Biological Treatment of Amyotrophic Lateral Sclerosis

Amyotrophic Lateral Sclerosis Clinical Trial

— NeuStem-ALS  

Official title:

Safety/Efficacy Study for the Biological Treatment of Amyotrophic Lateral Sclerosis With Autologous Stem/Progenitor Cells

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT02193893
• Other study ID #: ZPO 02
• Secondary ID: ZPO 02, ALS-BMSC
• Status: Enrolling by invitation
• Phase: Phase 1
• First received: July 16, 2014
• Last updated: July 17, 2014
• Start date: January 2010
• Est. completion date: December 2017

Study information

• Verified date: July 2014
• Source: Pomeranian Medical University Szczecin
• Contact: n/a
• Is FDA regulated: No
• Health authority: Poland: Ethics Committee
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 50
• Est. completion date: December 2017
• Est. primary completion date: July 2017
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 65 Years

Eligibility

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

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Other:
• Biological: Cell-based therapeutics
Human autologous bone marrow-derived stem/progenitor cell transplantation in ALS patients.
• Symptomatic treatment of ALS
Symptomatic neurological treatment of ALS

Locations

Country	Name	City	State
Poland	Department of Neurology of Pomeranian Medical University in Szczecin	Szczecin

Sponsors (1)

Lead Sponsor	Collaborator
Pomeranian Medical University Szczecin

Country where clinical trial is conducted

Poland, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety of autologous bone marrow stem/progenitor cell infusion in enrolled patients	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.	1 year	Yes
Secondary	Efficacy of autologous bone marrow stem/progenitor cell infusion in enrolled patients.	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.	1 year	No