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

This study is currently recruiting participants.
Verified May 2013 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 28, 2013
Last verified: May 2013

April 4, 2012
May 28, 2013
April 2012
April 2022   (final data collection date for primary outcome measure)
Overall Survival [ Time Frame: At 1 Year ] [ Designated as safety issue: No ]
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 ] [ 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.
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.

Not Provided
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
Hurler Syndrome Patients Treated with Laronidase
All patients receiving at least one dose of treatment with Laronidase.
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.
 
Recruiting
50
April 2022
April 2022   (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)
Both
Not Provided
No
Contact: Paul Orchard, M.D. 612-626-2313 orcha001@umn.edu
United States
 
NCT01572636
2011OC140, MT2011-21C
No
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
May 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP