Amyotrophic Lateral SclerosisPhase 1hNSCALSNCT01640067
Human Neural Stem Cell Transplantation in Amyotrophic Lateral Sclerosis (ALS)
A Phase 1 study testing Human Neural Stem Cells in people with Amyotrophic Lateral Sclerosis, run by Azienda Ospedaliera Santa Maria, Terni, Italy at 1 site in Italy.
Trial summary
Primary aim of the trial 1. to verify safety and tolerability of expanded human fetal neural stem cells 2. to verify safety and tolerability of a microsurgery human fetal neural stem cells transplantation model 3. to recognize each change in patient's quality of life Secondary aim of the trial 1. Read more about this study
- Who is it for?
- Ages 20 to 75 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- Human Neural Stem Cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteItaly
- Study dates
- Dec 2011to Dec 2015
- 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 20 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of definite or possible ALS according to revised EL Escorial criteria
- Age: 20-75 years
- documented progression of disease during the last 6 months.
- Absence of concomitant diseases
- Adequate assurances of adherence to protocol
- The patient must be able to communicate verbally or with the use of non-verbal communication system
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- Group 1
- maximum score of 1 on walking item of ALS functional rating scale
- forced vital capacity > or equal to 60
- Group 2
- forced vital capacity > or equal to 60
- ambulation difficulties (maximum score of 2 on walking item of ALS functional rating scale)
- Group 3
- Independent ambulation (score 4 on a scale ALS-FRS)
- forced vital capacity > or equal to 70
Who cannot join
- The presence of at least one of the following will lead to patient exclusion.
- psychiatric diseases or other neurological diseases different from ALS
- other systemic diseases
- Neoplasms or other diseases reducing life expectancy
- Antecedent polio infection
- corticosteroids, immunoglobulin or immunosuppressive treatment
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- involvement in other clinical trials
- 2 or more critical items in MMPI
- neuropsychological evaluation suggestive of mental deterioration or cognitive sphere disturbance.
- Impediments to MRI
- patients unable to understand informed consent form and study aims.
- women who are pregnant or childbearing potential for the duration of the study. Non-pregnant status will be documented by beta-HCG negative test 7 days before treatment start
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Intra-spinal Cord Delivery of Human Neural Stem Cells in ALS Patients: Proposal for a Phase I Study/ Trapianto Intramidollare di Cellule Staminali Neurali Umane Come Terapia Putativa Per la SLA: Proposta di un Trial Clinico di Fase I
Primary aim of the trial
- to verify safety and tolerability of expanded human fetal neural stem cells
- to verify safety and tolerability of a microsurgery human fetal neural stem cells transplantation model
- to recognize each change in patient's quality of life
Secondary aim of the trial
- assessment of the impact of human neural stem cells transplantation on the disability of ALS in a clinically significant and measurable way
- Assessment of the impact of human neural stem cells transplantation on mortality (all causes)
Read the detailed description
18 patients diagnosed as definite ALS according to the El Escorial criteria will be recruited during two years. The patients will be divided into 3 groups with different severity. Each group will be less compromised than the group that went before them. Their breathing measurements must be at least 60%. Subjects will receive injections of the human foetal neural stem cells into the spinal cord. A variety of tests and examinations will take place every month for the first year and every three months for the next two in order to monitor the course of the disease and the adverse events.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- Human Neural Stem Cells
- Keywords
- amyotrophic lateral sclerosis, motor neuron disease, stem cells, safety, ALS-FRS-Functional scale R and FVC (%) variation, Survival analysis, Changes in quality of life scales
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Human Neural Stem CellsBiological
Also called Human Neural Stem Cells Suspension
Surgical microinjection of human neural stem cells on spinal cord of ALS patients
| Study group | Role | What they receive |
|---|---|---|
| Human Neural Stem Cells Suspension | Experimental | 50,000 cells/µl. Patients received either unilateral or bilateral hNSCs microinjections (3 microinjections on each side) into the lumbar spinal cord. Each microinjection consisted of 15 µl of the above 50,000cells/µl suspension, yielding a total of 750,000 cells per injection site. Receives: Biological: Human Neural Stem Cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
safety of a microsurgery human neural stem cells transplantation into spinal cord of ALS patients
- Percentage of subjects (%) with treatment-related mortality defined as death due to procedure and not to the course of the disease
- Number of adverse events related to the procedure
- Changes in neuroradiological (MRI) and neurophysiological variables (SEP, MEP)
- Changes in neuropsychological variables
Measured: Participants will be followed for at least 3 years. Monthly in the first year and every three months the following two years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jul 2012 (estimated)
- Study startDec 2011
- Primary completionDec 2015
- Study completionDec 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)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
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Azienda Ospedaliera Santa Maria, Terni, Italy
- Sponsor type
- University, hospital or other
- Collaborators
- Azienda Ospedaliero Universitaria Maggiore della Carita, University of Padova
- Responsible party
- Principal investigator, Angelo Luigi Vescovi, Azienda Ospedaliera Santa Maria, Terni, Italy
- Investigators
- Angelo L Vescovi, Professor, Principal investigator, IRCCS Casa Sollievo della Soffernza and AOSP Terni, Italy
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01640067
- Sponsor's protocol ID
- EudraCT: 2009-014484-39
- Acronym
- hNSCALS
- Status last verified
- Dec 2015
- First submitted
- 9 Jul 2012
- Last updated
- 30 Dec 2015
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Gelati M, Profico D, Projetti-Pensi M, Muzi G, Sgaravizzi G, Vescovi AL. Culturing and expansion of "clinical grade" precursors cells from the fetal human central nervous system. Methods Mol Biol. 2013;1059:65-77. doi: 10.1007/978-1-62703-574-3_6.
