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Stem Cell Transplant w/Laronidase for Hurler

Primary Purpose

Mucopolysaccharidosis I, Hurler Syndrome

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Stem Cell Transplant
Laronidase ERT
Sponsored by
Masonic Cancer Center, University of Minnesota
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Mucopolysaccharidosis I focused on measuring Laronidase ERT, Stem cell transplant, storage disease, inborn errors of metabolism, hurler syndrome, glycosaminoglycans, enzyme replacement

Eligibility Criteria

undefined - 7 Years (Child)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Masonic Cancer Center, University of Minnesota

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Laronidase ERT Treatment

Arm Description

Weekly infusion of laronidase enzyme replacement therapy followed by hematopoietic stem cell transplant.

Outcomes

Primary Outcome Measures

Number of Patients Alive at One Year Post Transplant
Number of Patients Requiring Ventilator Support at One Year Post Transplant

Secondary Outcome Measures

Donor Engraftment
Patients With Grade III-IV Acute GVHD
Reduction in Glycosaminoglycans (GAG)
Data was not collected on this outcome measure and is not available for reporting.
Toxicity (Adverse Events) Associated With Infusions of Laronidase
Data was not collected on this outcome measure and is not available for reporting.
Development of Anti-iduronidase Antibodies in Serum
Patients With Improvement in Obstructive Apnea (Breathing) by Polysomnography

Full Information

First Posted
September 12, 2005
Last Updated
July 10, 2019
Sponsor
Masonic Cancer Center, University of Minnesota
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1. Study Identification

Unique Protocol Identification Number
NCT00176891
Brief Title
Stem Cell Transplant w/Laronidase for Hurler
Official Title
Phase II Study of Combined Laronidase (AldurazymeTM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH)
Study Type
Interventional

2. Study Status

Record Verification Date
July 2019
Overall Recruitment Status
Completed
Study Start Date
March 2004 (undefined)
Primary Completion Date
November 2012 (Actual)
Study Completion Date
November 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Masonic Cancer Center, University of Minnesota

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief 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.
Detailed 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Mucopolysaccharidosis I, Hurler Syndrome
Keywords
Laronidase ERT, Stem cell transplant, storage disease, inborn errors of metabolism, hurler syndrome, glycosaminoglycans, enzyme replacement

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
25 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Laronidase ERT Treatment
Arm Type
Experimental
Arm Description
Weekly infusion of laronidase enzyme replacement therapy followed by hematopoietic stem cell transplant.
Intervention Type
Procedure
Intervention Name(s)
Stem Cell Transplant
Other Intervention Name(s)
ERT
Intervention Description
enzyme replacement 10-12 weeks prior to hematopoietic stem cell transplant (HSCT), and 8 weeks following
Intervention Type
Drug
Intervention Name(s)
Laronidase ERT
Other Intervention Name(s)
Aldurazyme®
Intervention Description
Laronidase ERT will be administered 10-12 weeks prior to HSCT, and 8 weeks following.
Primary Outcome Measure Information:
Title
Number of Patients Alive at One Year Post Transplant
Time Frame
one year
Title
Number of Patients Requiring Ventilator Support at One Year Post Transplant
Time Frame
one year
Secondary Outcome Measure Information:
Title
Donor Engraftment
Time Frame
Day 100 post transplant
Title
Patients With Grade III-IV Acute GVHD
Time Frame
Day 100 post transplant
Title
Reduction in Glycosaminoglycans (GAG)
Description
Data was not collected on this outcome measure and is not available for reporting.
Time Frame
Prior to, During and After ERT
Title
Toxicity (Adverse Events) Associated With Infusions of Laronidase
Description
Data was not collected on this outcome measure and is not available for reporting.
Time Frame
1 year post transplant
Title
Development of Anti-iduronidase Antibodies in Serum
Time Frame
1 Year
Title
Patients With Improvement in Obstructive Apnea (Breathing) by Polysomnography
Time Frame
Baseline, 12 weeks after laronidase, after transplant

10. Eligibility

Sex
All
Maximum Age & Unit of Time
7 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Paul Orchard, MD
Organizational Affiliation
Masonic Cancer Center, University of Minnesota
Official's Role
Principal Investigator
Facility Information:
Facility Name
Masonic Cancer Center, University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States

12. IPD Sharing Statement

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Stem Cell Transplant w/Laronidase for Hurler

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