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

NCT03828123 | Completed Phase 1

• 1 other identifier: AMSC-ALS-001
• Study Type: interventional
• Target: 26
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Motor Neuron Disease, Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• Safety: Complications related to the medicinal product application - new neurological deficit and occurrence of other adverse events

  Complications at the site of intrathecal infusion of the medicinal product and no new neurological deficit (meningism, paraplegia, urinary incontinence) not attributed to the natural progression of the ALS disease will be recorded at Visits I, III, IV, V, VI, and IX. Occurrence of other potential adverse events, including headache, respiratory failure, leukocytosis, cervical spine stenosis, cystitis and hyperhydrosis will be evaluated on the severity scale (1=mild, 2=moderate, 3=severe). Brain and spinal cord MRI will be performed at Visits I and IX to exclude treatment-related tumor formation, pathological contrast enhancement or other structural pathology.

  1 year

Secondary Outcomes (3)

• Efficacy: Inhibition of the disease progression - ALS functional rating scale

  18 months

• Efficacy: Inhibition of the disease progression - Norris scale

  18 months

• Efficacy: Inhibition of the disease progression - Forced vital capacity (FVC)

  18 months

Study Arms (1)

Autologous Multipotent MSC

EXPERIMENTAL

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

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

Interventions

Suspension of human autologous MSC 3P in 1.5 ml BIOLOGICAL

Intrathecal application of Autologous Multipotent Mesenchymal Stromal Cells 3P suspension

Autologous Multipotent MSC

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• established diagnosis of definite ALS according to El Escorial criteria
• riluzole naive or stable dose for at least 2 months,
• life expectancy more than 2 years
• patients able to provide written informed consent.

You may not qualify if:

• FVC less than 70%
• in case of primary bulbar paralysis less than 15 points on Norris bulbar scale,
• less than 15 points on Norris spinal scale,
• pregnancy, breastfeeding
• coagulopathy,
• skin infection at the site of bone marrow aspiration or application of the cell product,
• gastrostomy,
• 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 .....),
• alcohol or drug abuse
• cancer.
• 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
• fertile men not using proven contraceptive measures including effective contraception of their partner (established oral contraception, intrauterine device, ligation of the uterine tube)

Sponsors & Collaborators

• Bioinova, s.r.o.: lead
• University Hospital, Motol: collaborator

Related Publications (1)

• 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.

  PMID: 27938483 RESULT

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 20, 2017
• First Posted: February 4, 2019
• Study Start: January 1, 2012
• Primary Completion: August 18, 2017
• Study Completion: August 18, 2017
• Last Updated: February 12, 2019

Record last verified: 2019-02