Post-Traumatic SyringomyeliaPhase 2CME-LEM4NCT02807142
Efficacy Assessment of the Cell Therapy Medicine NC1 in Patients With Post-traumatic Syringomyelia
A Phase 2 study testing NC 1 cell therapy in people with Post-Traumatic Syringomyelia, run by Puerta de Hierro University Hospital.
Trial summary
The objective of the study is to determine if the cell therapy NC1 administered in the spinal cord is effective for the treatment of a post-traumatic syringomyelia. The post-traumatic syringomyelia is the development and progression of cyst filled with cerebrospinal fluid (CSF) within the spinal cord. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Post-Traumatic Syringomyelia
- What is being tested?
- NC 1 cell therapy
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- No sites listed yet
- Study dates
- Mar 2016to 7 Feb 2018
- Phase
- Phase 2
- 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 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with traumatic medullar injury (levels A, B, C or D of the ASIA scale) and associated syringomyelia in at least three spinal segments, and with neurological deficit clinically stable at least 6 months prior to treatment.
- Previous studies in Neurophysiology, MR, Urology (if data on neurogenic bowel exists), defecatory function (if data on neurogenic intestine exists)
- Age between 18 and 70 years old.
- Presence of syringomyelia based on a neuro-image (MR)
- Patients will compromise to use anticonceptive measures from the cell extraction until 6 months after the administration of the treatment.
- Patients will compromise to a clinical follow-up and to perform physical therapy, one hour daily five days per week during the treatment period.
- Patients should sign an written informed consent.
- Haematological and creatinin parameters, SGOT and SGPT into the normal ranges.
Who cannot join
- Patients under 18 or above 70 years old
- Pregnancy or breastfeeding
- Neoplasia in the last 5 years
- Patients with systemic diseases that increase the risk of the surgical intervention
- Genetics alterations that could conduct to cellular transformation during the cellular expansion phase.
- Patients not really sure of their cooperation to follow the physical therapy or clinical controls during the study.
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- Additional neurodegenerative diseases
- Drug consuming, psychiatric disease or allergy to proteins used during cellular expansion
- HIV or syphilis positive serologies
- Active Hepatitis B or c, based on serologies
- Any other reasons according to the investigator criteria.
About this study
The objective of the study is to determine if the cell therapy NC1 administered in the spinal cord is effective for the treatment of a post-traumatic syringomyelia. The post-traumatic syringomyelia is the development and progression of cyst filled with cerebrospinal fluid (CSF) within the spinal cord. The cell therapy NC1 consist on cells obtained from the bone marrow of the patient, that are cultured in vitro and administered in the spinal cord of the same patient.
Read the detailed description
Phase II, Open, prospective, not controlled, not randomized clinical trial in patients affected by post-traumatic syringomyelia. The trial evaluates the efficacy of the cell therapy NC1 administered via intrathecal in the location of the injury. After the administration of the cell therapy, patients will undergo physiotherapy during the follow-up period (6 months). Patients will be evaluated at month 3 and month 6 after the NC1 administration.
- Conditions
- Post-Traumatic Syringomyelia
- Being tested
- NC 1 cell therapy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NC 1 cell therapyBiological
All patients will be treated with NC1
| Study group | Role | What they receive |
|---|---|---|
| NC 1 cell therapy | Experimental | All patients will be treated with the same treatment: NC1 cell therapy Receives: Biological: NC 1 cell therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia using the ASIA scale (American Spinal Injury Association
Neurological evaluation is done using ASIA scale (American Spinal Injury Association)
Measured: Pretreatment, month 3 post treatment, month 6 post treatment
Secondary outcomes (11)
Safety, by the assessment of the adverse events of the study
Adverse events
Measured: 1 year
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia assessed with the IANR-SCIFRS scale (Injury Functional Rating Scale)
Neurological evaluation is done using IANR-SCIFRS scale (Injury Functional Rating Scale)
Measured: Pretreatment, month 3 post treatment, month 6 post treatment
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia in terms of spams frequency
Neurological evaluation is done using PENN scale (spams frequency score)
Measured: Pretreatment, month 3 post treatment, month 6 post treatment
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia in term of spasticity
Neurological evaluation is done using Ashworth scale (spasticity)
Measured: Pretreatment, month 3 post treatment, month 6 post treatment
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia using the Visual Analog Scale
Neurological evaluation is done using EVA scale (Visual Analog Scale)
Measured: Pretreatment, month 3 post treatment, month 6 post treatment
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia in terms of bladder functionality
Neurological evaluation is done using GEFFNER scale (bladder functionality)
Measured: Pretreatment, month 3 post treatment, month 6 post treatment
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia using the Behavior Dimensions Scale
Neurological evaluation is done using BDS scales (Behavior Dimensions Scale)
Measured: Pretreatment, month 3 post treatment, month 6 post treatment
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia in terms of somatosensory or motor evoked potentials
Present of somatosensory or motor evoked potentials
Measured: Pretreatment, month 3 post treatment, month 6 post treatment
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia by electromyography
EMG (Electromyography)
Measured: Pretreatment, month 3 post treatment, month 6 post treatment
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia studying the spinal cord morphology
Spinal cord morphology (by Magnetic Resonance)
Measured: Pretreatment, month 3 post treatment, month 6 post treatment
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia in terms of defecatory function
Neurological evaluation is done assessing the defecatory function
Measured: Pretreatment, month 3 post treatment, month 6 post treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jun 2016 (estimated)
- Study startMar 2016
- Primary completion7 Feb 2018
- Study completion7 Feb 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Post-Traumatic SyringomyeliaStudies of the same condition, open and under way.
