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Autologous Bone Marrow Mesenchymal Stem Cells in the Treatment of Patients With Amyotrophic Lateral Sclerosis (UwmBmmscALS)

Primary Purpose

Amyotrophic Lateral Sclerosis

Status
Unknown status
Phase
Phase 1
Locations
Study Type
Interventional
Intervention
Biological: Cell-based therapy
Sponsored by
University of Warmia and Mazury
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • diagnosis of the ALS disease before the cell transplantation (diagnose established following the El Escorial criteria for definite ALS)
  • good understanding of the protocol and willingness to consent
  • signed informed consent
  • disease duration: up to 2 years
  • FVC > 50% / pulmonologist certificate about respiratory function of the patient

Exclusion Criteria:

  • cancer,
  • autoimmune diseases
  • renal failure,
  • subject is a respiratory dependent.
  • subject unwilling or unable to comply with the requirements of the protocol
  • pregnancy, breastfeeding

Sites / Locations

    Arms of the Study

    Arm 1

    Arm Type

    Experimental

    Arm Label

    Autologous BM-MSCs injection

    Arm Description

    Intervention: Biological: Cell-based therapy of autologous bone marrow-derived mesenchymal stem cells which are transplanted intrathecally (via a standard lumbar puncture) into the ALS subjects.

    Outcomes

    Primary Outcome Measures

    Changes in the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) between patients before and after stem cell transplantation.
    ALSFRS is ordinal rating scale questionnaire (rating 0-4 for each question, 4 is most functional, 0-48 total) of 12 functional activities. The most functional total score is 48. The First time the ALSFRS questionnaire has been dane after enrolment of the patients and then after 6 months of observation and stem cell injections the ALSFRS has been dane every 2 months up to 1,5 year of follow-up period.

    Secondary Outcome Measures

    Full Information

    First Posted
    April 22, 2016
    Last Updated
    August 23, 2016
    Sponsor
    University of Warmia and Mazury
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    1. Study Identification

    Unique Protocol Identification Number
    NCT02881489
    Brief Title
    Autologous Bone Marrow Mesenchymal Stem Cells in the Treatment of Patients With Amyotrophic Lateral Sclerosis
    Acronym
    UwmBmmscALS
    Official Title
    Evaluation of Mesenchymal Stem Cell Culturing Protocols in the Treatment of Amyotrophic Lateral Sclerosis
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    August 2016
    Overall Recruitment Status
    Unknown status
    Study Start Date
    November 2015 (undefined)
    Primary Completion Date
    April 2018 (Anticipated)
    Study Completion Date
    December 2018 (Anticipated)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Principal Investigator
    Name of the Sponsor
    University of Warmia and Mazury

    4. Oversight

    Data Monitoring Committee
    No

    5. Study Description

    Brief Summary
    The goal of this study is to investigate the safety and tolerability of autologous bone marrow-derived mesenchymal stem cells administration in the individuals with diagnosed amyotrophic lateral sclerosis.
    Detailed Description
    Amyotrophic lateral sclerosis (ALS) is one of the progressive neurodegenerative disorders, affecting upper and lower motor neurons in the cerebral cortex, brainstem and spinal cord. Hence, the signs of damage motor neurons are both at the peripheral (eg. atrophy), and central (eg. spasticity) level. There is no effective treatment for ALS and the majority of patients die within 5 years after diagnosis, usually due to respiratory failure. Numerous studies on murine models revealed that mesenchymal stem cells (MSCs) successfully improve the clinical and pathological features of ALS. The goal of this nonrandomized, open label study is to investigate the safety and tolerability of autologous bone marrow-derived mesenchymal stem cell transplantation into the individuals with diagnosed amyotrophic lateral sclerosis. This clinical trial is conducted to test the therapeutic (neuroprotective and paracrine) effect of autologous bone marrow-derived mesenchymal stem cells (BM-MSCs). All patients enrolled will have a documented history of ALS disease prior to study enrollment. Patients are recruited for a clinical trial not longer than 1 year from disease diagnosis. Then, patients are divided into two groups: Group I - patients receiving intrathecally one application of BM-MSCs and Group II - patients receiving intrathecally three applications (each administration every two months) of BM-MSCs. Subsequently, autologous bone marrow-derived mesenchymal stem cell transplantation to the cerebrospinal fluid at the site of the spinal cord will be performed. Finally, treatment safety, adverse events and exploratory parameters, including electromyographic (EMG) studies, forced vital capacity (FVC) and functional rating scale (FRS) to establish ALS progression rate will be recorded throughout the duration and in the post-treatment follow up period.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Amyotrophic Lateral Sclerosis

    7. Study Design

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

    8. Arms, Groups, and Interventions

    Arm Title
    Autologous BM-MSCs injection
    Arm Type
    Experimental
    Arm Description
    Intervention: Biological: Cell-based therapy of autologous bone marrow-derived mesenchymal stem cells which are transplanted intrathecally (via a standard lumbar puncture) into the ALS subjects.
    Intervention Type
    Other
    Intervention Name(s)
    Biological: Cell-based therapy
    Intervention Description
    Human autologous bone marrow-derived mesenchymal stem cell transplantation in ALS patients.
    Primary Outcome Measure Information:
    Title
    Changes in the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) between patients before and after stem cell transplantation.
    Description
    ALSFRS is ordinal rating scale questionnaire (rating 0-4 for each question, 4 is most functional, 0-48 total) of 12 functional activities. The most functional total score is 48. The First time the ALSFRS questionnaire has been dane after enrolment of the patients and then after 6 months of observation and stem cell injections the ALSFRS has been dane every 2 months up to 1,5 year of follow-up period.
    Time Frame
    From day of enrolment until the date of first stem cell injection (6 months - first time ALSFRS) + and then every 2 months up to 1,5 year of the trial

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Maximum Age & Unit of Time
    65 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: diagnosis of the ALS disease before the cell transplantation (diagnose established following the El Escorial criteria for definite ALS) good understanding of the protocol and willingness to consent signed informed consent disease duration: up to 2 years FVC > 50% / pulmonologist certificate about respiratory function of the patient Exclusion Criteria: cancer, autoimmune diseases renal failure, subject is a respiratory dependent. subject unwilling or unable to comply with the requirements of the protocol pregnancy, breastfeeding
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Wojciech Maksymowicz, MD, Prof.
    Organizational Affiliation
    Faculty of Medical Sciences, University of Warmia and Mazury in Olsztyn, Poland
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Plan to Share IPD
    Undecided

    Learn more about this trial

    Autologous Bone Marrow Mesenchymal Stem Cells in the Treatment of Patients With Amyotrophic Lateral Sclerosis

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