New treatment option for Smooth Muscle Dysfunction Syndrome (SMDS)
Official title Nicotinamide Riboside (NR) to Treat Moyamoya-like Cerebrovascular Disease in Smooth Muscle Dysfunction Syndrome (SMDS)
ClinicalTrials.gov ID: NCT06280482
What this study is testing
What is Nicotinamide riboside (NR)?
Nicotinamide riboside (NR) is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for smooth muscle dysfunction syndrome (smds).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 and older
You may be able to join if
- Individuals diagnosed with SMDS with confirmed ACTA2 mutation disrupting arginine 179
- Parental/guardian permission (informed consent) and, if appropriate, child assent.
You likely 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.
The study team makes the final eligibility decision.
Where it's taking place
- Houston, Texas, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 1 year and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Houston, Texas, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.