Gaucher Disease Trial, Recruiting (NCT04101968)

Gaucher Disease Trial, Recruiting NCT04101968 Sponsor: Pacific Parkinson's Research Centre Condition: Gaucher Disease

Printed from Voxsanity (voxsanity.com.au) — NCT04101968 — 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 carry a specific genetic variation in a gene called GBA (for example, the p.L444P or p.N370S variant), meaning you have one changed copy of this gene
You are between 18 and 80 years old

Who may not be able to join:

You have been diagnosed with another neurological (brain or nervous system) condition in addition to the one being studied
You have a metal implant or medical device in your body that makes MRI scans unsafe, such as a heart pacemaker or certain metal implants
You have severe claustrophobia (a strong fear of enclosed spaces, such as the inside of an MRI machine)
You are unable to temporarily stop taking your Parkinson's medications without serious problems — this applies only to participants who have Parkinson's disease linked to the GBA gene (confirm with trial site)
You are currently taking a type of medication called cholinergic drugs (confirm with trial site if you are unsure whether your medication falls into this category)

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: Michele Matarazzo, MD, Pacific Parkinson's Research Centre | University of British Columbia

Phone: 6048227764

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: NCT04101968

Sponsor: Pacific Parkinson's Research Centre

Phase: Not Applicable

Status: Recruiting

Condition: Gaucher Disease

Trial contact: 6048227764

More information: https://clinicaltrials.gov/study/NCT04101968

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 carry a specific genetic variation in a gene called GBA (for example, the p.L444P or p.N370S variant), meaning you have one changed copy of this gene
You are between 18 and 80 years old

Who may not be able to join:

You have been diagnosed with another neurological (brain or nervous system) condition in addition to the one being studied
You have a metal implant or medical device in your body that makes MRI scans unsafe, such as a heart pacemaker or certain metal implants
You have severe claustrophobia (a strong fear of enclosed spaces, such as the inside of an MRI machine)
You are unable to temporarily stop taking your Parkinson's medications without serious problems — this applies only to participants who have Parkinson's disease linked to the GBA gene (confirm with trial site)
You are currently taking a type of medication called cholinergic drugs (confirm with trial site if you are unsure whether your medication falls into this category)

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

Pacific Parkinson's Research Centre

Registry

ClinicalTrials.gov

Start date

1 May 2019

Est. completion

30 December 2025

Where this trial is recruiting

🇨🇦 Canada 🇺🇸 United States

Primary endpoints

Acetylcholinesterase activity; Tau protein deposition; Dopaminergic denervation; neuroQWERTY index

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