Clinical Efficacy of Autologous Mesenchymal Bone Marrow Stem Cells in Active & Progressive Multiple Sclerosis

NCT02166021 | Completed Phase 2

• 1 other identifier: MSC-MS-001-IL
• Study Type: interventional
• Target: 48
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to evaluate the clinical efficacy and the optimal way of administration of autologous mesenchymal bone marrow stem cells (MSC) compering intravenous injection and intrathecal injection vs. placebo, in active-progressive Multiple Sclerosis patients.

Conditions

Multiple Sclerosis (MS)

Keywords

Multiple Sclerosis (MS); Stem Cells

Outcome Measures

Primary Outcomes (2)

• Safety Assessment

  The proportions of the patients in the three treatment-groups (MSC-IV, MSC-IT and placebo) who experienced any adverse event.

  6 months for each treatment cycle; the two cycles (each of 6 months duration) will be combined together and provide a single measurement for each group

• Neurological efficacy

  The proportions of the patients with treatment failure (increase of the EDSS by 1 point for patients with baseline values of 5.0 or less and of 0.5 degree for baseline EDSS of more than 5.0), confirmed by two consecutive evaluations, in the three treatment-groups.

  6 months for each treatment cycle; the two cycles (each of 6 months duration) will be combined together and provide a single measurement for each group

Secondary Outcomes (15)

• EDSS score

  6 months for each treatment cycle; the two cycles (each of 6 months duration) will be combined together and provide a single measurement for each group

• Ambulation score

  6 months for each treatment cycle; the two cycles (each of 6 months duration) will be combined together and provide a single measurement for each group

• Functional scores

  6 months for each treatment cycle; the two cycles (each of 6 months duration) will be combined together and provide a single measurement for each group

• Single injection vs. repeated MSCs injection

  12 months: ie the total duration of the trial

• Relapse rate

  12 months: ie the total duration of the trial

Study Arms (3)

IT- Treated

EXPERIMENTAL

Injection to IT (Group 1). After 6 months, 8 patients (group 1A) will be treated with MSC once again in IT, and 8 additional patients (group 1B) will receive a placebo.

Biological: Mesenchymal stem cells

IV - Treated

EXPERIMENTAL

Injection to IV (Group 2). After 6 months, 8 patients (group 2A) will be treated with MSC once again in IV, and 8 additional patients (group 2B) will receive a placebo.

Biological: Mesenchymal stem cells

Placebo

PLACEBO COMPARATOR

Placebo at the first injection (group 3). After 6 months, 8 patients (group 3A) will be treated with MSC in IT, and 8 additional patients (group 3B) will be treated with MSC in IV.

Biological: Mesenchymal stem cells

Interventions

Mesenchymal stem cells BIOLOGICAL

A culture of purified MSCs was prepared under aseptic conditions, and cultured for 4 weeks, until they reached confluency, and were then harvested. After sterility was confirmed, the cells resuspended in normal saline at a concentration of 10 × 106/mL to 15 × 106/mL.

Also known as: Autologous MSC

IT- Treated; IV - Treated; Placebo

Eligibility Criteria

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

You may qualify if:

• Consenting patients fulfilling the Poser's clinical criteria for definite MS
• Age: 18-65, males and females
• Duration of disease: \>3 years
• Progressive form of MS: PPMS, SPMS (with/without relapses)
• EDSS score of 3.5 - 6.5
• Failure to currently available, registered - first and second line immunomodulatory treatments (at least one).
• Evidence for new activity of MS during the 3 months before the injection of MSC.

You may not qualify if:

• Patients suffering from significant cardiac, renal or hepatic failure or any other disease that may risk the patient or interfere with the ability to interpret the results
• Patients with active infections
• Patients with severe cognitive decline or inability to understand and sign the informed consent
• Patients who received any cellular treatment in the past

Sponsors & Collaborators

• Dimitrios Karussis: lead

Study Sites (1)

Hadassah Medical Organization

Jerusalem, 91120, Israel

Location

Related Publications (2)

• Petrou P, Kassis I, Ginzberg A, Hallimi M, Karussis D. Effects of Mesenchymal Stem Cell Transplantation on Cerebrospinal Fluid Biomarkers in Progressive Multiple Sclerosis. Stem Cells Transl Med. 2022 Mar 3;11(1):55-58. doi: 10.1093/stcltm/szab017.

  PMID: 35641166 DERIVED

• Petrou P, Kassis I, Levin N, Paul F, Backner Y, Benoliel T, Oertel FC, Scheel M, Hallimi M, Yaghmour N, Hur TB, Ginzberg A, Levy Y, Abramsky O, Karussis D. Beneficial effects of autologous mesenchymal stem cell transplantation in active progressive multiple sclerosis. Brain. 2020 Dec 1;143(12):3574-3588. doi: 10.1093/brain/awaa333.

  PMID: 33253391 DERIVED

MeSH Terms

Conditions

Multiple Sclerosis

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Hadas Lemberg, PhD

  Director, R&D Division

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Head of The Center for Multiple Sclerosis & Unit of Neuroimmunology

Model Details: Patients were randomised into three groups and treated intrathecally (IT) or intravenously (IV) with autologous MSCs or placebo. At 6-months, treatment groups were crossed over and patients re-treated with either MSC or placebo

Study Record Dates

• First Submitted: May 20, 2014
• First Posted: June 18, 2014
• Study Start: January 29, 2015
• Primary Completion: June 15, 2018
• Study Completion: December 24, 2018
• Last Updated: August 1, 2019

Record last verified: 2019-07

Locations

IL (1)