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

Study Overview
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Brief Summary
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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
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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.

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

Conditions
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Post-Traumatic Syringomyelia

Intervention / Treatment
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* Biological: NC 1 cell therapy


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: NC 1 cell therapy<br>All patients will be treated with the same treatment: NC1 cell therapy | Biological: NC 1 cell therapy<br>* All patients will be treated with NC1<br>|

What is the study measuring?
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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 |