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A Prospective Natural History Study of Patients With Syringomyelia

This study is currently recruiting participants. (see Contacts and Locations)
Verified January 2014 by National Institutes of Health Clinical Center (CC)
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute of Neurological Disorders and Stroke (NINDS) )
ClinicalTrials.gov Identifier:
NCT01150708
First received: June 24, 2010
Last updated: November 11, 2014
Last verified: January 2014
  Purpose

Background:

  • Syringomyelia is a disorder in which a cyst (syrinx) forms within the spinal cord and causes spinal cord injury, with symptoms worsening over many years, including paralysis, loss of sensation, and chronic pain. Researchers are interested in obtaining more knowledge about how a syrinx forms in order to develop safer and more effective treatments for syringomyelia and related conditions.
  • The goal of surgical treatment of syringomyelia is to eliminate the syrinx and prevent further spinal cord injury. In most patients, surgery results in the syrinx becoming smaller, but the effect of surgery on a patient s muscle strength, pain level, and overall function has not been studied over time. In addition, some individuals with syringomyelia or related conditions are not considered to be good candidates for surgery, and more information is needed about potential alternative treatments for these individuals.
  • By recording more than 5 years of symptoms, muscle strength, general level of functioning, and magnetic resonance imaging (MRI) scan findings from individuals who receive standard treatment for syringomyelia, researchers can obtain more information about factors that influence its development, progression, and relief of symptoms.

Objectives:

- To conduct a 5-year natural history study of individuals with syringomyelia and related conditions.

Eligibility:

- Individuals at least 18 years of age who have syringomyelia or related conditions (including pre-syringomyelia or Chiari I malformation without syringomyelia).

Design:

  • This study requires 7 outpatient visits to the National Institutes of Health Clinical Center: an initial visit; a visit 3 months later; and visits 1, 2, 3, 4, and 5 years after the initial visit. An additional 10 days of inpatient treatment and testing will be required if surgery is needed during the study.
  • The following tests will be performed during this study:
  • Medical history and physical examination, which may also determine eligibility for surgery
  • Detailed neurological history and examination
  • Blood and urine samples
  • MRI scans: Participants will have 2 scans at the initial evaluation, 2 scans at the 3-month visit, and 1 scan every year for the following 5 years.
  • Additional neurological and imaging tests if needed, including a lumbar puncture to collect spinal fluid, a myelogram (imaging study) of the spinal fluid, and a computed tomography scan of the skull and spine.
  • Participants who are surgical candidates will have additional tests along with the surgery, including diagnostic studies (electrocardiogram and chest X-ray) before surgery and an MRI scan 1 week after surgery.

Condition
Syringomyelia
Arnold Chiari Deformity

Study Type: Observational
Study Design: Time Perspective: Prospective
Official Title: A Prospective Natural History Study of Patients With Syringomyelia

Resource links provided by NLM:


Further study details as provided by National Institutes of Health Clinical Center (CC):

Estimated Enrollment: 120
Study Start Date: June 2010
Detailed Description:

OBJECTIVE

The natural history of patients who have syringomyelia has not been addressed in a prospective study. Present surgical treatment of patients with syringomyelia and neurologic deficit results in disease stabilization in many but not all patients, although objective improvement is less common. Delayed deterioration is not uncommon. The natural history of patients with syringomyelia and without a neurologic deficit or an associated lesion is also uncertain. By identifying factors that influence syringomyelia progression and that affect the response to surgical treatment, we can acquire knowledge that will enable us to provide more accurate recommendations to future patients with syringomyelia regarding optimal surgical or non-surgical treatment of their condition.

STUDY POPULATION

This study will enroll patients with syringomyelia and patients who are at risk of developing syringomyelia, including patients with Chiari I malformation and patients with presyringomyelia.

DESIGN

Prospective radiological and clinical data will be collected over a 5-year period from patients with syringomyelia and patients at risk of developing syringomyelia. Patients with syringomyelia and a neurological deficit will be considered to be surgical candidates. In this group, neurologic and radiographic outcomes in patients that undergo surgery will be compared to those in patients that refuse surgical treatment. Patients with syringomyelia but without neurological deficit will not be considered to be surgical candidates. In this group, initial neurologic and radiographic findings will be compared to those found one year after entrance into the study. Any patient with syringomyelia that develops progressive neurological deficit will be offered surgical treatment for his/her condition. In this study we will also prospectively collect information on processes that may influence the development and progression of syringomyelia, such as trauma and inflammatory diseases.

OUTCOME MEASURES

The primary outcome measure will be the change in motor strength, as measured by the American Spinal Injury (ASIA) Grading Scale, over 1 year. The secondary outcome measure will be the change in maximal syrinx diameter over 1 year in surgical candidates (surgically-treated vs. surgically untreated) and in non-surgical candidates.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria
  • INCLUSION CRITERIA:

To be eligible to participate in this research study, the subject must:

  1. Be 18 years of age or older.
  2. Have syringomyelia, presyringomyelia, or Chiari I malformation without syringomyelia.
  3. Be able to give informed consent.

SPECIFIC INCLUSION CRITERIA FOR PATIENTS THAT MAY UNDERGO SURGERY

  1. new or increased impairment in sensation, strength, or walking within the previous 2 years; and
  2. an MRI-scan showing a syrinx or the presence of abnormal fluid and swelling in the spinal cord; the abnormality in the spinal cord must be located in a part of the spinal cord that could produce the patient s symptoms; and
  3. evidence of abnormalities at the base of the skull or within the spine that are often associated with syringomyelia, or abnormalities in CSF circulation that increase the pressure of the cerebrospinal fluid; and
  4. medical health that is sufficient to allow the subject to withstand a major surgical procedure and to remain active during the recovery period after surgery.

EXCLUSION CRITERIA:

The subject will not be eligible to participate in this research study if the subject:

  1. Is pregnant (because multiple MRI scans might injure a fetus).
  2. Cannot have an MRI scan as determined by the radiologist (see the risks below).
  3. Has a problem with bleeding that cannot be corrected.

Note: Prior surgery for syringomyelia does not result in exclusion from the study if there is radiographic evidence of recurrent or persistent syringomyelia after surgery for this condition (failed previous surgery).

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01150708

Contacts
Contact: Gretchen C Scott, R.N. Not Listed SNBrecruiting@nih.gov

Locations
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 Patient Recruitment and Public Liaison Office (PRPL)    800-411-1222 ext TTY8664111010    prpl@mail.cc.nih.gov   
Sponsors and Collaborators
Investigators
Principal Investigator: John D Heiss, M.D. National Institute of Neurological Disorders and Stroke (NINDS)
  More Information

Additional Information:
Publications:
Responsible Party: National Institutes of Health Clinical Center (CC) ( National Institute of Neurological Disorders and Stroke (NINDS) )
ClinicalTrials.gov Identifier: NCT01150708     History of Changes
Other Study ID Numbers: 100143, 10-N-0143
Study First Received: June 24, 2010
Last Updated: November 11, 2014
Health Authority: United States: Federal Government

Keywords provided by National Institutes of Health Clinical Center (CC):
Syringomyelia
Magnetic Resonance Imaging
Natural History Study
Neurological Examination

Additional relevant MeSH terms:
Arnold-Chiari Malformation
Syringomyelia
Central Nervous System Diseases
Congenital Abnormalities
Nervous System Diseases
Nervous System Malformations
Neural Tube Defects
Spinal Cord Diseases

ClinicalTrials.gov processed this record on November 24, 2014