Autologous Multipotent Mesenchymal Stromal Cells in the Treatment of Amyotrophic Lateral Sclerosis

NCT ID: NCT03828123

Last Updated: 2019-02-12

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 26 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-01-31
• Study Completion Date: 2017-08-18

Brief Summary

Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disease that targets motor neurons. Prognosis is invariably fatal within 3-5 years since manifestation of the disease. Despite improved understanding of the mechanisms underlying ALS, the treatment remains essentially only supportive and focused on symptoms relief. Over the past few years, stem cell research has expanded greatly as a tool for developing new therapies to treat incurable diseases. Stem cell therapy has been shown as promising in several animal ALS models and human clinical trials.

Detailed Description

Subjects will be assigned to autologous mesenchymal stromal cell (AMSC) treatment according to inclusion and exclusion criteria (see below) screened four times prior to administration. Then the subjects will be observed for three consecutive yearsAfter a half year of screening period, the autologous multipotent mesenchymal stromal cells from bone marrow will be isolated. The cells will be cultivated for 3 passages (3 - 4 weeks) in order to get sufficient amount for therapy, cell suspension for intrathecal application will be prepared and introduced intrathecally through lumbar puncture. Subsequently, all the subjects will be observed at the range of standard medical care used at these types of interventions.

Conditions

Motor Neuron Disease, Amyotrophic Lateral Sclerosis

Study Design

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

Study Groups

Autologous Multipotent MSC

Patients with intrathecal administration of Suspension of human autologous MSC 3P in 1.5 ml

Group Type: EXPERIMENTAL

Suspension of human autologous MSC 3P in 1.5 ml

Intervention Type: BIOLOGICAL

Intrathecal application of Autologous Multipotent Mesenchymal Stromal Cells 3P suspension

Interventions

Suspension of human autologous MSC 3P in 1.5 ml

Intrathecal application of Autologous Multipotent Mesenchymal Stromal Cells 3P suspension

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

1. established diagnosis of definite ALS according to El Escorial criteria

2. riluzole naive or stable dose for at least 2 months,

3. life expectancy more than 2 years

4. patients able to provide written informed consent.

Exclusion Criteria

1. FVC less than 70%

2. in case of primary bulbar paralysis less than 15 points on Norris bulbar scale,

3. less than 15 points on Norris spinal scale,

4. pregnancy, breastfeeding

5. coagulopathy,

6. skin infection at the site of bone marrow aspiration or application of the cell product,

7. gastrostomy,

8. any significant medical condition that would compromise the safety of the patient (e.g. recent myocardial infarction, congestive heart failure, renal failure, liver failure, cancer, systemic infection, recurrent thromboembolic disease .....),

9. alcohol or drug abuse

10. cancer.

11. women of childbearing potential not using effective contraception (established oral contraception, intrauterine device, ligation of the uterine tube) including proven contraceptive measures taken by their sexual partners

12. fertile men not using proven contraceptive measures including effective contraception of their partner (established oral contraception, intrauterine device, ligation of the uterine tube)

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University Hospital, Motol

OTHER

Sponsor Role: collaborator

Bioinova, s.r.o.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

References

Sykova E, Rychmach P, Drahoradova I, Konradova S, Ruzickova K, Vorisek I, Forostyak S, Homola A, Bojar M. Transplantation of Mesenchymal Stromal Cells in Patients With Amyotrophic Lateral Sclerosis: Results of Phase I/IIa Clinical Trial. Cell Transplant. 2017 Apr 13;26(4):647-658. doi: 10.3727/096368916X693716. Epub 2016 Nov 7.

Reference Type: RESULT

PMID: 27938483 (View on PubMed)

Other Identifiers

AMSC-ALS-001

Identifier Type: -

Identifier Source: org_study_id