Clinical trial NCT01396343: Environmental and Genetic Risk Factors for Pediatric Multiple Sclerosis
Official title: Environmental and Genetic Risk Factors for Pediatric Multiple Sclerosis
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Pediatric Multiple Sclerosis
Keywords: Pediatric Multiple Sclerosis
Relapsing Remitting Multiple Sclerosis
Clinically Isolated Syndrome
Demyelinating Disease
Lead sponsor: University of California, San Francisco
Sponsor class: OTHER
Collaborators: name: New York University
name: Massachusetts General Hospital
name: State University of New York at Buffalo
name: University of Alabama at Birmingham
name: Mayo Clinic
name: Children's Hospital of Philadelphia
name: Boston Children's Hospital
name: Baylor College of Medicine
name: Loma Linda University
name: Children's Hospital Colorado
name: University of Texas
name: Ann & Robert H Lurie Children's Hospital of Chicago
name: Washington University School of Medicine
name: Children's National Med Center
name: Primary Children's Hospital
name: The Cleveland Clinic
First posted: 2011-07-18
Enrollment: 1276
Sex: ALL
Minimum age (years): 3
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: yes
Observational model: CASE_CONTROL
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Case patients seen at the 16 participating Pediatric MS Center Clinics. Control patients seen at the Pediatric Clinics of the same institution as MS cases.

Brief summary: The purpose of this study is to better understand multiple sclerosis (MS) in children and adolescents, to learn if it differs from adult MS and to investigate if genes or environmental exposures or a combination of both put children and adolescents at risk for getting MS.

Detailed description: The overall goal of this project is to determine whether well-established environmental and genetic risk factors for adult onset MS play an important role in susceptibility to pediatric-onset MS. Our study design is based on the hypothesis that genetic influences, specifically variation at HLA-DRB1 and other confirmed non-MHC MS loci, as well as environmental exposures including EBV infection and tobacco smoke, contribute to disease risk. In addition, we will also examine the relationship between serum levels of 25(OH) vitamin D3 and prior vitamin D status, and risk for pediatric onset MS. Finally, we will investigate whether specific G x E, and other multivariable relationships influencing risk exist for pediatric-onset MS. There are 16 collaborating sites other than UCSF that will enroll cases and controls for this study.

Eligibility criteria: Children are eligible for this study as cases if:

* They have MS or clinically isolated syndrome (CIS):

  * MS: As defined by the 2010 McDonald criteria for diagnosis of MS (Polman 2010),
  * CIS: A first demyelinating event indicating high risk for MS (i.e., one clinical event involving the spinal cord, the optic nerve, the brainstem or cerebellum, or occasionally the hemispheres) and at least 2 silent T2 bright areas on a brain or spinal cord MRI (at least one must be in the brain); AND
* They are three years of age or older; AND
* Disease onset occurred before 18 years of age.

Patients are not eligible for study participation if:

* Disease onset occurred more than 4 years prior to the opportunity to enroll; OR
* They have had an organ transplant; OR
* They are known to have neuromyelitis optica (NMO).

Children are not eligible to participate as pediatric controls if:

* They are two years of age or younger; OR
* They are 22 years of age or older; OR
* They are known to have MS or another demyelinating disease (for example, neuromyelitis optica or acute disseminated encephalomyelitis); OR
* They have a biological family member who has been enrolled as a control; OR
* They have an immediate, biological family member (parent/sibling) who has been diagnosed with MS; OR
* They have an autoimmune disorder (except asthma or eczema); OR
* They have had an organ transplant; OR
* They have a chronic neurological condition with major disability (this does not include, for example, migraine, controlled seizures, and mild learning disabilities such as ADD or ADHD).

Primary outcomes:
measure: Identify risk factors and their respective contribution to developing pediatric multiple sclerosis
timeFrame: 4 year data collection, 1 year analysis
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Center for pediatric-onset demyelinating diseases at the Children's Hospital of Alabama, Birmingham
state: Alabama
city: San Bernardino
country: United States
facility: Pediatric MS Clinic, Children's Hospital, Loma Linda University
state: California
city: San Francisco
country: United States
facility: UCSF Pediatric MS Center
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado, University of Colorado School of Medicine
state: Colorado
city: Washington
country: United States
facility: Pediatric MS Clinic, Children's National Medical Center
state: District of Columbia
city: Chicago
country: United States
facility: Pediatric MS Clinic, Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: Boston
country: United States
facility: Partners Pediatric MS Center at the Massachusetts General Hospital for Children
state: Massachusetts
city: Boston
country: United States
facility: Pediatric MS Clinic, Children's Hospital Boston
state: Massachusetts
city: Rochester
country: United States
facility: Regional Pediatric MS Center at Mayo Clinic
state: Minnesota
city: Saint Louis
country: United States
facility: Pediatric MS Clinic, Washington University School of Medicine
state: Missouri
city: Buffalo
country: United States
facility: Pediatric MS Center of the Jacobs Neurological Institute, University of Buffalo
state: New York
city: New York
country: United States
facility: New York University Langone Medical Center
state: New York
city: Cleveland
country: United States
facility: The Cleveland Clinic
state: Ohio
city: Philadelphia
country: United States
facility: Pediatric MS Clinic, Children's Hospital of Philadelphia
state: Pennsylvania
city: Dallas
country: United States
facility: Pediatric MS Center, University of Texas, Southwestern Medical Center
state: Texas
city: Houston
country: United States
facility: The Blue Bird Circle Clinic for MS at Texas Children's Hospital
state: Texas
city: Salt Lake City
country: United States
facility: Pediatric Neurology Clinic, Primary Children's Hospital
state: Utah
Study officials:
affiliation: University of California, San Francisco
name: Emmanuelle L Waubant, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: