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Biological Treatment of Amyotrophic Lateral Sclerosis (NeuStem-ALS)

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ClinicalTrials.gov Identifier: NCT02193893
Recruitment Status : Unknown
Verified July 2014 by Milosz Kawa, Pomeranian Medical University Szczecin.
Recruitment status was:  Enrolling by invitation
First Posted : July 18, 2014
Last Update Posted : July 18, 2014
Sponsor:
Information provided by (Responsible Party):
Milosz Kawa, Pomeranian Medical University Szczecin

Brief Summary:
The purpose of this study is to test the safety and effectiveness of an autologous bone marrow-derived stem/progenitor cells infusion in the subjects with diagnosed amyotrophic lateral sclerosis.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Other: Biological: Cell-based therapeutics Other: Symptomatic treatment of ALS Phase 1

Detailed Description:
Amyotrophic lateral sclerosis (ALS) is a universally fatal neurodegenerative condition that causes weakness leading to paralysis and death. Life expectancy is less that 5 years. The cause is generally unknown and there is no effective treatment. Patients with ALS typically exhibit a progressive paralysis due to the continued loss of motoneurons. Intraspinal injections of bone marrow mononuclear cells have been able to ameliorate the course of ALS in murine models. The purpose of this prospective, nonrandomized, open label, pilot study is to conduct the investigation of the safety and efficacy of infusion of autologous bone marrow-isolated stem/progenitor cells with different selected phenotypes into the subjects with diagnosed ALS. Especially, this clinical trial is designated to test the therapeutic (pro-regenerative and neuro-protective) functions of different stem/progenitor cell populations able to secrete bioactive neurotrophic factors. All patients enrolled will have a documented history of ALS disease prior to study enrollment. Patients diagnosed as early stage of ALS with the duration of disease less than 6 months and patients diagnosed with advanced stage of ALS disease with duration of 6-12 months will be recruited and allocated based on their disease severity to two treatment groups: Group I - patients of early ALS disease stage and Group II - patients of advanced ALS disease. Next, autologous bone marrow-isolated stem/progenitor cells administration 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), functional rating scale (FRS) and maximum voluntary isometric contraction-arm (MVIC-arm) evaluation, to establish ALS progression rate will be recorded throughout the duration of the post-treatment follow up period.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety/Efficacy Study for the Biological Treatment of Amyotrophic Lateral Sclerosis With Autologous Stem/Progenitor Cells
Study Start Date : January 2010
Estimated Primary Completion Date : July 2017
Estimated Study Completion Date : December 2017


Arm Intervention/treatment
Active Comparator: Stem/progenitor cells transplantation.
Intervention: Biological: Cell-based therapeutics Autologous bone marrow-derived stem/progenitor cells will be transplanted intrathecally (via a standard lumbar puncture) into early vs. progressive ALS subjects.
Other: Biological: Cell-based therapeutics
Human autologous bone marrow-derived stem/progenitor cell transplantation in ALS patients.

Sham Comparator: Standard treatment of ALS
Symptomatic treatment of ALS without biologic cell-based treatment
Other: Symptomatic treatment of ALS
Symptomatic neurological treatment of ALS




Primary Outcome Measures :
  1. Safety of autologous bone marrow stem/progenitor cell infusion in enrolled patients [ Time Frame: 1 year ]
    Confirm the safety of autologous bone marrow stem/progenitor cell infusion in enrolled patients by repeated follow-up over one year with clinical and laboratory evaluations.


Secondary Outcome Measures :
  1. Efficacy of autologous bone marrow stem/progenitor cell infusion in enrolled patients. [ Time Frame: 1 year ]
    Confirm the efficacy of autologous bone marrow stem/progenitor cell infusion in enrolled patients by repeated follow-up over one year with clinical and laboratory evaluation tests for neurodegeneration processes analysis and general health outcomes.



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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
  • patient is mentally intact and psychologically stable
  • signed informed consent

Exclusion Criteria:

Concomitant of other systemic disease or diseases:

  • inflammation (high protein or lymphocytosis in the CSF), active infections.
  • diabetes,
  • cardio-vascular disorders,
  • cancer,
  • autoimmune diseases
  • renal failure,
  • impaired hepatic function.
  • subject is a respiratory dependent.
  • subject unwilling or unable to comply with the requirements of the protocol.
  • patient has been treated previously with any cellular therapy.

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): NCT02193893


Locations
Poland
Department of Neurology of Pomeranian Medical University in Szczecin
Szczecin, Poland, 71-252
Sponsors and Collaborators
Pomeranian Medical University Szczecin
Investigators
Study Director: Boguslaw Machalinski, MD, PhD Pomeranian Medical University Szczecin
Study Chair: Przemyslaw Nowacki, MD, PhD Pomeranian Medical University Szczecin

Responsible Party: Milosz Kawa, Principal Investigator: Boguslaw Machalinski, Professor and Chief, Department of General Pathology, Pomeranian Medical University, Pomeranian Medical University Szczecin, Pomeranian Medical University Szczecin
ClinicalTrials.gov Identifier: NCT02193893     History of Changes
Other Study ID Numbers: ZPO 02
ZPO 02, ALS-BMSC #01 ( Other Identifier: Department of General Pathology, PMU in Szczecin )
First Posted: July 18, 2014    Key Record Dates
Last Update Posted: July 18, 2014
Last Verified: July 2014

Keywords provided by Milosz Kawa, Pomeranian Medical University Szczecin:
cell-based therapy
autologous bone marrow-derived stem/progenitor cells
stem cell infusion, stem cell transplantation
biological therapy with neurotrophic factors
bioactive neurotrophic factors

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
Coal Tar
Keratolytic Agents
Dermatologic Agents