SyringomyeliaPhase 2NCT06268093
The Therapeutic Effect of Thalidomide in Syringomyelia
A Phase 2 study testing Thalidomide in people with Syringomyelia and Thalidomide, run by Xuanwu Hospital, Beijing at 1 site in China.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Syringomyelia, Thalidomide
- What is being tested?
- Thalidomide
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteChina
- Study dates
- Feb 2024to Feb 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Contraindication to duraplasty and shunting treatment due to history or high risk of severe adverse effects,
- or non-effective response to duraplasty and shunting treatment in 12 months prior to study entry.
- Estimated life expectancy must be greater than 12 months.
- Routine laboratory studies: bilirubin </=1.0 * upper limits of normal (ULN); aspartate aminotransferase (AST or SGOT) or alanine aminotransferase (ALT)< 1.0 * ULN; creatinine <1.0 * ULN; white-cell count >/= 4,000 per cubic millimeter; neutrophils count >/=1500 per cubic millimeter platelets >/= 100,000 per cubic millimeter; Hb >/=110 gram per millilitres; PT, APTT, INR in a normal range.
- Ability to understand and willingness to sign a written informed consent document, or constant caregivers who well understand and willingness to sign a written informed consent document.
- Must be able to swallow tablets
Who cannot join
- Evidence of tumor metastasis, recurrence, or invasion;
- History of psychiatric diseases ;
- History of seizures;
- History of arteriosclerotic cardiovascular diseases (ASCVD), e.g. stroke, myocardial infaction, unstable angina, within 6 months;
- New York Heart Association Grade II or greater congestive heart failure;
- Serious and inadequately controlled cardiac arrhythmia;
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- Significant vascular disease, e.g. moderate or severe carotid stenosis, aortic aneurysm, history of aortic dissection;
- Severe infection;
- History of allergy to relevant drugs;
- Pregnancy, lactation, or fertility program in the following 12 months;
- History or current diagnosis of peripheral nerve disease;
- Abnormal in liver and renal function;
- Active tuberculosis;
- Transplanted organs;
- Human immunodeficiency virus;
- Participation in other experimental studies.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Fengzeng Jian
Beijing, Beijing City, 100032
fengzeng jian, md, jianfengzeng@xwh.ccmu.edu.cn
About this study
Official title: The Effect of Thalidomide in Refractory Syringomyelia（RS）: a Phase II Clinical Trial
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.
Read the detailed description
The pathogenesis of syringomyelia is poorly understood and duraplasty or shunting is not always effective. Although it is generally thought that syringomyelia is simply an accumulation of CSF from the subarachnoid space, the pathogenesis is likely to be more complex and may involve cellular and molecular processes.
The investigators supposed that blood spinal cord barrier(BSCB) might play a key role in the pathogenesis of syringomyelia, especially post-traumatic syringomyelia(PTS), and that thalidomide, as an BSCB protection-related drug, would reduce BSCB damage and protect BSCB in syringomyelia.
Primary objectives: This phase II clinical trial aims to evaluate the indications, therapeutic effects and safety of thalidomide in refractory syringomyelia.
- Conditions
- Syringomyelia, Thalidomide
- Being tested
- Thalidomide
- Medical subject headings
- Syringomyelia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ThalidomideDrug
Thalidomide 50 - 200 mg once at nightime
| Study group | Role | What they receive |
|---|---|---|
| Thalidomide | Experimental | Oral Thalidomide Receives: Drug: Thalidomide |
What is being measured
The primary outcome is the main question the study is designed to answer.
