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

Purpose

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.

Condition

  • Smooth Muscle Dysfunction Syndrome (SMDS)

Eligibility

Eligible Ages
Over 1 Year
Eligible Sex
All
Accepts Healthy Volunteers
No

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.

Study Design

Phase
Phase 1
Study Type
Interventional
Allocation
N/A
Intervention Model
Single Group Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Treatment
  • Drug: Nicotinamide riboside (NR)
    Participants will take their weight-based dose of NR once daily by mouth with food for 60 days.

Recruiting Locations

The University of Texas Health Science Center at Houston
Houston 4699066, Texas 4736286 77030
Contact:
Dianna Milewicz, MD, PhD
713-500-6725
Dianna.M.Milewicz@uth.tmc.edu

More Details

Status
Recruiting
Sponsor
The University of Texas Health Science Center, Houston

Study Contact

Dianna M Milewicz, MD, PhD
(713) 500-6725
Dianna.M.Milewicz@uth.tmc.edu