Efficacy Assessment of the Cell Therapy Medicine NC1 in Patients With Post-traumatic Syringomyelia (CME-LEM4)
Primary Purpose
Post-Traumatic Syringomyelia
Status
Completed
Phase
Phase 2
Locations
Study Type
Interventional
Intervention
NC 1 cell therapy
Sponsored by
About this trial
This is an interventional treatment trial for Post-Traumatic Syringomyelia
Eligibility Criteria
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
- 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.
Sites / Locations
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
NC 1 cell therapy
Arm Description
All patients will be treated with the same treatment: NC1 cell therapy
Outcomes
Primary Outcome Measures
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)
Secondary Outcome Measures
Safety, by the assessment of the adverse events of the study
Adverse events
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)
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)
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)
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)
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)
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)
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
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia by electromyography
EMG (Electromyography)
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)
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
Full Information
NCT ID
NCT02807142
First Posted
June 3, 2016
Last Updated
April 3, 2018
Sponsor
Puerta de Hierro University Hospital
1. Study Identification
Unique Protocol Identification Number
NCT02807142
Brief Title
Efficacy Assessment of the Cell Therapy Medicine NC1 in Patients With Post-traumatic Syringomyelia
Acronym
CME-LEM4
Official Title
Efficacy Assessment of the Cell Therapy Medicine NC1 in Patients With Post-traumatic Syringomyelia
Study Type
Interventional
2. Study Status
Record Verification Date
April 2018
Overall Recruitment Status
Completed
Study Start Date
March 2016 (undefined)
Primary Completion Date
February 7, 2018 (Actual)
Study Completion Date
February 7, 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Puerta de Hierro University Hospital
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief 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. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Post-Traumatic Syringomyelia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
6 (Actual)
8. Arms, Groups, and Interventions
Arm Title
NC 1 cell therapy
Arm Type
Experimental
Arm Description
All patients will be treated with the same treatment: NC1 cell therapy
Intervention Type
Biological
Intervention Name(s)
NC 1 cell therapy
Intervention Description
All patients will be treated with NC1
Primary Outcome Measure Information:
Title
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
Description
Neurological evaluation is done using ASIA scale (American Spinal Injury Association)
Time Frame
Pretreatment, month 3 post treatment, month 6 post treatment
Secondary Outcome Measure Information:
Title
Safety, by the assessment of the adverse events of the study
Description
Adverse events
Time Frame
1 year
Title
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)
Description
Neurological evaluation is done using IANR-SCIFRS scale (Injury Functional Rating Scale)
Time Frame
Pretreatment, month 3 post treatment, month 6 post treatment
Title
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia in terms of spams frequency
Description
Neurological evaluation is done using PENN scale (spams frequency score)
Time Frame
Pretreatment, month 3 post treatment, month 6 post treatment
Title
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia in term of spasticity
Description
Neurological evaluation is done using Ashworth scale (spasticity)
Time Frame
Pretreatment, month 3 post treatment, month 6 post treatment
Title
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia using the Visual Analog Scale
Description
Neurological evaluation is done using EVA scale (Visual Analog Scale)
Time Frame
Pretreatment, month 3 post treatment, month 6 post treatment
Title
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia in terms of bladder functionality
Description
Neurological evaluation is done using GEFFNER scale (bladder functionality)
Time Frame
Pretreatment, month 3 post treatment, month 6 post treatment
Title
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia using the Behavior Dimensions Scale
Description
Neurological evaluation is done using BDS scales (Behavior Dimensions Scale)
Time Frame
Pretreatment, month 3 post treatment, month 6 post treatment
Title
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
Description
Present of somatosensory or motor evoked potentials
Time Frame
Pretreatment, month 3 post treatment, month 6 post treatment
Title
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia by electromyography
Description
EMG (Electromyography)
Time Frame
Pretreatment, month 3 post treatment, month 6 post treatment
Title
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia studying the spinal cord morphology
Description
Spinal cord morphology (by Magnetic Resonance)
Time Frame
Pretreatment, month 3 post treatment, month 6 post treatment
Title
Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia in terms of defecatory function
Description
Neurological evaluation is done assessing the defecatory function
Time Frame
Pretreatment, month 3 post treatment, month 6 post treatment
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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.
Exclusion Criteria:
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.
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Jesús Vaquero Crespo, M.D.
Organizational Affiliation
Hospital Universitario Puerta de Hierro-Majadahonda
Official's Role
Principal Investigator
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
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.
IPD Sharing Time Frame
Starting at CTD submission to authorities.
IPD Sharing Access Criteria
Spanish competent authority.
Citations:
PubMed Identifier
19012250
Citation
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.
Results Reference
background
PubMed Identifier
21163325
Citation
Vaquero 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.
Results Reference
background
PubMed Identifier
21942842
Citation
Otero 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.
Results Reference
background
PubMed Identifier
21208021
Citation
Otero 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.
Results Reference
background
PubMed Identifier
22507677
Citation
Huang 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.
Results Reference
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Efficacy Assessment of the Cell Therapy Medicine NC1 in Patients With Post-traumatic Syringomyelia
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