AT RISK FOR MS - Clinical Conversion of Female Monozygotic Twins Discordant for CIS/MS

NCT00617383 | Completed

• 2 other identifiers: HSC-MS-07-0327NMSS Pilot grant PP1464
• Study Type: observational
• Target: 3
• Locations: 1 country
• Sites: 1

Brief Summary

The definition of the most 'at-risk' population within highly susceptible groups would provide an opportunity for preemptive therapeutics. A convenient, safe, and tolerable therapy that delays the onset of clinical disease during the pre-symptomatic stage of demyelinating disease would provide a therapeutic alternative to a 'wait and see' approach in subjects at 'high risk' for CIS (clinically isolated syndrome - monosymptomatic demyelinating disease) or MS. Identical twins share the same genes and have the highest rate of shared MS. An identical female with a sister twin with MS has a 34% chance of having MS. Non concordant (no MS yet) identical (monozygotic - from the same sperm-egg zygote) female twins provide an ideal population to find out what factors predict the onset of MS in the non-affected twin. We will recruit 30 identical female twins, one with MS and the other without MS, and obtain brain MRI and biological samples on the non-affected twin and determine if:

• the presence of characteristic MS-like lesion(s) on baseline MRI predisposes to MS.
• specific proteins in blood or cerebrospinal fluid predispose to the clinical expression of demyelinating disease If we can predict by simple tests (MR brain scan and blood tests) the likelihood of the onset of MS in 'at risk' subjects, and have safe and tolerable therapies, we may be able to prevent the clinical onset of demyelinating disease (MS).

Conditions

Multiple Sclerosis; Clinically Isolated Syndrome

Keywords

demyelinating disease; multiple sclerosis; female monozygotic identical twins; MS; non-concordant; clinically isolated syndrome - CIS; brain MRI; proteomics

Outcome Measures

Primary Outcomes (1)

• Determine if the presence of characteristic MS-like lesion(s) on baseline MRI predisposes to CIS/MS in female MZ twins discordant for CIS/MS.

  5 years or exit from study

Secondary Outcomes (1)

• Define the protein and microarray gene expression profile predictive of conversion to MS/CIS in female MZ twins discordant for CIS/MS.

  5 years or clinical conversion to MS

Eligibility Criteria

• Age: 10 Years - 45 Years
• Sex: female
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

female monozygotic twins discordant for CIS/MS

You may qualify if:

• 'at risk' individuals for MS - female co-twins discordant for CIS/MS.
• 'at risk' individuals for MS who at the time of randomization have not converted to MS or CIS.
• 'at risk' individuals will be treatment-naïve for immunomodulatory/suppressive medications.
• \< 46 years old.

You may not qualify if:

• Individuals diagnosed with MS or CIS.
• Other 'at risk' individuals who do not conform to the specific 'at risk' groups outlined above e.g., 1st degree, 2nd degree and 3rd degree relative of MS index cases.
• Subjects over 45.
• Use of immunomodulatory medications such as azathioprine, gold, sulfasalazine, minocycline, statins, and MTX or prednisone \> 7.5 mg/day within 30 days of randomization for any reason.
• Active drug use or dependence that, in the opinion of the site investigator, would interfere with adherence to study requirements or a psychiatric disorder that is unstable or would preclude reliable participation in the study.
• Serious illness (requiring systemic treatment and/or hospitalization) such as diabetes mellitus, renal, cardiac, or pulmonary disease. Subjects with a history of alcoholism, or in whom intellectual functioning is impaired sufficiently to interfere with the understanding of the protocol, or participation in the treatment and evaluation program.

Sponsors & Collaborators

• The University of Texas Health Science Center, Houston: lead
• National Multiple Sclerosis Society: collaborator

Study Sites (1)

University of Texas - Houston

Houston, Texas, 77030, United States

Location

Biospecimen

Retention: SAMPLES WITHOUT DNA

blood for serum and peripheral mononuclear cells; optional CSF - cerebrospinal fluid at entry and exit from trail

MeSH Terms

Conditions

Multiple Sclerosis; Demyelinating Diseases

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Staley A Brod, MD

  University of Texas

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 5, 2008
• First Posted: February 18, 2008
• Study Start: February 1, 2008
• Primary Completion: December 1, 2010
• Study Completion: December 1, 2010
• Last Updated: April 4, 2017

Record last verified: 2012-02

Locations

US (1)