PubMed 23934834Mazzini L, Gelati M, Profico DC, Sgaravizzi G, Projetti Pensi M, Muzi G, Ricciolini C, Rota Nodari L, Carletti S, Giorgi C, Spera C, Domenico F, Bersano E, Petruzzelli F, Cisari C, Maglione A, Sarnelli MF, Stecco A, Querin G, Masiero S, Cantello R, Ferrari D, Zalfa C, Binda E, Visioli A, Trombetta D, Novelli A, Torres B, Bernardini L, Carriero A, Prandi P, Servo S, Cerino A, Cima V, Gaiani A, Nasuelli N, Massara M, Glass J, Soraru G, Boulis NM, Vescovi AL. Human neural stem cell transplantation in ALS: initial results from a phase I trial. J Transl Med. 2015 Jan 27;13:17. doi: 10.1186/s12967-014-0371-2.
PubMed 25889343Gelati M, Profico DC, Ferrari D, Vescovi AL. Culturing and Expansion of "Clinical Grade" Neural Stem Cells from the Fetal Human Central Nervous System. Methods Mol Biol. 2022;2389:57-66. doi: 10.1007/978-1-0716-1783-0_5.
PubMed 34558001Robberecht W, Philips T. The changing scene of amyotrophic lateral sclerosis. Nat Rev Neurosci. 2013 Apr;14(4):248-64. doi: 10.1038/nrn3430. Epub 2013 Mar 6.
PubMed 23463272
Study at a glance
From the registry record, last updated by the sponsor on 30 Dec 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Intra-spinal Cord Delivery of Human Neural Stem Cells in ALS Patients: Proposal for a Phase I Study/ Trapianto Intramidollare di Cellule Staminali Neurali Umane Come Terapia Putativa Per la SLA: Proposta di un Trial Clinico di Fase I
- ClinicalTrials.gov ID
- NCT01640067
- Acronym / sponsor ID
- hNSCALS / EudraCT: 2009-014484-39
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- amyotrophic lateral sclerosis, motor neuron disease, stem cells, safety, ALS-FRS-Functional scale R and FVC (%) variation, Survival analysis, Changes in quality of life scales
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Azienda Ospedaliera Santa Maria, Terni, ItalyUniversity, hospital or other
- Collaborators
- Azienda Ospedaliero Universitaria Maggiore della Carita, University of Padova
- Study start
- Dec 2011
- Primary completion
- Dec 2015 (actual)
- Study completion
- Dec 2015 (actual)
- First posted
- 13 Jul 2012 (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.
Primary aim of the trial
- to verify safety and tolerability of expanded human fetal neural stem cells
- to verify safety and tolerability of a microsurgery human fetal neural stem cells transplantation model
- to recognize each change in patient's quality of life
Secondary aim of the trial
- assessment of the impact of human neural stem cells transplantation on the disability of ALS in a clinically significant and measurable way
- Assessment of the impact of human neural stem cells transplantation on mortality (all causes)
Detailed description
18 patients diagnosed as definite ALS according to the El Escorial criteria will be recruited during two years. The patients will be divided into 3 groups with different severity. Each group will be less compromised than the group that went before them. Their breathing measurements must be at least 60%. Subjects will receive injections of the human foetal neural stem cells into the spinal cord. A variety of tests and examinations will take place every month for the first year and every three months for the next two in order to monitor the course of the disease and the adverse events.
