Stem Cell Transplant w/Laronidase for Hurler

Mucopolysaccharidosis I Clinical Trial

Official title:

Phase II Study of Combined Laronidase (AldurazymeTM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00176891
• Other study ID #: MT2004-09
• Secondary ID: 0403M57728
• Status: Completed
• Phase: Phase 2
• 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
• 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.

Related Conditions & MeSH terms

• Hurler Syndrome
• Mucopolysaccharidoses
• Mucopolysaccharidosis I
• Syndrome

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