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
Between 5 Years and 18 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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 barrier method (diaphragm or condom), Depo-Provera, or an oral contraceptive, for the duration of the study

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. - Inability to tolerate PET/CT imaging and echocardiography without sedation. - 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, Texas 77030
Contact:
David Murdock, MD
713-500-6735
David.R.Murdock@uth.tmc.edu

More Details

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

Study Contact

David R Murdock, MD
(713) 500-6735
David.R.Murdock@uth.tmc.edu