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

April 3, 2018 updated by: Jesús Vaquero Crespo, M.D., Puerta de Hierro University Hospital
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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
6

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

14 years to 66 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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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
Time Frame: Pretreatment, month 3 post treatment, month 6 post treatment
Neurological evaluation is done using ASIA scale (American Spinal Injury Association)
Pretreatment, month 3 post treatment, month 6 post treatment

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Safety, by the assessment of the adverse events of the study
Time Frame: 1 year
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)
Time Frame: Pretreatment, month 3 post treatment, month 6 post treatment
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
Time Frame: Pretreatment, month 3 post treatment, month 6 post treatment
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
Time Frame: Pretreatment, month 3 post treatment, month 6 post treatment
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
Time Frame: Pretreatment, month 3 post treatment, month 6 post treatment
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
Time Frame: Pretreatment, month 3 post treatment, month 6 post treatment
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
Time Frame: Pretreatment, month 3 post treatment, month 6 post treatment
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
Time Frame: Pretreatment, month 3 post treatment, month 6 post treatment
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
Time Frame: Pretreatment, month 3 post treatment, month 6 post treatment
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
Time Frame: Pretreatment, month 3 post treatment, month 6 post treatment
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
Time Frame: Pretreatment, month 3 post treatment, month 6 post treatment
Neurological evaluation is done assessing the defecatory function
Pretreatment, month 3 post treatment, month 6 post treatment

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Puerta de Hierro University Hospital

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Jesús Vaquero Crespo, M.D., Hospital Universitario Puerta de Hierro-Majadahonda

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 1, 2016

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
February 7, 2018

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
February 7, 2018

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
June 3, 2016

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 20, 2016

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 21, 2016

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 5, 2018

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 3, 2018

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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.

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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