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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.


Found (20) clinical trials

A Long-term Follow-up Study of Gaucher Disease

The study aims (aims a-c) will be accomplished by the systematic monitoring of health records. Health information will be extracted from medical records for patients followed at Duke. Medical records will be requested from treating physicians outside of Duke. The PI, (Dr. Kishnani) a recognized expert in Gaucher disease, is ...

Phase N/A

Biomarker for Gaucher Disease

The unmetabolised Glucocerebrosides are stored throughout the whole reticulo-endothelial system. Accumulation of Glycolipid-enriched Macrophages establishes a pathoanatomical phenomenon, the so-called Gaucher cells, which can be verified by light microscopy. Activation markers of the Macrophages, like the enzyme Chitotriosidase or CCL18, are parameters which follow the course of Gaucher dis-ease. Gaucher ...

Phase N/A

Thrombocytopathy in Gaucher Disease Patients

Objectives Delineating the cause of the thrombocytopathy in Gaucher disease patients: Identifying thrombocytopathy among a cohort of 70 Gaucher patients managed (treated and untreated) in our clinic using a panel of platelets function tests. Understanding the etiology for platelets dysfunction in Gaucher disease. Evaluating the impact of Imiglucerase treatment duration ...

Phase N/A

Home Therapy With VPRIV in Gaucher's Disease

The purpose of this study is to proof increasing patient satisfaction and preservation of quality of life in patients with Gaucher's Disease receiving their enzyme replacement therapy with VPRIV (Velaglucerase alfa)at their home setting compared to receiving the infusions at the clinic or at doctor's practice.

Phase N/A

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 treatment received. No experimental intervention is involved. Patients undergo clinical assessments and receive care as determined by the patients' treating physician. The objectives of ...

Phase N/A

Clinical and Pathophysiological Investigations Into Erdheim Chester Disease

Rare and potentially lethal, Erdheim-Chester Disease (ECD) is a histiocytic neoplasm about which little is known, and for which there remains a paucity of effective treatments. Histologically, it is categorized as a non-Langerhans cell histiocytosis, and most commonly occurs in men between the ages of 50 and 70, although cases ...

Phase N/A

SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease

Introduction Lysosomes are small, cytoplasmic organelles that contain several acid hydrolase enzymes. These enzymes break down foreign materials and cellular debris allowing the lysosomes to act as recycling centers for the cells. Following DNA transcription, lysosomal enzymes are produced in the endoplasmic reticulum and targeted to lysosomes by specific recognition ...

Phase N/A

Oxidative Stress-Related Biomarkers in Gaucher Disease: A Preliminary Study

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 ...

Phase N/A

Gaucherite - A Study to Stratify Gaucher Disease

Gaucher disease is part of a rare group of genetic metabolic diseases which are caused by an inherited deficiency of the enzyme glucocerebrosidase. The most common form, Type 1 affects 1 in 40,000 to 60,000 individuals in the general population. In Type 1 symptoms may appear at any time from ...

Phase N/A

Investigating Lysosomal Storage Diseases in Minority Groups

Although lysosomal storage disorders, such as Fabry disease, Gaucher disease, and Pompe disease, represent serious challenges in the healthcare system, no study has yet investigated the prevalence of these diseases in the US. Frequently, patients show progressive worsening of symptoms for several years before they get diagnosed. Since many of ...

Phase N/A