Autologous Mesenchymal Bone Marrow Stromal Cells Secreting Neurotrophic Factors (MSC-NTF), in Patients With Amyotrophic Lateral Sclerosis (ALS)

NCT01777646 | Completed Phase 2

• 1 other identifier: MSC-NTF-002-HMO-CTIL
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 1

Brief Summary

The study will evaluate the safety, tolerability and therapeutic effects of transplantation of escalating doses of autologous cultured mesenchymal bone marrow stromal cells secreting neurotrophic factors (MSC-NTF), in patients with amyotrophic lateral sclerosis (ALS).

Conditions

Amyotrophic Lateral Sclerosis

Keywords

Mesenchymal Strmal Cells (MSC); Amyotrophic Lateral Sclerosis (ALS); Neuroprotection; Neurodegeneration; Stem cells

Outcome Measures

Primary Outcomes (1)

• Safety evaluation and tolerability of a single treatment administration in an escalating-dose of autologous cultured mesenchymal bone marrow stromal cells secreting neurotrophic factors (MSC-NTF)

  At baseline and up to 6 month following treatment administration

Secondary Outcomes (5)

• Changes in the progression rate of the disease as evidenced by changes in the ALS functional rating scale

  At baseline and up to 6 month following treatment administration

• Changes in muscle strength grading (MVIC) by muscle chart

  At baseline and up to 6 month following treatment administration

• Changes in muscle bulk estimated by MRI of the upper and lower extremities

  At baseline and up to 6 month following treatment administration

• Change in upper and lower extremities circumference (cm)

  At baseline and up to 6 month following treatment administration

• Changes in EMG parameters

  At baseline and up to 6 month following treatment administration

Study Arms (1)

MSC-NTF

EXPERIMENTAL

Biological: MSC_NTF cells transplantation by multiple intramuscular injections at 24 separate sites, in addition to a single intrathechal injection into the CSF

Interventions

MSC_NTF cells transplantation by multiple intramuscular injections at 24 separate sites, in addition to a single intrathechal injection into the CSF BIOLOGICAL

MSC-NTF

Eligibility Criteria

• Age: 20 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patient must fulfill El Escorial criteria (APPENDIX 2) for definite or probable ALS (sporadic and not familial)
• Participants, either men or non pregnant women are between 20 and 75 years of age.
• The patient is mentally intact and psychologically stable
• Patients will be with an ALS-FRS-R scale (APPENDIX 3) of at least 30 and disease duration of less than 2 years.
• Patient has sufficiently bulky muscles.
• Participant understands the nature of the procedure and provides written informed consent prior to any study procedure.

You may not qualify if:

• Patient has a positive test for HBV, HCV or HIV.
• Patient has high protein in the CSF (Protein \> 70 mg/ml).
• Patient has lymphocytosis in the CSF (lymphocytes \> 5/ml).
• Patient is positive for anti-GM1 antibodies.
• Patient has significant conduction blocks or slow nerve conduction velocities (a reduction of more than 30%) confirmed by nerve conduction velocity - EMG studies.
• The patient is a respiratory dependent patient.
• Patients with renal failure (Cr \> 2 mg/dl).
• Patients with impaired hepatic function (ALT, AST or GGT 2-fold higher than normal upper limit).
• Patients suffering from significant cardiac disease, malignant disease or any other disease that may risk the patient or interfere with the ability to interpret the results
• Patient with active infections.
• Participation in another clinical trial within 1 month prior to start of this study.
• Patient has not been treated previously with any cellular therapy.
• Subject unwilling or unable to comply with the requirements of the protocol.

Sponsors & Collaborators

• Brainstorm-Cell Therapeutics: lead
• Hadassah Medical Organization: collaborator

Study Sites (1)

Hadassah Medical Organization

Jerusalem, 91120, Israel

Location

Related Publications (2)

• Gothelf Y, Kaspi H, Abramov N, Aricha R. miRNA profiling of NurOwn(R): mesenchymal stem cells secreting neurotrophic factors. Stem Cell Res Ther. 2017 Nov 7;8(1):249. doi: 10.1186/s13287-017-0692-1.

  PMID: 29116031 DERIVED

• Petrou P, Gothelf Y, Argov Z, Gotkine M, Levy YS, Kassis I, Vaknin-Dembinsky A, Ben-Hur T, Offen D, Abramsky O, Melamed E, Karussis D. Safety and Clinical Effects of Mesenchymal Stem Cells Secreting Neurotrophic Factor Transplantation in Patients With Amyotrophic Lateral Sclerosis: Results of Phase 1/2 and 2a Clinical Trials. JAMA Neurol. 2016 Mar;73(3):337-44. doi: 10.1001/jamaneurol.2015.4321.

  PMID: 26751635 DERIVED

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Nerve Degeneration

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Dimitrios Karusis, MD, PhD

  Hadassah Medical Organization

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 12, 2012
• First Posted: January 29, 2013
• Study Start: December 1, 2012
• Primary Completion: September 1, 2014
• Study Completion: September 1, 2015
• Last Updated: March 8, 2018

Record last verified: 2018-03

Locations

IL (1)