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RecruitingPhase 1NCT07572825

Assessing the Safety and Tolerability of NMN in DHDDS-CDG

Small Cohort, Off Label Treatment Assessing the Safety and Tolerability of NMN in DHDDS-Congenital Disorder of Glycosylation (DHDDS-CDG)

Sponsor

Eva Morava-Kozicz

Enrollment

8 participants

Start Date

May 8, 2026

Study Type

INTERVENTIONAL

Conditions

DHDDS-Congenital Disorder of GlycosylationDHDDS-CDGCongenital Disorder of Glycosylation

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.

Eligibility

Min Age: 4 Years

Inclusion Criteria3

  • 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 Criteria14

  • 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.

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Interventions

DRUGNicotinamide Mononucleotide (NMN) Nucleosidase

Name: nicotinamide mononucleotide (NMN) Form: measured powder Dose: 250 mg/day Frequency: Daily Route of administration: Oral

Locations(1)

Icahn School of Medicine at Mount Sinai

New York, New York, United States

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NCT07572825