NCT06280482PHASE1Recruiting
Nicotinamide Riboside (NR) to Treat Moyamoya-like Cerebrovascular Disease in Smooth Muscle Dysfunction Syndrome (SMDS)
The University of Texas Health Science Center, Houston
Start Date
3/6/2024
Completion Date
7/25/2027
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
Age Range: 1 years to No maximum
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.
Interventions
DRUG
Nicotinamide riboside (NR)
Conditions
Smooth Muscle Dysfunction Syndrome (SMDS)
Locations
The University of Texas Health Science Center at Houston
Houston, Texas 77030
United States