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Completed

NCT Number: NCT00176891

Stem Cell Transplant w/Laronidase for Hurler

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.

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Key information

Conditions

Mucopolysaccharidosis I Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Hurler Syndrome Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Nutritional and Metabolic Diseases Skin and Connective Tissue Diseases

Age range

Up to 7 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Masonic Cancer Center, University of Minnesota

Minneapolis, Minnesota, 55455, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Stem Cell Transplant

Procedure

enzyme replacement 10-12 weeks prior to hematopoietic stem cell transplant (HSCT), and 8 weeks following

Other names: ERT

Laronidase ERT

Drug

Laronidase ERT will be administered 10-12 weeks prior to HSCT, and 8 weeks following.

Other names: Aldurazyme®

Primary outcomes

  1. Number of Patients Alive at One Year Post Transplant

    Time frame: one year

  2. Number of Patients Requiring Ventilator Support at One Year Post Transplant

    Time frame: one year

Secondary outcomes

  1. Donor Engraftment

    Time frame: Day 100 post transplant

  2. Patients With Grade III-IV Acute GVHD

    Time frame: Day 100 post transplant

  3. Reduction in Glycosaminoglycans (GAG)

    Time frame: Prior to, During and After ERT

    Data was not collected on this outcome measure and is not available for reporting.

  4. Toxicity (Adverse Events) Associated With Infusions of Laronidase

    Time frame: 1 year post transplant

    Data was not collected on this outcome measure and is not available for reporting.

  5. Development of Anti-iduronidase Antibodies in Serum

    Time frame: 1 Year

  6. Patients With Improvement in Obstructive Apnea (Breathing) by Polysomnography

    Time frame: Baseline, 12 weeks after laronidase, after transplant

Sponsors and collaborators

Lead sponsor

Masonic Cancer Center, University of Minnesota

Other

Registry information

Official study title

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

Important dates

Study start
2004
Primary completion
2012
Study completion
2012
First posted
Sep 15, 2005
Registry last updated
Jul 11, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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