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Bone Marrow-Derived Stem Cell Transplantation for the Treatment of Cerebral Palsy

Primary Purpose

Cerebral Palsy

Status
Unknown status
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
Stem Cell Therapy
Sponsored by
Stem Cells Arabia
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cerebral Palsy focused on measuring stem cells

Eligibility Criteria

2 Years - 12 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Must be more than 2 year of age and less than 12 years of age at the time of screening for inclusion in the study.
  • Clinical evidence of a non-progressive motor disability due to brain dysfunction.
  • Willing to comply with all study procedures.
  • Gross Motor Function Classification Score level II-V

Exclusion Criteria:

Known history of:

  • Intractable seizures
  • Traumatic brain injury
  • Genetic disorder
  • Current Infection
  • Renal insufficiency
  • Hepatic disease
  • HIV+ (as demonstrated by positive blood test)
  • Immunosuppression
  • Infectious related neurological injury
  • Other etiologies such as degenerative, mitochondrial, and metabolic disorders
  • Normal brain MRI
  • Evidence of acute illness such as fever (temperature > 37.5 C), vomiting, diarrhea, wheezing or crackles
  • Progressing neurological disease (Batten Disease, Leukodystrophies, Neurotransmitter disorders)
  • Microcephaly, macrocephaly, cortical malformations, or genetic disorders of dysgenesis
  • Pulmonary disease requiring ventilator support
  • Unwillingness to return for follow-up visits
  • Contraindications to MRI

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    Stem Cells

    Arm Description

    Intravenous and Intrathecal transplantation of specific populations of purified bone marrow-derived stem cells and mesenchymal stem cells.

    Outcomes

    Primary Outcome Measures

    Changes in Motor Performance.
    GMPM (Gross Motor Performance Measure) as a standard measurement tool for assessing quality of movement.

    Secondary Outcome Measures

    Motor function studies
    Total volumes and specific tract lesions will be studied and correlated with functional outcomes.
    Specific white matter tract analysis using MRI
    Changes on brain Diffusion Tensor Image (DTI).

    Full Information

    First Posted
    February 25, 2017
    Last Updated
    March 15, 2020
    Sponsor
    Stem Cells Arabia
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    1. Study Identification

    Unique Protocol Identification Number
    NCT03078621
    Brief Title
    Bone Marrow-Derived Stem Cell Transplantation for the Treatment of Cerebral Palsy
    Official Title
    Safety and Efficacy of Transplantation of Specific Populations of Stem Cells and Mesenchymal Stem Cells for the Treatment of Cerebral Palsy
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    March 2020
    Overall Recruitment Status
    Unknown status
    Study Start Date
    September 2016 (undefined)
    Primary Completion Date
    October 2020 (Anticipated)
    Study Completion Date
    January 2021 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Stem Cells Arabia

    4. Oversight

    Data Monitoring Committee
    No

    5. Study Description

    Brief Summary
    This study is a single arm, single center trial to study the safety and efficacy of purified autologous bone marrow derived stem cells and mesenchymal stem cells for the treatment of children with cerebral palsy.
    Detailed Description
    Cerebral palsy (CP) is a chronic childhood disorder defined by a range of motor and cognitive impairments and results in a substantial suffering to the patient and a tremendous socio-economic burden to the individual, family, and healthcare system. With no effective treatments or interventions, therapies for CP are currently focused on supportive and management strategies. Stem cell transplantation has been suggested as a putative intervention for neural pathology, as mesenchymal and neural stem cells, as well as olfactory ensheathing glia and Schwann cells, which have shown some regenerative and functional efficacy in experimental central nervous system disorders. In this trial, the investigators study the safety and efficacy of the intrathecal transplantation of specific populations of purified autologous bone marrow-derived stem cells and mesenchymal stem cells in patients with cerebral palsy.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Cerebral Palsy
    Keywords
    stem cells

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 1, Phase 2
    Interventional Study Model
    Single Group Assignment
    Masking
    None (Open Label)
    Allocation
    N/A
    Enrollment
    50 (Anticipated)

    8. Arms, Groups, and Interventions

    Arm Title
    Stem Cells
    Arm Type
    Experimental
    Arm Description
    Intravenous and Intrathecal transplantation of specific populations of purified bone marrow-derived stem cells and mesenchymal stem cells.
    Intervention Type
    Biological
    Intervention Name(s)
    Stem Cell Therapy
    Intervention Description
    Intravenous and Intrathecal transplantation of purified autologous bone marrow-derived stem cells and mesenchymal stem cells.
    Primary Outcome Measure Information:
    Title
    Changes in Motor Performance.
    Description
    GMPM (Gross Motor Performance Measure) as a standard measurement tool for assessing quality of movement.
    Time Frame
    6 months
    Secondary Outcome Measure Information:
    Title
    Motor function studies
    Description
    Total volumes and specific tract lesions will be studied and correlated with functional outcomes.
    Time Frame
    1 year
    Title
    Specific white matter tract analysis using MRI
    Description
    Changes on brain Diffusion Tensor Image (DTI).
    Time Frame
    6 months

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    2 Years
    Maximum Age & Unit of Time
    12 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: Must be more than 2 year of age and less than 12 years of age at the time of screening for inclusion in the study. Clinical evidence of a non-progressive motor disability due to brain dysfunction. Willing to comply with all study procedures. Gross Motor Function Classification Score level II-V Exclusion Criteria: Known history of: Intractable seizures Traumatic brain injury Genetic disorder Current Infection Renal insufficiency Hepatic disease HIV+ (as demonstrated by positive blood test) Immunosuppression Infectious related neurological injury Other etiologies such as degenerative, mitochondrial, and metabolic disorders Normal brain MRI Evidence of acute illness such as fever (temperature > 37.5 C), vomiting, diarrhea, wheezing or crackles Progressing neurological disease (Batten Disease, Leukodystrophies, Neurotransmitter disorders) Microcephaly, macrocephaly, cortical malformations, or genetic disorders of dysgenesis Pulmonary disease requiring ventilator support Unwillingness to return for follow-up visits Contraindications to MRI

    12. IPD Sharing Statement

    Plan to Share IPD
    Undecided

    Learn more about this trial

    Bone Marrow-Derived Stem Cell Transplantation for the Treatment of Cerebral Palsy

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