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

NCT ID: NCT04651855

Last Updated: 2022-05-03

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-12-02
• Study Completion Date: 2023-04-30

Brief 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.

Detailed Description

Clinical Phase: I/II

Population: Patients with Amyotrophic Lateral Sclerosis.

Project Design: One arm, non-blinded, open label study

Planned Sample Size: 20 patients

Investigational Medicinal Product: active IMP - mesenchymal stem cells isolated from Wharton's jelly

Screening:

Three visits on site to check the eligibility criteria (around 90, 60 and 30 days before first IMP administration)

Treatment (IMP administration):

Each patient will receive IMP three times: on baseline (day 0), 30 and 60 days after baseline (+/- 7 days).

Administration route: intrathecal

Follow up:

Duration: 18 months after first IMP administration Four on-site visits (3, 6, 9, 12 months after first IMP administration) and seven phone visits (4, 5, 7, 8, 10, 11 and 18 months after first IMP administration)

Conditions

Amyotrophic Lateral Sclerosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treatment arm

It is planned that IMP administration will be performed three times for each enrolled patient. IMP administration could be performed only if the patients does not have any contraindications for lumbar puncture.

Group Type: EXPERIMENTAL

Mesenchymal stem cells isolated from Wharton's jelly

Intervention Type: DRUG

Intrathecal administration of mesenchymal stem cells

Interventions

Mesenchymal stem cells isolated from Wharton's jelly

Intrathecal administration of mesenchymal stem cells

Intervention Type: DRUG

Eligibility 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 assessed in ALSFRS-R scale

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(2x 50mg per 24h) 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 (HbA1c > 8%)

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)

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Center for Research and Development, Poland

OTHER

Sponsor Role: collaborator

Polski Bank Komorek Macierzystych JSC (PBKM)

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

JST sp. z o.o.

Częstochowa, , Poland

Countries

Poland

Other Identifiers

ALSTEM

Identifier Type: -

Identifier Source: org_study_id