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

NCT ID: NCT02881489

Last Updated: 2016-08-29

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE1
• Total Enrollment: 30 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-11-30
• Study Completion Date: 2018-12-31

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.

Conditions

Amyotrophic Lateral Sclerosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

Biological: Cell-based therapy

Intervention Type: OTHER

Human autologous bone marrow-derived mesenchymal stem cell transplantation in ALS patients.

Interventions

Biological: Cell-based therapy

Human autologous bone marrow-derived mesenchymal stem cell transplantation in ALS patients.

Intervention Type: OTHER

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of Warmia and Mazury

OTHER

Sponsor Role: lead

Responsible Party

Wojciech Maksymowicz

MD, PhD, Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Wojciech Maksymowicz, MD, Prof.

Role: PRINCIPAL_INVESTIGATOR

Faculty of Medical Sciences, University of Warmia and Mazury in Olsztyn, Poland

Other Identifiers

UWM/ALS-MSC.2015/002

Identifier Type: -

Identifier Source: org_study_id