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Nicotinamide Riboside (NR) for Moyamoya-like Cerebrovascular Disease in SMDS

This study is looking at Nicotinamide Riboside (NR) to treat Moyamoya-like cerebrovascular disease in people with Smooth Muscle Dysfunction Syndrome (SMDS). Researchers want to see if NR can improve blood pressure, brain oxygen and blood flow, and thinking skills. They also want to check if NR is safe and well-tolerated, and if it affects glucose uptake in the aorta or NAD+ levels in the blood. You may be able to join if you have SMDS with a specific ACTA2 gene change (arginine 179 mutation) and have not had certain aortic surgeries. The study plans to enroll 15 participants, but its current status is unclear.

Study design
This is an interventional study with a planned enrollment of 15 participants. It is not specified if it is randomized or blinded.
What's involved
Participants will take a weight-based dose of Nicotinamide Riboside (NR) once daily by mouth with food for 60 days. Measurements for primary endpoints will be taken at baseline and 8 weeks.
Compensation
Not stated in the trial record.
Follow-up
Participants will be assessed at 8 weeks after starting treatment to measure changes in blood pressure, cerebral oxygenation, and cognitive function.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06280482

Nicotinamide Riboside (NR) to Treat Moyamoya-like Cerebrovascular Disease in Smooth Muscle Dysfunction Syndrome (SMDS)

Recruiting
PHASE1Ages 1+InterventionalTreatment
The University of Texas Health Science Center, Houston
~15 participants
Updated 2025-09-12 on ClinicalTrials.gov
What's tested:Nicotinamide riboside (NR)

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Change in systolic blood
Measured over Baseline , 8 weeks
+6 more outcomes measured

Conditions

Smooth Muscle Dysfunction Syndrome (SMDS)

Explore related trials

Multisystemic smooth muscle dysfunction syndromeACTA2 Gene

Where it's being run

1 sites across 1 states
Texas1

Study leadership

  • Dianna Milewicz, MD, PhD · PRINCIPAL_INVESTIGATOR · The University of Texas Health Science Center, Houston

Who to contact

Dianna M Milewicz, MD, PhD
[email protected]
(713) 500-6725
Email the study team

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Eligibility criteria

Inclusion

Individuals diagnosed with SMDS with confirmed ACTA2 mutation disrupting arginine 179
Parental/guardian permission (informed consent) and, if appropriate, child assent.

Exclusion

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.

What this trial measures

  • Change in systolic bloodBaseline , 8 weeks
  • Change in cerebral oxygenation and perfusion as assessed by the Head-up tilt table testing (HUTT) with near-infrared spectroscopy (NIRS) monitoring.Baseline , 8 weeks
  • Change in cognitive function as assessed by the NIH Toolbox assessmentBaseline , 8 weeks

    NIH Toolbox assessment evaluates multiple domains of cognitive performance including executive function, attention, episodic memory, language, processing speed, and working memory. Individual test scores are combined into a composite cognition score and reported as age-adjusted T-scores (mean = 50, SD = 10), where higher scores indicate better cognitive function.

  • Change in autonomic symptoms as assessed by the Composite Autonomic Symptom Score 31 (COMPASS-31) surveyBaseline , 8 weeks

    This is a 31 item questionnaire and each is scored from 0 (no symptoms) to 100(maximum symptom burden), with higher scores indicating more severe autonomic dysfunction.

  • Change in Impact of headaches as assessed by the Headache Impact Test (HIT-6) questionnaireBaseline, 8 weeks

    HIT-6 questionnaire consists of 6 questions, each scored from 6(never) to 13(always), with total scores ranging from 36 to 78. Higher scores indicate a greater impact of headaches on quality of life.

  • Pulmonary function tests (PFTs), including spirometry and lung volume measurementsBaseline, 8 weeks
  • Change in Aortic diameter as assessed by the echocardiographyBaseline , 8 weeks