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Immunologic Effects of Supplemental Monosaccharide and Nucleoside Derivatives in Patients With Inherited Disorders of Glycosylation

NCT02511041 · Status: TERMINATED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 2

Last updated 2017-07-02

No results posted yet for this study

Summary

Background:

\- A congenital disorder of glycosylation (CDG) affects the cells that make up the organs and tissues. In these cells, sugar molecules do not properly attach to other molecules, which are the basic building blocks of cells. Changes in sugars seen in people with CDGs may lead to allergies and can change people s ability to fight infections. Researchers want to see if a sugar supplement called N-acetylglucosamine can help people with CDGs who have detectable changes in their immune systems.

Objective:

\- To see if N-acetylglucosamine can help cells to function in a healthy way in people with CDGs.

Eligibility:

\- People at least 2 years of age who have a CDG and immune system changes.

Design:

* Participants will be screened with a physical exam, medical history, and blood tests.
* One month later, participants will repeat the blood tests from the screening visit. Blood will be drawn on 2 different days in the same week.
* Participants will get N-acetylglucosamine supplements and instructions for how to take them. N-acetylglucosamine is a powder that can be added to food or drink.
* Participants will have a physical exam and blood tests every month during the study.
* After taking N-acetylglucosamine for about 4 months, participants will have more blood tests. They will get more N-acetylglucosamine supplements and a nucleoside supplement. The second supplement may be a powder or tablets that may be crushed and added to food. Participants will take both supplements for 5 months.
* After about 10 months in the study, participants will have 2 more visits in the same week for the same blood tests.

Conditions

  • PGM3

Interventions

DRUG

N-Acetylglucosamine (GlcNAc)

N-acetylglucosamine (GlcNAc), 2-acetamino-2-deoxy- \<=-Dglucose, or 2- acetylamino)-2- deoxy-D-glucose, is a monosaccharide derivative of glucose. It is classified as dietary supplement in the United States. In general, it is a white and slightly sweet powder that melts at 221 degrees C. The solubility of GlcNAc is 25% in water, and 1% aqueous solutions are colorless and clear. GlcNAc is contraindicated for patients receiving warfarin, chemotherapy or diabetes drugs. As GlcNAc may interfere with blood sugar control during and after surgery, it is contraindicated during the two weeks prior to major surgery.

DRUG

Uridine

Uridine 5 -monophosphate (UMP) disodium salt is a water soluble colorless crystalline powder which melts at 202oC.

Sponsors & Collaborators

  • National Institute of Allergy and Infectious Diseases (NIAID)

    lead NIH

Principal Investigators

  • Jonathan J Lyons, M.D. · National Institute of Allergy and Infectious Diseases (NIAID)

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-06-30
Primary Completion
2017-01-24
Completion
2017-01-24

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02511041 on ClinicalTrials.gov