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 26 clinical trials
Ambroxol as a Treatment for Parkinson's Disease Dementia

The present study will test the hypothesis that the medication Ambroxol is safe and well tolerated and will improve cognitive and motor symptoms of Parkinson's Disease Dementia (PDD). Ambroxol has been shown to raise the levels of the enzyme beta-glucocerebrosidase resulting in lower the levels of the protein alpha-synuclein, both …

  • 633 views
  • 08 Feb, 2021
  • 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
  • 27 Jan, 2021
  • 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

imiglucerase
type 1 gaucher disease
alglucerase
eliglustat
deficiency
  • 522 views
  • 24 Jan, 2021
  • 221 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.

genetic disorder
palliative care
stem cell transplantation
hematopoetic
cell transplantation
  • 359 views
  • 26 Jan, 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
  • 87 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
  • 26 Jan, 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

type 1 gaucher disease
velaglucerase alfa
substrate reduction therapy
replacement therapy
vpriv
  • 12 views
  • 22 Aug, 2021
  • 1 location
Survey Study for Velaglucerase Alfa (VPRIV) in Japan

The objective of this post-marketing survey study is to collect data to determine the safety and efficacy of velaglucerase alfa (VPRIV) in participants with Gaucher disease who are new to

velaglucerase alfa
vpriv
  • 74 views
  • 27 Jan, 2021
  • 31 locations