SyringomyeliaNCT01150708
A Prospective Natural History Study of Patients With Syringomyelia
An observational study of Syringomyelia and Arnold Chiari Deformity, run by National Institute of Neurological Disorders and Stroke (NINDS) at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Syringomyelia, Arnold Chiari Deformity
- How many people
- 180 participants
- Where
- 1 siteUnited States
- Study dates
- 3 Aug 2010to not given
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: The study population is 180 patients with syringomyelia or with a condition that predisposes to the formation of syringomyelia such as presyringomyelia or Chiari I malformation without syringomyelia. A syrinx or syringomyelia is defined as an intramedullary cyst that extends / length > 1 spinal segment. Presyringomyelia will be defined as T2 hyperintensity > 4mm diameter extending over one spinal segment and associated with either Chiari I Malformation or a spinal lesion.A Chiari I Malformation without syringomyelia is defined as descent of the cerebellar tonsils > 5 mm below the foramen magnum without associated syringomyelia.
Who may be able to join
- To be eligible to participate in this research study, the subject must:
- Be 18 years of age or older.
- Have syringomyelia, presyringomyelia, or Chiari I malformation without syringomyelia.
- Be able to give informed consent.
- ADDITIONAL CRITERIA FOR PATIENTS THAT MAY UNDERGO SURGERY (not required for study inclusion)
- new or increased impairment in sensation, strength, or walking within the previous 2 years; and
Show all 9Show fewer
- 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
- 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
- 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.
Who cannot join
- The subject will not be eligible to participate in this research study if the subject:
- Is pregnant (because multiple MRI scans might injure a fetus).
- Cannot have an MRI scan as determined by the radiologist (see the risks below).
- 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).
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
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.
Read more
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.
Read the 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.
- Conditions
- Syringomyelia, Arnold Chiari Deformity
- Keywords
- Syringomyelia, Magnetic Resonance Imaging, Natural History Study, Neurological Examination, Natural History
- Medical subject headings
- Syringomyelia, Arnold-Chiari Malformation
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Chiari 1 with syringomyelia | Chiari I malformation with syringomyelia. | |
| Chiari 1 without syringomyelia | A Chiari I Malformation without syringomyelia is defined as descent of the cerebellar tonsils > 5 mm below the foramen magnum 79 without associated syringomyelia. | |
| Syringomyelia without chiari | A syrinx or syringomyelia is defined as an intramedullary cyst that extends / length > 1spinal segment. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in ASIA Score
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 neurologic and radiographic outcomes in patients that refuse surgical treatment. The primary outcome variable will be the change in motor strength in muscles of the upper and lower extremities as assessed by the American Spinal Injury (ASIA) Grading Scale. The change in ASIA score in patients between before surgery and one year after surgery will be compared to the one-year change in ASIA score in patients not receiving surgery (see Section 11, Statistical Analysis).
Measured: 1 year
Secondary outcomes (1)
Change in maximal Syrinx Anteroposterior Diameter
A secondary outcome measure will be the change in maximal syrinx anteroposterior diameter over 1 year in the surgical candidate group.Comparison will be made between groups that either undergo surgery or refuse surgical treatment. Another secondary outcome measure will be the change in maximal syrinx anteroposterior diameter over 1 year in the group of subjects that are not candidates for surgery.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jun 2010 (estimated)
- Study start3 Aug 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for SyringomyeliaStudies of the same condition, open and under way.
- More trials run by National Institute of Neurological Disorders and Stroke (NINDS)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 180 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- John D Heiss, M.D., Principal investigator, National Institute of Neurological Disorders and Stroke (NINDS)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01150708
- Sponsor's protocol ID
- 100143
- Other IDs
- 10-N-0143
- Status last verified
- 29 Sep 2026
- First submitted
- 24 Jun 2010
- Last updated
- 26 Oct 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Agarwalla PK, Dunn IF, Scott RM, Smith ER. Tethered cord syndrome. Neurosurg Clin N Am. 2007 Jul;18(3):531-47. doi: 10.1016/j.nec.2007.04.001.
PubMed 17678753Anderson NE, Willoughby EW, Wrightson P. The natural history of syringomyelia. Clin Exp Neurol. 1986;22:71-80.
