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

Hurler Syndrome Clinical Trial

Official title:

MT2011-21C Laronidase (Aldurazyme TM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH).

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01572636
• Other study ID #: 2011OC140
• Secondary ID: MT2011-21C
• Status: Terminated
• Start date: March 28, 2012
• Est. completion date: May 1, 2018

Study information

• Verified date: September 2018
• Source: Masonic Cancer Center, University of Minnesota
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 20
• Est. completion date: May 1, 2018
• Est. primary completion date: May 1, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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)

Related Conditions & MeSH terms

• Hurler Syndrome
• MPS I
• Mucopolysaccharidoses
• Mucopolysaccharidosis I
• Mucopolysaccharidosis Type IH
• Syndrome

Intervention

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.

Locations

Country	Name	City	State
United States	Masonic Cancer Center, University of Minnesota	Minneapolis	Minnesota

Sponsors (1)

Lead Sponsor	Collaborator
Masonic Cancer Center, University of Minnesota

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Overall Survival	Patients alive at 1 year post transplantation.	At 1 Year
Secondary	Incidence of Engraftment	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.	1 Year Post Transplant
Secondary	Incidence of Grade III-IV Acute Graft Versus Host Disease	Cumulative incidence will be used to estimate grade III-IV acute GvHD, treating death as a competing risk.	Day 100
Secondary	Proportion of patients in need of ventilator support	Count of patients using ventilator by 1 year.	1 Year