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Administration of IV Laronidase Post Bone Marrow Transplant in Hurler

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota Identifier:
First received: July 28, 2010
Last updated: February 23, 2016
Last verified: February 2016
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.

Condition Intervention Phase
Hurler Syndrome
Drug: Laronidase
Phase 1

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Pilot Study of Administration of Intravenous Laronidase Following Allogeneic Transplantation for Hurler Syndrome

Resource links provided by NLM:

Further study details as provided by Masonic Cancer Center, University of Minnesota:

Primary Outcome Measures:
  • Toxicities [ Time Frame: Prior to Starting Enzyme Throughout 2 Years on Therapy ]
    Adverse events that occur after administration with Laronidase.

Secondary Outcome Measures:
  • Change in Orthopedic Measures [ Time Frame: From baseline every 6 months for 2 Years ]
    Measurements from pQCT, bone mineral density, bone mineral content, bone geometry and strength.

  • Change in Markers of Bone Metabolism [ Time Frame: From baseline every 6 months through 2 years ]
    Measurements of Serum osteocalcin (OCN) and bone-specific alkaline phosphatase (BSAP), carboxyterminal telopeptide of type I collagen (ICTP) and carboxyterminal telopeptide α1 chain of type I collagen (CTX)

  • Change in Flexibility and Muscle Strength [ Time Frame: From baseline every 6 months through 2 years ]
    determined by Biodex and Physical Therapy evaluations, including a 6 minute walk study. The Biodex III isokinetic strength testing system will be used to assess strength at the knee and elbow for each participant.

  • Change in 0xygen Utilization to Monitor "Fitness" [ Time Frame: From baseline every 6 months through 2 years ]
    The measurement of peak oxygen uptake (VO2 peak) during exercise testing is considered the best single physiologic indicator of an individual's cardiorespiratory fitness and be also be used to monitor changes in cardiorespiratory fitness over time .

  • Change in Neuropsychological Function [ Time Frame: From baseline every 6 months through 2 years ]
    Continuous scores gathered regarding tests of intelligence, attention, learning and memory, visual processing, fine motor, and quality of life

  • Change in Laboratory Measurements [ Time Frame: From baseline every 3 months through 2 years ]
    including biomarkers, cytokines, markers of inflammation and the development of antibody

  • Change in Bone Density [ Time Frame: From baseline to 1 and 2 Years ]
    Measurements from dual energy x-ray absorptiometry (DXA)

Estimated Enrollment: 10
Study Start Date: May 2012
Estimated Study Completion Date: March 2016
Estimated Primary Completion Date: March 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Laronidase After Transplantation
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.
Drug: Laronidase
Laronidase 0.58 mg/kg intravenously (IV) once a week for a maximum of 2 years
Other Name: Aldurazyme

Detailed Description:

The primary objective of this pilot study is to determine the feasibility of giving weekly Laronidase for 2 years in patients with Hurler syndrome after allogeneic transplantation. Specifically, i) the ability to enroll patients, ii) continued compliance throughout the study with drug administration and testing, as well as iii) the relevance of various endpoint determinations will be assessed. The findings of the pilot study will be used to assess whether a subsequent larger study will be conducted.

Secondary Objectives: The secondary objectives of this study will focus on the toxicity associated with weekly Laronidase in this patient population, and the evaluation of a variety of testing and efficacy parameters that would be utilized to measure outcomes and determine benefit in patients treated on a subsequent larger study.

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.


Ages Eligible for Study:   up to 14 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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
  Contacts and Locations
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Please refer to this study by its identifier: NCT01173016

United States, Minnesota
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
Masonic Cancer Center, University of Minnesota
Principal Investigator: Paul Orchard, MD Masonic Cancer Center, University of Minnesota
  More Information

Responsible Party: Masonic Cancer Center, University of Minnesota Identifier: NCT01173016     History of Changes
Other Study ID Numbers: 2009LS090
MT2009-20 ( Other Identifier: Blood and Marrow Transplantation Program )
1004M80513 ( Other Identifier: IRB, University of Minnesota )
Study First Received: July 28, 2010
Last Updated: February 23, 2016

Keywords provided by Masonic Cancer Center, University of Minnesota:
mucopolysaccharide IH (MPS IH)

Additional relevant MeSH terms:
Mucopolysaccharidosis I
Pathologic Processes
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Connective Tissue Diseases
Metabolic Diseases processed this record on April 28, 2017