Stem Cell Transplant w/Laronidase for Hurler - Full Text View

Sponsors and Collaborators:	University of Minnesota Masonic Cancer Center, University of Minnesota
Information provided by:	University of Minnesota
ClinicalTrials.gov Identifier:	NCT00176891

Purpose

We 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 GAG burden that is associated with decreased Hurler and transplant related pulmonary complications following transplant.

Condition	Intervention	Phase
Mucopolysaccharidosis I Hurler Syndrome	Procedure: Stem Cell Transplant Drug: Laronidase ERT	Phase II

Genetics Home Reference related topics: mucopolysaccharidosis type I

Drug Information available for: Laronidase

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title:	Phase II Study of Combined Laronidase (AldurazymeTM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH)

Further study details as provided by University of Minnesota:

Primary Outcome Measures:

Survival at one year and the proportion of patients in need of ventilator support by one year [ Time Frame: one year ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Donor engraftment over one year post transplant [ Time Frame: five years ] [ Designated as safety issue: No ]
Grade III-IV acute GvHD [ Time Frame: 5 years ] [ Designated as safety issue: No ]
Disease specific measurements: [ Time Frame: 5 years ] [ Designated as safety issue: No ]
(reduction in GAG, improvement in polysomnography) [ Time Frame: 5 years ] [ Designated as safety issue: No ]
Toxicity associated with infusions of enzyme [ Time Frame: 1 year ] [ Designated as safety issue: No ]
Development of anti-iduronidase antibodies in serum [ Time Frame: 5 years ] [ Designated as safety issue: No ]

Estimated Enrollment:	25
Study Start Date:	March 2004
Estimated Study Completion Date:	March 2012
Estimated Primary Completion Date:	March 2009 (Final data collection date for primary outcome measure)

Intervention Details:

enzyme replacement 10-12 weeks prior to HSCT, and 8 weeks following

10-12 weeks prior to HSCT, 8 weeks following

Detailed Description:

Subjects will receive laronidase once a week 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 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 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, we 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.

Eligibility

Ages Eligible for Study:	up to 7 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

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

Exclusion Criteria:

Not eligible for University of Minnesota myeloablative HSCT protocol. Refer to Appendix I.
Previous administration of laronidase enzyme
Second or subsequent HSCT.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00176891

Contacts

Contact: Paul Orchard, M.D.

612-626-2961

orcha001@umn.edu

Locations

United States, Minnesota
University of Minnesota Medical Center	Recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Paul Orchard, MD 612-626-2961 orcha001@umn.edu

Sponsors and Collaborators

University of Minnesota

Masonic Cancer Center, University of Minnesota

Investigators

Principal Investigator:

Paul Orchard, MD

University of Minnesota Medical Center

More Information

Responsible Party:	Masonic Cancer Center at the University of Minnesota ( Paul Orchard, M.D. )
Study ID Numbers:	0403M57728, MT2004-09
Study First Received:	September 12, 2005
Last Updated:	September 12, 2008
ClinicalTrials.gov Identifier:	NCT00176891
Health Authority:	United States: Institutional Review Board

Keywords provided by University of Minnesota:

Laronidase ERT
Stem cell transplant
storage disease
inborn errors of metabolism

hurler syndrome
glycosaminoglycans
enzyme replacement

Study placed in the following topic categories:

Alpha-L-iduronidase deficiency
Metabolism, Inborn Errors
Mucopolysaccharidoses
Metabolic Diseases
Genetic Diseases, Inborn
Lysosomal Storage Diseases

Connective Tissue Diseases
Mucopolysaccharidosis
Metabolic disorder
Hurler syndrome
Mucopolysaccharidosis I

Additional relevant MeSH terms:

Pathologic Processes
Disease
Syndrome
Mucinoses
Carbohydrate Metabolism, Inborn Errors

ClinicalTrials.gov processed this record on January 16, 2009