- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05402345
A Study of GlcNAc on Tear Production in NGLY1-CDDG
March 28, 2024 updated by: Eva Morava-Kozicz
A Phase II Randomized, Multicenter, Double-Blind, Placebo-Controlled Study Evaluating Effect Of GlcNAc On Tear Production In Individuals With NGLY1-CDDG
In patients with NGLY1-CDDG, the disorder can lead to eye damage due to not being able to produce enough tears.
This study is being done to see if the dietary supplement, GlcNAc, improves tear production in patients with NGLY1-CDDG.
Study Overview
Status
Not yet recruiting
Conditions
Intervention / Treatment
Detailed Description
This study is a multicenter randomized, double-blind, placebo-controlled trial of GlcNAc supplementation for improvement of tear production in NGLY1-CDDG.
Clinical history and screening data will be reviewed to determine subject eligibility.
If a subject is already on GlcNAc, a washout period of 1 month will be required prior to consent and randomization.
Interested subjects who have a molecularly confirmed diagnosis of NGLY1-CDDG will be consented.
Baseline data will be collected prior to randomization at treatment initiation.
Subjects will then be randomized to placebo or GlcNAc.
They will be administered weight-dependent doses of GlcNAc or an equivalent volume of placebo enterally for 6 weeks, followed by open label weight-dependent doses of GlcNAc for 6 weeks.
A visit for evaluation and collection of lab samples will be conducted at 6 and 12 weeks.
Study Type
Interventional
Enrollment (Estimated)
18
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Mary Freeman, MS, CGC
- Phone Number: 212-659-1434
- Email: mary.freeman@mssm.edu
Study Locations
-
-
Pennsylvania
-
Philadelphia, Pennsylvania, United States, 19104
- Children's Hospital of Philadelphia
-
Principal Investigator:
- Andrew Edmondson, MD, PhD
-
-
Washington
-
Seattle, Washington, United States, 98105
- Seattle Children's Hospital
-
Principal Investigator:
- Christina Lam, MD, FACMG
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
2 years to 18 years (Child, Adult)
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
- Previously molecularly confirmed NGLY1-CDDG
- Parent or legal guardian available to provide consent on behalf of minor subjects or adult subjects who are unable to give informed consent due to developmental disabilities. Willingness of subject or legal guardian to provide consent.
Exclusion Criteria
- Hypersensitivity to any of the components of the placebo
- History of treatment with GlcNAc within 28 days of Visit 1
- Participation in another therapeutic trial - the subject will not be permitted to participate in any other drug trial during the blinded phase and during the 28 days prior to Visit 1
- Shellfish allergy
- Planned eye surgery within 3 months of enrollment
- • Females that are pregnant, nursing or less than 6 months postpartum or attempting to conceive
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Triple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: GlcNAc
GlcNAc powder
|
GlcNAc powder - weight-dependent dose
|
Placebo Comparator: Placebo
Placebo glucose powder
|
Placebo glucose powder
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Difference in tear production from baseline in placebo vs GlcNAc group
Time Frame: 6 weeks
|
The primary endpoint of the study is the difference in tear production from baseline in individuals with NGLY1-CDDG between the placebo and the GlcNAc group after 6 weeks of blinded therapy, as measured by Schirmer II test which is a tool that helps assess the amount of tears in the eyes.
|
6 weeks
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Frequency of eye infections needing treatments
Time Frame: 6 weeks, 12 weeks
|
Patient/family reported number of eye infections needing treatment in last month after 6 weeks of blinded therapy and at 12 weeks after 6 weeks of open-label administration of GlcNAc.
|
6 weeks, 12 weeks
|
Frequency of eye redness needing treatments
Time Frame: 6 weeks, 12 weeks
|
Patient/family reported frequency of eye redness after 6 weeks of blinded therapy and at 12 weeks after 6 weeks of open-label administration of GlcNAc.
|
6 weeks, 12 weeks
|
Frequency of eye tearing/watering
Time Frame: 6 weeks, 12 weeks
|
Patient/family reported frequency of eye tearing/watering after 6 weeks of blinded therapy and at 12 weeks after 6 weeks of open-label administration of GlcNAc.
|
6 weeks, 12 weeks
|
Frequency of light sensitivity
Time Frame: 6 weeks, 12 weeks
|
Patient/family reported frequency of light sensitivity after 6 weeks of blinded therapy and at 12 weeks after 6 weeks of open-label administration of GlcNAc.
|
6 weeks, 12 weeks
|
Frequency of wind sensitivity
Time Frame: 6 weeks, 12 weeks
|
Patient/family reported frequency of wind sensitivity after 6 weeks of blinded therapy and at 12 weeks after 6 weeks of open-label administration of GlcNAc.
|
6 weeks, 12 weeks
|
Difference in tear production from baseline
Time Frame: 12 weeks
|
Difference in tear production, as measured by Schirmer II test, at 12 weeks after 6 weeks of open-label administration of GlcNAc.
|
12 weeks
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Eva Morava-Kozicz, MD, PhD, Icahn School of Medicine at Mount Sinai
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Helpful Links
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Estimated)
May 1, 2024
Primary Completion (Estimated)
June 1, 2025
Study Completion (Estimated)
June 1, 2025
Study Registration Dates
First Submitted
May 28, 2022
First Submitted That Met QC Criteria
May 28, 2022
First Posted (Actual)
June 2, 2022
Study Record Updates
Last Update Posted (Actual)
April 1, 2024
Last Update Submitted That Met QC Criteria
March 28, 2024
Last Verified
March 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- 23-020868
- 8404 (Other Identifier: FCDGC)
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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