identificazione dello studio
- 2020-002
Piano per i dati dei singoli partecipanti (IPD)
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Informazioni su farmaci e dispositivi, documenti di studio
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