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Intrathecal Stem Cells for Cerebral Palsy Phase II (ISCII)

Primary Purpose

Cerebral Palsy

Status
Unknown status
Phase
Phase 2
Locations
Mexico
Study Type
Interventional
Intervention
Autologous Stem Cell Transplantation
Sponsored by
Hospital Universitario Dr. Jose E. Gonzalez
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional supportive care trial for Cerebral Palsy focused on measuring brain, children, cerebral palsy, bone marrow stem cells, intrathecal autologous total nucleated cells, quadriparesis

Eligibility Criteria

7 Years - 9 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Patients with quadriplegic cerebral palsy, with an age from 7 to 9 years of age, regardless the age at the time of injury, time post-injury, or previously received therapies, different from ours

Exclusion Criteria:

  • Patients with neurodegenerative or autoimmune diseases
  • Patients with active infection in any organ or tissue at the time of entering the study, the onset of stimulation with G-CSF or at the procedure
  • Patients who do not sign the informed consent form

Sites / Locations

  • Hematology Service, Hospital Universitario Dr. Jose E. GonzalezRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

No Intervention

Arm Label

Intrathecal Autologous Bone Marrow TNC

Control group

Arm Description

Procedure/Surgery: Intrathecal Autologous Bone Marrow TNC. Other Names: Autologous Stem Cell Transplantation Patients will be stimulated with Granulocyte Colony Stimulating Factor (G-CSF) (10mcgr/kg of body weight) for 3 consecutive days. Bone marrow will be harvested under sedation and, after being processed in the laboratory, the autologous TNC concentrate of 10 mL will be infused intrathecally.

Patients will be evaluated with the "Gross Motor Functional Classification System" initially, at one, three and six months, and then cross to the intervention arm.

Outcomes

Primary Outcome Measures

Number of participants who show progress in GMFCS scale after autologous bonemarrow TNC intrathecal infusion.
Patients, in an outpatient basis, will be stimulated with Granulocyte Colony Stimulating Factor (G-CSF) for 3 consecutive days. Their bone marrow will be harvested under sedation and, after being processed in the laboratory, the buffy coat of 10 mL will be infused intrathecally. Patient will be monitored during acute phase to register any adverse effect (malaise, headache, fever, and nausea or vomit, etc).Gross Motor Function Classification System (GMFCS) scale is going to be monitored at baseline, one, three and six months to measured differences between the scales. GMFCS is based on patient's self-initiated movement with particular emphasis on sitting, walking, and wheeled mobility. Distinctions between levels are based on functional abilities, the need for assistive technology, including hand-held mobility devices (walkers, crutches, or canes) or wheeled mobility, and to a much lesser extent, quality of movement.

Secondary Outcome Measures

Full Information

First Posted
July 14, 2014
Last Updated
September 3, 2014
Sponsor
Hospital Universitario Dr. Jose E. Gonzalez
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1. Study Identification

Unique Protocol Identification Number
NCT02231242
Brief Title
Intrathecal Stem Cells for Cerebral Palsy Phase II
Acronym
ISCII
Official Title
Intrathecal Autologous Stem Cells for Children With Cerebral Palsy Phase II
Study Type
Interventional

2. Study Status

Record Verification Date
September 2014
Overall Recruitment Status
Unknown status
Study Start Date
September 2013 (undefined)
Primary Completion Date
December 2015 (Anticipated)
Study Completion Date
June 2016 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Hospital Universitario Dr. Jose E. Gonzalez

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this study is to determine whether the infusion of intrathecal autologous bone marrow total nucleated cells would improve the neurologic evolution of pediatric patients with quadriparetic cerebral palsy.
Detailed Description
There is accumulated evidence that shows that the administration of bone marrow total nucleated cells (TNC) into the brain may produce some benefits by different mechanisms like cytokine production, in several neurological areas such as motor, social, adaptative and cognitive. It has been found that after introducing TNC in the subarachnoid space of the spinal cord, these cells may be transported through the cerebrospinal fluid and can be delivered more efficiently to the injured area, compared with intravenous route in patients with neurologic injury. Patients will be stimulated for 3 consecutive days with subcutaneous granulocyte colony stimulating factor (G-CSF) and then their bone marrow will be harvested according to their weight. Bone marrow will be processed in order to obtain the buffy coat and minimize the amount of red blood cells. An inoculum of 10mL of this TNC will be infused intrathecally. Patients will be evaluated with the "Gross Motor Functional Classification System" before the procedure and one, three and six months after that.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cerebral Palsy
Keywords
brain, children, cerebral palsy, bone marrow stem cells, intrathecal autologous total nucleated cells, quadriparesis