ASIA Score
American Spinal Injury Association(ASIA) Score for evaluating the spinal cord function， degree of the spinal cord function, motor1-100, sensory 1-224, higher scores mean a better outcome
Measured: 1 day before and 3 days, 3 months after drug treatment
Secondary outcomes (7)
syringomyelia remission
syringomyelia remission is defined as ≥ 25% reduction in syringomyelia volume on T2 images at week 12, as compared with that before thalidomide usage
Measured: 1 day before and 3 days, 3 months after drug treatment
The evoked electromyographic signal (eEMG) potential
The evoked electromyographic signal (eEMG) potential is the standard index
Measured: 1 day before and 3 days, 3 months after drug treatment
Visual Analog Scale (VAS)
degree of the pain, 1-10, higher scores mean a worse outcome
Measured: 1 day before and 3 days, 3 months after drug treatment
Klekamp and Sammi syringomyelia scale
for evaluating the spinal cord function, higher scores mean a better outcome
Measured: 1 day before and 3 days, 3 months after drug treatment
modified Japanese Orthopaedic Association Scores (mJOA)
Motor function， sensory， bladder function；for evaluating the spinal cord function；0-17， higher scores mean a better outcome
Measured: 1 day before and 3 days, 3 months after drug treatment
xuanwu syringomyelia scale
for evaluating the spinal cord function, for evaluating the spinal cord function；0-18， higher scores mean a worse outcome
Measured: 1 day before and 3 days, 3 months after drug treatment
Incidence of complications
Incidence of complications
Measured: 1 day before and 3 days, 3 months after drug treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Feb 2024
- Study startFeb 2024 (estimated)
- Primary completionFeb 2027 (estimated)
- Study completionFeb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (7)Papers the sponsor has linked to this study.
- Other trials for SyringomyeliaStudies of the same condition, open and under way.
- More trials run by Xuanwu Hospital, BeijingThe 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Xuanwu Hospital, Beijing
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- fengzeng jian, Study chair, Xuanwu Hospital, Beijing
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06268093
- Sponsor's protocol ID
- XWTETS
- Status last verified
- Feb 2024
- First submitted
- 7 Feb 2024
- Last updated
- 20 Feb 2024
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Chen H, Wu S, Tang M, Zhao R, Zhang Q, Dai Z, Gao Y, Yang S, Li Z, Du Y, Yang A, Zhong L, Lu L, Xu L, Shen X, Liu S, Zhong J, Li X, Lu H, Xiong H, Shen Y, Chen H, Gong S, Xue H, Ge Z. Thalidomide for Recurrent Bleeding Due to Small-Intestinal Angiodysplasia. N Engl J Med. 2023 Nov 2;389(18):1649-1659. doi: 10.1056/NEJMoa2303706.
PubMed 37913505Guan J, Yuan C, Yao Q, Du Y, Fang Z, Zhang L, Jia S, Zhang C, Liu Z, Wang K, Duan W, Wang Z, Wang X, Wu H, Chen Z, Jian F. A novel scoring system for assessing adult syringomyelia associated with CM I treatment outcomes. Acta Neurol Belg. 2023 Jun;123(3):807-814. doi: 10.1007/s13760-023-02264-4. Epub 2023 Apr 12.
PubMed 37046133Hemley SJ, Bilston LE, Cheng S, Stoodley MA. Aquaporin-4 expression and blood-spinal cord barrier permeability in canalicular syringomyelia. J Neurosurg Spine. 2012 Dec;17(6):602-12. doi: 10.3171/2012.9.SPINE1265. Epub 2012 Oct 19.
PubMed 23082850Yuan C, Xia P, Duan W, Wang J, Guan J, Du Y, Zhang C, Liu Z, Wang K, Wang Z, Wang X, Wu H, Chen Z, Jian F. Long-Term Impairment of the Blood-Spinal Cord Barrier in Patients With Post-Traumatic Syringomyelia and its Effect on Prognosis. Spine (Phila Pa 1976). 2024 Mar 15;49(6):E62-E71. doi: 10.1097/BRS.0000000000004884. Epub 2023 Nov 28.
PubMed 38014747Heiss JD. Cerebrospinal Fluid Hydrodynamics in Chiari I Malformation and Syringomyelia: Modeling Pathophysiology. Neurosurg Clin N Am. 2023 Jan;34(1):81-90. doi: 10.1016/j.nec.2022.08.007. Epub 2022 Nov 3.
