Progressive Multiple SclerosisPhase 1STEMSNCT03269071
Neural Stem Cell Transplantation in Multiple Sclerosis Patients
A Phase 1 study testing human fetal-derived Neural Stem Cells (hNSCs) in people with Progressive Multiple Sclerosis, run by IRCCS San Raffaele at 1 site in Italy.
Trial summary
This is a phase I study evaluating the feasibility, safety and tolerability of intrathecally administered human Neural Stem Cells (hNSCs), at an escalating dose ranging from 0.7x10^6±10% cells to 5.7x10^6±10% cells/kg of body weight, in patients affected by Progressive Multiple Sclerosis Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Progressive Multiple Sclerosis
- What is being tested?
- human fetal-derived Neural Stem Cells (hNSCs)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteItaly
- Study dates
- 17 May 2017to 31 Jul 2021
- 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 55 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Signature of the informed consent by the patient or patients' legal tutors
- Age 18 to 55 years
- Diagnosis of a. Progressive MS as per the revised MC Donald 2010 criteria with a progressive course according to 2013 Lublin phenotypes classification (PMS) with failure or intolerance to all approved therapies according to the disease course or without any alternative approved therapy
- Evidence of progression of disease defined by an increase of ≥ 0.5 Expanded Disability Status Scale (EDSS) points in the last 12 months
- Disease duration 2 to 20 years (included)
- Expanded Disability Status Scale (EDSS) ≥ 6.5
- Presence of oligoclonal band in the cerebrospinal fluid (CSF) is required for Primary Progressive MS
Who cannot join
- They will be excluded from the study patients:
- with any active or chronic infection or diseases other than MS including but not limited to infection with HIV1-2, Hepatitis B or Hepatitis C and tuberculosis or immune deficiency syndromes;
- treated with any immunosuppressive therapy, including but not limited to natalizumab and fingolimod, within the 3 months prior to screening;
- treated with interferon-beta or glatiramer acetate within the 30 days prior to screening;
- treated with corticosteroids within the 30 days prior to screening;
- if relapse occurred during the 30 days prior to screening;
Show all 10Show fewer
- with contraindications for or intolerance to any medication, treatments and procedures that will be used in the study;
- pregnant or in lactation or of childbearing age who are not willing to use a contraceptive method effective* for the entire duration of the study;
- who, in the opinion of the investigator, showing any condition that would preclude study participation.
- refer to guideline http://www.hma.eu/fileadmin/dateien/Human_Medicines/01 About_HMA/Working_Groups/CTFG/2014_09_HMA_CTFG_Contraception.pdf
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Neural Stem Cell Transplantation in Multiple Sclerosis Patient: a Phase I Study
This is a phase I study evaluating the feasibility, safety and tolerability of intrathecally administered human Neural Stem Cells (hNSCs), at an escalating dose ranging from 0.7x10^6±10% cells to 5.7x10^6±10% cells/kg of body weight, in patients affected by Progressive Multiple Sclerosis
Read the detailed description
This is a prospective, monocentric, national, therapeutic exploratory, phase I, not randomized, open label, not controlled, single dose escalation clinical trial.
Each subject will participate in the study for approximately 96 weeks. Participation will include a screening evaluation between -28 and -7 days before the Advance Therapy Investigational Medicinal Products (ATIMP) administration.
A follow-up with clinical visits will be performed from 1 to 96 weeks.
The protocol will consist of a total of four treatment cohorts (TCs), labeled from A to D, each receiving a single escalating dose of allogenic fetal-derived human Neural Stem Cells (hNSCs) injected intrathecally, as it follows:
- TC-A: 0.7x10^6 ± 10% cells/kg of body weight;
- TC-B: 1.4x10^6 ± 10% cells/kg of body weight;
- TC-C: 2.8x10^6 ± 10% cells/kg of body weight;
- TC-D: 5.7x10^6 ± 10% cells/kg of body weight.
The intrathecal injection of hNSCs will be performed in a hospitalized setting. The trial will start with TC-A and will go through the subsequent enrolment of patients to be included in TCs from B to D.
Each cohort will consist of three patients at minimum. In case of safety issue the number in each TC will be increased to six patients.
After the inclusion of the first patient of the TC the investigators will wait at least 14 days to treat the second patient. The same interval time will be used for all the following patient within the same TC. After the inclusion of all the planned patients within a TC and with no Dose Limiting Toxicity (DLT) within the TC, the investigators will wait at least 3 months before switching to the upper TC. In case of 1 DLT within the TC, the cohort will be extended to six patients. If another DLT will be observed, the current dosage will be considered excessive and the immediate lower dosage will be considered the Maximum Tolerated Dose (MTD).
The safety monitoring board will review all safety date in the case of evaluated Adverse Event (AE) ≥ 3 "possibly related to human Neural Stem Cells" by investigators and in any case before the shifts between TCs.
This approach will be repeated for every TC up to the end of the study. At the end of the total follow-up, a long term follow up is planned for all enrolled and treated patients in the study, in accordance with the national applicable laws and the international guidelines.
