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

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ClinicalTrials.gov Identifier: NCT01572636
Recruitment Status : Terminated (Research Cancelled)
First Posted : April 6, 2012
Last Update Posted : September 27, 2018
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota

Study Description
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Brief 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.

Condition or disease Intervention/treatment
Mucopolysaccharidosis Type IH MPS I Hurler Syndrome Drug: Laronidase

Detailed Description:
Laronidase Enzyme Replacement Therapy will be performed using laronidase once a week for 12 weeks prior to hematopoietic stem cell transplantation and for 8 weeks post-transplant to reduce pulmonary complications.
Study Design
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Study Type : Observational
Actual Enrollment : 20 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: MT2011-21C Laronidase (Aldurazyme TM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH).
Actual Study Start Date : March 28, 2012
Actual Primary Completion Date : May 1, 2018
Actual Study Completion Date : May 1, 2018

Resource links provided by the National Library of Medicine

Drug Information available for: Laronidase

Groups and Cohorts
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Group/Cohort Intervention/treatment
Laronidase use in Hurler Syndrome
Laronidase receiving prior and post transplant
 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.
Other Name: Aldurazyme


Outcome Measures
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Primary Outcome Measures :
  1. Overall Survival [ Time Frame: At 1 Year ]
    Patients alive at 1 year post transplantation.


Secondary Outcome Measures :
  1. Incidence of Engraftment [ Time Frame: 1 Year Post Transplant ]
    The incidence of donor engraftment will be estimated by taking the simple proportion of patients achieving donor engraftment over the number of evaluable patients. Donor engraftment will be defined as achieving an absolute neutrophil count ≥ 5x10^8/kg for three consecutive days before day 42 and maintenance of >10% donor chimerism through one year post transplant or death.

  2. Incidence of Grade III-IV Acute Graft Versus Host Disease [ Time Frame: Day 100 ]
    Cumulative incidence will be used to estimate grade III-IV acute GvHD, treating death as a competing risk.

  3. Proportion of patients in need of ventilator support [ Time Frame: 1 Year ]
    Count of patients using ventilator by 1 year.


Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Diagnosis of mucopolysaccharidosis type IH (MPS I, Hurler syndrome) and being considered as a candidate for first transplant according to a University of Minnesota myeloablative hematopoietic stem cell transplant (HSCT) protocol
Criteria

Inclusion Criteria:

  • Diagnosis of mucopolysaccharidosis type IH (MPS I, Hurler syndrome) and being considered as a candidate for first transplant according to a University of Minnesota myeloablative hematopoietic stem cell transplant (HSCT) protocol

Exclusion Criteria:

  • No prior therapy with laronidase enzyme replacement therapy (ERT)
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01572636


Locations
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United States, Minnesota
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
Masonic Cancer Center, University of Minnesota
Investigators
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Principal Investigator: Paul Orchard, M.D. Masonic Cancer Center, University of Minnesota
More Information
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Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT01572636    
Other Study ID Numbers: 2011OC140
MT2011-21C ( Other Identifier: Blood and Marrow Transplant Program )
First Posted: April 6, 2012    Key Record Dates
Last Update Posted: September 27, 2018
Last Verified: September 2018
Keywords provided by Masonic Cancer Center, University of Minnesota:
Hurler Syndrome
Additional relevant MeSH terms:
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Syndrome
Pathologic Processes
Mucopolysaccharidoses
Mucopolysaccharidosis I
Disease
Carbohydrate Metabolism, Inborn Errors
 Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases