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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT00176891
Other study ID # MT2004-09
Secondary ID 0403M57728
Status Completed
Phase Phase 2
First received
Last updated
Start date March 2004
Est. completion date November 2012

Study information

Verified date July 2019
Source Masonic Cancer Center, University of Minnesota
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

The investigators hypothesize that weekly infusions of Laronidase ERT for 10-12 weeks prior to transplant and 8 weeks following transplant will result in a reduction of glycosaminoglycans (GAG) burden that is associated with decreased complications following transplant.


Description:

Subjects will receive laronidase once a week intravenously for 10-12 weeks prior to transplant and for approximately 8 weeks after transplant. Laronidase will be given by intravenous infusion (IV) through a catheter and from there to your child's body's cells and organs to break down the glycosaminoglycans (GAG) buildup.

Prior to starting ERT, subjects will have a complete physical examination, which includes a complete assessment of your child's airway and lungs. In addition to standard treatment evaluations and tests, which are done prior to hematopoietic stem cell transplant (HSCT), subjects will have the following tests: an additional teaspoon of blood for a baseline test for serum antibodies against laronidase, before and after the fourth dose of laronidase, the investigators will collect 2 teaspoons of blood for an alpha-L-iduronidase enzyme level; to watch for side effects to laronidase and the development of antibodies to laronidase, approximately 2 teaspoons of blood will be collected every 3 weeks while the subject is receiving laronidase ERT.


Recruitment information / eligibility

Status Completed
Enrollment 25
Est. completion date November 2012
Est. primary completion date November 2012
Accepts healthy volunteers No
Gender All
Age group N/A to 7 Years
Eligibility Inclusion Criteria:

- Patients with the diagnosis of mucopolysaccharidosis type IH (MPS I, Hurler syndrome) who are candidates for first hematopoietic stem cell transplant (HSCT) according to a University of Minnesota myeloablative HSCT protocol.

Exclusion Criteria:

- Not being considered for University of Minnesota myeloablative HSCT protocol.

- Previous administration of laronidase enzyme

- Second or subsequent HSCT.

Study Design


Intervention

Procedure:
Stem Cell Transplant
enzyme replacement 10-12 weeks prior to hematopoietic stem cell transplant (HSCT), and 8 weeks following
Drug:
Laronidase ERT
Laronidase ERT will be administered 10-12 weeks prior to HSCT, and 8 weeks following.

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 Number of Patients Alive at One Year Post Transplant one year
Primary Number of Patients Requiring Ventilator Support at One Year Post Transplant one year
Secondary Donor Engraftment Day 100 post transplant
Secondary Patients With Grade III-IV Acute GVHD Day 100 post transplant
Secondary Reduction in Glycosaminoglycans (GAG) Data was not collected on this outcome measure and is not available for reporting. Prior to, During and After ERT
Secondary Toxicity (Adverse Events) Associated With Infusions of Laronidase Data was not collected on this outcome measure and is not available for reporting. 1 year post transplant
Secondary Development of Anti-iduronidase Antibodies in Serum 1 Year
Secondary Patients With Improvement in Obstructive Apnea (Breathing) by Polysomnography Baseline, 12 weeks after laronidase, after transplant
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