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

Polski Bank Komorek Macierzystych JSC (PBKM)

Status and phase: Unknown; Phase 2; Phase 1

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: Mesenchymal stem cells isolated from Wharton's jelly

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT04651855

ALSTEM

Details and patient eligibility

About

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.

Full 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)

Enrollment: 20 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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)

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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20 participants in 1 patient group

Treatment arm

Experimental group

Description:

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.

Treatment:

Drug: Mesenchymal stem cells isolated from Wharton's jelly