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

Phase 1

Recruiting

• Conditions: Smooth Muscle Dysfunction Syndrome (SMDS)
• Interventions: Drug: Nicotinamide riboside (NR)

• Registration Number: NCT06280482
• Lead Sponsor: The University of Texas Health Science Center, Houston

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-02-19
• Study First Submitted QC: 2024-02-19
• Study First Posted: 2024-02-28
• Study Start: 2024-03-06
• Status Verified: 2024-04
• Last Update Submitted: 2024-04-03
• Last Update Posted: 2024-04-05
• Primary Completion: 2025-07-25
• Study Completion: 2025-07-25

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 5

Inclusion Criteria

• bodyweight ≥ 12 kg
• native (not surgically replaced) ascending aorta
• diagnosed with SMDS with confirmed ACTA2 mutation disrupting arginine 179
• females with negative urine pregnancy test
• must agree to use acceptable method of contraception, including abstinence, a barrier method (diaphragm or condom), Depo-Provera, or an oral contraceptive, for the duration of the study

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.
• Inability to tolerate PET/CT imaging and echocardiography without sedation.
• Concurrent participation in another intervention trial.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Treatment	Nicotinamide riboside (NR)	-

Primary Outcome Measures

Name	Time	Method
Change in Fluoro-18-Deoxyglucose (18F-FDG ) uptake in aortic wall as measured by positron emission tomography (PET)/computerized tomography (CT) imaging	Baseline , 8 weeks

Secondary Outcome Measures

Name	Time	Method
Change in Levels of Nicotinamide adenine dinucleotide (NAD+) in whole blood as measured by high-performance liquid chromatography (HPLC)	Baseline , 8 weeks
Tolerability as assessed by the number of patients who complete study	end of study( 8 weeks after baseline)
Change in Levels of NR in whole blood as measured by high-performance liquid chromatography (HPLC)	Baseline , 8 weeks
Change in aortic measurements as assessed by echocardiography	Baseline , 8 weeks
Safety as assessed by number of participants that show drug toxicity as shown in bloodwork	end of study (8 weeks after baseline)

Trial Locations

Locations (1)

The University of Texas Health Science Center at Houston; 🇺🇸 Houston, Texas, United States