Ignite Creation Date:
2024-05-05 @ 11:25 AM
Last Modification Date:
2024-10-26 @ 9:07 AM
Study NCT ID:
NCT00030212
Status:
COMPLETED
Last Update Posted:
2017-07-02
First Post:
2002-02-07
Brief Title:
Immune Abnormalities in Sporadic Inclusion Body Myositis
Sponsor:
National Institute of Neurological Disorders and Stroke NINDS
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Study of Immune Dysregulation in Patients With Sporadic Inclusion Body Myositis s-IBM
Status:
COMPLETED
Status Verified Date:
2007-12-10
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:
This study will examine the abnormal immune response in patients with sporadic inclusion body myositis s-IBM-the most common inflammatory muscle disease in people over the age of 50 s-IBM progresses steadily and may lead to severe weakness and wasting of arm and leg muscles Patients may become unable to perform daily living activities and be confined to wheelchairs s-IBM is thought to be an autoimmune disease in which the bodys own immune system attacks healthy muscles This study will explore the causes of the muscle tissue inflammation that is responsible for destruction of muscle fibers and weakness in this disease Information from the study may help in the development of an effective treatment for this disease
Patients with s-IBM may be eligible for this study Those who are unable to travel or who have severe cardiovascular renal or other end-stage organ disease will be excluded Candidates will be screened for eligibility with a medical history and physical and neurological examinations
Participants will be seen at the NIH Clinical Center every six months over a 12-month period visits at enrollment 6 months and 12 months either on an inpatient or outpatient basis depending on their disease severity Each 2- to 3-day visit will involve the following tests and evaluations
Blood samples for routine laboratory tests are collected at every visit Additional blood for research studies is collected at 12 months
Quantitative muscle strength testing is done at every visit The patient pulls against straps connected to dynamometers devices that measure muscle power to evaluate strength of the main muscle groups in the arms and legs
Lymphapheresis is done at enrollment and at 12 months This is a procedure for collecting quantities of lymphocytes white blood cells that are an important part of the immune system Blood is collected through a needle placed in an arm vein and circulated through a machine that spins it separating it into its components The lymphocytes are removed and the rest of the blood red cells platelets and plasma is returned to the body through the same needle or another needle placed in the other arm
Electrophysiologic studies electromyography and nerve conduction testing are done at enrollment and 12 months Electromyography evaluates the electrical activity of muscles A small needle is inserted into the muscle and the patient is asked to relax or to contract the muscle For nerve conduction testing nerves are stimulated by electrodes small wires taped to the skin over the muscle
Muscle biopsy is done at enrollment and 12 months A sample of muscle tissue about the size of a lima bean from an arm or leg is surgically removed to confirm the diagnosis of s-IBM and for analysis of proteins involved in the muscle inflammation process A local anesthetic is used to numb the area before the surgery and the wound is closed with stitches
Patients with s-IBM may be eligible for this study Those who are unable to travel or who have severe cardiovascular renal or other end-stage organ disease will be excluded Candidates will be screened for eligibility with a medical history and physical and neurological examinations
Participants will be seen at the NIH Clinical Center every six months over a 12-month period visits at enrollment 6 months and 12 months either on an inpatient or outpatient basis depending on their disease severity Each 2- to 3-day visit will involve the following tests and evaluations
Blood samples for routine laboratory tests are collected at every visit Additional blood for research studies is collected at 12 months
Quantitative muscle strength testing is done at every visit The patient pulls against straps connected to dynamometers devices that measure muscle power to evaluate strength of the main muscle groups in the arms and legs
Lymphapheresis is done at enrollment and at 12 months This is a procedure for collecting quantities of lymphocytes white blood cells that are an important part of the immune system Blood is collected through a needle placed in an arm vein and circulated through a machine that spins it separating it into its components The lymphocytes are removed and the rest of the blood red cells platelets and plasma is returned to the body through the same needle or another needle placed in the other arm
Electrophysiologic studies electromyography and nerve conduction testing are done at enrollment and 12 months Electromyography evaluates the electrical activity of muscles A small needle is inserted into the muscle and the patient is asked to relax or to contract the muscle For nerve conduction testing nerves are stimulated by electrodes small wires taped to the skin over the muscle
Muscle biopsy is done at enrollment and 12 months A sample of muscle tissue about the size of a lima bean from an arm or leg is surgically removed to confirm the diagnosis of s-IBM and for analysis of proteins involved in the muscle inflammation process A local anesthetic is used to numb the area before the surgery and the wound is closed with stitches
Detailed Description:
Sporadic Inclusion Body Myositis s-IBM is the most common acquired myopathy above the age of 50 years It is a disabling disease that leads to wheelchair confinement The cause is unknown but autoimmune mechanisms have been implicated The primary goal of the study is to search for candidate autoantigens that drive the autoimmune response and identify candidate pathogenic T-cell epitopes among the endomysial T-cells extracted from the patients muscle biopsies It is anticipated that the search for the antigens implicated in the clonal expansion of endomysial T-cells over time will elucidate the autoimmune pathogenesis of the disease and lead to the development of antigen-specific immunomodulatory therapeutic strategies This is an investigative study intended to better define the pathogenesis of s-IBM No new therapy will be provided except of standard care
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 |
|---|---|---|---|
| 02-N-0121 | None | None | None |