Clinical trial NCT02807142: Efficacy Assessment of the Cell Therapy Medicine NC1 in Patients With Post-traumatic Syringomyelia
Official title: Efficacy Assessment of the Cell Therapy Medicine NC1 in Patients With Post-traumatic Syringomyelia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Post-Traumatic Syringomyelia
Lead sponsor: Puerta de Hierro University Hospital
Sponsor class: OTHER
First posted: 2016-06-21
Enrollment: 6
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
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
timeFrame: Pretreatment, month 3 post treatment, month 6 post treatment
Secondary outcomes:
measure: Safety, by the assessment of the adverse events of the study
timeFrame: 1 year
measure: 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)
timeFrame: Pretreatment, month 3 post treatment, month 6 post treatment
measure: Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia in terms of spams frequency
timeFrame: Pretreatment, month 3 post treatment, month 6 post treatment
measure: Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia in term of spasticity
timeFrame: Pretreatment, month 3 post treatment, month 6 post treatment
measure: Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia using the Visual Analog Scale
timeFrame: Pretreatment, month 3 post treatment, month 6 post treatment
measure: Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia in terms of bladder functionality
timeFrame: Pretreatment, month 3 post treatment, month 6 post treatment
measure: Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia using the Behavior Dimensions Scale
timeFrame: Pretreatment, month 3 post treatment, month 6 post treatment
measure: 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
timeFrame: Pretreatment, month 3 post treatment, month 6 post treatment
measure: Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia by electromyography
timeFrame: Pretreatment, month 3 post treatment, month 6 post treatment
measure: Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia studying the spinal cord morphology
timeFrame: Pretreatment, month 3 post treatment, month 6 post treatment
measure: Efficacy of the treatment with NC1 in terms of improvement of neurological sequelae in patients with post traumatic syringomyelia in terms of defecatory function
timeFrame: Pretreatment, month 3 post treatment, month 6 post treatment
Other outcomes:
Locations:
Study officials:
affiliation: Hospital Universitario Puerta de Hierro-Majadahonda
name: Jesús Vaquero Crespo, M.D.
role: PRINCIPAL_INVESTIGATOR
References:
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.
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.
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.
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.
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.