Assessing the Safety and Tolerability of NMN in DHDDS-CDG (NCT07572825) | Clinical Trial Compass
RecruitingPhase 1
Assessing the Safety and Tolerability of NMN in DHDDS-CDG
United States8 participantsStarted 2026-05-08
Plain-language summary
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.
Who can participate
Age range
4 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.