Gaucher Disease Trial, Recruiting (NCT03240653)

Gaucher Disease Trial, Recruiting NCT03240653 Sponsor: Cambridge University Hospitals NHS Foundation Trust Condition: Gaucher Disease

Printed from Voxsanity (voxsanity.com.au) — NCT03240653 — Data sourced from ClinicalTrials.gov. Verify directly with the trial site before attending. Not medical advice.

Back to Gaucher Disease

Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

You have a confirmed diagnosis of Type I, Type II, or Type III Gaucher disease
You (or your parent or legal guardian) have given written agreement to take part, as approved by an ethics committee
You can be male or female, and there is no age limit for this trial
You are willing and able to follow the study schedule and attend the required visits
In some cases, data from patients who have passed away may be included if an ethics committee decides this is appropriate without needing new consent

Who may not be able to join:

You have a separate, unrelated health condition that means your life expectancy is less than 6 months
You (or your parent or legal guardian, if applicable) are not able to understand, sign, and date the required consent forms
The doctor running the study decides that you are not a suitable candidate for any reason (confirm with trial site)

⚠️ Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.

Last synced 28 July 2026

This study is not part of the standard drug-approval phase pathway (for example an observational, device, behavioural, or registry study), so a phase success rate does not apply.

Contact this trial

Principal Investigator: Timothy M Cox, MD, University of Cambridge

Phone: +441223274634

Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.

GP referral letter

Print a one-page summary to share with your doctor.

Voxsanity

Clinical trials and treatments, explained plainly · voxsanity.com.au

Date:

To: My Doctor

Re: Clinical trial enquiry

I am writing to enquire about the following clinical trial which may be relevant to my care:

Trial: NCT03240653

Sponsor: Cambridge University Hospitals NHS Foundation Trust

Phase: Not Applicable

Status: Recruiting

Condition: Gaucher Disease

Trial contact: +441223274634

More information: https://clinicaltrials.gov/study/NCT03240653

Eligibility summary (AI generated)

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

You have a confirmed diagnosis of Type I, Type II, or Type III Gaucher disease
You (or your parent or legal guardian) have given written agreement to take part, as approved by an ethics committee
You can be male or female, and there is no age limit for this trial
You are willing and able to follow the study schedule and attend the required visits
In some cases, data from patients who have passed away may be included if an ethics committee decides this is appropriate without needing new consent

Who may not be able to join:

You have a separate, unrelated health condition that means your life expectancy is less than 6 months
You (or your parent or legal guardian, if applicable) are not able to understand, sign, and date the required consent forms
The doctor running the study decides that you are not a suitable candidate for any reason (confirm with trial site)

⚠️ Important: Always verify eligibility with the trial site directly before applying.

Access in Australia: A treatment being studied in a clinical trial is generally not yet listed on the PBS. Where a medicine is not approved or not available through a trial, the TGA Special Access Scheme may allow a doctor to access unapproved therapeutic goods for individual patients. See voxsanity.com.au/sas-navigator/ for a plain English explainer.

This information was sourced from ClinicalTrials.gov via Voxsanity (data last synced 28 July 2026). It is not medical advice. Please verify the trial's current status directly with the trial site before acting.

Trial details

Status

Recruiting

Phase

Not Applicable

Sponsor

Cambridge University Hospitals NHS Foundation Trust

Registry

ClinicalTrials.gov

Start date

1 January 2014

Est. completion

30 September 2028

Where this trial is recruiting

🇬🇧 United Kingdom

Primary endpoints

Neurological outcome; Fragility Fracture; Disease Severity; Bone Marrow Involvement; Bone avascular necrosis; Cognitive Function; Cognitive Function; Neurological Physical Assessment; Multiple Myeloma

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov