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

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01572636
First Posted: April 6, 2012
Last Update Posted: September 15, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota
April 4, 2012
April 6, 2012
September 15, 2017
March 28, 2012
July 17, 2018   (Final data collection date for primary outcome measure)
Overall Survival [ Time Frame: At 1 Year ]
Patients alive at 1 year post transplantation.
Same as current
Complete list of historical versions of study NCT01572636 on ClinicalTrials.gov Archive Site
  • 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.
  • 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.
  • Proportion of patients in need of ventilator support [ Time Frame: 1 Year ]
    Count of patients using ventilator by 1 year.
Same as current
Not Provided
Not Provided
 
Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome
MT2011-21C Laronidase (Aldurazyme TM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH).
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.
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.
Observational
Observational Model: Cohort
Time Perspective: Prospective
Not Provided
Not Provided
Probability Sample
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
  • Mucopolysaccharidosis Type IH
  • MPS I
  • Hurler Syndrome
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
Laronidase use in Hurler Syndrome
Laronidase receiving prior and post transplant
Intervention: Drug: Laronidase
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
20
July 17, 2018
July 17, 2018   (Final data collection date for primary outcome measure)

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)
Sexes Eligible for Study: All
Child, Adult, Senior
No
Contact information is only displayed when the study is recruiting subjects
United States
 
 
NCT01572636
2011OC140
MT2011-21C ( Other Identifier: Blood and Marrow Transplant Program )
No
Not Provided
Not Provided
Masonic Cancer Center, University of Minnesota
Masonic Cancer Center, University of Minnesota
Not Provided
Principal Investigator: Paul Orchard, M.D. Masonic Cancer Center, University of Minnesota
Masonic Cancer Center, University of Minnesota
September 2017