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

Last updated: April 3, 2024
Sponsor: The University of Texas Health Science Center, Houston
Overall Status: Active - Recruiting

Phase

1

Condition

Neurologic Disorders

Treatment

Nicotinamide riboside (NR)

Clinical Study ID

NCT06280482
HSC-MS-23-0332
  • Ages 5-18
  • 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:

  • bodyweight ≥ 12 kg
  • native (not surgically replaced) ascending aorta
  • diagnosed with SMDS with confirmed ACTA2 mutation disrupting arginine 179
  • females with negative urine pregnancy test
  • must agree to use acceptable method of contraception, including abstinence, a barriermethod (diaphragm or condom), Depo-Provera, or an oral contraceptive, for the durationof the study

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 in thestudy.
  • Known allergy or sensitivity to niacin or nicotinamide riboside.
  • Prior consumption of niacin or nicotinamide riboside supplement within the prior eightweeks.
  • Failure to provide informed consent.
  • Inability to tolerate PET/CT imaging and echocardiography without sedation.
  • Concurrent participation in another intervention trial.

Study Design

Total Participants: 5
Treatment Group(s): 1
Primary Treatment: Nicotinamide riboside (NR)
Phase: 1
Study Start date:
March 06, 2024
Estimated Completion Date:
July 25, 2025

Connect with a study center

  • The University of Texas Health Science Center at Houston

    Houston, Texas 77030
    United States

    Active - Recruiting

Not the study for you?

Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.