Ignite Creation Date:
2024-05-05 @ 11:30 AM
Last Modification Date:
2024-10-26 @ 9:09 AM
Study NCT ID:
NCT00069329
Status:
TERMINATED
Last Update Posted:
2016-12-01
First Post:
2003-09-22
Brief Title:
Anakinra to Treat Patients With Neonatal Onset Multisystem Inflammatory Disease
Sponsor:
National Institute of Arthritis and Musculoskeletal and Skin Diseases NIAMS
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
A Long-Term Outcome Study With the IL-1 Receptor Antagonist AnakinraKineret in Patients With Neonatal Onset Multisystem Inflammatory Disease NOMIDCINCA Syndrome A Therapeutic Approach to Study the Pathogenesis of This Disease
Status:
TERMINATED
Status Verified Date:
2016-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
data submitted for Food and Drug Administration FDA approval
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This study will evaluate the safety and effectiveness of anakinra Kineret for treating patients with neonatal-onset multisystem inflammatory disease NOMID also known as chronic infantile neurological cutaneous and arthropathy CINCA syndrome This disease can cause rash joint deformities brain inflammation eye problems and learning difficulties Immune suppressing medicines commonly used to treat other pediatric rheumatologic diseases do not suppress NOMID symptoms and if used long-term and in high doses can cause harmful side effects Anakinra approved by The Food and Drug Administration for treating rheumatoid arthritis in adults blocks a substance called IL-1 that may be an important factor in causing the inflammation in NOMID
Detailed Description:
This study uses the IL-1 receptor antagonist anakinra to treat children and adults with Neonatal-Onset Multisystem Inflammatory Disease NOMID also known as chronic infantile neurological cutaneous and arthropathy CINCA syndrome NOMIDCINCA syndrome is a rare genetic systemic auto-inflammatory disease that is characterized by a triad of symptoms including a persistent urticaria-like skin rash an arthropathy associated with patellar and epiphyseal osseous overgrowth and neurological manifestations including chronic aseptic meningitis optic disc edema high frequency hearing loss and mental retardation Spontaneous genetic mutations in the NACHT domain of CIAS1 a gene located on chromosome 1 have been recently identified in about half of the patients with NOMIDCINCA syndrome CIAS1 encodes a protein cryopyrin that is associated with up-regulation of IL-1 production in vitro which has formed the rationale to target the IL-1 pathway in children with NOMID During an up to 3- week enrollment period before initiating therapy we will collect selfparent reported daily diary data and serological samples on up to 3 occasions one week apart to determine baseline disease activity These data may be gathered by collaborating centers At the end of the observation period patients will be admitted to the NIH for a standardized clinical evaluation and initiation of treatment with anakinra administered at 1 mgkgday by regular daily subcutaneous injections If patients do not fulfill improvement criteria at 1 month the dose will be escalated between 05 and 1 mgkgday increments to obtain inflammatory remission An initial withdrawal study in a subset of 11 patients was performed The clinical improvement at 3-4 months and the change in serum amyloid A levels SAA a sensitive inflammatory marker from before treatment to 3-4 months post treatment and drug safety are the primary clinical outcomes of this study To assess long-term safety and efficacy all patients will be observed during an open ended extension phase of the study Clinical and laboratory parameters will be used to assess safety and efficacy throughout the trial All patients will be seen every 6 months and annually as calculated from initiation of anakinra treatment to further evaluate safety and long term outcomes During the open ended extension phase of the study patients who have residual clinical or laboratory evidence of active inflammation may have their dose increased between 05 and 1 mgkgday increments to a maximum dose of 10 mgkgper day to achieve clinical remission In addition since no data on the pharmacokinetics PK of anakinra in pediatric patients is available with doses exceeding 2 mgkgday we plan to determine the PK of anakinra with each dose escalation
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 |
|---|---|---|---|
| 03-AR-0298 | OTHER | NIHCC | httpsreporternihgovquickSearchZIAAR041138-08 |
| ZIAAR041138-08 | NIH | None | None |
| NCT00069329 | OTHER | None | None |