Overview

Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I

Status:
Terminated
Trial end date:
2011-10-01
Target enrollment:
0
Participant gender:
All
Summary
The investigators are studying the use of enzyme replacement therapy into the spinal fluid for treatment of spinal cord compression in the Hurler-Scheie and Scheie forms of mucopolysaccharidosis I (MPS I). Funding source -- FDA OOPD
Phase:
Phase 1
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Patricia I. Dickson, M.D.
Collaborators:
FDA Office of Orphan Products Development
The Ryan Foundation
University of California, Los Angeles
Criteria
Inclusion Criteria:

- Hurler-Scheie,Scheie form of MPS I, of Hurler 2 years after hematopoietic stem cell
transplantation

- Spinal cord compression

- 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 (non-sterile females of child-bearing
potential only)

- Currently using two acceptable methods of birth control (non-sterile females of
child-bearing potential who are sexually active only)

- Willing and able to comply with study procedures

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