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 range14 Years

SexALL

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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

What they're measuring

Percentage of Adherence to the Scheduled Weekly Infusion by the Participants

Timeframe: 24 months

Number of Participants Experiencing Severe Adverse Events

Timeframe: 24 months

Trial details

NCT IDNCT01173016

SponsorMasonic Cancer Center, University of Minnesota

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2016-03-04

Results submitted2017-04-13

View on ClinicalTrials.gov More Hurler Syndrome trials