Gaucher Disease Clinical Trials

A listing of Gaucher Disease medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

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Found 28 clinical trials
GSL Synthetase Inhibitor or in Combination With Immune Checkpoint Inhibitor in Previously Treated Blood and Solid Tumor

Immune checkpoint blockade has made great but unsatisfied success in treating cancers. One important reason is the hijacked HLA-I antigen presentation. Eliglustat could inhibit glycosphingolipids synthesis and restore HLA-I antigen presentation and transform the immunogenicity of tumor cells. Therefore,GSL synthetase inhibitor eliglustat monotherapy or in combination with immune checkpoint inhibitor …

cancer
solid tumor
  • 0 views
  • 12 Jan, 2022
  • 1 location
Gaucher Disease Outcome Survey (GOS)

The Gaucher Outcomes Survey (GOS) is an ongoing observational, international, multi-center, long-term Registry of Patients with Gaucher Disease irrespective of their treatment status or type of

velaglucerase alfa
  • 126 views
  • 08 Jan, 2022
  • 1 location
International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher disease, irrespective of

deficiency
alglucerase
imiglucerase
type 1 gaucher disease
eliglustat
  • 0 views
  • 22 Jan, 2022
  • 281 locations
International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher disease, irrespective of

deficiency
alglucerase
imiglucerase
type 1 gaucher disease
eliglustat
  • 522 views
  • 23 Jan, 2022
  • 173 locations
Longitudinal Study of Neurodegenerative Disorders

The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the effects of interventions.

hematopoetic
genetic disorders
palliative care
stem cell transplantation
genetic disorder
  • 359 views
  • 13 Nov, 2021
  • 1 location
Gaucherite - A Study to Stratify Gaucher Disease

The purpose of this research is to review data already collected and to collect new data from adults and children in England with Gaucher Disease to determine clinical factors which predict

enzyme therapy
cancer
  • 117 views
  • 28 Jul, 2021
  • 8 locations
Biomarkers for Inborn Errors of Metabolism

International, multicenter, observational, longitudinal study to identify or monitor Inborn Error of Metabolism disease biomarkers and to explore the clinical robustness, specificity, and long-term variability of these biomarkers

  • 15 views
  • 02 Jul, 2021
  • 18 locations
A Long-term Follow-up Study of Gaucher Disease

The study aims are to: a) identify the long-term natural history of Gaucher disease, b) evaluate long-term treatment efficacy of enzyme replacement therapy (ERT) and substrate reduction therapy

substrate reduction therapy
replacement therapy
  • 26 views
  • 30 Sep, 2021
  • 1 location
Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease

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

antioxidants
  • 8 views
  • 01 Jul, 2021
  • 1 location
A Study of VPRIV in Participants With Gaucher Disease Previously Treated With Other Enzyme Replacement Therapies or Substrate Reduction Therapies

The main aim of this study is to describe the safety profile of velaglucerase alfa (VPRIV) in participants with Gaucher disease type 1. Participants will be transitioning from other enzyme

velaglucerase alfa
vpriv
substrate reduction therapy
type 1 gaucher disease
replacement therapy
  • 16 views
  • 22 Aug, 2021
  • 1 location