NCT03240653Recruiting
Gaucherite - A Study to Stratify Gaucher Disease
Predictive Measures to Stratify Clinical Outcomes in Children and Adults With Gaucher Disease and Responses to Specific Therapies
Source: ClinicalTrials.gov · Last updated 6 Aug 2024 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
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 severity and response to therapy of Gaucher disease especially in the areas of bone, cancer and brain conditions.
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 250 (estimated)
- First submitted
- 12 Jul 2017
- Start date
- 1 Jan 2014
- Primary completion date
- 30 Sept 2028
- Lead sponsor
- Cambridge University Hospitals NHS Foundation Trust
- Diseases
- Gaucher disease
- NCT number
- NCT03240653
What you commit to
- Study type: Observational
- Planned participants: 250
- From 1 Jan 2014 to the primary completion date, 30 Sept 2028
- 8 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- Unrelated co-morbid condition limiting life expectancy to less than 6 months
Other
- Patients meeting any of the following criteria will be excluded from the study:
- 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
- If determined unsuitable for the study by the investigator
Who the study is looking for
- Each patient must meet all of the following criteria to be enrolled in this study:
- Confirmed biochemical diagnosis of Type I, Type II or Type III Gaucher disease
- Written Ethics Committee (EC) approved informed consent obtained from the patient, or patient's parent or legal guardian and patient assent if appropriate
- Male or Female patients, no age limitation
- Willing and able to comply with study schedule and procedures
- Deceased patients for whom the EC determines that patient data can be collected without a new consent from the patient
Sex: All · Does not accept healthy volunteers
Sites (8)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| New Queen Elizabeth Hospital Coordinator of this site? Confirm its status | Birmingham | United Kingdom | Recruiting |
| Birmingham Childrens Hospital Coordinator of this site? Confirm its status | Birmingham | United Kingdom | Terminated |
| Cambridge university hospital Coordinator of this site? Confirm its status | Cambridge | United Kingdom | Recruiting |
| Royal Free Hospital Coordinator of this site? Confirm its status | London | United Kingdom | Recruiting |
| National Hospital For Neurology and Neurosurgery Coordinator of this site? Confirm its status | London | United Kingdom | Recruiting |
| Great Ormond Street Hospital Coordinator of this site? Confirm its status | London | United Kingdom | Recruiting |
| Royal Manchester Childrens Hospital Coordinator of this site? Confirm its status | Manchester | United Kingdom | Recruiting |
| Salford Royal NHS Foundation Trust Coordinator of this site? Confirm its status | Salford | United Kingdom | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.