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Autologous Multipotent Mesenchymal Stromal Cells in the Treatment of Amyotrophic Lateral Sclerosis (AMSC-ALS-001)

ClinicalTrials.gov ID NCT03828123
Sponsor Bioinova, s.r.o.
Information provided by Bioinova, s.r.o. (Responsible Party)
Last Update Posted 2019-02-12
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Study Overview

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.
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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.
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Official 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
Conditions
Motor Neuron Disease, Amyotrophic Lateral Sclerosis
Intervention / Treatment
  • Biological: Suspension of human autologous MSC 3P in 1.5 ml
  • Biological: Suspension of human autologous MSC 3P in 1.5 ml
Other Study ID Numbers
  • AMSC-ALS-001
Study Start (Actual)
2012-01
Primary Completion (Actual)
2017-08-18
Study Completion (Actual)
2017-08-18
Enrollment (Actual)
26
Study Type
Interventional
Phase
Phase 1Phase 2

Contacts and Locations

This section provides contact details for people who can answer questions about joining this study, and information on where this study is taking place.

To learn more, please see the Contacts and Locations section in How to Read a Study Record(https://clinicaltrials.gov/study-basics/how-to-read-study-record#contacts-and-locations).

No location data

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies(https://clinicaltrials.gov/study-basics/learn-about-studies).
Eligibility Criteria
Description

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)
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Ages Eligible for Study
18 Years to 65 Years (AdultOlder Adult )
Sexes Eligible for Study
All
Accepts Healthy Volunteers
No

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

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Design Details
Primary Purpose : Treatment
Allocation : N/A
Interventional Model : Single Group Assignment
Masking : None (Open Label)

Arms and Interventions

Participant Group/Arm Intervention/Treatment
Participant Group/Arm Experimental: Autologous Multipotent MSC
Patients with intrathecal administration of Suspension of human autologous MSC 3P in 1.5 ml
Intervention/Treatment Biological: Suspension of human autologous MSC 3P in 1.5 ml
  • Intrathecal application of Autologous Multipotent Mesenchymal Stromal Cells 3P suspension

Primary Outcome Measures
Outcome Measure Measure Description Time Frame
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 Outcome Measures
Outcome Measure Measure Description Time Frame
Efficacy: Inhibition of the disease progression - ALS functional rating scale

Inhibition of the disease progression will be recorded by ALS functional rating scale (ALSFRS) at Visits I, III, and VI through X.

Measures (all 4-0):

  1. speech
  2. salivation
  3. swallowing
  4. handwriting
  5. cutting food and handling utensils (with or without gastrostomy)
  6. dressing and hygiene
  7. turning in bed and adjusting bed clothes
  8. walking
  9. climbing stairs
  10. breathing

ALSFRS = SUM (points for all 10 measures)

Interpretation:

minimum score: 0 maximum score: 40 The higher the score the more function is retained.

18 months
Efficacy: Inhibition of the disease progression - Norris scale

Inhibition of the disease progression will be recorded by Norris scale at Visits I, III, and VI through X.

Norris scal has has 22 items examining bulbar, respiratory, trunk, arm, leg, and general domains involving reflexes, fasciculation, and muscle atrophy. The scale also measures emotional lability, fatigability and leg rigidity. The Norris scale has a linear decline during the course of ALS.

18 months
Efficacy: Inhibition of the disease progression - Forced vital capacity (FVC)FVC (%) will be measured at Visits I, and VI through X.18 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.
Sponsor
Bioinova, s.r.o.
Collaborators
  • Department of Neurology, University Hospital Motol, Prague, Czech Republic

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Registration Dates
First Submitted
2017-07-20
First Submitted that Met QC Criteria
2019-02-01
First Posted
2019-02-04
Study Record Updates
Last Update Submitted that met QC Criteria
2019-02-11
Last Update Posted
2019-02-12
Last Verified
2019-02

More Information

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Additional Relevant MeSH Terms