Using Nicotinamide Riboside to Treat Smooth Muscle Dysfunction Syndrome
Official title: Nicotinamide Riboside (NR) to Treat Moyamoya-like Cerebrovascular Disease in Smooth Muscle Dysfunction Syndrome (SMDS)
Run by The University of Texas Health Science Center, Houston · NCT06280482 · Data updated 2026-11-12
This study is testing if Nicotinamide Riboside can help people with Smooth Muscle Dysfunction Syndrome and a specific genetic mutation by looking at its effects on glucose levels and safety.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Ages | 1 Year and up |
| Sex | All |
| Enrollment | 15 (estimated) |
| Sponsor | The University of Texas Health Science Center, Houston Academic / other |
| Locations | 1 site (Houston, Texas) |
| Status | Recruiting |
| Started | March 6, 2024 |
| Main results expected | July 25, 2027 |
| Study ends | July 25, 2027 |
| Registry record updated | September 12, 2025 |
| Trial ID | NCT06280482 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates for this study are patients with Smooth Muscle Dysfunction Syndrome who have a confirmed ACTA2 mutation and meet specific inclusion criteria.
✕Probably not a fit
Patients who have undergone aortic surgery or have other medical conditions that impair their ability to participate may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This study aims to evaluate the effects of Nicotinamide Riboside (NR) on patients diagnosed with Smooth Muscle Dysfunction Syndrome (SMDS) who have a specific genetic mutation. The research will assess whether NR treatment can lead to decreased glucose uptake in the aorta and measurable changes in blood levels of NAD+ and NR. Additionally, the study will monitor the stability of aortic measurements post-treatment and evaluate the safety and tolerability of NR in this patient population.
Why it matters
Potential benefit: If successful, this treatment could improve vascular health and reduce the risk of cerebrovascular events in patients with SMDS.
How similar studies have gone: While there is limited data on the specific use of NR for SMDS, similar approaches targeting metabolic pathways have shown promise in other cerebrovascular conditions.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Individuals diagnosed with SMDS with confirmed ACTA2 mutation disrupting arginine 179 * Parental/guardian permission (informed consent) and, if appropriate, child assent. Exclusion Criteria: * Individuals who have undergone surgery to replace aneurysmal or dissected ascending and root aortic tissue with a graft. * Additional medical conditions that impair the patient's ability to participate in the study. * Known allergy or sensitivity to niacin or nicotinamide riboside. * Prior consumption of niacin or nicotinamide riboside supplement within the prior eight weeks. * Failure to provide informed consent. * Concurrent participation in another intervention trial.
Where it's running
-
The University of Texas Health Science Center at Houston
Houston, Texas, United States Recruiting
Study contacts
- Principal investigator: Dianna Milewicz, MD, PhD — The University of Texas Health Science Center, Houston
- Study coordinator: Dianna M Milewicz, MD, PhD
- Email: Dianna.M.Milewicz@uth.tmc.edu
- Phone: (713) 500-6725
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.