Amyotrophic Lateral SclerosisPhase 1ADIPOSTEMNCT03296501
Intraspinal Transplantation of Autologous ADRC in ALS Patients
A Phase 1 study testing Cell-based therapy of autologous adipose derived regenerative cells transplanted intraspinally and intrathecally in ALS patients in people with Amyotrophic Lateral Sclerosis, run by Mossakowski Medical Research Centre Polish Academy of Sciences at 1 site in Poland.
Trial summary
The goal of our nonrandomized, open label study is to investigate the safety and efficacy of autologous adipose derived mesenchymal regenerative cells (ADRC) transplantation into the individuals with diagnosed amyotrophic lateral sclerosis (ALS). 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?
- Cell-based therapy of autologous adipose derived regenerative cells transplanted intraspinally and intrathecally in ALS patients
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 sitePoland
- Study dates
- 13 Oct 2015to 31 Oct 2022
- 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
- clinically definite or probable ALS according to El Escorial criteria
- life expectancy of more than 1 year
- INR ≤2 before liposuction
- lack of treatment with immune-suppressants and/or corticosteroids within min. 20 days prior to recruitment
- constant riluzole treatment (50 mg/bid) throughout the study period
- compliance with treatment regimen e.g. will and possibility to attend check-up visits
- Polish citizens
Who cannot join
- primary haematological disease, including hypercoagulable states
- Presence of comorbidity that would stand in the way of neurosurgical treatment-
- previous history of a spinal-cord surgery at the clinically affected level
- previous/current history of neoplasm or comorbidity that could impact upon patient's survival
- PEG
- pregnancy /lactation
- noninvasive/invasive mechanical ventilation at time of recruitment
- alcohol abuse, cocaine amphetamine, etc.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Safety and Efficacy of Intraspinal Transplantation of Autologous ADRC in ALS Patients
The goal of our nonrandomized, open label study is to investigate the safety and efficacy of autologous adipose derived mesenchymal regenerative cells (ADRC) transplantation into the individuals with diagnosed amyotrophic lateral sclerosis (ALS). All enrolled patients will have a documented at least 3-months clinical and electrophisiological observation of ALS disease course prior to study enrollment. Each patient will recive 3 injections of ADRC every 3 months: an intraspinal injection followed by 2 subsequent intrathecal infusions. Safety, adverse events and efficacy will be confirmed by clinical, elecrophisiological ( EMG, MUNIX), neuroimmaging and spirometry together with functional (ALSFRS-R) and objective motor assesment (MRC and dynamometer).
Read the detailed description
Amyotrophic Lateral Sclerosis (ALS) is an incurable disease of unknown etiology that in a short time leads to significant impairment of motor functions and death. The frequency of ALS is 4-8/100 000. Mostly it affects people between 40 and 70 years old, but it can occur at a younger age. Since the symptom onset is most frequently in the fifth or sixth decade, ALS is a huge economic burden for the society. There are many studies conducted to treat the disease and prevent it, but currently the medicine offers only one drug that can slow the appearance of disease symptoms but could not stop the progression. Yet, improvements in medical management, including nutrition and breathing, regularly increase patient survival - 50% of affected patients live at least 3 or more years after diagnosis; 20% live 5 years or more; and up to 10% will survive more than 10 years. The stem-cell-based therapies could be therefore a new waited strategy for ALS clinical treatment.
Since the clinical course of ALS may vary substantially between patients, we are planning to qualify them with a primarily established clinical course based on a detailed anamnesis and clinical assessment. The goal of our nonrandomized, open label study is to investigate the safety and efficacy of autologous adipose derived mesenchymal regenerative cells (ADRC) transplantation into the individuals with diagnosed amyotrophic lateral sclerosis. In order to select a group of 30 ALS patients for the ADRC treatment, approximately 50 ALS patients will be examined. All patients enrolled will have a documented at least 3-months clinical and electrophisiological observation of ALS disease course prior to study enrollment. Each patient will recive 3 injections of ADRC every 3 months: an intraspinal injection followed by 2 subsequent intrathecal infusions. Safety, adverse events and efficacy will be confirmed by clinical, elecrophisiological ( EMG, MUNIX), neuroimmaging and spirometry together with functional (ALSFRS-R) and objective motor assesment (MRC and dynamometer).
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Cell-based therapy of autologous adipose derived regenerative cells transplanted intraspinally and intrathecally in ALS patients
- Keywords
- ALS, ADRC, adipose derived regenerative cells, intraspinal transplantation, mesenchymal cells
- 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.
Cell-based therapy of autologous adipose derived regenerative cells transplanted intraspinally and intrathecally in ALS patientsBiological
3 injections of ADRC: 1 intraspinal and 2 intrathecal
| Study group | Role | What they receive |
|---|---|---|
| Autologous ADRC injection | Experimental | Not described Receives: Biological: Cell-based therapy of autologous adipose derived regenerative cells transplanted intraspinally and intrathecally in ALS patients |
What is being measured
The primary outcome is the main question the study is designed to answer.
Functional progression rate
Delta ALS Functional Rating Scale (ALSFRS-R)/month
Measured: 3 months
Secondary outcomes (1)
Electrophisiological progression rate
electromyography (EMG)/ Motor unit number index (MUNIX)
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Sep 2017
- Study start13 Oct 2015
- Primary completion31 Oct 2020 (estimated)
- Study completion31 Oct 2022 (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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Mossakowski Medical Research Centre Polish Academy of Sciences
- Sponsor type
- University, hospital or other
- Collaborators
- Medical University of Warsaw
- Responsible party
- Principal investigator, Anna Sarnowska, Mossakowski Medical Research Centre Polish Academy of Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03296501
- Sponsor's protocol ID
- DPO/014/33/09/2015
- Acronym
- ADIPOSTEM
- Status last verified
- Apr 2020
- First submitted
- 21 Sep 2017
- Last updated
- 15 Apr 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Kuzma-Kozakiewicz M, Marchel A, Kaminska A, Gawel M, Sznajder J, Figiel-Dabrowska A, Nowak A, Maj E, Krzesniak NE, Noszczyk BH, Domanska-Janik K, Sarnowska A. Intraspinal Transplantation of the Adipose Tissue-Derived Regenerative Cells in Amyotrophic Lateral Sclerosis in Accordance with the Current Experts' Recommendations: Choosing Optimal Monitoring Tools. Stem Cells Int. 2018 Aug 12;2018:4392017. doi: 10.1155/2018/4392017. eCollection 2018.
PubMed 30158984
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Intraspinal Transplantation of Autologous ADRC in ALS Patients
- ClinicalTrials.gov ID
- NCT03296501
- Acronym / sponsor ID
- ADIPOSTEM / DPO/014/33/09/2015
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- ALS, ADRC, adipose derived regenerative cells, intraspinal transplantation, mesenchymal cells
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Mossakowski Medical Research Centre Polish Academy of SciencesUniversity, hospital or other
- Collaborators
- Medical University of Warsaw
- Study start
- 13 Oct 2015 (actual)
- Primary completion
- 31 Oct 2020 (estimated)
- Study completion
- 31 Oct 2022 (estimated)
- First posted
- 28 Sep 2017 (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.
The goal of our nonrandomized, open label study is to investigate the safety and efficacy of autologous adipose derived mesenchymal regenerative cells (ADRC) transplantation into the individuals with diagnosed amyotrophic lateral sclerosis (ALS). All enrolled patients will have a documented at least 3-months clinical and electrophisiological observation of ALS disease course prior to study enrollment. Each patient will recive 3 injections of ADRC every 3 months: an intraspinal injection followed by 2 subsequent intrathecal infusions. Safety, adverse events and efficacy will be confirmed by clinical, elecrophisiological ( EMG, MUNIX), neuroimmaging and spirometry together with functional (ALSFRS-R) and objective motor assesment (MRC and dynamometer).
Detailed description
Amyotrophic Lateral Sclerosis (ALS) is an incurable disease of unknown etiology that in a short time leads to significant impairment of motor functions and death. The frequency of ALS is 4-8/100 000. Mostly it affects people between 40 and 70 years old, but it can occur at a younger age. Since the symptom onset is most frequently in the fifth or sixth decade, ALS is a huge economic burden for the society. There are many studies conducted to treat the disease and prevent it, but currently the medicine offers only one drug that can slow the appearance of disease symptoms but could not stop the progression. Yet, improvements in medical management, including nutrition and breathing, regularly increase patient survival - 50% of affected patients live at least 3 or more years after diagnosis; 20% live 5 years or more; and up to 10% will survive more than 10 years. The stem-cell-based therapies could be therefore a new waited strategy for ALS clinical treatment.
Since the clinical course of ALS may vary substantially between patients, we are planning to qualify them with a primarily established clinical course based on a detailed anamnesis and clinical assessment. The goal of our nonrandomized, open label study is to investigate the safety and efficacy of autologous adipose derived mesenchymal regenerative cells (ADRC) transplantation into the individuals with diagnosed amyotrophic lateral sclerosis. In order to select a group of 30 ALS patients for the ADRC treatment, approximately 50 ALS patients will be examined. All patients enrolled will have a documented at least 3-months clinical and electrophisiological observation of ALS disease course prior to study enrollment. Each patient will recive 3 injections of ADRC every 3 months: an intraspinal injection followed by 2 subsequent intrathecal infusions. Safety, adverse events and efficacy will be confirmed by clinical, elecrophisiological ( EMG, MUNIX), neuroimmaging and spirometry together with functional (ALSFRS-R) and objective motor assesment (MRC and dynamometer).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Autologous ADRC injection | Experimental | Biological: Cell-based therapy of autologous adipose derived regenerative cells transplanted intraspinally and intrathecally in ALS patients |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- clinically definite or probable ALS according to El Escorial criteria
- life expectancy of more than 1 year
- INR ≤2 before liposuction
- lack of treatment with immune-suppressants and/or corticosteroids within min. 20 days prior to recruitment
- constant riluzole treatment (50 mg/bid) throughout the study period
Key exclusion criteria (8 listed)
- primary haematological disease, including hypercoagulable states
- Presence of comorbidity that would stand in the way of neurosurgical treatment-
- previous history of a spinal-cord surgery at the clinically affected level
- previous/current history of neoplasm or comorbidity that could impact upon patient's survival
- PEG
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 |
|---|---|---|
| Cell-based therapy of autologous adipose derived regenerative cells transplanted intraspinally and intrathecally in ALS patients3 injections of ADRC: 1 intraspinal and 2 intrathecal | Biological | Autologous ADRC injection |
Primary outcome
Functional progression rate
Time frame: 3 months
Delta ALS Functional Rating Scale (ALSFRS-R)/month
1 secondary outcome
Electrophisiological progression rate
Time frame: 3 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Apr 2020.
- Results publication: PubMed 30158984Kuzma-Kozakiewicz M, Marchel A, Kaminska A, Gawel M, Sznajder J, Figiel-Dabrowska A, Nowak A, Maj E, Krzesniak NE, Noszczyk BH, Domanska-Janik K, Sarnowska A. Intraspinal Transplantation of the Adipose Tissue-Derived Regenerative Cells in Amyotrophic Lateral Sclerosis in Accordance with the Current Experts' Recommendations: Choosing Optimal Monitoring Tools. Stem Cells Int. 2018 Aug 12;2018:4392017. doi: 10.1155/2018/4392017. eCollection 2018.
- Individual participant dataSharing planned: No
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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.
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 Cell-based therapy of autologous adipose derived regenerative cells transplanted intraspinally and intrathecally in ALS patients in people with Amyotrophic Lateral Sclerosis, run by Mossakowski Medical Research Centre Polish Academy of Sciences 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 7 inclusion and 8 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 15 Apr 2020. 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. 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?
Functional progression rate (time frame: 3 months). 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 NCT03296501, 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 15 Apr 2020.