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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03828123
• Date of registration: 20/07/2017
• Prospective Registration: No
• Primary sponsor: Bioinova, s.r.o.
• Public title: Autologous Multipotent Mesenchymal Stromal Cells in the Treatment of Amyotrophic Lateral Sclerosis AMSC-ALS-001
• Scientific title: A Prospective, Non-randomized, Open Label Study to Assess the Safety and the Efficacy of Autologous Multipotent Mesenchymal Stromal Cells in the Treatment of Amyotrophic Lateral Sclerosis
• Date of first enrolment: January 2012
• Target sample size: 26
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03828123
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Key inclusion & exclusion 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)



Age minimum: 18 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Motor Neuron Disease, Amyotrophic Lateral Sclerosis

Intervention(s)

Biological: Suspension of human autologous MSC 3P in 1.5 ml

Primary Outcome(s)

Safety: Complications related to the medicinal product application - new neurological deficit and occurrence of other adverse events [Time Frame: 1 year]

Secondary Outcome(s)

Efficacy: Inhibition of the disease progression - Norris scale [Time Frame: 18 months]

Efficacy: Inhibition of the disease progression - Forced vital capacity (FVC) [Time Frame: 18 months]

Efficacy: Inhibition of the disease progression - ALS functional rating scale [Time Frame: 18 months]

Secondary ID(s)

AMSC-ALS-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Department of Neurology, University Hospital Motol, Prague, Czech Republic