What this study is
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.
What's being tested
- Nicotinamide riboside (NR)
Intervention · DRUG
Participants will take their weight-based dose of NR once daily by mouth with food for 60 days.
Study details
- Sponsor
- The University of Texas Health Science Center, Houston
- Conditions
- Smooth Muscle Dysfunction Syndrome (SMDS)
- Enrolling
- 15 people
- Starts
- 2024-03-06
- Est. completion
- 2027-07-25
- Design
- Open-label, single group, Phase 1
- Record
- View on ClinicalTrials.gov ↗
Who can join
Age 1 Year and overSex ALLHealthy volunteers Not acceptedYou may qualify if
- Individuals diagnosed with SMDS with confirmed ACTA2 mutation disrupting arginine 179
- Parental/guardian permission (informed consent) and, if appropriate, child assent.
You can't join if
- 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.
Sign in and we'll check these against your records.Full criteria on ClinicalTrials.gov ↗
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Study locations (1)
1 openThe University of Texas Health Science Center at Houston
Houston, Texas, United States
- Dianna Milewicz, MD, PhD · Contact713-500-6725Dianna.M.Milewicz@uth.tmc.edu
- David Murdock, MD · Contact(713) 500-6735David.R.Murdock@uth.tmc.edu
Recruiting
Study team
Sponsor
The University of Texas Health Science Center, Houston
Study ID HSC-MS-23-0332
Principal investigator
Dianna Milewicz, MD, PhD
The University of Texas Health Science Center, Houston
Frequently asked questions
The sponsor hasn't published answers to common questions for this trial.