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Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome

NCT01572636 · Status: TERMINATED · Type: OBSERVATIONAL · Enrollment: 20

Last updated 2018-09-27

No results posted yet for this study

Summary

This is a standard of care treatment guideline for patients with the diagnosis of mucopolysaccharidosis type IH (MPS I, Hurler syndrome) who are being considered as candidates for first hematopoietic stem cell transplantation (HSCT) according to a University of Minnesota myeloablative HSCT protocol.

Conditions

Interventions

DRUG

Laronidase

Administered 0.58 mg/kg/dose intravenously (IV) once a week beginning 12 weeks before planned hematopoietic stem cell transplant (HSCT) and resume same dosing regimen for 8 weeks after HSCT.

Sponsors & Collaborators

  • Masonic Cancer Center, University of Minnesota

    lead OTHER

Principal Investigators

  • Paul Orchard, M.D. · Masonic Cancer Center, University of Minnesota

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-03-28
Primary Completion
2018-05-01
Completion
2018-05-01

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 NCT01572636 on ClinicalTrials.gov