- 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
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Puerta de Hierro University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jesús Vaquero Crespo, M.D., Puerta de Hierro University Hospital
- Investigators
- Jesús Vaquero Crespo, M.D., Principal investigator, Hospital Universitario Puerta de Hierro Majadahonda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02807142
- Sponsor's protocol ID
- CME-LEM4
- Acronym
- CME-LEM4
- Status last verified
- Apr 2018
- First submitted
- 3 Jun 2016
- Last updated
- 5 Apr 2018
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Yes: Anonymized individual data of participants will be shared with Authorities at the end of the Clinical Development Plan by the CTD (Common Technical Document). Results will be published in a scientific publication.
Publications
Vaquero J, Zurita M. Bone marrow stromal cells for spinal cord repair: a challenge for contemporary neurobiology. Histol Histopathol. 2009 Jan;24(1):107-16. doi: 10.14670/HH-24.107.
PubMed 19012250Vaquero J, Zurita M. Functional recovery after severe CNS trauma: current perspectives for cell therapy with bone marrow stromal cells. Prog Neurobiol. 2011 Mar;93(3):341-9. doi: 10.1016/j.pneurobio.2010.12.002. Epub 2010 Dec 14.
PubMed 21163325Otero L, Zurita M, Bonilla C, Aguayo C, Rico MA, Rodriguez A, Vaquero J. Allogeneic bone marrow stromal cell transplantation after cerebral hemorrhage achieves cell transdifferentiation and modulates endogenous neurogenesis. Cytotherapy. 2012 Jan;14(1):34-44. doi: 10.3109/14653249.2011.608349. Epub 2011 Sep 23.
PubMed 21942842Otero L, Zurita M, Bonilla C, Aguayo C, Vela A, Rico MA, Vaquero J. Late transplantation of allogeneic bone marrow stromal cells improves neurologic deficits subsequent to intracerebral hemorrhage. Cytotherapy. 2011 May;13(5):562-71. doi: 10.3109/14653249.2010.544720. Epub 2011 Jan 5.
PubMed 21208021Huang H, Xi H, Chen L, Zhang F, Liu Y. Long-term outcome of olfactory ensheathing cell therapy for patients with complete chronic spinal cord injury. Cell Transplant. 2012;21 Suppl 1:S23-31. doi: 10.3727/096368912X633734.
PubMed 22507677
Study at a glance
From the registry record, last updated by the sponsor on 5 Apr 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy Assessment of the Cell Therapy Medicine NC1 in Patients With Post-traumatic Syringomyelia
- ClinicalTrials.gov ID
- NCT02807142
- Acronym / sponsor ID
- CME-LEM4 / CME-LEM4
- Conditions
- Post-Traumatic Syringomyelia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Puerta de Hierro University HospitalUniversity, hospital or other
- Study start
- Mar 2016
- Primary completion
- 7 Feb 2018 (actual)
- Study completion
- 7 Feb 2018 (actual)
- First posted
- 21 Jun 2016 (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.
The objective of the study is to determine if the cell therapy NC1 administered in the spinal cord is effective for the treatment of a post-traumatic syringomyelia. The post-traumatic syringomyelia is the development and progression of cyst filled with cerebrospinal fluid (CSF) within the spinal cord. The cell therapy NC1 consist on cells obtained from the bone marrow of the patient, that are cultured in vitro and administered in the spinal cord of the same patient.
Detailed description
Phase II, Open, prospective, not controlled, not randomized clinical trial in patients affected by post-traumatic syringomyelia. The trial evaluates the efficacy of the cell therapy NC1 administered via intrathecal in the location of the injury. After the administration of the cell therapy, patients will undergo physiotherapy during the follow-up period (6 months). Patients will be evaluated at month 3 and month 6 after the NC1 administration.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| NC 1 cell therapy | Experimental | Biological: NC 1 cell therapyAll patients will be treated with the same treatment: NC1 cell therapy |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Patients with traumatic medullar injury (levels A, B, C or D of the ASIA scale) and associated syringomyelia in at least three spinal segments, and with neurological deficit clinically stable at least 6 months prior to treatment.
