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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01777646
• Date of registration: 12/12/2012
• Prospective Registration: No
• Primary sponsor: Brainstorm-Cell Therapeutics
• Public title: Autologous Mesenchymal Bone Marrow Stromal Cells Secreting Neurotrophic Factors (MSC-NTF), in Patients With Amyotrophic Lateral Sclerosis (ALS)
• Scientific title: A Phase IIa, Open Label, Dose-escalating Clinical Study to Evaluate the Safety, Tolerability and Therapeutic Effects of Transplantation of Autologous Mesenchymal Bone Marrow Stromal Cells Secreting Neurotrophic Factors (MSC-NTF), in Patients With Amyotrophic Lateral Sclerosis (ALS).
• Date of first enrolment: December 2012
• Target sample size: 14
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01777646
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Israel

Contacts

• Name: Dimitrios Karusis, MD, PhD
• Affiliation: Hadassah Medical Organization

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Patient must fulfill El Escorial criteria (APPENDIX 2) for definite or probable ALS
(sporadic and not familial)

2. Participants, either men or non pregnant women are between 20 and 75 years of age.

3. The patient is mentally intact and psychologically stable

4. Patients will be with an ALS-FRS-R scale (APPENDIX 3) of at least 30 and disease
duration of less than 2 years.

5. Patient has sufficiently bulky muscles.

6. Participant understands the nature of the procedure and provides written informed
consent prior to any study procedure.

Exclusion Criteria:

1. Patient has a positive test for HBV, HCV or HIV.

2. Patient has high protein in the CSF (Protein > 70 mg/ml).

3. Patient has lymphocytosis in the CSF (lymphocytes > 5/ml).

4. Patient is positive for anti-GM1 antibodies.

5. Patient has significant conduction blocks or slow nerve conduction velocities (a
reduction of more than 30%) confirmed by nerve conduction velocity - EMG studies.

6. The patient is a respiratory dependent patient.

7. Patients with renal failure (Cr > 2 mg/dl).

8. Patients with impaired hepatic function (ALT, AST or GGT 2-fold higher than normal
upper limit).

9. 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

10. Patient with active infections.

11. Participation in another clinical trial within 1 month prior to start of this study.

12. Patient has not been treated previously with any cellular therapy.

13. Subject unwilling or unable to comply with the requirements of the protocol.



Age minimum: 20 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

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

Primary Outcome(s)

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) [Time Frame: At baseline and up to 6 month following treatment administration]

Secondary Outcome(s)

Changes in muscle strength grading (MVIC) by muscle chart [Time Frame: At baseline and up to 6 month following treatment administration]

Changes in EMG parameters [Time Frame: At baseline and up to 6 month following treatment administration]

Changes in the progression rate of the disease as evidenced by changes in the ALS functional rating scale [Time Frame: At baseline and up to 6 month following treatment administration]

Change in upper and lower extremities circumference (cm) [Time Frame: At baseline and up to 6 month following treatment administration]

Changes in muscle bulk estimated by MRI of the upper and lower extremities [Time Frame: At baseline and up to 6 month following treatment administration]

Secondary ID(s)

MSC-NTF-002-HMO-CTIL

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Hadassah Medical Organization