Clinical trial NCT02881489: Autologous Bone Marrow Mesenchymal Stem Cells in the Treatment of Patients With Amyotrophic Lateral Sclerosis
Official title: Evaluation of Mesenchymal Stem Cell Culturing Protocols in the Treatment of Amyotrophic Lateral Sclerosis
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
Conditions: Amyotrophic Lateral Sclerosis
Lead sponsor: University of Warmia and Mazury
Sponsor class: OTHER
First posted: 2016-08-29
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Changes in the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) between patients before and after stem cell transplantation.
timeFrame: 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
Secondary outcomes:
Other outcomes:
Locations:
Study officials:
affiliation: Faculty of Medical Sciences, University of Warmia and Mazury in Olsztyn, Poland
name: Wojciech Maksymowicz, MD, Prof.
role: PRINCIPAL_INVESTIGATOR
References: