NCT07572825 · Eva Morava-Kozicz
Assessing the Safety and Tolerability of NMN in DHDDS-CDG
What this study is about
The primary objective of this study is to evaluate the safety and how well patients handle the treatment of the dietary supplement, nicotinamide mononucleotide (NMN), in individuals with dehydrodolichol diphosphate synthase congenital disorder of glycosylation (DHDDS-CDG).
View original scientific description
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.
Interventions
DRUG
Nicotinamide Mononucleotide (NMN) Nucleosidase
Name: nicotinamide mononucleotide (NMN) Form: measured powder Dose: 250 mg/day Frequency: Daily Route of administration: Oral
Primary outcome measures
Incidence of adverse events (AEs)
Time frame: up to 12 months
Incidence of AEs will be collected throughout the treatment period and optional long-term safety follow up period.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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:
- 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.
Where
- New York, New York
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 22, 2026 · Source of record for eligibility and locations