The Evaluation of the Effect of Mesenchymal Stem Cells on the Immune System of Patients With ALS

• Status: Recruiting
• Trial end date: 2023-02-01
• Target enrollment: 0
• Participant gender: All

Summary

The objective of this study is to evaluate the safety of intrathecal administration of Wharton's Jelly Mesenchymal Stem Cells (WJMSC) and the impact on the immune system of patients with Amyotrophic Lateral Sclerosis.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Polski Bank Komorek Macierzystych JSC (PBKM)
• Collaborators: National Center for Research and Development, Poland; The National Centre for Research and Development

Criteria

Inclusion Criteria:

1. Adult patients (at least 18 years old)

2. The minimum patient's weight is not less than 40 kg

3. Diagnosis of sporadic ALS, definite or probable, as defined by El Escorial World Federation of Neurology criteria

4. History of ALS symptoms less than 2 years duration from the first symptoms of the disease

5. More than 6 months from diagnosis of the disease

6. Disease progression at 6 past months at least 3 points during this period of time

7. ALSFRS-R scale of at least 30 at screening appointment

8. Forced vital capacity >70% of predicted value for age, gender and height

9. Treatment with stable dose of riluzole before baseline visit (for at least 1 month)

10. Capable of providing written informed consent

11. Able to comply with study requirements and willing to follow all study procedures and follow-up visits

12. Women of child-bearing age and men with partners of child-bearing potential must agree to use two forms of contraceptive therapy throughout the course of the trial

13. Women of child-bearing age must undergo pregnancy test

14. Polish-language native speakers or patients who are proficient in the Polish language

Exclusion Criteria:

1. Pregnancy or breastfeeding

2. Tracheostomy

3. Ventilator dependence

4. Renal disease with creatinine >2mg/dl

5. Liver disease with ALT, AST or GGTP 2-fold higher than upper normal limit

6. Positive test for HBV, HCV, HIV with NAT method

7. Positive tests for syphilis

8. Any other clinically significant abnormalities on laboratory evaluation

9. Any condition that would compromise ability of undergoing lumbar puncture

10. Active systemic disease

11. Autoimmune disease (Hashimoto disease under control is allowed)

12. Uncontrolled diabetes

13. Pulmonary disease that could affect interpretation of spirometry

14. Neurological concomitant disease

15. Unstable psychiatric concomitant disease

16. High risk of suicide

17. History of substance abuse within past year

18. History of malignancy, within the previous 5 years, including melanoma with exception of localized skin cancers

19. Any other clinically significant medical condition that can compromise patient's safety in the opinion of the investigator

20. Treatment with immunomodulatory drugs (for example immunoglobulins, corticosteroids or other immunosuppressant) in last 6 months

21. Participation in another clinical trial in last 6 months

22. Previous cellular therapy of any kind

23. Hypersensitivity to any component used in the cell culture

24. Nuchal rigidity and other signs of meningitis

25. Patients on chronic anticoagulation treatment (heparin/ warfarin/acenocoumarol/(N)OAC)