Biological Treatment of Amyotrophic Lateral Sclerosis

Phase 1

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Other: Symptomatic treatment of ALS

• Registration Number: NCT02193893
• Lead Sponsor: 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.

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 Timeline

• Study Start: 2010-01
• Status Verified: 2014-07
• Study First Submitted: 2014-07-16
• Study First Submitted QC: 2014-07-17
• Last Update Submitted: 2014-07-17
• Study First Posted: 2014-07-18
• Last Update Posted: 2014-07-18
• Primary Completion: 2017-07
• Study Completion: 2017-12

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 50

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Standard treatment of ALS	Symptomatic treatment of ALS	Symptomatic treatment of ALS without biologic cell-based treatment

Primary Outcome Measures

Name	Time	Method
Safety of autologous bone marrow stem/progenitor cell infusion in enrolled patients	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

Name	Time	Method
Efficacy of autologous bone marrow stem/progenitor cell infusion in enrolled patients.	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.

Trial Locations

Locations (1)

Department of Neurology of Pomeranian Medical University in Szczecin; 🇵🇱 Szczecin, Poland