PubMed 3581513Andrews BT, Weinstein PR, Rosenblum ML, Barbaro NM. Intradural arachnoid cysts of the spinal canal associated with intramedullary cysts. J Neurosurg. 1988 Apr;68(4):544-9. doi: 10.3171/jns.1988.68.4.0544.
PubMed 3280748
Study at a glance
From the registry record, last updated by the sponsor on 26 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Natural History Study of Patients With Syringomyelia
- ClinicalTrials.gov ID
- NCT01150708
- Acronym / sponsor ID
- 100143
- Conditions
- Syringomyelia, Arnold Chiari Deformity
- Keywords
- Syringomyelia, Magnetic Resonance Imaging, Natural History Study, Neurological Examination, Natural History
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 180 participants (actual)
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)US National Institutes of Health
- Study start
- 3 Aug 2010 (actual)
- First posted
- 25 Jun 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Chiari 1 with syringomyelia | – | –Chiari I malformation with syringomyelia. |
| Chiari 1 without syringomyelia | – | –A Chiari I Malformation without syringomyelia is defined as descent of the cerebellar tonsils > 5 mm below the foramen magnum 79 without associated syringomyelia. |
| Syringomyelia without chiari | – | –A syrinx or syringomyelia is defined as an intramedullary cyst that extends / length > 1spinal segment. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- The study population is 180 patients with syringomyelia or with a condition that predisposes to the formation of syringomyelia such as presyringomyelia or Chiari I malformation without syringomyelia. A syrinx or syringomyelia is defined as an intramedullary cyst that extends / length > 1 spinal segment. Presyringomyelia will be defined as T2 hyperintensity > 4mm diameter extending over one spinal segment and associated with either Chiari I Malformation or a spinal lesion.A Chiari I Malformation without syringomyelia is defined as descent of the cerebellar tonsils > 5 mm below the foramen magnum without associated syringomyelia.
Key inclusion criteria (9 listed)
- Be 18 years of age or older.
- Have syringomyelia, presyringomyelia, or Chiari I malformation without syringomyelia.
- Be able to give informed consent.
- ADDITIONAL CRITERIA FOR PATIENTS THAT MAY UNDERGO SURGERY (not required for study inclusion)
- new or increased impairment in sensation, strength, or walking within the previous 2 years; and
Key exclusion criteria (5 listed)
- Is pregnant (because multiple MRI scans might injure a fetus).
- Cannot have an MRI scan as determined by the radiologist (see the risks below).
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Change in ASIA Score
Time frame: 1 year
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 neurologic and radiographic outcomes in patients that refuse surgical treatment. The primary outcome variable will be the change in motor strength in muscles of the upper and lower extremities as assessed by the American Spinal Injury (ASIA) Grading Scale. The change in ASIA score in patients between before surgery and one year after surgery will be compared to the one-year change in ASIA score in patients not receiving surgery (see Section 11, Statistical Analysis).
1 secondary outcome
Change in maximal Syrinx Anteroposterior Diameter
Time frame: 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Oct 2026.
- Background: PubMed 17678753Agarwalla PK, Dunn IF, Scott RM, Smith ER. Tethered cord syndrome. Neurosurg Clin N Am. 2007 Jul;18(3):531-47. doi: 10.1016/j.nec.2007.04.001.
- Background: PubMed 3581513Anderson NE, Willoughby EW, Wrightson P. The natural history of syringomyelia. Clin Exp Neurol. 1986;22:71-80.
- Background: PubMed 3280748Andrews BT, Weinstein PR, Rosenblum ML, Barbaro NM. Intradural arachnoid cysts of the spinal canal associated with intramedullary cysts. J Neurosurg. 1988 Apr;68(4):544-9. doi: 10.3171/jns.1988.68.4.0544.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
Related trials for Syringomyelia
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Subarachnoid-Subarachnoid (S-S) Bypass Versus Adhesion Lysis in Spinal Arachnoiditis and Syringomyelia
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The Therapeutic Effect of Betaine in Syringomyelia
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The Therapeutic Effect of Thalidomide in Syringomyelia
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
John D Heiss, M.D. principal investigator
National Institute of Neurological Disorders and Stroke (NINDS)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Syringomyelia and Arnold Chiari Deformity, run by National Institute of Neurological Disorders and Stroke (NINDS) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in ASIA Score (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01150708, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Oct 2026.