Biological Treatment of Amyotrophic Lateral Sclerosis

NCT ID: NCT02193893

Last Updated: 2014-07-18

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE1
• Total Enrollment: 50 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-01-31
• Study Completion Date: 2017-12-31

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.

Conditions

Amyotrophic Lateral Sclerosis

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: ACTIVE_COMPARATOR

Biological: Cell-based therapeutics

Intervention Type: OTHER

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

Standard treatment of ALS

Symptomatic treatment of ALS without biologic cell-based treatment

Group Type: SHAM_COMPARATOR

Symptomatic treatment of ALS

Intervention Type: OTHER

Symptomatic neurological treatment of ALS

Interventions

Biological: Cell-based therapeutics

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

Intervention Type: OTHER

Symptomatic treatment of ALS

Symptomatic neurological treatment of ALS

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
• 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.

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

Sponsors

Pomeranian Medical University Szczecin

OTHER

Sponsor Role: lead

Responsible Party

Milosz Kawa

Principal Investigator: Boguslaw Machalinski, Professor and Chief, Department of General Pathology, Pomeranian Medical University, Pomeranian Medical University Szczecin

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Boguslaw Machalinski, MD, PhD

Role: STUDY_DIRECTOR

Pomeranian Medical University Szczecin

Przemyslaw Nowacki, MD, PhD

Role: STUDY_CHAIR

Pomeranian Medical University Szczecin

Locations

Department of Neurology of Pomeranian Medical University in Szczecin

Szczecin, Poland, Poland

Countries

Poland

References

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.

Reference Type: DERIVED

PMID: 31629403 (View on PubMed)

Other Identifiers

ZPO 02, ALS-BMSC #01

Identifier Type: OTHER

Identifier Source: secondary_id

ZPO 02

Identifier Type: -

Identifier Source: org_study_id