Nicotinamide Riboside (NR) to Treat Moyamoya-like Cerebrovascular Disease in Smooth Muscle Dysfunction Syndrome (SMDS)
Recruiting
Phase 1
Interventional Study
Smooth Muscle Dysfunction Syndrome (SMDS)
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 1 and older
- Sex
- Any
- Trial phase
- Phase 1
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 15 (estimated)
- Sponsor
- The University of Texas Health Science Center, Houston · Other
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About This Trial
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.
Trial Locations
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Eligibility Criteria
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 ascendin…
Contacts