Assessing the Safety and Tolerability of NMN in DHDDS-CDG

What Was Tested

In this phase 1 trial, researchers set out to test NAD+.

The primary objective of this study is to evaluate the safety and tolerability of the dietary supplement, nicotinamide mononucleotide (NMN), in individuals with dehydrodolichol diphosphate synthase congenital disorder of glycosylation (DHDDS-CDG).

This will to contribute to knowledge that will allow healthcare providers to make informed decisions about recommending this dietary supplement in this population.

It had 1 arm (groups): nicotinamide mononucleotide (NMN) (experimental).

The registry classes the interventions as drug.

More on the Plan

This is a small cohort, off label treatment study assessing the safety, and tolerability of the over-the-counter supplement, nicotinamide mononucleotide (NMN) in individuals with heterozygous DHDDS-CDG.

There is no treatment currently available for this progressive disease and there is evidence that this supplement may be a viable supportive therapy.

This study will assess the safety and tolerability of this supplement, as well as examine NMN's effect on clinical manifestations of DHDDS-CDG, including gait abnormalities and hand tremor.

Study assessments will involve physician assessments, lab tests, physical exams, vital signs, height/weight measurements, participant goal-setting, and tests to assess gait performance and hand tremor.

Who Took Part

8 planned participants were the target.

It was open to people of any sex aged from 4 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Subject is ≥ 4 years old. Subject has biologically and genetically proven heterozygous DHDDS-CDG. Subject/legally authorized representative (LAR) is able to understand and provide written informed consent, and assent (as applicable) to participate in this study.

The full record lists 3 inclusion and 14 exclusion criteria; among the exclusions: subject has intellectual disability with iq\<52 (moderate or lower iq intellectual disability); subject has any of the following:; liver failure.

How the Study Was Run

The set-up was single-group and open-label.

Nothing was hidden in this open-label NAD+ study: the participants and staff both knew the treatment.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2026-05-08 with completion listed for 2027-08-01.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Incidence of adverse events (AEs) (time frame: up to 12 months).

It also tracked 3 secondary outcomes, for example International Cooperative Ataxia Rating Scale (ICARS) Score, Composite gait stability score (Cord walking test performance) and Composite tremor severity score (Archimedes spiral test performance).

What This Means in Plain Language

Put simply, this is a phase 1 trial of NAD+ in 8 people.

Its size is small, so on its own it can only give an early look.

It is still recruiting; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the NAD+ compound page, all human trials.