Evaluate the efficacy of the cell therapy with NC1 medication in patients with post-traumatic syringomyelia

• Register: EUCTR
• Last refreshed on: 19 February 2018
• Main ID: EUCTR2015-002383-16-ES
• Date of registration: 15/10/2015
• Prospective Registration: Yes
• Primary sponsor: Fundación Investigación Biomédica Hospital Universitario Puerta de Hierro
• Public title: Evaluate the efficacy of the cell therapy with NC1 medication in patients with post-traumatic syringomyelia
• Scientific title: Evaluate the efficacy of the cell therapy with NC1 medication in patients with post-traumatic syringomyelia
• Date of first enrolment: 19/01/2016
• Target sample size: 6
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-002383-16
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Spain

Contacts

• Name: Unidad de puesta en marcha
• Affiliation: SERMES PLANIFICACION. S.L

• Name: Unidad de puesta en marcha
• Affiliation: SERMES PLANIFICACION. S.L

Key inclusion & exclusion criteria

Inclusion criteria:
1.Post-traumatic spinal cord injury patients (Level A, B, C or D in ASIA scales), with associated syringomyelia extended at least in three vertebral segments, and neurological stable clinic for at least 6 months prior to study start.
2.Previous studies of Neurophysiology, MRI and Urology (if neurogenic bladder data exist) and defecatory activity (if neurogenic intestine signs exist) to allow useful baseline, in order that they can be compared with the same scans at the end of the follow uo period, and to obtain objective data of potential efficacy.
3.Age between 18 and 70 years.
4.Neuroimagin (MR) that allow to confirm the syringomyelic cavity existence extended at lest 3 vertebral segments.
5.Men and women of childbearing age must compromisse to use contraceptives from the time at which the removal of cells from the bone marrow is performed until 6 months after the celular administration.
6.Possibility of follow up and commitment to perform ambulatory physical therapy, at least one hour per day, five days per week, throughout all treatment period.
7.Written informed consent, according to the law in force.
8.Hematologic, creatinine, SGOT and SGPT parameters, within the normal range, according to laboratory standards. However, slight modifications that are considered significant in the context of treatment to be performed, according to the criterion of the research team, are accepted.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 5
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion criteria:
1. Age below 18 years or above 70.
2. Pregnancy or lactation.
3. Current neoplastic disease or in the previous 5 years (diagnosed or treated).
4. Patients with systemic disease or other pathology that represents an added risk to the cellular therapy treatment or to the surgical operation.
5. Alterations in the genetic study performed to discard risk cell transformation in the expansion process.
6. Patients with doubts about possible cooperation in the maintenance physical therapy or in the controls carried out during the study
7. Neurodegenerative disease added.
8. History of substance abuse, psychiatric disease or allergy to protein products used in the process of cell expansion.
9. Positive serology for HIV and syphilis.
10. Active Hepatitis B or Hepatitis C, according to serology analysis.
11. If in the opinion of the researcher there is some other reason why the patient is not considered candidate for the study.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Traumatic spinal cord injury, chronically established, associated with syringomyelia and neurological deficit considered irreversible.

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Intervention(s)

Product Name: Células mesenquimales troncales adultas autólogas de medula ósea expandidas
Product Code: CME-LEM4
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: células mesenquimales troncales adultas autólogas de médula ósea expandidas
Current Sponsor code: CME-LEM4
Concentration unit: U/ml unit(s)/millilitre
Concentration type: equal
Concentration number: 300000000-

Primary Outcome(s)

Primary end point(s): -Changes in ASIA scales and its subsections, as well as in IANR-SCIFRS, PENN, ASHWORTH, EVA, GEFFNER and BDS scales.
-Changes in the neurophysiological records (somato-sensory evoked potentials, motor evoked potentials and EMG).
-Changes in spinal cord morphology in neuroimaging studies (MRI).
-Changes in urodynamic records and defecatory function.

Timepoint(s) of evaluation of this end point: Efficacy will be assessed taking into account the changes in score of the different scales between the patient inclusion moment in the study and the scores obtained at the end of the follow up period, according to the clinic scales assessment and the objective study results (MR, neurophysiological records, urodynamic records and defecatory activity).

Main Objective: To analyze the potential clinical efficacy of the administration in the intrathecal compartment, ( intramedullary and in the subarachnoid space), of the medication NC1, to improve the neurological sequels of patients with established chronic spinal cord injury (LEM) and post-traumatic syringomyelia.

Secondary Objective: Confirm the treatment security.

Secondary Outcome(s)

Secondary end point(s): Will be evaluated the possible adverse effects during CME administration, development of complications and other adverse effects after it and during the follow up period.

Timepoint(s) of evaluation of this end point: During follow up period.

Secondary ID(s)

CME-LEM4

Source(s) of Monetary Support

Instituto de Salud Carlos III