Administration of IV Laronidase Post Bone Marrow Transplant in Hurler

University of Minnesota (UMN)

Status and phase: Completed; Phase 1

Conditions: Hurler Syndrome

Treatments: Drug: Laronidase

Study type: Interventional

Funder types: Other

Identifiers

NCT01173016

1004M80513 (Other Identifier)

2009LS090

MT2009-20 (Other Identifier)

Details and patient eligibility

About

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.

Full description

This 2-year open-label pilot study of laronidase includes patients (age 5-13 years) who are at least 2 years post-hematopoietic cell transplantation (HCT) and donor engrafted. Outcomes are assessed semi-annually and compared to historic controls. Eligible patients will receive Laronidase as an infusion over several hours once a week at a local site. The dosing of enzyme will be the standard doses recommended by Genzyme.

The findings of this Pilot Study will be used to assess whether a subsequent larger study can be conducted.

Enrollment: 11 patients

Sex: All

Ages: Under 14 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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11 participants in 1 patient group

Laronidase After Transplantation

Experimental group

Description:

Patients with Mucopolysaccharidosis type IH (MPS I, Hurler syndrome) treated with a prior allogeneic transplant \>2 years previously and treated with Laronidase weekly for 2 years after transplant.

Treatment:

Drug: Laronidase