RecruitingNot Applicable

Gaucher Disease Outcome Survey (GOS)

United States1,257 participantsStarted 2010-12-29

Plain-language summary

The Gaucher Outcomes Survey (GOS) is an ongoing observational, international, multi-center, long-term Registry of Patients with Gaucher Disease irrespective of their treatment status or type of treatment received. No experimental intervention is involved. Patients undergo clinical assessments and receive care as determined by the patients' treating physician. The objectives of the registry include to evaluate the safety and long-term effectiveness of velaglucerase alfa, to characterize patients receiving velaglucerase alfa or other Gaucher Disease-specific treatments, to gain a better understanding of the natural history of GD and to serve as a database for evidence-based management of Gaucher Disease over time in real-life clinical practice.

Who can participate

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: * Patients of any age or gender with confirmed diagnosis (biochemical and/or genetic) of Gaucher disease * Signed and dated written informed consent from the patient or, for patients aged \<18 years (\<16 years in the United Kingdom \[UK\]), their parent and/or legally authorized representatives (LAR), and assent of the minor where applicable. Legally authorized representatives are also applicable for cognitively impaired patients. Exclusion Criteria: \- Patients currently enrolled in ongoing blinded clinical trials (drugs or devices; includes all blinded trials)

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

Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

Timeframe: Baseline to one year for up to 20 years

Number of Participants With Infusion-related Reactions (IRRs)

Timeframe: Baseline to one year for up to 20 years

Increase of Hemoglobin Concentration

Timeframe: Baseline to one year for up to 20 years

Increase of Platelet Count

Timeframe: Baseline to one year for up to 20 years

Decrease in Liver Volume

Timeframe: Baseline to one year for up to 20 years

Decrease in Spleen Volume

Timeframe: Baseline to one year for up to 20 years

Increase in Bone Mineral Density (BMD)

Timeframe: Baseline to one year for up to 20 years

Trial details

NCT IDNCT03291223

SponsorShire

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2026-09-30

Contact for this trial

Shire Contact

+1 866 842 5335 ClinicalTransparency@shire.com

View on ClinicalTrials.gov More Gaucher Disease trials

Plain-language summary

Who can participate

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.

What they're measuring

Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

Timeframe: Baseline to one year for up to 20 years

Number of Participants With Infusion-related Reactions (IRRs)

Timeframe: Baseline to one year for up to 20 years

Increase of Hemoglobin Concentration

Timeframe: Baseline to one year for up to 20 years

Increase of Platelet Count

Timeframe: Baseline to one year for up to 20 years

Decrease in Liver Volume

Timeframe: Baseline to one year for up to 20 years

Decrease in Spleen Volume

Timeframe: Baseline to one year for up to 20 years

Increase in Bone Mineral Density (BMD)

Timeframe: Baseline to one year for up to 20 years