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

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2014 by Masonic Cancer Center, University of Minnesota
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier:
NCT01572636
First received: April 4, 2012
Last updated: May 15, 2014
Last verified: May 2014
  Purpose

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 Intervention
Mucopolysaccharidosis Type IH
MPS I
Hurler Syndrome
Drug: Laronidase

Study Type: Observational
Study Design: 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).

Resource links provided by NLM:


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

Primary Outcome Measures:
  • Overall Survival [ Time Frame: At 1 Year ] [ Designated as safety issue: No ]
    Patients alive at 1 year post transplantation.


Secondary Outcome Measures:
  • Incidence of Engraftment [ Time Frame: 1 Year Post Transplant ] [ Designated as safety issue: No ]
    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.

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

  • Proportion of patients in need of ventilator support [ Time Frame: 1 Year ] [ Designated as safety issue: Yes ]
    Count of patients using ventilator by 1 year.


Estimated Enrollment: 50
Study Start Date: April 2012
Estimated Study Completion Date: April 2022
Estimated Primary Completion Date: April 2022 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
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

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.

  Eligibility

Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01572636

Contacts
Contact: Paul Orchard, M.D. 612-626-2313 orcha001@umn.edu

Locations
United States, Minnesota
Masonic Cancer Center, University of Minnesota Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Paul Orchard, M.D.    612-626-2313    orcha001@umn.edu   
Principal Investigator: Paul Orchard, M.D.         
Sponsors and Collaborators
Masonic Cancer Center, University of Minnesota
Investigators
Principal Investigator: Paul Orchard, M.D. Masonic Cancer Center, University of Minnesota
  More Information

No publications provided

Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT01572636     History of Changes
Other Study ID Numbers: 2011OC140, MT2011-21C
Study First Received: April 4, 2012
Last Updated: May 15, 2014
Health Authority: United States: Institutional Review Board

Keywords provided by Masonic Cancer Center, University of Minnesota:
Hurler Syndrome

Additional relevant MeSH terms:
Mucopolysaccharidoses
Mucopolysaccharidosis I
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases

ClinicalTrials.gov processed this record on July 22, 2014