Clinical Research Directory

A Prospective Natural History Study of Patients With Syringomyelia

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.

Extracellular Vesicles for the Treatment of Syringomyelia

This is a open-label, single-arm, dose escalation phase I clinical trial. The goal of this clinical trial is to evaluate the safety and preliminary efficacy of Intrathecal injection human umbilical cord-derived mesenchymal stromal cell-derived extracellular vesicle (hUC-MSC-sEV) in syringomyelia.

Subarachnoid-Subarachnoid (S-S) Bypass Versus Adhesion Lysis in Spinal Arachnoiditis and Syringomyelia

To determine whether Subarachnoid-Subarachnoid (S-S) Bypass results in better patient outcomes with fewer complications and improved quality of life compared to intradural adhesion lysis in individuals with Spinal Arachnoiditis and Syringomyelia.

The Therapeutic Effect of Betaine in Syringomyelia

Purpose: This clinical trial aims to evaluate the indications, therapeutic effects and side effects of betaine in refractory syringomyelia. Primary outcome measure: The primary endpoint is the change of ASIA at week 12. The clinical efficacy is defined as ASIA increase ≥ 1 at week 12, as compared with that before betaine usage.

The Therapeutic Effect of Thalidomide in Syringomyelia

Purpose: This phase II clinical trial aims to evaluate the indications, therapeutic effects and side effects of thalidomide in refractory syringomyelia. Primary outcome measure: The primary endpoint is the change of ASIA at week 12. The clinical efficacy is defined as ASIA increase ≥ 1 at week 12, as compared with that before thalidomide usage.

Development of a Patient-reported Outcome Measure for Chiari Malformation and Syringomyelia

Chiari malformation corresponds to the herniation of cerebellar tonsils into the foramen magnum resulting in obstruction of cerebrospinal fluid circulation, which may eventually lead to the formation of an intramedullary cavity called syringomyelia. Chiari and syringomyelia can be responsible of variable symptoms, based on which neurosurgeons might propose surgical treatment. Yet, there is no properly developped and validated patient reported outcome measure (PROM) to assess the clinical severity of Chiari malformation and/or syringomyelia. The lack of such evaluation tool is a major issue to determine the optimal therapeutic strategy and to achieve a standardized and reproducible follow-up.