Clinical trial NCT01737372: A Pilot Study to Assess microRNA Biomarkers in Early and Later Stage Multiple Sclerosis
Official title: A Pilot Study to Assess microRNA Biomarkers in Early and Later Stage Multiple Sclerosis
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Multiple Sclerosis
Secondary Progressive Multiple Sclerosis
Clinically Isolated Syndrome
Keywords: Multiple Sclerosis
Secondary Progressive Multiple Sclerosis
Clinically Isolated Syndrome
Lead sponsor: Rocky Mountain MS Research Group, LLC
Sponsor class: OTHER
Collaborators: name: Genzyme, a Sanofi Company
First posted: 2012-11-29
Enrollment: 49
Sex: ALL
Minimum age (years): 18
Maximum age (years): 68
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: 45 participants will be enrolled into a study to examine the microRNA and cell products profile in both cerebrospinal fluid (CSF) and blood. Study population will consist of a total of: 20 CIS patients defined as patients having a single attack (or the appearance of one or more symptoms characteristic of MS) high risk of developing MS, when no other diseases or causes are apparent.

20 SPMS patients 5 normal, non-diseased controls

Brief summary: This study will look at the blood and cerebrospinal fluid of consented participants who either have early stage multiple sclerosis (clinically isolated syndrome) or who have later stage (secondary progressive multiple sclerosis), or participants who do not have any neurological or autoimmune illness. Biomarkers and microRNA will be assessed for group differences.

Detailed description: Multiple Sclerosis is a chronic autoimmune disorder of the central nervous system. While there are current immunomodulatory therapies that have been shown to be efficacious in the early stages of MS, these therapies are less potent in the later phases of MS. We can look at magnetic resonance imaging with gadolinium to measure active disease but it does not detect axonal degeneration. Therefore, there is a need to identify other biomarkers that may be used to diagnose MS and predict disease progression. Biomarkers found in the cerebrospinal fluid (CSF) are in closer proximity to the inflammatory lesion sites and are more sensitive.

This pilot study seeks to characterize differences in microRNA profiles and cell products in the early and later stages of MS, with the hope that that microRNA profiles could then be correlated to clinical and CSF inflammation indexes. CSF will be obtained from 45 participants.

Eligibility criteria: Inclusion Criteria:

* able to understand and agree to informed consent;
* male or female patients 18-68 years of age
* no disease modifying therapy 60 days prior to Baseline
* EDSS score of less than or equal to 3.0 if a CIS patient; or less than or equal to 7.5 if SPMS patient
* Labs within normal range no greater than 2 times the ULN or at the discretion of the PI
* weight 46 kilograms to 127 kilograms inclusive
* no active systemic infection
* not currently pregnant or breast feeding
* no history of corticosteroid treatment or relapse within 60 days prior to Baseline.

Exclusion Criteria:

* not able to understand informed consent
* if any of the inclusion criteria is not met
* HIV infection, or current active Hepatitis B or C infection, positive HCVAb or HBsAG or HBcAB
* positive pregnancy test
* patient withdraws consent
* Coumadin use within 60 days prior to Baseline

Primary outcomes:
measure: To characterize differences in microRNA profile and cell product patterns between early and later stage multiple sclerosis.
timeFrame: baseline
Secondary outcomes:
measure: Correlate microRNA profiles with clinical and CSF inflammation indexes
timeFrame: baseline
Other outcomes:
Locations:
city: Salt Lake City
country: United States
facility: Rocky Mountain MS Clinic
state: Utah
Study officials:
affiliation: Rocky Mountain MS Research Group, LLC
name: John F Foley, MD
role: PRINCIPAL_INVESTIGATOR
References: