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Monosaccharide

GlcNAc for NGLY1 Deficiency

Phase 2

Waitlist Available

Led By Eva Morava-Kozicz, MD, PhD

Research Sponsored by Eva Morava-Kozicz

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Previously molecularly confirmed NGLY1-CDDG

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 6 weeks, 12 weeks

Awards & highlights

Study Summary

This trial studies if a dietary supplement can help improve tear production in people with a rare disorder, NGLY1-CDDG, which can cause eye damage.

Who is the study for?

This trial is for individuals with NGLY1-CDDG, a condition that impairs tear production and can damage the eyes. Participants must have a confirmed diagnosis, be willing to consent (or have a guardian who can consent), and not be planning eye surgery soon or participating in other drug trials.Check my eligibility

What is being tested?

The study tests whether GlcNAc, a dietary supplement, increases tear production in those with NGLY1-CDDG. Patients will either receive GlcNAc-GlcN or a placebo without knowing which one they're getting to compare the effects fairly.See study design

What are the potential side effects?

Potential side effects are not specified but may include reactions related to hypersensitivity to the components of GlcNAc-GlcN or placebo. Those with shellfish allergies should avoid participation due to potential risks.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

My condition is confirmed to be NGLY1-CDDG.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 6 weeks, 12 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~6 weeks, 12 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 weeks, 12 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Difference in tear production from baseline in placebo vs GlcNAc group

Secondary outcome measures

Difference in tear production from baseline

Frequency of eye infections needing treatments

Frequency of eye redness needing treatments

+3 more

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: GlcNAcExperimental Treatment1 Intervention

GlcNAc powder

Group II: PlaceboPlacebo Group1 Intervention

Placebo glucose powder

0 / 1Eligibility criteria met

Find a Location

Who is running the clinical trial?

Children's Hospital of PhiladelphiaOTHER

708 Previous Clinical Trials

8,581,285 Total Patients Enrolled

Eva Morava-KoziczLead Sponsor

3 Previous Clinical Trials

69 Total Patients Enrolled

Seattle Children's HospitalOTHER

301 Previous Clinical Trials

5,216,959 Total Patients Enrolled

Media Library

GlcNAc-GlcN (Monosaccharide) Clinical Trial Eligibility Overview. Trial Name: NCT05402345 — Phase 2

NGLY1 Deficiency Research Study Groups: Placebo, GlcNAc

NGLY1 Deficiency Clinical Trial 2023: GlcNAc-GlcN Highlights & Side Effects. Trial Name: NCT05402345 — Phase 2

GlcNAc-GlcN (Monosaccharide) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05402345 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Could I potentially partake in this medical research?

"This research programme is looking to accept 18 people with ngly1 deficiency, aged between 2 and 18 years old."

Answered by AI

Has the administration given its sanction to GlcNAc?

"Due to its Phase 2 status, meaning there is evidence of safety but no proof yet of efficacy, the team has assessed GlcNAc's security level at a score of two."

Answered by AI

Does the qualifying criteria for this research include individuals below sixty years of age?

"The study requires its participants to be between the ages of 2 and 18. Additionally, there is a separate trial for those under two years old and another one for those over 65."

Answered by AI

Is recruitment for this clinical experiment still underway?

"Data observed on clinicaltrials.gov indicates that the recruiting phase for this medical trial has ended, as it was first posted on November 1st 2023 and last updated August 25th 2023. However, there are currently another one of these trials enrolling patients at present."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Study of GlcNAc on Tear Production in NGLY1-CDDG

Eva Morava-Kozicz 2024. "A Study of GlcNAc on Tear Production in NGLY1-CDDG". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05402345.