Marfan SyndromeNCT07495267
Nutritional Ketosis Marfan
A study testing Ketogenic diet in people with Marfan Syndrome and Aortic Dissection, run by Washington University School of Medicine.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 50 years
- Conditions the study is for
- Marfan Syndrome, Aortic Dissection
- What is being tested?
- Ketogenic diet
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- No sites listed yet
- Study dates
- 13 Mar 2026to 1 Mar 2028
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 50 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 9Show fewer
- 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.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Nutritional Ketosis as a Novel Therapeutic Strategy to Stabilize Chronic Aortic Dissection in Marfan Syndrome
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.
- Conditions
- Marfan Syndrome, Aortic Dissection
- Being tested
- Ketogenic diet
- Medical subject headings
- Marfan Syndrome, Aortic Dissection
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ketogenic dietOther
Participants will continue ketogenic diet for 12 months.
| Study group | Role | What they receive |
|---|---|---|
| Prospective Cohort | Experimental | Participants will initiate a supervised ketogenic diet formulated to provide approximately 70-75% of calories from fat, 20% from protein, and less than 10% from carbohydrates, consistent with protocols successfully used in therapeutic epilepsy trials but adapted for adult cardiovascular patients.
They 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. Receives: Other: Ketogenic diet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Freedom from aortic intervention
Freedom from aortic intervention at 12 months compared with historical controls.
Measured: 12 months
Secondary outcomes (2)
Aortic dissection and inflammatory markers
Observation of changes related to aortic dissection and inflammatory markers
Measured: 12 months
Number of Participants who Maintained Diet Adherence measured by Ketone Monitor
In order to measure adherence, participants will use a handheld ketone meter (Keto-Mojo) to measure β-hydroxybutyrate levels three times per week via saliva as well as finger-stick blood testing.
Measured: From day 1 to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Mar 2026
- Study start13 Mar 2026 (estimated)
- Primary completion1 Mar 2028 (estimated)
- Study completion1 Mar 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (27)Papers the sponsor has linked to this study.
- Other trials for Marfan SyndromeStudies of the same condition, open and under way.
- More trials run by Washington University School of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Washington University School of Medicine
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07495267
- Sponsor's protocol ID
- 202510143
- Status last verified
- Mar 2026
- First submitted
- 12 Mar 2026
- Last updated
- 27 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 40411493Huang 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.
PubMed 36794812Neves 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.
PubMed 33230563Roehl 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.
PubMed 28754198Campello 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.
PubMed 40008501Loeys 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.
PubMed 20591885Wagner 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.
PubMed 30926475Wheeler 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.
PubMed 34807419Huang K, Wang Y, Siu KL, Zhang Y, Cai H. Targeting feed-forward signaling of TGFbeta/NOX4/DHFR/eNOS uncoupling/TGFbeta axis with anti-TGFbeta and folic acid attenuates formation of aortic aneurysms: Novel mechanisms and therapeutics. Redox Biol. 2021 Jan;38:101757. doi: 10.1016/j.redox.2020.101757. Epub 2020 Oct 13.
PubMed 33126053Zhu H, Bi D, Zhang Y, Kong C, Du J, Wu X, Wei Q, Qin H. Ketogenic diet for human diseases: the underlying mechanisms and potential for clinical implementations. Signal Transduct Target Ther. 2022 Jan 17;7(1):11. doi: 10.1038/s41392-021-00831-w.
PubMed 35034957Patikorn C, Saidoung P, Pham T, Phisalprapa P, Lee YY, Varady KA, Veettil SK, Chaiyakunapruk N. Effects of ketogenic diet on health outcomes: an umbrella review of meta-analyses of randomized clinical trials. BMC Med. 2023 May 25;21(1):196. doi: 10.1186/s12916-023-02874-y.
PubMed 37231411Chamma L, Chamma N, Mattar L, Slaybe M, Haidar S, Rizk R. Beyond epilepsy management: A narrative review of the health effects of ketogenic diets. Nutrition. 2025 Sep;137:112804. doi: 10.1016/j.nut.2025.112804. Epub 2025 Apr 16.
PubMed 40403346Augustin K, Khabbush A, Williams S, Eaton S, Orford M, Cross JH, Heales SJR, Walker MC, Williams RSB. Mechanisms of action for the medium-chain triglyceride ketogenic diet in neurological and metabolic disorders. Lancet Neurol. 2018 Jan;17(1):84-93. doi: 10.1016/S1474-4422(17)30408-8. Epub 2017 Dec 16.
