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Taking people in United StatesPhase 1

Tests nicotinamide riboside in people with smooth muscle dysfunction syndrome

Tests nicotinamide riboside in people with a rare disease affecting smooth muscle function.

Key facts

Who runs it
The University of Texas Health Science Center, Houston
Where
Houston, United StatesSee the site
Trial code
Registry title
Nicotinamide Riboside (NR) to Treat Moyamoya-like Cerebrovascular Disease in Smooth Muscle Dysfunction Syndrome (SMDS)
Status
Recruiting
Phase
Phase 1
People taking part
15 people, planned
Started
6 Mar 2024
Expected to end
25 Jul 2027
Type of study
Interventional
Treatments tested
  • Nicotinamide riboside (NR)
Record updated
12 September 2025

Who can take part

AI summary

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for people with smooth muscle dysfunction syndrome (SMDS) who have a confirmed ACTA2 gene change at arginine 179. It tests nicotinamide riboside (NR).

Age
1 year and older
Sex
All sexes
Disease stage
Diagnosed smooth muscle dysfunction syndrome (SMDS).
Earlier treatment
Must not have taken niacin or nicotinamide riboside supplements in the prior eight weeks. Must not have had surgery to replace the ascending or root aorta with a graft.
Biomarker or test result
Confirmed ACTA2 mutation disrupting arginine 179.

Main reasons for not taking part

  • Surgery to replace aneurysmal or dissected ascending and root aortic tissue with a graft
  • Other medical conditions that impair the ability to take part
  • Known allergy or sensitivity to niacin or nicotinamide riboside
  • Niacin or nicotinamide riboside supplement in the prior eight weeks
  • Taking part in another intervention trial at the same time

The registry criteria, word for word

Show all 8 criteriaShow less

Who can take part

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

Who cannot take part

  • Individuals who have undergone surgery to replace aneurysmal or dissected ascending and root aortic tissue with a graft
  • Prior consumption of niacin or nicotinamide riboside supplement within the prior eight weeks
  • Concurrent participation in another intervention trial
  • Additional medical conditions that impair the patient's ability to participate in the study
  • Known allergy or sensitivity to niacin or nicotinamide riboside
  • Failure to provide informed consent

Where in United States

  • The University of Texas Health Science Center at HoustonHouston, United StatesContact: Dianna Milewicz, MD, PhDTaking people

About the trial

What the trial wants to find out

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.

The group in the trial

  • TreatmentTreatment being tested

    Treatment: Nicotinamide riboside (NR)

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov.
Official title
Nicotinamide Riboside (NR) to Treat Moyamoya-like Cerebrovascular Disease in Smooth Muscle Dysfunction Syndrome (SMDS)
Plain-language title
Tests nicotinamide riboside in people with smooth muscle dysfunction syndromeGenerated from the registry record. The registry title is the official one.
AI summary
From the trial's eligibility criteria. Read by machine on 12 November 2026.
Record updated
12 September 2025