Gaucherite - A Study to Stratify Gaucher Disease

Launched by CAMBRIDGE UNIVERSITY HOSPITALS NHS FOUNDATION TRUST · Aug 4, 2017

Keywords

ClinConnect Summary

This clinical trial, called Gaucherite, is studying Gaucher Disease, which affects how the body processes certain fats. The researchers want to understand why some patients experience more severe symptoms than others and how well they respond to treatments. They will look at information from both adults and children in England who have been diagnosed with Gaucher Disease, focusing on issues related to bones, cancer, and brain health.

To participate in this study, patients must have a confirmed diagnosis of either Type I, Type II, or Type III Gaucher Disease and provide consent, which can also be given by a parent or legal guardian for minors. There are no age limits for participants, and both men and women can join. Participants will need to follow the study schedule and procedures, and some information may be collected from deceased patients if approved. It’s essential that potential participants do not have other serious health issues that could significantly shorten their life expectancy. Overall, this study hopes to gather important data that could lead to better treatment strategies for those living with Gaucher Disease.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• Each patient must meet all of the following criteria to be enrolled in this study:
• • 1. Confirmed biochemical diagnosis of Type I, Type II or Type III Gaucher disease
• • 2. Written Ethics Committee (EC) approved informed consent obtained from the patient, or patient's parent or legal guardian and patient assent if appropriate
• • 3. Male or Female patients, no age limitation
• • 4. Willing and able to comply with study schedule and procedures
• • 5. Deceased patients for whom the EC determines that patient data can be collected without a new consent from the patient
• Exclusion Criteria:
• Patients meeting any of the following criteria will be excluded from the study:
• • 1. Unrelated co-morbid condition limiting life expectancy to less than 6 months
• • 2. Patient or if applicable, parent or legal guardian is unable to comprehend, sign and date the EC approved informed consent form and patient assent as appropriate
• • 3. If determined unsuitable for the study by the investigator