Clinical trial NCT02193893: Biological Treatment of Amyotrophic Lateral Sclerosis
Official title: Safety/Efficacy Study for the Biological Treatment of Amyotrophic Lateral Sclerosis With Autologous Stem/Progenitor Cells
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
Conditions: Amyotrophic Lateral Sclerosis
Keywords: cell-based therapy
autologous bone marrow-derived stem/progenitor cells
stem cell infusion, stem cell transplantation
biological therapy with neurotrophic factors
bioactive neurotrophic factors
Lead sponsor: Pomeranian Medical University Szczecin
Sponsor class: OTHER
First posted: 2014-07-18
Enrollment: 50
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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.

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
* 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.

Primary outcomes:
measure: Safety of autologous bone marrow stem/progenitor cell infusion in enrolled patients
timeFrame: 1 year
Secondary outcomes:
measure: Efficacy of autologous bone marrow stem/progenitor cell infusion in enrolled patients.
timeFrame: 1 year
Other outcomes:
Locations:
city: Szczecin
country: Poland
facility: Department of Neurology of Pomeranian Medical University in Szczecin
Study officials:
affiliation: Pomeranian Medical University Szczecin
name: Boguslaw Machalinski, MD, PhD
role: STUDY_DIRECTOR
affiliation: Pomeranian Medical University Szczecin
name: Przemyslaw Nowacki, MD, PhD
role: STUDY_CHAIR
References:
citation: Pawlukowska W, Baumert B, Golab-Janowska M, Meller A, Machowska-Sempruch K, Welnicka A, Paczkowska E, Rotter I, Machalinski B, Nowacki P. Comparative assessment and monitoring of deterioration of articulatory organs using subjective and objective tools among patients with amyotrophic lateral sclerosis. BMC Neurol. 2019 Oct 19;19(1):241. doi: 10.1186/s12883-019-1484-2.