Efficacy Assessment of the Cell Therapy Medicine NC1 in Patients With Post-traumatic Syringomyelia (CME-LEM4)

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.

Study Overview

• Status: Completed
• Conditions: Post-Traumatic Syringomyelia
• Intervention / Treatment: Biological: NC 1 cell therapy

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 Type: Interventional
• Enrollment (Actual): 6
• Phase: Phase 2

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 68 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: NC 1 cell therapy; All patients will be treated with the same treatment: NC1 cell therapy	Biological: NC 1 cell therapy; All patients will be treated with NC1

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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)	Pretreatment, month 3 post treatment, month 6 post treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety, by the assessment of the adverse events of the study	Adverse events	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)	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)	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)	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)	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)	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)	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	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)	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)	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	Pretreatment, month 3 post treatment, month 6 post treatment

Collaborators and Investigators

• Sponsor: Puerta de Hierro University Hospital
• Investigators: Principal Investigator: Jesús Vaquero Crespo, M.D., Hospital Universitario Puerta de Hierro-Majadahonda

Publications and helpful links

General Publications

• 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.
• 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.
• 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.
• 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.
• 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.

Study record dates

Study Major Dates

• Study Start: March 1, 2016
• Primary Completion (Actual): February 7, 2018
• Study Completion (Actual): February 7, 2018

Study Registration Dates

• First Submitted: June 3, 2016
• First Submitted That Met QC Criteria: June 20, 2016
• First Posted (Estimate): June 21, 2016

Study Record Updates

• Last Update Posted (Actual): April 5, 2018
• Last Update Submitted That Met QC Criteria: April 3, 2018
• Last Verified: April 1, 2018

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Syringomyelia
• Other Study ID Numbers: CME-LEM4

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes
• IPD 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.