Ignite Creation Date:
2024-05-05 @ 12:02 PM
Last Modification Date:
2024-10-26 @ 9:18 AM
Study NCT ID:
NCT00215527
Status:
TERMINATED
Last Update Posted:
2013-02-21
First Post:
2005-09-19
Brief Title:
Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis MPS I
Sponsor:
Patricia I Dickson MD
Organization:
Dickson Patricia I MD
Study Overview
Official Title:
A Study of Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis I
Status:
TERMINATED
Status Verified Date:
2013-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Due to slow enrolment
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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
Detailed Description:
Enzyme replacement therapy ERT has been developed for mucopolysaccharidosis I MPS I a lysosomal storage disorder ERT helps many physical ailments due to the disease but does not treat the central nervous system due to inability to cross the blood brain barrier Our purpose is to test delivery of ERT to the spinal fluid via intrathecal injection in patients with MPS I In this pilot study we will use recombinant human α-L-iduronidase administered intrathecally once per month for four months to individuals with the Hurler-Scheie and Scheie forms of MPS I and spinal cord compression If successful intrathecal delivery could represent a practical straightforward method of treating central nervous system disease due to lysosomal storage
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 12069-01 | OTHER | Los Angeles Biomedical Research Institute at Harbor-UCLA | None |