Nicotinamide Riboside for Diabetic Neuropathy

NCT03685253 | Terminated Phase 1 DMC FDA Drug

• 1 other identifier: HP-00080331
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

At the current time there is no effective disease modifying therapy for diabetic neuropathy (DN). The proposed study design employs a quantifiable early measure of DN, intraepidermal nerve fiber density (IENFD), allowing for accurate assessment of actual nerve fiber density. Preclinical data supports the use of Niagen® (3-(Aminocarbonyl)-1-β-D-ribofuranosyl-pyridinium chloride - NR) as a potential therapy for diabetic neuropathy. Phase I data indicates safety in humans. This study seeks to investigate the use of Niagen® (NR) as a potential treatment for diabetic neuropathy in subjects with type 2 diabetes mellitus or impaired glucose tolerance over a 6 month period. The endpoint measures in addition to the IENFD with determine changes in clinical and electrophysiological outcomes, quality of life and biochemical measures.

Conditions

Diabetic Neuropathy Peripheral

Keywords

diabetes; neuropathy; treatment; drug

Outcome Measures

Primary Outcomes (1)

• Thigh Intraepidermal Nerve Fiber Density

  Change in the proximal thigh Intra-Epidermal Nerve Fiber Density as assessed by skin biopsy. Smaller values indicate worse neuropathy. Normative ranges depend on age and gender.

  6 months.

Secondary Outcomes (1)

• Distal lower limb intraepidermal nerve fiber density

  6 months.

Other Outcomes (8)

• Modified Toronto Clinical Neuropathy Scale

  6 months.

• Total Neuropathy Score (Clinical)

  6 months.

• The Neuropathy Total Symptom Score (NTSS-6)

  6 months.

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Participants randomized to placebo will take 2 capsules by mouth twice a day for 6 months. The placebo capsules are matched to the NR capsules.

Drug: Placebo

Niagen®

EXPERIMENTAL

Participants randomized to Niagen® (3-(Aminocarbonyl)-1-β-D-ribofuranosyl-pyridinium chloride - NR) will take 250 mg capsules. Participants will take 2 capsules by mouth twice a day (1000 mg) for 6 months

Drug: Niagen

Interventions

Niagen DRUG

Niagen® (3-(Aminocarbonyl)-1-β-D-ribofuranosyl-pyridinium chloride - NR) will be the experimental treatment, at a dose of 1000 mg/day taken as two 250 mg capsules twice daily for 6 months.

Also known as: Nicotinamide Riboside, 3-(Aminocarbonyl)-1-β-D-ribofuranosyl-pyridinium chloride

Niagen®

Placebo DRUG

Placebo capsules matched to the experimental drug and taken orally as 2 capsules twice daily for 6 months.

Placebo

Eligibility Criteria

• Age: 30 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Impaired glucose tolerance or controlled type 2 diabetes mellitus at the time of screening or within three months of screening\*.
• The hemoglobin A1c may be normal, but should be less than 9%.
• If diabetic participants are on medication, they should be stable on medication for at least 3 months prior to entering the study. Addition or change in antidiabetic medication (if on medication) after enrollment does not affect participation or group assignment.
• Impaired glucose regulation is the most likely cause of the neuropathy.
• Mild diabetic polyneuropathy as defined by the Toronto Diabetic Neuropathy Expert Group consensus criteria.
• Age 30 (to exclude patients with type 1 diabetes) to 80 years inclusive.
• Medically stable at the time of enrollment.
• Willing to accept randomization assignment and compliance with the study procedures.

You may not qualify if:

• Women of childbearing potential must be using an acceptable method of contraception to prevent pregnancy when they are enrolled in the study.
• Patient must agree to take an alternative medication to Warfarin or Factor X inhibitors when undergoing a skin biopsy.
• Neuropathy due to factors other than type 2 diabetes mellitus based on careful clinical and laboratory evaluation by the study physicians.
• Abnormal liver function tests, including alanine transaminase, aspartate transaminase, alkaline phosphatase, and bilirubin.
• Current severe medical conditions that are active on the day of screening.

Sponsors & Collaborators

• University of Maryland, Baltimore: lead
• US Department of Veterans Affairs: collaborator

Study Sites (1)

University of Maryland

Baltimore, Maryland, 21201, United States

Location

MeSH Terms

Conditions

Diabetes Mellitus

Interventions

nicotinamide-beta-riboside

Condition Hierarchy (Ancestors)

Glucose Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Endocrine System Diseases

Study Officials

• James W Russell, MD

  University of Maryland School of Medicine & Department of Veterans' Affairs

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: September 21, 2018
• First Posted: September 26, 2018
• Study Start: January 24, 2019
• Primary Completion: May 1, 2022
• Study Completion: May 1, 2022
• Last Updated: January 6, 2026

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)