UnknownNot Applicable

SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease

United States30 participantsStarted 2015-05

Plain-language summary

This project is expected to elucidate role of different therapeutic interventions: SRT in comparison to ERT in influencing immune aspects of GD pathology, as well as bone involvement.

Who can participate

Age range

18 Years

Sex

ALL

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 diagnosis of Gaucher disease
. Subjects on SRT using Cerdelga ( must fulfil the pharmacogenomics criteria for Cerdelga)

Exclusion criteria

. Unconfirmed diagnosis of Gaucher disease
. Subject or guardian unable to provide consent
. Any chronic immunosuppressive state or therapy

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Delineate T-, B- and NK cell fractions as well as dendritic cell and monocyte numbers and compare between patients undergoing ERT vs. SRT.

Timeframe: 2 Years

Trial details

NCT IDNCT02605603

SponsorLysosomal and Rare Disorders Research and Treatment Center, Inc.

Sponsor typeOTHER

Study typeOBSERVATIONAL

Primary completion2020-02

View on ClinicalTrials.gov More Gaucher Disease trials