Nutritional Ketosis Marfan

Официальное название

Nutritional Ketosis as a Novel Therapeutic Strategy to Stabilize Chronic Aortic Dissection in Marfan Syndrome

Обзор

In this pilot clinical trial, the investigators will test whether a carefully supervised ketogenic diet can safely help people with Marfan syndrome who already have a chronic or residual aortic dissection. Fifteen participants will work with a study coordinator and dietitian to learn how to follow the diet and will have regular guidance and check-ins. The participants will measure their blood ketone levels at home with a simple finger-stick device and relay their readings to the research team. Every three months, blood samples will also be collected to confirm the body's level of ketosis. Participants will continue their usual heart and blood-pressure medicines and regular visits with their doctors. Standard of care computed tomography (CT) scans of the aorta will be performed at the start of the study and then again at 6 and 12 months to see if the aorta changes in size. The main goal is to see how many patients can avoid needing aortic surgery within one year, compared with what is normally expected from past studies. The investigators will also track how well patients follow the diet, how their aorta changes, and whether inflammation in their blood decreases.

Вмешательства

• Другое Ketogenic diet
  Participants will continue ketogenic diet for 12 months.

Первичные конечные точки

• Freedom from aortic intervention [Срок оценки: 12 months]

Вторичные конечные точки (2)

• Aortic dissection and inflammatory markers [Срок оценки: 12 months]
• Number of Participants who Maintained Diet Adherence measured by Ketone Monitor [Срок оценки: From day 1 to 12 months]

Критерии участия

Критерии включения

• Adults between 18 and 50 years old.
• Ability to understand and willingness to sign an Institutional Review Board (IRB) approved written informed consent document.
• Genetically or clinically confirmed Marfan syndrome
• Chronic descending thoracic aortic dissection present for at least three months
• Maximal descending thoracic aortic diameter must measure between 4.0 and 5.0 cm at baseline on contrast-enhanced computed tomography angiography (CTA)
• Have been clinically stable and consistent antihypertensive regimen for at least four weeks

Критерии исключения

• Acute or rapidly enlarging dissection (>0.5 cm increase over the preceding three months)
• Prior descending thoracic aortic repair or endograft placement
• Advanced renal or hepatic dysfunction
• Poorly controlled diabetes requiring insulin
• Active malignancy, pregnancy, or other metabolic or nutritional disorders that would contraindicate ketogenic therapy
• Body mass index below 18 kg/m²
• Inability to comply with dietary restrictions or follow-up imaging
• Current enrollment in another interventional study, which in the opinion of the principal investigator, may confound study results.
• Patients who, in the opinion of the principal investigator, are not suitable candidates for study participation.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Другое

Центры проведения

Список центров уточняется — проверьте первичный протокол.

Публикации

• Kaul N, Duan J, Cui D, Erlichster M, Chen Z, Anderson D, Chan J, Scheffer IE, Skafidas E, Liao J, Kwan P. Serial correlation between saliva and blood beta-hydroxybutyrate levels in children commencing the ketogenic diet for epilepsy. Epilepsia. 2025 Sep;66(9):3282-3292. doi: 10.1111/epi.18465. Epub 2025 May 24. PMID 40411493
• Huang J, Yeung AM, Bergenstal RM, Castorino K, Cengiz E, Dhatariya K, Niu I, Sherr JL, Umpierrez GE, Klonoff DC. Update on Measuring Ketones. J Diabetes Sci Technol. 2024 May;18(3):714-726. doi: 10.1177/19322968231152236. Epub 2023 Feb 16. PMID 36794812
• Neves GS, Lunardi MS, Lin K, Rieger DK, Ribeiro LC, Moreira JD. Ketogenic diet, seizure control, and cardiometabolic risk in adult patients with pharmacoresistant epilepsy: a review. Nutr Rev. 2021 Jul 7;79(8):931-944. doi: 10.1093/nutrit/nuaa112. PMID 33230563
• Roehl K, Sewak SL. Practice Paper of the Academy of Nutrition and Dietetics: Classic and Modified Ketogenic Diets for Treatment of Epilepsy. J Acad Nutr Diet. 2017 Aug;117(8):1279-1292. doi: 10.1016/j.jand.2017.06.006. PMID 28754198
• Campello Jorge CA, Marway PS, Tjahjadi NS, Knauer HA, Patel HJ, Hofmann Bowman M, Eagle K, Burris NS. Growth Rate Assessed by Vascular Deformation Mapping Predicts Type B Aortic Dissection in Marfan Syndrome. J Am Heart Assoc. 2025 Mar 18;14(6):e039179. doi: 10.1161/JAHA.124.039179. Epub 2025 Feb 26. PMID 40008501
• Loeys BL, Dietz HC, Braverman AC, Callewaert BL, De Backer J, Devereux RB, Hilhorst-Hofstee Y, Jondeau G, Faivre L, Milewicz DM, Pyeritz RE, Sponseller PD, Wordsworth P, De Paepe AM. The revised Ghent nosology for the Marfan syndrome. J Med Genet. 2010 Jul;47(7):476-85. doi: 10.1136/jmg.2009.072785. PMID 20591885
• Wagner AH, Zaradzki M, Arif R, Remes A, Muller OJ, Kallenbach K. Marfan syndrome: A therapeutic challenge for long-term care. Biochem Pharmacol. 2019 Jun;164:53-63. doi: 10.1016/j.bcp.2019.03.034. Epub 2019 Mar 27. PMID 30926475
• Wheeler JB, Ikonomidis JS, Jones JA. Connective Tissue Disorders and Cardiovascular Complications: The Indomitable Role of Transforming Growth Factor-beta Signaling. Adv Exp Med Biol. 2021;1348:161-184. doi: 10.1007/978-3-030-80614-9_7. PMID 34807419

Идентификаторы

NCT: NCT07495267 · 202510143

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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