PubMed 29263011Weng X, Pan L, Ma X, Luo W, Su H, Pei Z, Dong Z, Liu L, Yang J, Gao P, Sun A. Ketogenic diet and beta-hydroxybutyrate inhibit HDAC1 to preserve vascular smooth muscle cell function in thoracic aortic aneurysm. J Adv Res. 2026 Feb;80:475-486. doi: 10.1016/j.jare.2025.05.035. Epub 2025 May 19.
PubMed 40398746Sastriques-Dunlop S, Elizondo-Benedetto S, Arif B, Meade R, Zaghloul MS, English SJ, Liu Y, Zayed MA. Ketosis Prevents Abdominal Aortic Aneurysm Rupture Through CCR2 Downregulation and Enhanced MMP Balance. bioRxiv [Preprint]. 2023 Feb 22:2023.02.21.529460. doi: 10.1101/2023.02.21.529460.
PubMed 36865192
Study at a glance
From the registry record, last updated by the sponsor on 27 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Nutritional Ketosis as a Novel Therapeutic Strategy to Stabilize Chronic Aortic Dissection in Marfan Syndrome
- ClinicalTrials.gov ID
- NCT07495267
- Acronym / sponsor ID
- 202510143
- Conditions
- Marfan Syndrome, Aortic Dissection
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Washington University School of MedicineUniversity, hospital or other
- Study start
- 13 Mar 2026 (estimated)
- Primary completion
- 1 Mar 2028 (estimated)
- Study completion
- 1 Mar 2028 (estimated)
- First posted
- 27 Mar 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Prospective Cohort | Experimental | Other: Ketogenic dietParticipants will initiate a supervised ketogenic diet formulated to provide approximately 70-75% of calories from fat, 20% from protein, and less than 10% from carbohydrates, consistent with protocols successfully used in therapeutic epilepsy trials but adapted for adult cardiovascular patients. They 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. |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- 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)
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Ketogenic dietParticipants will continue ketogenic diet for 12 months. | Other | Prospective Cohort |
Primary outcome
Freedom from aortic intervention
Time frame: 12 months
Freedom from aortic intervention at 12 months compared with historical controls.
2 secondary outcomes
Aortic dissection and inflammatory markers
Time frame: 12 months
Number of Participants who Maintained Diet Adherence measured by Ketone Monitor
Time frame: From day 1 to 12 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Mar 2026.
- Background: PubMed 40411493Kaul 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.
- Background: PubMed 36794812Huang 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.
- Background: PubMed 33230563Neves 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.
- Background: PubMed 28754198Roehl 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.
- Background: PubMed 40008501Campello 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.
- Background: PubMed 20591885Loeys 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.
- Background: PubMed 30926475Wagner 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.
- Background: PubMed 34807419Wheeler 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.
- Background: PubMed 33126053Huang K, Wang Y, Siu KL, Zhang Y, Cai H. Targeting feed-forward signaling of TGFbeta/NOX4/DHFR/eNOS uncoupling/TGFbeta axis with anti-TGFbeta and folic acid attenuates formation of aortic aneurysms: Novel mechanisms and therapeutics. Redox Biol. 2021 Jan;38:101757. doi: 10.1016/j.redox.2020.101757. Epub 2020 Oct 13.
- Background: PubMed 35034957Zhu H, Bi D, Zhang Y, Kong C, Du J, Wu X, Wei Q, Qin H. Ketogenic diet for human diseases: the underlying mechanisms and potential for clinical implementations. Signal Transduct Target Ther. 2022 Jan 17;7(1):11. doi: 10.1038/s41392-021-00831-w.
- Background: PubMed 37231411Patikorn C, Saidoung P, Pham T, Phisalprapa P, Lee YY, Varady KA, Veettil SK, Chaiyakunapruk N. Effects of ketogenic diet on health outcomes: an umbrella review of meta-analyses of randomized clinical trials. BMC Med. 2023 May 25;21(1):196. doi: 10.1186/s12916-023-02874-y.
- Background: PubMed 40403346Chamma L, Chamma N, Mattar L, Slaybe M, Haidar S, Rizk R. Beyond epilepsy management: A narrative review of the health effects of ketogenic diets. Nutrition. 2025 Sep;137:112804. doi: 10.1016/j.nut.2025.112804. Epub 2025 Apr 16.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A study testing Ketogenic diet in people with Marfan Syndrome and Aortic Dissection, run by Washington University School of Medicine.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 27 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Mohamed Zayed, MD, PhD, MBA, DFSVS, FAHA, FAC ((314) 362-5648, zayedm@wustl.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Freedom from aortic intervention (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07495267, which also lists 27 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Mar 2026.