Back to results

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

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

NCT ID

NCT00176891

First Posted

September 12, 2005

Last Updated

July 10, 2019

Sponsor

Masonic Cancer Center, University of Minnesota

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

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

Eligibility Criteria

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

Learn more about this trial

Stem Cell Transplant w/Laronidase for Hurler

We'll reach out to this number within 24 hrs