Amyotrophic Lateral SclerosisPhase 1NeuStem-ALSNCT02193893
Biological Treatment of Amyotrophic Lateral Sclerosis
A Phase 1 study testing Biological: Cell-based therapeutics and Symptomatic treatment of ALS in people with Amyotrophic Lateral Sclerosis, run by Pomeranian Medical University Szczecin at 1 site in Poland.
Trial summary
The purpose of this study is to test the safety and effectiveness of an autologous bone marrow-derived stem/progenitor cells infusion in the subjects with diagnosed amyotrophic lateral sclerosis. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Biological: Cell-based therapeutics and Symptomatic treatment of ALS
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 50 participants
- Where
- 1 sitePoland
- Study dates
- Jan 2010to Dec 2017
- Phase
- Phase 1
- 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
- diagnosis of the ALS disease before the cell transplantation (diagnose established following the El Escorial criteria for definite ALS)
- good understanding of the protocol and willingness to consent
- patient is mentally intact and psychologically stable
- signed informed consent
Who cannot join
- Concomitant of other systemic disease or diseases:
- inflammation (high protein or lymphocytosis in the CSF), active infections.
- diabetes,
- cardio-vascular disorders,
- cancer,
- autoimmune diseases
Show all 11Show fewer
- renal failure,
- impaired hepatic function.
- subject is a respiratory dependent.
- subject unwilling or unable to comply with the requirements of the protocol.
- patient has been treated previously with any cellular therapy.
Trial locations and contacts
1 site in 1 country.
Poland1 site
Department of Neurology of Pomeranian Medical University in Szczecin
Szczecin, Poland, 71-252
About this study
Official title: Safety/Efficacy Study for the Biological Treatment of Amyotrophic Lateral Sclerosis With Autologous Stem/Progenitor Cells
The purpose of this study is to test the safety and effectiveness of an autologous bone marrow-derived stem/progenitor cells infusion in the subjects with diagnosed amyotrophic lateral sclerosis.
Read the detailed description
Amyotrophic lateral sclerosis (ALS) is a universally fatal neurodegenerative condition that causes weakness leading to paralysis and death. Life expectancy is less that 5 years. The cause is generally unknown and there is no effective treatment. Patients with ALS typically exhibit a progressive paralysis due to the continued loss of motoneurons. Intraspinal injections of bone marrow mononuclear cells have been able to ameliorate the course of ALS in murine models. The purpose of this prospective, nonrandomized, open label, pilot study is to conduct the investigation of the safety and efficacy of infusion of autologous bone marrow-isolated stem/progenitor cells with different selected phenotypes into the subjects with diagnosed ALS. Especially, this clinical trial is designated to test the therapeutic (pro-regenerative and neuro-protective) functions of different stem/progenitor cell populations able to secrete bioactive neurotrophic factors. All patients enrolled will have a documented history of ALS disease prior to study enrollment. Patients diagnosed as early stage of ALS with the duration of disease less than 6 months and patients diagnosed with advanced stage of ALS disease with duration of 6-12 months will be recruited and allocated based on their disease severity to two treatment groups: Group I - patients of early ALS disease stage and Group II - patients of advanced ALS disease. Next, autologous bone marrow-isolated stem/progenitor cells administration to the cerebrospinal fluid at the site of the spinal cord will be performed. Finally, treatment safety, adverse events and exploratory parameters, including electromyographic (EMG) studies, forced vital capacity (FVC), functional rating scale (FRS) and maximum voluntary isometric contraction-arm (MVIC-arm) evaluation, to establish ALS progression rate will be recorded throughout the duration of the post-treatment follow up period.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Biological: Cell-based therapeutics, Symptomatic treatment of ALS
- Keywords
- cell-based therapy, autologous bone marrow-derived stem/progenitor cells, stem cell infusion, stem cell transplantation, biological therapy with neurotrophic factors, bioactive neurotrophic factors
- 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.
Biological: Cell-based therapeuticsOther
Human autologous bone marrow-derived stem/progenitor cell transplantation in ALS patients.
Symptomatic treatment of ALSOther
Symptomatic neurological treatment of ALS
| Study group | Role | What they receive |
|---|---|---|
| Stem/progenitor cells transplantation. | Active comparator | Intervention: Biological: Cell-based therapeutics Autologous bone marrow-derived stem/progenitor cells will be transplanted intrathecally (via a standard lumbar puncture) into early vs. progressive ALS subjects. Receives: Other: Biological: Cell-based therapeutics |
| Standard treatment of ALS | Sham comparator | Symptomatic treatment of ALS without biologic cell-based treatment Receives: Other: Symptomatic treatment of ALS |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of autologous bone marrow stem/progenitor cell infusion in enrolled patients
Confirm the safety of autologous bone marrow stem/progenitor cell infusion in enrolled patients by repeated follow-up over one year with clinical and laboratory evaluations.
Measured: 1 year
Secondary outcomes (1)
Efficacy of autologous bone marrow stem/progenitor cell infusion in enrolled patients.
Confirm the efficacy of autologous bone marrow stem/progenitor cell infusion in enrolled patients by repeated follow-up over one year with clinical and laboratory evaluation tests for neurodegeneration processes analysis and general health outcomes.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jul 2014 (estimated)
- Study startJan 2010
- Primary completionJul 2017 (estimated)
- Study completionDec 2017 (estimated)
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 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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Pomeranian Medical University Szczecin
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Milosz Kawa, Pomeranian Medical University Szczecin
- Investigators
- Boguslaw Machalinski, MD, PhD, Study director, Pomeranian Medical University SzczecinPrzemyslaw Nowacki, MD, PhD, Study chair, Pomeranian Medical University Szczecin
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02193893
- Sponsor's protocol ID
- ZPO 02
- Other IDs
- ZPO 02, ALS-BMSC #01
- Acronym
- NeuStem-ALS
- Status last verified
- Jul 2014
- First submitted
- 16 Jul 2014
- Last updated
- 18 Jul 2014
- Results posted
- No
- Data monitoring committee
- No
Publications
Pawlukowska W, Baumert B, Golab-Janowska M, Meller A, Machowska-Sempruch K, Welnicka A, Paczkowska E, Rotter I, Machalinski B, Nowacki P. Comparative assessment and monitoring of deterioration of articulatory organs using subjective and objective tools among patients with amyotrophic lateral sclerosis. BMC Neurol. 2019 Oct 19;19(1):241. doi: 10.1186/s12883-019-1484-2.
PubMed 31629403
Study at a glance
From the registry record, last updated by the sponsor on 18 Jul 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety/Efficacy Study for the Biological Treatment of Amyotrophic Lateral Sclerosis With Autologous Stem/Progenitor Cells
- ClinicalTrials.gov ID
- NCT02193893
- Acronym / sponsor ID
- NeuStem-ALS / ZPO 02
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- cell-based therapy, autologous bone marrow-derived stem/progenitor cells, stem cell infusion, stem cell transplantation, biological therapy with neurotrophic factors, bioactive neurotrophic factors
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Pomeranian Medical University SzczecinUniversity, hospital or other
- Study start
- Jan 2010
- Primary completion
- Jul 2017 (estimated)
- Study completion
- Dec 2017 (estimated)
- First posted
- 18 Jul 2014 (estimated)
- 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.
The purpose of this study is to test the safety and effectiveness of an autologous bone marrow-derived stem/progenitor cells infusion in the subjects with diagnosed amyotrophic lateral sclerosis.
Detailed description
Amyotrophic lateral sclerosis (ALS) is a universally fatal neurodegenerative condition that causes weakness leading to paralysis and death. Life expectancy is less that 5 years. The cause is generally unknown and there is no effective treatment. Patients with ALS typically exhibit a progressive paralysis due to the continued loss of motoneurons. Intraspinal injections of bone marrow mononuclear cells have been able to ameliorate the course of ALS in murine models. The purpose of this prospective, nonrandomized, open label, pilot study is to conduct the investigation of the safety and efficacy of infusion of autologous bone marrow-isolated stem/progenitor cells with different selected phenotypes into the subjects with diagnosed ALS. Especially, this clinical trial is designated to test the therapeutic (pro-regenerative and neuro-protective) functions of different stem/progenitor cell populations able to secrete bioactive neurotrophic factors. All patients enrolled will have a documented history of ALS disease prior to study enrollment. Patients diagnosed as early stage of ALS with the duration of disease less than 6 months and patients diagnosed with advanced stage of ALS disease with duration of 6-12 months will be recruited and allocated based on their disease severity to two treatment groups: Group I - patients of early ALS disease stage and Group II - patients of advanced ALS disease. Next, autologous bone marrow-isolated stem/progenitor cells administration to the cerebrospinal fluid at the site of the spinal cord will be performed. Finally, treatment safety, adverse events and exploratory parameters, including electromyographic (EMG) studies, forced vital capacity (FVC), functional rating scale (FRS) and maximum voluntary isometric contraction-arm (MVIC-arm) evaluation, to establish ALS progression rate will be recorded throughout the duration of the post-treatment follow up period.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Stem/progenitor cells transplantation. | Active comparator | Other: Biological: Cell-based therapeuticsIntervention: Biological: Cell-based therapeutics Autologous bone marrow-derived stem/progenitor cells will be transplanted intrathecally (via a standard lumbar puncture) into early vs. progressive ALS subjects. |
| Standard treatment of ALS | Sham comparator | Other: Symptomatic treatment of ALSSymptomatic treatment of ALS without biologic cell-based treatment |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- diagnosis of the ALS disease before the cell transplantation (diagnose established following the El Escorial criteria for definite ALS)
- good understanding of the protocol and willingness to consent
- patient is mentally intact and psychologically stable
- signed informed consent
Key exclusion criteria (11 listed)
- inflammation (high protein or lymphocytosis in the CSF), active infections.
- diabetes,
- cardio-vascular disorders,
- cancer,
- autoimmune diseases
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 |
|---|---|---|
| Biological: Cell-based therapeuticsHuman autologous bone marrow-derived stem/progenitor cell transplantation in ALS patients. | Other | Stem/progenitor cells transplantation. |
| Symptomatic treatment of ALSSymptomatic neurological treatment of ALS | Other | Standard treatment of ALS |
Primary outcome
Safety of autologous bone marrow stem/progenitor cell infusion in enrolled patients
Time frame: 1 year
Confirm the safety of autologous bone marrow stem/progenitor cell infusion in enrolled patients by repeated follow-up over one year with clinical and laboratory evaluations.
1 secondary outcome
Efficacy of autologous bone marrow stem/progenitor cell infusion in enrolled patients.
Time frame: 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jul 2014.
- Derived from this study: PubMed 31629403Pawlukowska W, Baumert B, Golab-Janowska M, Meller A, Machowska-Sempruch K, Welnicka A, Paczkowska E, Rotter I, Machalinski B, Nowacki P. Comparative assessment and monitoring of deterioration of articulatory organs using subjective and objective tools among patients with amyotrophic lateral sclerosis. BMC Neurol. 2019 Oct 19;19(1):241. doi: 10.1186/s12883-019-1484-2.
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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.
Boguslaw Machalinski, MD, PhD study director
Pomeranian Medical University Szczecin
Przemyslaw Nowacki, MD, PhD study chair
Pomeranian Medical University Szczecin
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Biological: Cell-based therapeutics and Symptomatic treatment of ALS in people with Amyotrophic Lateral Sclerosis, run by Pomeranian Medical University Szczecin at 1 site in Poland.
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 11 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: status not confirmed, as of the sponsor's last update on 18 Jul 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Poland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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 of autologous bone marrow stem/progenitor cell infusion in enrolled patients (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 NCT02193893, 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 18 Jul 2014.