RECRUITING

Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1)

Conditions

Gaucher Disease Type 1 Gaucher Disease GD1 N-acetylcysteine glutathione GSH

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

The purpose of this study is to measure levels of blood and brain chemicals related to oxidative stress and inflammation in healthy volunteers and individuals with Type 1 Gaucher disease (GD1) to see if these levels are altered by GD1.

Official Title

Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1): Potential Use of Antioxidant/Anti-inflammatory Medications

Quick Facts

Study Start:2015-09

Study Completion:2025-07-31

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT02583672

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.

Ages Eligible for Study:18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:Yes

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
1. All participants must be 18 years or older. 2. All participants must understand and cooperate with requirements of the study in the opinion of the investigators and must be able to provide written informed consent. 3. Individuals with GD1 who are medically stable for participation in the study in the opinion of the investigator. 4. GD1 patients must be on a stable, specific ERT and/or SRT therapy at a specific dose (e.g. on a units/kg basis) for at least 2 years. 5. GD1 patients who have had a change in therapy, i.e. a change in dose or switch from one drug to another, can be enrolled after at least 6 months have elapsed since the change and is considered stable in the opinion of the clinician providing care to the patient. 6. Healthy subjects who will be frequency-matched for age. 7. All participants must not have taken antioxidants coenzyme Q-10, vitamin C, or vitamin E for 3 weeks prior to the study and during the course of the study.	1. Medically unstable conditions in any group as determined by the investigators. 2. Concurrent disease; medical condition; or an extenuating circumstance that, in the opinion of the investigator, might compromise subject safety, study compliance, completion of the study, or the integrity of the data collected for the study. 3. Women who are pregnant or lactating or of child-bearing age who are not using acceptable forms of contraception. 4. History of asthma that is presently being treated. 5. Patients enrolled in another interventional study. 6. Allergy to N-acetylcysteine. 7. Patients who cannot or are unwilling to have blood drawn. 8. Inability to undergo MRI scanning, including but not limited to: unable to remain still in an MRI scanner for more than 30 minutes, claustrophobia, presence of paramagnetic substances or pacemakers in body, weight over 300 lbs. 9. Unable to adhere to study protocol for whatever reason.

Inclusion Criteria

Exclusion Criteria

1. All participants must be 18 years or older.
2. All participants must understand and cooperate with requirements of the study in the opinion of the investigators and must be able to provide written informed consent.
3. Individuals with GD1 who are medically stable for participation in the study in the opinion of the investigator.
4. GD1 patients must be on a stable, specific ERT and/or SRT therapy at a specific dose (e.g. on a units/kg basis) for at least 2 years.
5. GD1 patients who have had a change in therapy, i.e. a change in dose or switch from one drug to another, can be enrolled after at least 6 months have elapsed since the change and is considered stable in the opinion of the clinician providing care to the patient.
6. Healthy subjects who will be frequency-matched for age.
7. All participants must not have taken antioxidants coenzyme Q-10, vitamin C, or vitamin E for 3 weeks prior to the study and during the course of the study.

1. Medically unstable conditions in any group as determined by the investigators.
2. Concurrent disease; medical condition; or an extenuating circumstance that, in the opinion of the investigator, might compromise subject safety, study compliance, completion of the study, or the integrity of the data collected for the study.
3. Women who are pregnant or lactating or of child-bearing age who are not using acceptable forms of contraception.
4. History of asthma that is presently being treated.
5. Patients enrolled in another interventional study.
6. Allergy to N-acetylcysteine.
7. Patients who cannot or are unwilling to have blood drawn.
8. Inability to undergo MRI scanning, including but not limited to: unable to remain still in an MRI scanner for more than 30 minutes, claustrophobia, presence of paramagnetic substances or pacemakers in body, weight over 300 lbs.
9. Unable to adhere to study protocol for whatever reason.

Contacts and Locations

Study Contact

Reena V. Kartha, Ph.D.

CONTACT

612-626-2436

rvkartha@umn.edu

Principal Investigator

Reena V. Kartha, PharmD

PRINCIPAL_INVESTIGATOR

University of Minnesota

Study Locations (Sites)

University of Minnesota

Minneapolis, Minnesota, 55455

United States

New York University

New York, New York, 10016

United States

Collaborators and Investigators

Sponsor: University of Minnesota

Reena V. Kartha, PharmD, PRINCIPAL_INVESTIGATOR, University of Minnesota

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 Registration Dates

Study Start Date2015-09

Study Completion Date2025-07-31

Study Record Updates

Study Start Date2015-09

Study Completion Date2025-07-31

Terms related to this study

Keywords Provided by Researchers

Gaucher Disease
GD1
N-acetylcysteine
glutathione
GSH

Additional Relevant MeSH Terms

Gaucher Disease Type 1