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Administration of IV Laronidase Post Bone Marrow Transplant in Hurler

NCT01173016 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 11

Last updated 2020-03-20

Study results available
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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.

Conditions

Interventions

DRUG

Laronidase

Laronidase 0.58 mg/kg intravenously (IV) once a week for a maximum of 2 years

Sponsors & Collaborators

  • Masonic Cancer Center, University of Minnesota

    lead OTHER

Principal Investigators

  • Paul Orchard, MD · Masonic Cancer Center, University of Minnesota

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
14 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-05-29
Primary Completion
2016-03-04
Completion
2016-03-04

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01173016 on ClinicalTrials.gov