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

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ClinicalTrials.gov Identifier: NCT02881489
Recruitment Status : Enrolling by invitation
First Posted : August 29, 2016
Last Update Posted : August 29, 2016
Sponsor:
Information provided by (Responsible Party):
Wojciech Maksymowicz, University of Warmia and Mazury

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.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Other: Biological: Cell-based therapy Phase 1

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.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Evaluation of Mesenchymal Stem Cell Culturing Protocols in the Treatment of Amyotrophic Lateral Sclerosis
Study Start Date : November 2015
Estimated Primary Completion Date : April 2018
Estimated Study Completion Date : December 2018


Arm Intervention/treatment
Experimental: Autologous BM-MSCs injection
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.
Other: Biological: Cell-based therapy
Human autologous bone marrow-derived mesenchymal stem cell transplantation in ALS patients.




Primary Outcome Measures :
  1. Changes in the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) between patients before and after stem cell transplantation. [ 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 ]
    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.



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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02881489


Sponsors and Collaborators
University of Warmia and Mazury
Investigators
Principal Investigator: Wojciech Maksymowicz, MD, Prof. Faculty of Medical Sciences, University of Warmia and Mazury in Olsztyn, Poland

Responsible Party: Wojciech Maksymowicz, MD, PhD, Professor, University of Warmia and Mazury
ClinicalTrials.gov Identifier: NCT02881489     History of Changes
Other Study ID Numbers: UWM/ALS-MSC.2015/002
First Posted: August 29, 2016    Key Record Dates
Last Update Posted: August 29, 2016
Last Verified: August 2016
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Additional relevant MeSH terms:
Sclerosis
Amyotrophic Lateral Sclerosis
Motor Neuron Disease
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases