Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I

• Status: Terminated
• Trial end date: 2011-10-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Patricia I. Dickson, M.D.
• Collaborator: The Ryan Foundation

Criteria

Inclusion Criteria:

• 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)

Exclusion Criteria:

• 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