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

Description

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.

Conditions

Smooth Muscle Dysfunction Syndrome (SMDS)

Study Overview

Study Details

Study overview

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.

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

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

Condition
Smooth Muscle Dysfunction Syndrome (SMDS)
Intervention / Treatment

-

Contacts and Locations

Houston

The University of Texas Health Science Center at Houston, Houston, Texas, United States, 77030

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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 barrier method (diaphragm or condom), Depo-Provera, or an oral contraceptive, for the duration of the study
  • * 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.
  • * Inability to tolerate PET/CT imaging and echocardiography without sedation.
  • * Concurrent participation in another intervention trial.

Ages Eligible for Study

5 Years to 18 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

The University of Texas Health Science Center, Houston,

David Murdock, MD, PRINCIPAL_INVESTIGATOR, The University of Texas Health Science Center, Houston

Study Record Dates

2025-07-25