Recruiting
Phase 1

Nicotinamide Riboside

Sponsor:

The University of Texas Health Science Center, Houston

Code:

NCT06280482

Conditions

Smooth Muscle Dysfunction Syndrome (SMDS)

Eligibility Criteria

Sex: All

Age: 1+

Healthy Volunteers: Not accepted

Interventions

Nicotinamide riboside (NR)

Study Details

Brief 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.

Conditions

Smooth Muscle Dysfunction Syndrome (SMDS)

Study ID

NCT06280482

Start date

Mar 6, 2024

Status verified date

Sep, 2025

Completion date

Jul 25, 2027

Anticipated

Primary completion date

Jul 25, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 1+

Healthy Volunteers: Not accepted

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

Enrollment

15 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Treatment

Interventions

Nicotinamide riboside (NR)

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

Primary outcome measure

  • Change in systolic blood [ Time Frame: Baseline , 8 weeks ]
  • Change in cerebral oxygenation and perfusion as assessed by the Head-up tilt table testing (HUTT) with near-infrared spectroscopy (NIRS) monitoring. [ Time Frame: Baseline , 8 weeks ]
  • Change in cognitive function as assessed by the NIH Toolbox assessment [ Time Frame: Baseline , 8 weeks ]
  • Change in autonomic symptoms as assessed by the Composite Autonomic Symptom Score 31 (COMPASS-31) survey [ Time Frame: Baseline , 8 weeks ]
  • Change in Impact of headaches as assessed by the Headache Impact Test (HIT-6) questionnaire [ Time Frame: Baseline, 8 weeks ]
  • Pulmonary function tests (PFTs), including spirometry and lung volume measurements [ Time Frame: Baseline, 8 weeks ]
  • Change in Aortic diameter as assessed by the echocardiography [ Time Frame: Baseline , 8 weeks ]

Central Contacts and Locations

Central contacts

Dianna M Milewicz, MD, PhD

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

David R Murdock, MD

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

Locations

The University of Texas Health Science Center at Houston

Recruiting

Houston, Texas, United States, 77030

Contacts

Dianna Milewicz, MD, PhD

713-500-6725Dianna.M.Milewicz@uth.tmc.edu

David Murdock, MD

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

More Information

Sponsor

The University of Texas Health Science Center, Houston

Last update posted

Sep 12, 2025

Last verified

Sep, 2025

Keywords

  • smooth muscle aortic alpha-actin (ACTA2)
  • arginine 179 residue
  • Nicotinamide Riboside
  • Stroke
  • Cerebrovascular Disease
  • Thoracic Aortic Aneurysms and Dissections

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by The University of Texas Health Science Center, Houston on 2025-09-12.