Nicotinamide Riboside (NR) to Treat Moyamoya-like Cerebrovascular Disease in Smooth Muscle Dysfunction Syndrome (SMDS)
RecruitingPhase 1
Who can join
Ages 1 and older · All sexes
Full eligibility 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.
About the study
The purpose of this study is to determine whether SMDS patients treated with NR at the proposed dose exhibit decreased glucose uptake in the aorta, to determine if NR treatment results in measurable changes of blood NAD+ and NR levels, to determine if aortic measurements are stable after treatment with NR and to evaluate the safety and tolerability of NR in SMDS patients.
What is being tested
- Nicotinamide riboside (NR) (drug)
Sponsor: The University of Texas Health Science Center, Houston · Participants: 15 · Started: Mar 6, 2024
Contact the study team
- Dianna M Milewicz, MD, PhD · Phone: (713) 500-6725
- David R Murdock, MD · Phone: (713) 500-6735
Official record on ClinicalTrials.gov — NCT06280482
Locations in the U.S.
| Texas | The University of Texas Health Science Center at Houston, Houston |
Conditions
From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.