Administration of IV Laronidase Post Bone Marrow Transplant in Hurler (NCT01173016) | Clinical Trial Compass
CompletedPhase 1
Administration of IV Laronidase Post Bone Marrow Transplant in Hurler
United States11 participantsStarted 2012-05-29
Plain-language summary
This is a single center pilot study in which Laronidase will be given weekly for two years in patients with Hurler syndrome, also known as mucopolysaccharide IH (MPS I, Hurler syndrome), that have previously been treated with an allogeneic transplant.
Who can participate
Age range
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:
* Mucopolysaccharidosis type IH (MPS I, Hurler syndrome) treated with a prior allogeneic transplant \>2 years previously
* Age \<14 years old
* \>10% engrafted based on recent testing (\<4 months prior to enrollment)
* Willing to commit to traveling to the University of Minnesota every 6 months
* Written informed consent prior to the performance of any study related procedures
Exclusion Criteria:
* Previous administration of Laronidase enzyme \> 3 months post transplantation
* Anticipated survival less than 2 years
* History of cardiac or pulmonary insufficiency, including an ejection fraction (EF) \< 40% or those requiring continuous supplemental oxygen
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
1
Percentage of Adherence to the Scheduled Weekly Infusion by the Participants
Timeframe: 24 months
2
Number of Participants Experiencing Severe Adverse Events
Timeframe: 24 months
Trial details
NCT IDNCT01173016
SponsorMasonic Cancer Center, University of Minnesota