- Conditions
- Progressive Multiple Sclerosis
- Being tested
- human fetal-derived Neural Stem Cells (hNSCs)
- Keywords
- Multiple Sclerosis, Cell Therapy, Human Neural Stem Cells
- Medical subject headings
- Multiple Sclerosis, Chronic Progressive, Multiple Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
human fetal-derived Neural Stem Cells (hNSCs)Drug
Also called human Neural Stem Cells (hNCSs)
The Drug Product (DP) can be classified as an ATIMP belonging to the class of Cell Therapy Medicinal Products (EU law). The ATIMP consists of human fetal-derived Neural Stem Cells (hNSCs) re-suspended in their final formulation medium as defined in the Investigational Medical Product Dossier (IMPD).
For dosage indications, see specific Treatment Cohorts (TC), from A to D.
The product will be administered intrathecally through lumbar puncture.
| Study group | Role | What they receive |
|---|---|---|
| Treatment Cohort A | Experimental | See Study Description
TC-A: 0.7 x 10^6 ± 10% human fetal-derived Neural Stem Cells (hNSCs) / kg of body weight Receives: Drug: human fetal-derived Neural Stem Cells (hNSCs) |
| Treatment Cohort B | Experimental | See Study Description
TC-B: 1.4 x 10^6 ± 10% human fetal-derived Neural Stem Cells (hNSCs) / kg of body weight Receives: Drug: human fetal-derived Neural Stem Cells (hNSCs) |
| Treatment Cohort C | Experimental | See Study Description
TC-C: 2.8 x 10^6 ± 10% human fetal-derived Neural Stem Cells (hNSCs) / kg of body weight Receives: Drug: human fetal-derived Neural Stem Cells (hNSCs) |
| Treatment Cohort D | Experimental | See Study Description
TC-D: 5.7 x 10^6 ± 10% human fetal-derived Neural Stem Cells (hNSCs) / kg of body weight Receives: Drug: human fetal-derived Neural Stem Cells (hNSCs) |
What is being measured
The primary outcome is the main question the study is designed to answer.
SHORT TERM (0-24 hours) Overall survival
Number of patients alive all over the trial
Measured: 0-24 hours after intrathecal injection of human Neural Stem Cells (hNSCs)
SHORT TERM (0-24 hours) Overall safety and tolerability measured by Adverse Event (AE) recording
Number of AEs in alive patients all over the trial
Measured: 0-24 hours after intrathecal injection of human Neural Stem Cells (hNSCs)
SHORT TERM (0-24 hours) Changes in neurological conditions not related to disease
Number of changes in neurological conditions not related to disease of alive patients all over the trial
Measured: 0-24 hours after intrathecal injection of human Neural Stem Cells (hNSCs)
SHORT TERM (0-24 hours) Proportion of successful intrathecal administration procedure (feasibility)
Number of successful intrathecal administration procedures versus all intrathecal administration procedures in the whole trial
Measured: 0-24 hours after intrathecal injection of human Neural Stem Cells (hNSCs)
MID TERM (day 1- day 14) Overall survival
Number of alive patients in the whole trial
Measured: from 1 to 14 days after intrathecal injection of human Neural Stem Cells (hNSCs)
MID TERM (day 1- day 14) Overall safety and tolerability measured by AE recording
Number of AEs of alive patients in the whole trial
Measured: from 1 to 14 days after intrathecal injection of human Neural Stem Cells (hNSCs)
MID TERM (day 1- day 14) Changes in neurological conditions not related to disease
Number of changes in neurological conditions not related to disease in alive patients of the whole trial
Measured: from 1 to 14 days after intrathecal injection of human Neural Stem Cells (hNSCs)
LONG TERM (day 15 - week 96) Overall survival
Number of alive patients in the whole trial
Measured: from day 15 to week 96 after intrathecal injection of human Neural Stem Cells (hNSCs)
LONG TERM (day 15 - week 96) Overall safety and tolerability measured by AE recording
Number of AEs in alive patients of the whole trial
Measured: from day 15 to week 96 after intrathecal injection of human Neural Stem Cells (hNSCs)
Long term incidence of malignancies
Incidence of malignancies in alive patients of the whole trial
Measured: from day 0 to week 96 after intrathecal injection of human Neural Stem Cells (hNSCs)
Evaluation of changes in quality of life measures
Health-related quality of life will be assessed by standardized questionnaires
Measured: 12, 24, 48, 72 and 96 weeks
LONG TERM (day 15 - week 96) Changes in neurological conditions not related to the disease
Number of changes in the neurological conditions not related to disease in alive patients of the whole trial
Measured: from day 15 to week 96 after intrathecal injection of human Neural Stem Cells (hNSCs)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Aug 2017
- Study start17 May 2017
- Primary completion31 Jul 2021
- Study completion31 Jul 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Progressive Multiple SclerosisStudies of the same condition, open and under way.
- More trials run by IRCCS San RaffaeleThe sponsor's other registered studies.