PubMed 36424067Guan J, Yuan C, Zhang C, Ma L, Yao Q, Cheng L, Liu Z, Wang K, Duan W, Wang X, Wu H, Chen Z, Jian F. Intradural Pathology Causing Cerebrospinal Fluid Obstruction in Syringomyelia and Effectiveness of Foramen Magnum and Foramen of Magendie Dredging Treatment. World Neurosurg. 2020 Dec;144:e178-e188. doi: 10.1016/j.wneu.2020.08.068. Epub 2020 Aug 15.
PubMed 32805463Liu S, Ma L, Qi B, Li Q, Chen Z, Jian F. Suppression of TGFbetaR-Smad3 pathway alleviates the syrinx induced by syringomyelia. Cell Biosci. 2023 May 29;13(1):98. doi: 10.1186/s13578-023-01048-w.
PubMed 37248485
Study at a glance
From the registry record, last updated by the sponsor on 20 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effect of Thalidomide in Refractory Syringomyelia（RS）: a Phase II Clinical Trial
- ClinicalTrials.gov ID
- NCT06268093
- Acronym / sponsor ID
- XWTETS
- Conditions
- Syringomyelia, Thalidomide
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Xuanwu Hospital, BeijingUniversity, hospital or other
- Study start
- Feb 2024 (estimated)
- Primary completion
- Feb 2027 (estimated)
- Study completion
- Feb 2028 (estimated)
- First posted
- 20 Feb 2024 (actual)
- 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.
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.
Detailed description
The pathogenesis of syringomyelia is poorly understood and duraplasty or shunting is not always effective. Although it is generally thought that syringomyelia is simply an accumulation of CSF from the subarachnoid space, the pathogenesis is likely to be more complex and may involve cellular and molecular processes.
The investigators supposed that blood spinal cord barrier(BSCB) might play a key role in the pathogenesis of syringomyelia, especially post-traumatic syringomyelia(PTS), and that thalidomide, as an BSCB protection-related drug, would reduce BSCB damage and protect BSCB in syringomyelia.
Primary objectives: This phase II clinical trial aims to evaluate the indications, therapeutic effects and safety of thalidomide in refractory syringomyelia.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Thalidomide | Experimental | Drug: ThalidomideOral Thalidomide |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Contraindication to duraplasty and shunting treatment due to history or high risk of severe adverse effects,
- or non-effective response to duraplasty and shunting treatment in 12 months prior to study entry.
- Estimated life expectancy must be greater than 12 months.
- Routine laboratory studies: bilirubin </=1.0 * upper limits of normal (ULN); aspartate aminotransferase (AST or SGOT) or alanine aminotransferase (ALT)< 1.0 * ULN; creatinine <1.0 * ULN; white-cell count >/= 4,000 per cubic millimeter; neutrophils count >/=1500 per cubic millimeter platelets >/= 100,000 per cubic millimeter; Hb >/=110 gram per millilitres; PT, APTT, INR in a normal range.
- Ability to understand and willingness to sign a written informed consent document, or constant caregivers who well understand and willingness to sign a written informed consent document.
Key exclusion criteria (16 listed)
- Evidence of tumor metastasis, recurrence, or invasion;
- History of psychiatric diseases ;
- History of seizures;
- History of arteriosclerotic cardiovascular diseases (ASCVD), e.g. stroke, myocardial infaction, unstable angina, within 6 months;
- New York Heart Association Grade II or greater congestive heart failure;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ThalidomideThalidomide 50 - 200 mg once at nightime | Drug | Thalidomide |
Primary outcome
ASIA Score
Time frame: 1 day before and 3 days, 3 months after drug treatment
American Spinal Injury Association(ASIA) Score for evaluating the spinal cord function， degree of the spinal cord function, motor1-100, sensory 1-224, higher scores mean a better outcome
7 secondary outcomes
syringomyelia remission
Time frame: 1 day before and 3 days, 3 months after drug treatment
The evoked electromyographic signal (eEMG) potential
Time frame: 1 day before and 3 days, 3 months after drug treatment
Visual Analog Scale (VAS)
Time frame: 1 day before and 3 days, 3 months after drug treatment
Klekamp and Sammi syringomyelia scale
Time frame: 1 day before and 3 days, 3 months after drug treatment
modified Japanese Orthopaedic Association Scores (mJOA)
Time frame: 1 day before and 3 days, 3 months after drug treatment
xuanwu syringomyelia scale
Time frame: 1 day before and 3 days, 3 months after drug treatment
Incidence of complications
Time frame: 1 day before and 3 days, 3 months after drug treatment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Feb 2024.
