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

The University of Texas System (UT)

Status and phase: Enrolling; Phase 1

Conditions: Smooth Muscle Dysfunction Syndrome (SMDS)

Treatments: Drug: Nicotinamide riboside (NR)

Study type: Interventional

Funder types: Other

Identifiers

NCT06280482

HSC-MS-23-0332

Details and patient eligibility

About

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.

Enrollment: 15 estimated patients

Sex: All

Ages: 1 to 18 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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15 participants in 1 patient group

Treatment

Experimental group

Treatment:

Drug: Nicotinamide riboside (NR)