A Study of GlcNAc on Tear Production in NGLY1-CDDG
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ClinicalTrials.gov Identifier: NCT05402345 |
Recruitment Status :
Not yet recruiting
First Posted : June 2, 2022
Last Update Posted : April 1, 2024
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
NGLY1 Deficiency | Drug: GlcNAc-GlcN Other: Placebo | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 18 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Triple (Participant, Care Provider, Investigator) |
Primary Purpose: | Treatment |
Official Title: | A Phase II Randomized, Multicenter, Double-Blind, Placebo-Controlled Study Evaluating Effect Of GlcNAc On Tear Production In Individuals With NGLY1-CDDG |
Estimated Study Start Date : | May 2024 |
Estimated Primary Completion Date : | June 2025 |
Estimated Study Completion Date : | June 2025 |
Arm | Intervention/treatment |
---|---|
Experimental: GlcNAc
GlcNAc powder
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Drug: GlcNAc-GlcN
GlcNAc powder - weight-dependent dose |
Placebo Comparator: Placebo
Placebo glucose powder
|
Other: Placebo
Placebo glucose powder |
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
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Ages Eligible for Study: | 1 Year to 60 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05402345
Contact: Mary Freeman, MS, CGC | 212-659-1434 | mary.freeman@mssm.edu |
United States, Pennsylvania | |
Children's Hospital of Philadelphia | |
Philadelphia, Pennsylvania, United States, 19104 | |
Principal Investigator: Andrew Edmondson, MD, PhD | |
United States, Washington | |
Seattle Children's Hospital | |
Seattle, Washington, United States, 98105 | |
Principal Investigator: Christina Lam, MD, FACMG |
Principal Investigator: | Eva Morava-Kozicz, MD, PhD | Icahn School of Medicine at Mount Sinai |
Responsible Party: | Eva Morava-Kozicz, Principal Investigator, Icahn School of Medicine at Mount Sinai |
ClinicalTrials.gov Identifier: | NCT05402345 |
Other Study ID Numbers: |
23-020868 8404 ( Other Identifier: FCDGC ) |
First Posted: | June 2, 2022 Key Record Dates |
Last Update Posted: | April 1, 2024 |
Last Verified: | March 2024 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |