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Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease

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ClinicalTrials.gov Identifier: NCT02437396
Recruitment Status : Recruiting
First Posted : May 7, 2015
Last Update Posted : July 13, 2022
Sponsor:
Information provided by (Responsible Party):
University of Minnesota

Study Description
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Brief Summary:
The objective of this study is to evaluate oxidative stress and/or inflammation in patients with Gaucher disease type I using a series of biomarkers and correlate with measurements of currently used diagnostic biomarkers.

Condition or disease
Gaucher Disease Type I Oxidative Stress Inflammation

Detailed Description:
We will determine oxidative stress and/or inflammation related biomarkers in whole blood and/or plasma in adult subjects with Gaucher disease. Fifteen milliliter blood sample will be collected during three independent visits over a period of approximately 3 months. These samples will be processed to separate plasma from red blood cells and frozen until assays are performed. Standardized immunoassay methods and LC/MS based methods will be adopted to assay a series of biomarkers in these samples. These data will be correlated with currently used diagnostic biomarkers.
Study Design
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Study Type : Observational
Estimated Enrollment : 35 participants
Observational Model: Case-Control
Time Perspective: Prospective
Official Title: Novel Inflammatory Biomarkers Complement 5A and Hepcidin in Patients With Gaucher Disease (GD)
Actual Study Start Date : October 2015
Estimated Primary Completion Date : June 2023
Estimated Study Completion Date : June 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Gaucher Disease

Groups and Cohorts
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Group/Cohort
Treatment naive GD1
Type 1 Gaucher disease subjects who are naive to any treatment
Treated GD1
Type 1 Gaucher disease who are stable on therapy (on the specific ERT and/or SRT and specific dose for at least 2 years)
Healthy Volunteers (No longer recruiting)
Age matched healthy controls. No new participants will be enrolled to this arm.


Outcome Measures
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Primary Outcome Measures :
  1. Change in Plasma C5a Concentration [ Time Frame: 6 months ]
    Outcome will be reported as the change in plasma concentration of C5a (ng/ml) from baseline to 6 months in participants with Gaucher disease type 1

  2. Change in Plasma Hepcidin Concentration [ Time Frame: 6 months ]
    Outcome will be reported as the change in plasma concentration of hepcidin (ng/ml) from baseline to 6 months in participants with Gaucher disease type 1


Secondary Outcome Measures :
  1. Change in Blood Glutathione Concentration [ Time Frame: 6 months ]
    Outcome will be reported as the change in blood concentration of glutathione (umol/g) from baseline to 6 months in participants with Gaucher disease type 1

  2. Change in Plasma Tumor Necrosis Factor (TNF)-alpha Concentration [ Time Frame: 6 months ]
    Outcome will be reported as the change in blood concentration of TNF-alpha (pg/ml) from baseline to 6 months in participants with Gaucher disease type 1


Biospecimen Retention:   Samples Without DNA
Blood samples

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Type 1 Gaucher disease subjects who are either naive to treatment or are stable on therapy (specific Enzyme Replacement Therapy (ERT) / Substrate Reduction Therapy (SRT) formulation at a specific dose) for at least 2 years.
Criteria

Inclusion criteria:

  1. All participants must be 18 years or older.
  2. All enrollees 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 Gaucher disease who are medically stable for participation in study in the opinion of the investigator.
  4. GD subjects must be stable on a specific ERT and/or SRT therapy at a specific dose (for e.g. on a units/kg basis) for at least 2 years or be naïve to these therapies (no therapy for 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. All participants must not have taken antioxidants coenzyme Q-10, vitamin C, or vitamin E for 3 weeks prior to the study.

Exclusion Criteria:

  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. Females 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. Subjects who cannot or are unwilling to have blood drawn
  6. Unable to adhere to study protocol for whatever reason
Contacts and Locations
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Information from the National Library of Medicine

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): NCT02437396


Contacts
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Contact: Reena Kartha, PhD 612-626-2436 rvkartha@umn.edu

Locations
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United States, Minnesota
University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Reena Kartha, PhD    612-626-2436    rvkartha@umn.edu   
Principal Investigator: Reena Kartha, PhD         
Sponsors and Collaborators
University of Minnesota
Investigators
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Principal Investigator: Reena Kartha, PhD University of Minnesota
More Information
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Responsible Party: University of Minnesota
ClinicalTrials.gov Identifier: NCT02437396    
Other Study ID Numbers: 1305M34501
First Posted: May 7, 2015    Key Record Dates
Last Update Posted: July 13, 2022
Last Verified: July 2022
Additional relevant MeSH terms:
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Gaucher Disease
Inflammation
Pathologic Processes
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
 Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders