Motor Neuron Disease, Amyotrophic Lateral SclerosisPhase 1/2AMSC-ALS-001NCT03828123
Autologous Multipotent Mesenchymal Stromal Cells in the Treatment of Amyotrophic Lateral Sclerosis
A Phase 1/2 study testing Suspension of human autologous MSC 3P in 1.5 ml in people with Motor Neuron Disease, Amyotrophic Lateral Sclerosis, run by Bioinova, s.r.o..
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Motor Neuron Disease, Amyotrophic Lateral Sclerosis
- What is being tested?
- Suspension of human autologous MSC 3P in 1.5 ml
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- No sites listed yet
- Study dates
- Jan 2012to 18 Aug 2017
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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,
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- 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)
About this study
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
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.
Read the 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.
- Being tested
- Suspension of human autologous MSC 3P in 1.5 ml
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Suspension of human autologous MSC 3P in 1.5 mlBiological
Intrathecal application of Autologous Multipotent Mesenchymal Stromal Cells 3P suspension
| Study group | Role | What they receive |
|---|---|---|
| Autologous Multipotent MSC | Experimental | Patients with intrathecal administration of Suspension of human autologous MSC 3P in 1.5 ml Receives: Biological: Suspension of human autologous MSC 3P in 1.5 ml |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 1 year
Secondary outcomes (3)
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):
- speech
- salivation
- swallowing
- handwriting
- cutting food and handling utensils (with or without gastrostomy)
- dressing and hygiene
- turning in bed and adjusting bed clothes
- walking
- climbing stairs
- breathing
ALSFRS = SUM (points for all 10 measures)
Interpretation:
minimum score: 0 maximum score: 40 The higher the score the more function is retained.
Measured: 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.
Measured: 18 months
Efficacy: Inhibition of the disease progression - Forced vital capacity (FVC)
FVC (%) will be measured at Visits I, and VI through X.
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Feb 2019
- Study startJan 2012
- Primary completion18 Aug 2017
- Study completion18 Aug 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Motor Neuron Disease, Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bioinova, s.r.o.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- University Hospital, Motol
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03828123
- Sponsor's protocol ID
- AMSC-ALS-001
- Acronym
- AMSC-ALS-001
- Status last verified
- Feb 2019
- First submitted
- 20 Jul 2017
- Last updated
- 12 Feb 2019
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 27938483
Study at a glance
From the registry record, last updated by the sponsor on 12 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03828123
- Acronym / sponsor ID
- AMSC-ALS-001 / AMSC-ALS-001
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Bioinova, s.r.o.Industry (pharma, biotech, devices)
- Collaborators
- University Hospital, Motol
- Study start
- Jan 2012 (actual)
- Primary completion
- 18 Aug 2017 (actual)
- Study completion
- 18 Aug 2017 (actual)
- First posted
- 4 Feb 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Autologous Multipotent MSC | Experimental | Biological: Suspension of human autologous MSC 3P in 1.5 mlPatients with intrathecal administration of Suspension of human autologous MSC 3P in 1.5 ml |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (12 listed)
- 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,
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Suspension of human autologous MSC 3P in 1.5 mlIntrathecal application of Autologous Multipotent Mesenchymal Stromal Cells 3P suspension | Biological | Autologous Multipotent MSC |
Primary outcome
Safety: Complications related to the medicinal product application - new neurological deficit and occurrence of other adverse events
Time frame: 1 year
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.
3 secondary outcomes
Efficacy: Inhibition of the disease progression - ALS functional rating scale
Time frame: 18 months
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Feb 2019.
- Results publication: PubMed 27938483Sykova 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Suspension of human autologous MSC 3P in 1.5 ml in people with Motor Neuron Disease, Amyotrophic Lateral Sclerosis, run by Bioinova, s.r.o..
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 12 Feb 2019. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety: Complications related to the medicinal product application - new neurological deficit and occurrence of other adverse events (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03828123, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Feb 2019.