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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
Puerta de Hierro University Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Post-Traumatic Syringomyelia

Eligibility Criteria

18 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

    First Posted
    June 3, 2016
    Last Updated
    April 3, 2018
    Sponsor
    Puerta de Hierro University Hospital
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    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
    background

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    Efficacy Assessment of the Cell Therapy Medicine NC1 in Patients With Post-traumatic Syringomyelia

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