Comprehensive Multimodal Analysis of Neuroimmunological Diseases of the Central Nervous System
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00794352|
Recruitment Status : Recruiting
First Posted : November 20, 2008
Last Update Posted : September 14, 2020
Inflammatory or degenerative diseases of the brain and spinal cord, such as multiple sclerosis, may be related to problems with an individual s immune system. However, more information is needed on the ways in which the cells of the immune system interact with the central nervous system (CNS). This study will compare tests performed on both healthy volunteers and individuals who have signs or symptoms of immune-related damage to their CNS.
This study will include two groups of subjects at least 12 years old. Subjects will either have symptoms of immune-related CNS damage, or will be healthy volunteers selected for comparison purposes.
Study participants will visit the NIH Clinical Center on an outpatient basis for an initial evaluation visit. During the visit, patients will provide a comprehensive medical history and undergo a neurological examination, and will provide blood samples for research purposes. The healthy volunteers will be asked to schedule a return visit for a magnetic resonance imaging (MRI) procedure, and may be asked to undergo other tests requested by the study researchers on an as-needed basis. The group of patients with symptoms of immune-related CNS damage will be asked to undergo a series of tests, including the following:
- MRI procedures, with a minimum of three brain MRIs and one spinal cord MRI taken approximately 4 weeks apart
- A diagnostic lumbar puncture, performed on an outpatient basis
- Tests of brain and vision activity
- Additional blood and tissue samples
All study participants will return for a followup visit 1 year after the initial evaluation visit. Patients with symptoms of immune-related CNS damage may be offered the opportunity to participate in additional followup tests with NIH researchers.
|Condition or disease|
|Central Nervous System Disease Multiple Sclerosis|
Objective: The goal of this study is to define the pathophysiological mechanisms underlying the development of disability in immune-mediated disorders of the central nervous system (CNS) and to distinguish these from physiological (and often beneficial) responses of the human immune system to CNS injury. The long-term objective of the study is to acquire knowledge that would allow us to therapeutically inhibit the pathogenic mechanisms and enhance repair mechanisms in immune-mediated CNS diseases, thereby minimizing the extent of CNS tissue damage and promoting recovery.
The protocol also serves as a screening tool for NDU clinical trials and enables development of clinically-useful tools such as diagnostic tests and new, sensitive scales of neurological disability, disease severity and CNS tissue destruction.
Study Population: Patients with evidence, or suspicion of immune-mediated CNS injury will be enrolled. In addition, healthy volunteers will be included as controls for immunological and imaging and biomarkers and to obtain normative data for development of new clinical scales and smartphone apps.
Design: We will collect, in a standardized manner, multimodal data (standardized clinical/functional, neuroimaging and molecular/immunological data) during the diagnostic work-up of patients with varied disorders of the CNS in which immunemediated
processes are expected to play a pathophysiological role.
For the patient cohort, a comprehensive initial evaluation will be performed to establish a definitive diagnosis or confirm diagnosis and subtype of multiple sclerosis (MS) as a pre-requisite for enrollment into NDU clinical trials, but also to collect consistent multimodal research data. Additional diagnostic tests, tailored to individual patients, may be performed if required for the diagnostic process. The protocol stipulates a mandatory follow-up visit for all adult patients inclusive of clinical, functional and MRI examinations. Depending on the specific diagnosis, treatment decisions and clinical/research needs, patients may be offered additional follow-up visits. The maximum frequency of the follow-up visits and research samples to be collected is specified to ensure patient safety is not compromised. Patients age 12-17 may also be included in the patient cohort to establish a definitive diagnosis, or to provide non-standard assays to help with diagnostic and therapeutic decisions as part of extraordinary clinical care.
The volunteer cohort will provide sex and age-matched (to adult patients) normative values.
Additionally, we will also enroll patients from whom only biological samples collected by outside clinicians will be processed. This will be done to provide non-standard assays to help with diagnostic and therapeutic decisions as a part of extraordinary clinical care.
Outcome Measures: Clinical, MRI and immunological measures will be the outcome measures. However, no pre-defined research questions will be addressed other than to establish the diagnosis, determine the level of disease activity, and monitor the natural history.
|Study Type :||Observational|
|Estimated Enrollment :||1200 participants|
|Official Title:||Comprehensive Multimodal Analysis of Neuroimmunological Diseases of the CNS|
|Actual Study Start Date :||October 1, 2008|
Healthy patients with NO inflammatory and/or demyelinating/dysmyelinating diseases of theCN
Patients who present with CNS white matter injury (including inflammatory and/or demyelinating/dysmyelinating diseases of the CNS)
- Definite diagnosis of MS or another disorder. [ Time Frame: 12 weeks ]To identify MS-specific markers, biomarkers from peripheral blood and CSF will be compared between patients who fulfilled diagnostic criteria for MS versus those who were found to have alternative diagnoses.
- Disease progression as assessed by clinical and MRI criteria. [ Time Frame: 1-2 years ]1. Sustained (i.e. > 3 months) progression of disability as measured by greater than or equal to 0.5 CombiWISE (Kosa,Ghazali et al. 2016) points or2. Development of new /clearly enlarged distinct lesions on T2WI
- MRI measures of lesion load and CNS tissue destruction [ Time Frame: 12 weeks ]
- Immunological biomarkers [ Time Frame: 12 weeks ]
- Changes in clinical measures of disability from baseline [ Time Frame: 1 year ]
- Clinical measures of disability [ Time Frame: 12 weeks ]
- Changes in MRI measure of lesion load and CNS tissue destruction from baseline [ Time Frame: 1 year ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00794352
|Contact: Tiffany C Hauser||(301) firstname.lastname@example.org|
|Contact: Bibiana Bielekova, M.D.||(240) email@example.com|
|United States, Maryland|
|National Institutes of Health Clinical Center, 9000 Rockville Pike||Recruiting|
|Bethesda, Maryland, United States, 20892|
|Contact: For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR) 800-411-1222 ext TTY8664111010 firstname.lastname@example.org|
|Principal Investigator:||Bibiana Bielekova, M.D.||National Institute of Allergy and Infectious Diseases (NIAID)|