RecruitingNot Applicable

Gaucherite - A Study to Stratify Gaucher Disease

United Kingdom250 participantsStarted 2014-01-01

Plain-language summary

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.

Who can participate

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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

Exclusion criteria

✕. Unrelated co-morbid condition limiting life expectancy to less than 6 months
✕. 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

What they're measuring

Neurological outcome

Timeframe: 2029

Fragility Fracture

Timeframe: 2029

Disease Severity

Timeframe: 2029

Bone Marrow Involvement

Timeframe: 2029

Bone avascular necrosis

Timeframe: 2029

Cognitive Function

Timeframe: 2019

Cognitive Function

Timeframe: 2029

Neurological Physical Assessment

Timeframe: 2029

Multiple Myeloma

Timeframe: 2029

Trial details

NCT IDNCT03240653

SponsorCambridge University Hospitals NHS Foundation Trust

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2028-09-30

Contact for this trial

Elizabeth M MORRIS, RN

+441223274634 liz.morris@addenbrookes.nhs.uk

View on ClinicalTrials.gov More Gaucher Disease, Type I trials

Plain-language summary

Who can participate

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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

Exclusion criteria

✕. Unrelated co-morbid condition limiting life expectancy to less than 6 months
✕. 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

What they're measuring

Neurological outcome

Timeframe: 2029

Fragility Fracture

Timeframe: 2029

Disease Severity

Timeframe: 2029

Bone Marrow Involvement

Timeframe: 2029

Bone avascular necrosis

Timeframe: 2029

Cognitive Function

Timeframe: 2019

Cognitive Function

Timeframe: 2029

Neurological Physical Assessment

Timeframe: 2029

Multiple Myeloma

Timeframe: 2029