Assessing the Safety and Tolerability of NMN in DHDDS-CDG
Recruiting
Phase 1
Interventional Study
DHDDS-Congenital Disorder of Glycosylation
DHDDS-CDG
Congenital Disorder of Glycosylation
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 4 and older
- Sex
- Any
- Trial phase
- Phase 1
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 8 (estimated)
- Sponsor
- Eva Morava-Kozicz · Other
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About This Trial
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 …
Trial Locations
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Eligibility Criteria
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 …
Contacts