- Background: PubMed 37913505Chen H, Wu S, Tang M, Zhao R, Zhang Q, Dai Z, Gao Y, Yang S, Li Z, Du Y, Yang A, Zhong L, Lu L, Xu L, Shen X, Liu S, Zhong J, Li X, Lu H, Xiong H, Shen Y, Chen H, Gong S, Xue H, Ge Z. Thalidomide for Recurrent Bleeding Due to Small-Intestinal Angiodysplasia. N Engl J Med. 2023 Nov 2;389(18):1649-1659. doi: 10.1056/NEJMoa2303706.
- Background: PubMed 37046133Guan J, Yuan C, Yao Q, Du Y, Fang Z, Zhang L, Jia S, Zhang C, Liu Z, Wang K, Duan W, Wang Z, Wang X, Wu H, Chen Z, Jian F. A novel scoring system for assessing adult syringomyelia associated with CM I treatment outcomes. Acta Neurol Belg. 2023 Jun;123(3):807-814. doi: 10.1007/s13760-023-02264-4. Epub 2023 Apr 12.
- Results publication: PubMed 23082850Hemley SJ, Bilston LE, Cheng S, Stoodley MA. Aquaporin-4 expression and blood-spinal cord barrier permeability in canalicular syringomyelia. J Neurosurg Spine. 2012 Dec;17(6):602-12. doi: 10.3171/2012.9.SPINE1265. Epub 2012 Oct 19.
- Results publication: PubMed 38014747Yuan C, Xia P, Duan W, Wang J, Guan J, Du Y, Zhang C, Liu Z, Wang K, Wang Z, Wang X, Wu H, Chen Z, Jian F. Long-Term Impairment of the Blood-Spinal Cord Barrier in Patients With Post-Traumatic Syringomyelia and its Effect on Prognosis. Spine (Phila Pa 1976). 2024 Mar 15;49(6):E62-E71. doi: 10.1097/BRS.0000000000004884. Epub 2023 Nov 28.
- Results publication: PubMed 36424067Heiss JD. Cerebrospinal Fluid Hydrodynamics in Chiari I Malformation and Syringomyelia: Modeling Pathophysiology. Neurosurg Clin N Am. 2023 Jan;34(1):81-90. doi: 10.1016/j.nec.2022.08.007. Epub 2022 Nov 3.
- Results publication: PubMed 32805463Guan J, Yuan C, Zhang C, Ma L, Yao Q, Cheng L, Liu Z, Wang K, Duan W, Wang X, Wu H, Chen Z, Jian F. Intradural Pathology Causing Cerebrospinal Fluid Obstruction in Syringomyelia and Effectiveness of Foramen Magnum and Foramen of Magendie Dredging Treatment. World Neurosurg. 2020 Dec;144:e178-e188. doi: 10.1016/j.wneu.2020.08.068. Epub 2020 Aug 15.
- Results publication: PubMed 37248485Liu S, Ma L, Qi B, Li Q, Chen Z, Jian F. Suppression of TGFbetaR-Smad3 pathway alleviates the syrinx induced by syringomyelia. Cell Biosci. 2023 May 29;13(1):98. doi: 10.1186/s13578-023-01048-w.
- Individual participant dataSharing planned: Undecided
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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.
fengzeng jian study chair
Xuanwu Hospital, Beijing
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Thalidomide in people with Syringomyelia and Thalidomide, run by Xuanwu Hospital, Beijing at 1 site in China.
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 6 inclusion and 16 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: looking for participants now, as of the sponsor's last update on 20 Feb 2024. 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 China. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact fengzeng jian (+861083198899, jianfengzeng@xwh.ccmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
ASIA Score (time frame: 1 day before and 3 days, 3 months after drug treatment). 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 NCT06268093, which also lists 7 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 20 Feb 2024.