drugset / Trial / NCT00215527
Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I
Phase 1
Terminated
4 enrolled
Patricia I. Dickson, M.D.
FDA Office of Orphan Products Development · collabThe Ryan Foundation · collabUniversity of California, Los Angeles · collab
NaSingle-groupOpen-labelTreatment
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
Timeline
- Start
- 2005-11
- Primary completion
- 2011-10
- Completion
- 2011-10
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | laronidase | Protein / enzyme biologic | 1.74 mg | Intrathecal |