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Completed

NCT Number: NCT00638547

Intrathecal Enzyme Replacement for Hurler Syndrome

This protocol will examine whether the enzyme alpha-L-iduronidase (Laronidase), delivered into the spinal fluid of patients with Hurler syndrome at intervals before and after bone marrow transplant, is a safe and effective approach to slow the neurologic degeneration seen in Hurler patients undergoing transplantation.

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Key information

Conditions

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

Age range

6 month–3 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

University of Minnesota, Fairview

Minneapolis, Minnesota, 55455, United States

About this study

Subjects will receive an infusion of Laronidase into his/her spinal fluid approximately 12 weeks before, 2 weeks before, 100 days after and 6 months after transplant. This procedure is done by lumbar puncture (also called a "spinal tap").

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Patients with a diagnosis of MPS IH (Hurler syndrome) are candidates for this protocol if they are being considered for hematopoietic stem cell transplantation according the University of Minnesota guidelines.

Exclusion criteria

  • Patients are less than 6 months old, or older than 3 years of age.
  • There is a history of clinically-severe hypersensitivity to Laronidase.
  • There is a contraindication for repeated lumbar puncture.
  • The family is not willing to undergo the necessary procedures and evaluations inherent in the study.
  • Consent has not been signed for participation in the 2004-09 study of intravenous Laronidase administration.

Treatment and study plan

IRT Laronidase

Drug

Laronidase belongs to a class of drugs called enzyme replacement therapies or ERT that provides people with sufficient quantities of an important enzyme that they cannot create on their own. The main ingredient in laronidase is a protein that is identical to a naturally occurring form of the human enzyme alpha-L-iduronidase. Laronidase replaces the missing enzyme alpha-L-iduronidase and restores sufficient enzyme activity to break down GAG buildup.

Subjects will receive an infusion of Laronidase into his/her spinal fluid approximately 12 weeks before, 2 weeks before, 100 days after and 6 months after transplant. This procedure is done by lumbar puncture

Other names: Aldurazyme

Primary outcomes

  1. To demonstrate the efficacy of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I in decreasing neurodevelopmental deterioration

    Time frame: 1 year

Secondary outcomes

  1. To determine the safety and toxicity of intrathecally delivering alpha-L-iduronidase in patients with mucopolysaccharidosis type I

    Time frame: 1 year

  2. To determine brain changes with magnetic resonance imaging

    Time frame: 1 and 2 years

  3. To determine neurocognitive changes present in patients with Hurler syndrome

    Time frame: 6, 12, and 24 months

  4. To determine cerebral spinal fluid levels of glycosaminoglycans, cytokines and antibodies to Laronidase at baseline and at each point CSF is obtained

    Time frame: through 1 year

Sponsors and collaborators

Lead sponsor

Masonic Cancer Center, University of Minnesota

Other

Registry information

Official study title

Intrathecal Enzyme Replacement Therapy For Patients With Mucopolysaccharidosis Type I (Hurler Syndrome)

Important dates

Study start
2008
Primary completion
2017
Study completion
2018
First posted
Mar 19, 2008
Registry last updated
Dec 3, 2018

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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