Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I

Stopped early · Phase 1

Conditions studied: Spinal Cord Compression, Mucopolysaccharidosis I, Hurler-Scheie Syndrome, Scheie Syndrome, Lysosomal Storage Disease

In brief

This is a one-year extension study of the use of laronidase into the spinal fluid to treat spinal cord compression in mucopolysaccharidosis I. Mucopolysaccharidosis I is a rare genetic condition due to deficiency of the enzyme alpha-l-iduronidase. Spinal cord compression occurs in this condition due to accumulation of material called glycosaminoglycans (GAG). Laronidase is the manufactured form of the enzyme alpha-l-iduronidase that is deficient in mucopolysaccharidosis I patients. The aim of this study is to determine whether laronidase is safe and effective when given into the spinal fluid as a potential non-surgical treatment for spinal cord compression due to mucopolysaccharidosis I disease. Funding Source -- FDA OOPD

Key facts

Study ID

NCT00786968

Run by

Patricia I. Dickson, M.D.

People needed

3

Starts

2008-01-01

Expected to finish

2011-10-01

Last updated by the study team

2013-02-21

Who can join

Age: 8 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Hurler-Scheie, Scheie form of MPS I, or Hurler 2 years after hematopoietic stem cell transplantation
• Spinal cord compression
• Has received intrathecal laronidase previously with good response and no significant safety concerns
• Age greater than 8 years
• Able to provide legal informed consent
• Aware of clinical treatment option of observation without treatment or surgical decompression
• Negative urine pregnancy test at screening (nonsterile females of child-bearing potential who are sexually active only)

You may not qualify if…

• Severe (Hurler) form of MPS I
• Desires surgical or medical treatment of spinal cord compression
• Spinal cord compression that warrants immediate surgical intervention
• Pregnancy or lactation
• Hematopoietic stem cell transplantation within 2 years of study enrollment
• Receipt of an investigational drug within 30 days of enrollment
• Infusion reactions to laronidase that required medical intervention, prophylaxis, or altered enzyme administration
• Significant anti-iduronidase antibody titer
• Recent initiation of intravenous laronidase (within past 6 months)
• Presence of cervical subluxation or similar external pathology as the major cause of cord compression symptoms for which surgical intervention should be immediately undertaken

Where it is running

• Los Angeles Biomedical Research Institute at Harbor-UCLA — Torrance, California, United States
• Helsinki University Central Hospital — Helsinki, Finland

Full record on ClinicalTrials.gov

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