Recruiting
Phase 1

Nicotinamide Mononucleotide

Sponsor:

Eva Morava-Kozicz

Code:

NCT07572825

Conditions

DHDDS-Congenital Disorder of Glycosylation

DHDDS-CDG

Congenital Disorder of Glycosylation

Eligibility Criteria

Sex: All

Age: 4+

Healthy Volunteers: Not accepted

Interventions

Nicotinamide Mononucleotide (NMN) Nucleosidase

Study Details

Brief 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.

Conditions

DHDDS-Congenital Disorder of Glycosylation

DHDDS-CDG

Congenital Disorder of Glycosylation

Study ID

NCT07572825

Start date

May 8, 2026

Status verified date

Jun, 2026

Completion date

Aug 1, 2027

Anticipated

Primary completion date

Aug 1, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 4+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

8 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: nicotinamide mononucleotide (NMN)

Participants will take 250mg NMN daily.

Interventions

Nicotinamide Mononucleotide (NMN) Nucleosidase

Name: nicotinamide mononucleotide (NMN)

Form: measured powder

Dose: 250 mg/day

Frequency: Daily

Route of administration: Oral

Primary outcome measure

  • Incidence of adverse events (AEs) [ Time Frame: up to 12 months ]

Central Contacts and Locations

Central contacts

Georgia MacDonald, MS, CGC

646-946-6923Georgia.macdonald@mssm.edu

Locations

Icahn School of Medicine at Mount Sinai

Recruiting

New York, New York, United States, 10029

Contacts

Georgia MacDonald

646-946-6923Georgia.macdonald@mssm.edu

Principal Investigator:

Eva Morava

More Information

Sponsor

Eva Morava-Kozicz

Last update posted

Jun 22, 2026

Last verified

Jun, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Eva Morava-Kozicz on 2026-06-22.