RecruitingPhase 1NCT06280482

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


Sponsor

The University of Texas Health Science Center, Houston

Enrollment

15 participants

Start Date

Mar 6, 2024

Study Type

INTERVENTIONAL

Conditions

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

Min Age: 1 Year

Inclusion Criteria2

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

Exclusion Criteria6

  • 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.

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Interventions

DRUGNicotinamide riboside (NR)

Participants will take their weight-based dose of NR once daily by mouth with food for 60 days.


Locations(1)

The University of Texas Health Science Center at Houston

Houston, Texas, United States

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NCT06280482