Study design
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Human Neural Stem Cells Suspension | Experimental | Biological: Human Neural Stem Cells50,000 cells/µl. Patients received either unilateral or bilateral hNSCs microinjections (3 microinjections on each side) into the lumbar spinal cord. Each microinjection consisted of 15 µl of the above 50,000cells/µl suspension, yielding a total of 750,000 cells per injection site. |
Who may qualify
- Ages
- 20 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Diagnosis of definite or possible ALS according to revised EL Escorial criteria
- Age: 20-75 years
- documented progression of disease during the last 6 months.
- Absence of concomitant diseases
- Adequate assurances of adherence to protocol
Key exclusion criteria (12 listed)
- The presence of at least one of the following will lead to patient exclusion.
- psychiatric diseases or other neurological diseases different from ALS
- other systemic diseases
- Neoplasms or other diseases reducing life expectancy
- Antecedent polio infection
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 |
|---|---|---|
| Human Neural Stem CellsAlso: Human Neural Stem Cells SuspensionSurgical microinjection of human neural stem cells on spinal cord of ALS patients | Biological | Human Neural Stem Cells Suspension |
Primary outcome
safety of a microsurgery human neural stem cells transplantation into spinal cord of ALS patients
Time frame: Participants will be followed for at least 3 years. Monthly in the first year and every three months the following two years
* Percentage of subjects (%) with treatment-related mortality defined as death due to procedure and not to the course of the disease * Number of adverse events related to the procedure * Changes in neuroradiological (MRI) and neurophysiological variables (SEP, MEP) * Changes in neuropsychological variables
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 30 Dec 2015.
- Background: PubMed 23934834Gelati M, Profico D, Projetti-Pensi M, Muzi G, Sgaravizzi G, Vescovi AL. Culturing and expansion of "clinical grade" precursors cells from the fetal human central nervous system. Methods Mol Biol. 2013;1059:65-77. doi: 10.1007/978-1-62703-574-3_6.
- Results publication: PubMed 25889343Mazzini L, Gelati M, Profico DC, Sgaravizzi G, Projetti Pensi M, Muzi G, Ricciolini C, Rota Nodari L, Carletti S, Giorgi C, Spera C, Domenico F, Bersano E, Petruzzelli F, Cisari C, Maglione A, Sarnelli MF, Stecco A, Querin G, Masiero S, Cantello R, Ferrari D, Zalfa C, Binda E, Visioli A, Trombetta D, Novelli A, Torres B, Bernardini L, Carriero A, Prandi P, Servo S, Cerino A, Cima V, Gaiani A, Nasuelli N, Massara M, Glass J, Soraru G, Boulis NM, Vescovi AL. Human neural stem cell transplantation in ALS: initial results from a phase I trial. J Transl Med. 2015 Jan 27;13:17. doi: 10.1186/s12967-014-0371-2.
- Derived from this study: PubMed 34558001Gelati M, Profico DC, Ferrari D, Vescovi AL. Culturing and Expansion of "Clinical Grade" Neural Stem Cells from the Fetal Human Central Nervous System. Methods Mol Biol. 2022;2389:57-66. doi: 10.1007/978-1-0716-1783-0_5.
- Derived from this study: PubMed 23463272Robberecht W, Philips T. The changing scene of amyotrophic lateral sclerosis. Nat Rev Neurosci. 2013 Apr;14(4):248-64. doi: 10.1038/nrn3430. Epub 2013 Mar 6.
- No profit Association that grant this studyLink provided by the sponsor.
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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.
Angelo L Vescovi, Professor principal investigator
IRCCS Casa Sollievo della Soffernza and AOSP Terni, Italy
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 Human Neural Stem Cells in people with Amyotrophic Lateral Sclerosis, run by Azienda Ospedaliera Santa Maria, Terni, Italy at 1 site in Italy.
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 15 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 30 Dec 2015. 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 Italy. 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?
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 of a microsurgery human neural stem cells transplantation into spinal cord of ALS patients (time frame: Participants will be followed for at least 3 years. Monthly in the first year and every three months the following two years). 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 NCT01640067, which also lists 4 linked publications.
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 30 Dec 2015.