Ignite Creation Date:
2024-05-05 @ 12:06 PM
Last Modification Date:
2024-10-26 @ 9:20 AM
Study NCT ID:
NCT00244361
Status:
COMPLETED
Last Update Posted:
2011-05-10
First Post:
2005-10-24
Brief Title:
Effectiveness of Rituximab in Pediatric OMS Patients
Sponsor:
National Pediatric Neuroinflammation Organization Inc
Organization:
National Pediatric Neuroinflammation Organization Inc
Study Overview
Official Title:
A Phase I Clinical Trial of Rituximab for Pediatric Opsoclonus-Myoclonus Syndrome
Status:
COMPLETED
Status Verified Date:
2011-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of this study is to reduce the symptoms of OMS by testing rituximab Rituxan to remove B lymphocytes that make antibodies and trigger brain inflammation Evidence suggests that autoimmune brain inflammation causes the symptoms of OMS This study of blood and spinal fluid intends to find out what effect rituximab has on OMS and on the spinal fluid B-cells
Rituximab targets and destroys B-cells which make antibodies that can attack the brain and cause may OMS It is infused through a vein over a period of several hours Rituximab has been used widely and studied extensively since its approval in 1997 by the US Food and Drug Administration FDA for non-Hodgkins B-cell Lymphoma NHL Today more than 300000 patients have received rituximab and it is part of more than 200 completed ongoing or planned clinical trials Rituximab is not FDA-approved for OMS
Rituximab targets and destroys B-cells which make antibodies that can attack the brain and cause may OMS It is infused through a vein over a period of several hours Rituximab has been used widely and studied extensively since its approval in 1997 by the US Food and Drug Administration FDA for non-Hodgkins B-cell Lymphoma NHL Today more than 300000 patients have received rituximab and it is part of more than 200 completed ongoing or planned clinical trials Rituximab is not FDA-approved for OMS
Detailed Description:
Opsoclonus-myoclonus syndrome OMS is a rare but pervasive paraneoplastic neurological disorder purported to be autoantibody-mediated We demonstrated expansion of B-cells in cerebrospinal fluid CSF despite tumor resection chemotherapy or conventional immunotherapy Whether B-cells can be purged from the CSF compartment with benefit to the patient is unknown Targeting of CSF B lymphocytes represents a novel and valuable paradigm shift in the therapy of centrally-mediated paraneoplastic disorders The objective of this preliminary study is to determine if rituximab a monoclonal antibody against CD20 B-cells reduces or eliminates CSF B-cells in OMS and whether the reduction results in clinical improvement B lymphocyte subsets and relevant T-cell subsets will be immunophenotyped in the CSF and peripheral blood of children with OMS by four-color dual-laser flow cytometry Sixteen children with an increased percentage of CSF B-cells will be treated with rituximab 375 mgm2 IV once weekly for four consecutive weeks and CSF testing will be repeated at six months with more frequent clinical evaluations and blood testing out to 12 months Clinical outcome will be rated blindly from videotapes by an experienced observer using a validated 12-item motor evaluation scale and quantifiable parameters of sleep behavior and motor function Immunological outcome variables will include percentages of B-cell subsets and quantitative immunoglobulins Post-treatment results will be compared to pre-treatment values statistically If rituximab proves to be an efficacious and safe method of treating CSF B-cell expansion and the neurological syndrome this study will lead to a phase II trial with the eventual aim of gaining FDA approval of rituximab for this indication
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 |
|---|---|---|---|
| SCRIHS 04-112 | None | None | None |