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Found 31 clinical trials
Digital Health Platform Customized for Patients With Gaucher Disease

families in managing their health care. In a rare disease, such as Gaucher disease, patient involvement through digital technology is of great importance. Gaucher patients come for an inspection at

  • 0 views
  • 05 Jun, 2022
  • 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
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
  • 12 Feb, 2022
  • 31 locations
A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease

The main aim of this study is to learn if velaglucerase alfa (VPRIV) improves growth and symptoms in participants under 5 years old with Gaucher disease. Symptoms will be checked with blood

velaglucerase alfa
vpriv
  • 0 views
  • 20 Jul, 2021
  • 1 location
Post Marketing Surveillance (PMS) Study for Velaglucerase Alfa (VPRIV) in India

record genetic mutation data from participants with Gaucher disease.

velaglucerase alfa
vpriv
  • 0 views
  • 21 Aug, 2021
  • 2 locations
The GBA Multimodal Study in Parkinson's Disease

This study plans to analyze the molecular and clinical mechanisms of the relationship between the GBA mutations and Parkinson's disease. This will be assessed through the use of advanced neuroimaging techniques called PET (positron emission tomography) to study the accumulation of the tau protein and the dysfunction of acetylcholine and …

acetylcholine
gba gene
positron emission tomography
dopamine
parkinson's disease
  • 0 views
  • 25 Jan, 2022
  • 3 locations
World Data on Ambroxol for Patients With GD and GBA Related PD

and efficacy of ambroxol for patients with Gaucher disease (GD).

alglucerase
type 1 gaucher disease
  • 0 views
  • 05 Jun, 2022
  • 2 locations
A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen (OPPUS)

Primary Objective: To assess prevalence of Gaucher disease (GD) diagnosed in pediatric patients presenting with unexplained splenomegaly (SMG) after exclusion of first intention-diagnoses

  • 0 views
  • 23 May, 2022
  • 1 location
A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen (OPPUS)

Primary Objective: To assess prevalence of Gaucher disease (GD) diagnosed in pediatric patients presenting with unexplained splenomegaly (SMG) after exclusion of first intention-diagnoses

  • 0 views
  • 24 Jun, 2022
  • 2 locations
Natural History of Glycosphingolipid and Glycoprotein Lysosomal Storage Disorders

treatment. Patients of any age with Tay-Sachs disease, Sandhoff disease, GM1 gangliosidosis, or type 2 Gaucher disease may be eligible for this study. Participants will be admitted to the NIH

MRI
deficiency
  • 76 views
  • 17 Jun, 2022
  • 1 location