Nicotinamide Riboside (NR) to Treat Moyamoya-like Cerebrovascular Disease in Smooth Muscle Dysfunction Syndrome (SMDS)

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

15 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-03-06

Study Completion Date

2027-07-25

Brief Summary

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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.

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Detailed Description

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Conditions

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Smooth Muscle Dysfunction Syndrome (SMDS)

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Treatment

Group Type EXPERIMENTAL

Nicotinamide riboside (NR)

Intervention Type DRUG

Participants will take their weight-based dose of NR once daily by mouth with food for 60 days.

Interventions

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Nicotinamide riboside (NR)

Participants will take their weight-based dose of NR once daily by mouth with food for 60 days.

Intervention Type DRUG

Eligibility Criteria

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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.

Minimum Eligible Age

1 Year

Eligible Sex

ALL

Accepts Healthy Volunteers

No