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

Phase

Condition

Stroke

Cerebral Ischemia

Neurologic Disorders

Treatment

Nicotinamide riboside (NR)

Clinical Study ID

NCT06280482

HSC-MS-23-0332

Ages > 1
All Genders

Study Summary

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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

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

Exclusion

Exclusion Criteria:

Individuals who have undergone surgery to replace aneurysmal or dissected ascendingand root aortic tissue with a graft.
Additional medical conditions that impair the patient's ability to participate inthe study.
Known allergy or sensitivity to niacin or nicotinamide riboside.
Prior consumption of niacin or nicotinamide riboside supplement within the prioreight weeks.
Failure to provide informed consent.
Concurrent participation in another intervention trial.

Study Design