Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

Could a common supplement help a rare disease? early trial begins

NCT ID NCT07572825

Disease control Sponsor: Eva Morava-Kozicz Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage trial is testing the safety of a dietary supplement called NMN in 8 people with a rare genetic disorder known as DHDDS-CDG, which affects movement and development. Participants will take 250 mg of NMN daily for 6 months, with an optional 12-month follow-up. The main goal is to see if NMN is safe and tolerable, and to gather information that may help doctors decide whether to recommend it for this condition.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
nicotinamide mononucleotide (NMN), a dietary supplement
What this could lead to
If it works, this could point toward a way to manage symptoms of DHDDS-CDG, a rare genetic disorder that affects movement and development.
What could go wrong
This is a very small, early-phase trial with only 8 participants. It primarily tests safety, not effectiveness, so it may not lead to a proven treatment. NMN is a supplement and its benefits for this condition are unproven.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 8 people

The number the study aims to enrol. It can still change while the study runs.

Started

May 2026

Expected to finish

Aug 2027

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

4 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * 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. Exclusion Criteria: * Subject has intellectual disability with IQ\<52 (moderate or lower IQ intellectual disability). * In the site Principal Investigator's opinion, subject has a history of intolerance to NMN or other niacin metabolite supplement that precludes the subject from participation in this study. * Subject has any of the following: * Liver failure * ALT level \>5x ULN * AST level \>5x ULN * eGFR \< 30 OR creatinine \>180 mmol/L * Subject is pregnant. * Use of investigational compounds within the previous 6 months or current enrollment in another trial involving investigational compounds. * Concomitant use of the following medications that could interact with orally administered NMN: * Aspirin * Metformin * Statins or other cholesterol-lowering drugs * In the site Principal Investigator's opinion, subject is not able or willing to comply with the trial requirements.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Congenital disorder of glycosylation are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

congenital disorder of glycosylation DHDDS-CDG

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Congenital disorder of glycosylation DHDDS-CDG DHDDS-congenital disorder of glycosylation

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Icahn School of Medicine at Mount Sinai

    RECRUITING

    New York, New York, 10029, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.