7. Study Design

Primary Purpose
Supportive Care
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Masking
Outcomes Assessor
Allocation
Randomized
Enrollment
60 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Intrathecal Autologous Bone Marrow TNC
Arm Type
Experimental
Arm Description
Procedure/Surgery: Intrathecal Autologous Bone Marrow TNC. Other Names: Autologous Stem Cell Transplantation Patients will be stimulated with Granulocyte Colony Stimulating Factor (G-CSF) (10mcgr/kg of body weight) for 3 consecutive days. Bone marrow will be harvested under sedation and, after being processed in the laboratory, the autologous TNC concentrate of 10 mL will be infused intrathecally.
Arm Title
Control group
Arm Type
No Intervention
Arm Description
Patients will be evaluated with the "Gross Motor Functional Classification System" initially, at one, three and six months, and then cross to the intervention arm.
Intervention Type
Biological
Intervention Name(s)
Autologous Stem Cell Transplantation
Intervention Description
Patients will be stimulated with Granulocyte Colony Stimulating Factor (G-CSF) 3 consecutive days. Bone marrow will be harvested under sedation and, after being processed in the laboratory, the buffy coat (TNC) of 10 mL will be infused intrathecally. Intrathecal Autologous Bone Marrow TNC
Primary Outcome Measure Information:
Title
Number of participants who show progress in GMFCS scale after autologous bonemarrow TNC intrathecal infusion.
Description
Patients, in an outpatient basis, will be stimulated with Granulocyte Colony Stimulating Factor (G-CSF) for 3 consecutive days. Their bone marrow will be harvested under sedation and, after being processed in the laboratory, the buffy coat of 10 mL will be infused intrathecally. Patient will be monitored during acute phase to register any adverse effect (malaise, headache, fever, and nausea or vomit, etc).Gross Motor Function Classification System (GMFCS) scale is going to be monitored at baseline, one, three and six months to measured differences between the scales. GMFCS is based on patient's self-initiated movement with particular emphasis on sitting, walking, and wheeled mobility. Distinctions between levels are based on functional abilities, the need for assistive technology, including hand-held mobility devices (walkers, crutches, or canes) or wheeled mobility, and to a much lesser extent, quality of movement.
Time Frame
six months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
7 Years
Maximum Age & Unit of Time
9 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patients with quadriplegic cerebral palsy, with an age from 7 to 9 years of age, regardless the age at the time of injury, time post-injury, or previously received therapies, different from ours Exclusion Criteria: Patients with neurodegenerative or autoimmune diseases Patients with active infection in any organ or tissue at the time of entering the study, the onset of stimulation with G-CSF or at the procedure Patients who do not sign the informed consent form
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Consuelo Mancias-Guerra, MD
Phone
+52 81 83 48 61 36
Ext
413
Email
consuelomanciasg@gmail.com
First Name & Middle Initial & Last Name or Official Title & Degree
Oscar Gonzalez-Llano, MD
Phone
+52 86 75 67 18
Email
ogzzll25@hotmail.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Consuelo Mancias-Guerra, MD
Organizational Affiliation
Hospital Universitario Dr. Jose E. Gonzalez
Official's Role
Principal Investigator
Facility Information:
Facility Name
Hematology Service, Hospital Universitario Dr. Jose E. Gonzalez
City
Monterrey
State/Province
Nuevo Leon
ZIP/Postal Code
64460
Country
Mexico
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Consuelo Mancias-Guerra, MD
Phone
+52 81 83 48 61 36
Ext
413
Email
consuelomanciasg@gmail.com
First Name & Middle Initial & Last Name & Degree
David Gomez-Almaguer, MD
Phone
+52 81 83 48 61 36
Email
dgomezalmaguer@gmail.com
First Name & Middle Initial & Last Name & Degree
Consuelo Mancias-Guerra, MD

12. IPD Sharing Statement

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Links:
URL
http://www.hematologia-uanl.com/
Description
web page

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Intrathecal Stem Cells for Cerebral Palsy Phase II

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