- Previous studies in Neurophysiology, MR, Urology (if data on neurogenic bowel exists), defecatory function (if data on neurogenic intestine exists)
- Age between 18 and 70 years old.
- Presence of syringomyelia based on a neuro-image (MR)
- Patients will compromise to use anticonceptive measures from the cell extraction until 6 months after the administration of the treatment.
Key exclusion criteria (11 listed)
- Patients under 18 or above 70 years old
- Pregnancy or breastfeeding
- Neoplasia in the last 5 years
- Patients with systemic diseases that increase the risk of the surgical intervention
- Genetics alterations that could conduct to cellular transformation during the cellular expansion phase.
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 |
|---|---|---|
| NC 1 cell therapyAll patients will be treated with NC1 | Biological | NC 1 cell therapy |
Primary outcome
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia using the ASIA scale (American Spinal Injury Association
Time frame: Pretreatment, month 3 post treatment, month 6 post treatment
Neurological evaluation is done using ASIA scale (American Spinal Injury Association)
11 secondary outcomes
Safety, by the assessment of the adverse events of the study
Time frame: 1 year
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia assessed with the IANR-SCIFRS scale (Injury Functional Rating Scale)
Time frame: Pretreatment, month 3 post treatment, month 6 post treatment
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia in terms of spams frequency
Time frame: Pretreatment, month 3 post treatment, month 6 post treatment
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia in term of spasticity
Time frame: Pretreatment, month 3 post treatment, month 6 post treatment
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia using the Visual Analog Scale
Time frame: Pretreatment, month 3 post treatment, month 6 post treatment
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia in terms of bladder functionality
Time frame: Pretreatment, month 3 post treatment, month 6 post treatment
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia using the Behavior Dimensions Scale
Time frame: Pretreatment, month 3 post treatment, month 6 post treatment
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia in terms of somatosensory or motor evoked potentials
Time frame: Pretreatment, month 3 post treatment, month 6 post treatment
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia by electromyography
Time frame: Pretreatment, month 3 post treatment, month 6 post treatment
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia studying the spinal cord morphology
Time frame: Pretreatment, month 3 post treatment, month 6 post treatment
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia in terms of defecatory function
Time frame: Pretreatment, month 3 post treatment, month 6 post treatment
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 5 Apr 2018.
- Background: PubMed 19012250Vaquero J, Zurita M. Bone marrow stromal cells for spinal cord repair: a challenge for contemporary neurobiology. Histol Histopathol. 2009 Jan;24(1):107-16. doi: 10.14670/HH-24.107.
- Background: PubMed 21163325Vaquero J, Zurita M. Functional recovery after severe CNS trauma: current perspectives for cell therapy with bone marrow stromal cells. Prog Neurobiol. 2011 Mar;93(3):341-9. doi: 10.1016/j.pneurobio.2010.12.002. Epub 2010 Dec 14.
- Background: PubMed 21942842Otero L, Zurita M, Bonilla C, Aguayo C, Rico MA, Rodriguez A, Vaquero J. Allogeneic bone marrow stromal cell transplantation after cerebral hemorrhage achieves cell transdifferentiation and modulates endogenous neurogenesis. Cytotherapy. 2012 Jan;14(1):34-44. doi: 10.3109/14653249.2011.608349. Epub 2011 Sep 23.
- Background: PubMed 21208021Otero L, Zurita M, Bonilla C, Aguayo C, Vela A, Rico MA, Vaquero J. Late transplantation of allogeneic bone marrow stromal cells improves neurologic deficits subsequent to intracerebral hemorrhage. Cytotherapy. 2011 May;13(5):562-71. doi: 10.3109/14653249.2010.544720. Epub 2011 Jan 5.
- Background: PubMed 22507677Huang H, Xi H, Chen L, Zhang F, Liu Y. Long-term outcome of olfactory ensheathing cell therapy for patients with complete chronic spinal cord injury. Cell Transplant. 2012;21 Suppl 1:S23-31. doi: 10.3727/096368912X633734.
- Individual participant dataSharing planned: Yes. Anonymized individual data of participants will be shared with Authorities at the end of the Clinical Development Plan by the CTD (Common Technical Document). Results will be published in a scientific publication.
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.
Jesús Vaquero Crespo, M.D. principal investigator
Hospital Universitario Puerta de Hierro Majadahonda
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing NC 1 cell therapy in people with Post-Traumatic Syringomyelia, run by Puerta de Hierro University Hospital.
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 8 inclusion and 11 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 5 Apr 2018. Status can change between updates, so confirm with the study contact before making plans.
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?
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?
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia using the ASIA scale (American Spinal Injury Association (time frame: Pretreatment, month 3 post treatment, month 6 post 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 NCT02807142, which also lists 5 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 5 Apr 2018.