WithdrawnPhase 4

The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease

Stopped: No subjects accrued and no potential subjects were identified..

United States0Started 2015-07

Plain-language summary

The purpose of this trial is to study the effect of Velaglucerase Alfa on skeletal bone development of children with Type 1 or Type 3 Gaucher Disease. In addition, the natural history and neurological status of children with Type 3 Gaucher Disease will be studied.

Who can participate

Age range

4 Years – 14 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: * Enzyme Replacement Therapy naive, * confirmed diagnosis of Gaucher disease type 1 or 3, * able to travel to Dallas, Texas 1x per year for baseline plus 3 consecutive years, * able to tolerate all study procedures, * skeleton not fully formed as confirmed by DXA and MRI), * and willing to receive velaglucerase alfa infusions every other week for the duration of the study. Exclusion Criteria: * Clinically unstable, * taking or have taken bisphosphonates, * Gaucher type 2, * pregnant female, * or deemed inappropriate for participation by the principal investigator.

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

Change in Z-scores from baseline in Skeletal Bone Mineral Density by Duel-energy X-ray absorptiometry (DXA) in children with Gaucher Disease type 1 and 3 receiving velaglucerase alfa.

Timeframe: Baseline pre-intervention and yearly thereafter for 3 years

Trial details

NCT IDNCT02528617

SponsorBaylor Research Institute

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2017-10

View on ClinicalTrials.gov More Gaucher Disease Type 1 trials

Who can participate

Age range

4 Years – 14 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.