Recruiting NCT06280482
Nicotinamide Riboside (NR) to Treat Moyamoya-like Cerebrovascular Disease in Smooth Muscle Dysfunction Syndrome (SMDS)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Nicotinamide riboside (NR).
- Who it may be relevant to
- Registry conditions: Smooth Muscle Dysfunction Syndrome (SMDS). Basic parameters: from 1 year · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Overview
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.
Interventions
- Drug Nicotinamide riboside (NR)
Participants will take their weight-based dose of NR once daily by mouth with food for 60 days.
Primary outcome measures
- 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]
Secondary outcome measures (5)
- Change in Levels of Nicotinamide adenine dinucleotide (NAD+) in whole blood as measured by high-performance liquid chromatography (HPLC) [Time frame: Baseline , 8 weeks]
- Change in Levels of NR in whole blood as measured by high-performance liquid chromatography (HPLC) [Time frame: Baseline , 8 weeks]
- Safety as assessed by number of participants that show drug toxicity as shown in bloodwork [Time frame: Baseline]
- Safety as assessed by number of participants that show drug toxicity as shown in bloodwork [Time frame: end of study (8 weeks after baseline)]
- Tolerability as assessed by the number of patients who complete study [Time frame: end of study( 8 weeks after baseline)]
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 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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 1 center
- The University of Texas Health Science Center at Houston — Houston
Identifiers
NCT: NCT06280482 · HSC-MS-23-0332