- 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
- Sequential
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- IRCCS San Raffaele
- Sponsor type
- University, hospital or other
- Collaborators
- Fondazione Italiana Sclerosi Multipla
- Responsible party
- Principal investigator, Gianvito MARTINO, IRCCS San Raffaele
- Investigators
- Gianvito Martino, Principal investigator, IRCCS San Raffaele
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03269071
- Sponsor's protocol ID
- 2016-002020-86 (EudraCT)
- Other IDs
- 2016
- Acronym
- STEMS
- Status last verified
- Sep 2021
- First submitted
- 30 Aug 2017
- Last updated
- 5 Sep 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: It is not yet known the best way to exploit the Individual Participant data (IPD) that will become available
Publications
Kokaia Z, Martino G, Schwartz M, Lindvall O. Cross-talk between neural stem cells and immune cells: the key to better brain repair? Nat Neurosci. 2012 Jul 26;15(8):1078-87. doi: 10.1038/nn.3163.
PubMed 22837038Martino G, Franklin RJ, Baron Van Evercooren A, Kerr DA; Stem Cells in Multiple Sclerosis (STEMS) Consensus Group. Stem cell transplantation in multiple sclerosis: current status and future prospects. Nat Rev Neurol. 2010 May;6(5):247-55. doi: 10.1038/nrneurol.2010.35. Epub 2010 Apr 20.
PubMed 20404843Pluchino S, Gritti A, Blezer E, Amadio S, Brambilla E, Borsellino G, Cossetti C, Del Carro U, Comi G, 't Hart B, Vescovi A, Martino G. Human neural stem cells ameliorate autoimmune encephalomyelitis in non-human primates. Ann Neurol. 2009 Sep;66(3):343-54. doi: 10.1002/ana.21745.
PubMed 19798728Pluchino S, Quattrini A, Brambilla E, Gritti A, Salani G, Dina G, Galli R, Del Carro U, Amadio S, Bergami A, Furlan R, Comi G, Vescovi AL, Martino G. Injection of adult neurospheres induces recovery in a chronic model of multiple sclerosis. Nature. 2003 Apr 17;422(6933):688-94. doi: 10.1038/nature01552.
PubMed 12700753Pluchino S, Zanotti L, Rossi B, Brambilla E, Ottoboni L, Salani G, Martinello M, Cattalini A, Bergami A, Furlan R, Comi G, Constantin G, Martino G. Neurosphere-derived multipotent precursors promote neuroprotection by an immunomodulatory mechanism. Nature. 2005 Jul 14;436(7048):266-71. doi: 10.1038/nature03889.
PubMed 16015332Genchi A, Brambilla E, Sangalli F, Radaelli M, Bacigaluppi M, Furlan R, Andolfo A, Drago D, Magagnotti C, Scotti GM, Greco R, Vezzulli P, Ottoboni L, Bonopane M, Capilupo D, Ruffini F, Belotti D, Cabiati B, Cesana S, Matera G, Leocani L, Martinelli V, Moiola L, Vago L, Panina-Bordignon P, Falini A, Ciceri F, Uglietti A, Sormani MP, Comi G, Battaglia MA, Rocca MA, Storelli L, Pagani E, Gaipa G, Martino G. Neural stem cell transplantation in patients with progressive multiple sclerosis: an open-label, phase 1 study. Nat Med. 2023 Jan;29(1):75-85. doi: 10.1038/s41591-022-02097-3. Epub 2023 Jan 9.
PubMed 36624312
Study at a glance
From the registry record, last updated by the sponsor on 5 Sep 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Neural Stem Cell Transplantation in Multiple Sclerosis Patient: a Phase I Study
- ClinicalTrials.gov ID
- NCT03269071
- Acronym / sponsor ID
- STEMS / 2016-002020-86 (EudraCT)
- Conditions
- Progressive Multiple Sclerosis
- Keywords
- Multiple Sclerosis, Cell Therapy, Human Neural Stem Cells
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- IRCCS San RaffaeleUniversity, hospital or other
- Collaborators
- Fondazione Italiana Sclerosi Multipla
- Study start
- 17 May 2017 (actual)
- Primary completion
- 31 Jul 2021 (actual)
- Study completion
- 31 Jul 2021 (actual)
- First posted
- 31 Aug 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.
This is a phase I study evaluating the feasibility, safety and tolerability of intrathecally administered human Neural Stem Cells (hNSCs), at an escalating dose ranging from 0.7x10^6±10% cells to 5.7x10^6±10% cells/kg of body weight, in patients affected by Progressive Multiple Sclerosis
Detailed description
This is a prospective, monocentric, national, therapeutic exploratory, phase I, not randomized, open label, not controlled, single dose escalation clinical trial.
Each subject will participate in the study for approximately 96 weeks. Participation will include a screening evaluation between -28 and -7 days before the Advance Therapy Investigational Medicinal Products (ATIMP) administration.
A follow-up with clinical visits will be performed from 1 to 96 weeks.
The protocol will consist of a total of four treatment cohorts (TCs), labeled from A to D, each receiving a single escalating dose of allogenic fetal-derived human Neural Stem Cells (hNSCs) injected intrathecally, as it follows:
- TC-A: 0.7x10^6 ± 10% cells/kg of body weight;
- TC-B: 1.4x10^6 ± 10% cells/kg of body weight;
- TC-C: 2.8x10^6 ± 10% cells/kg of body weight;
- TC-D: 5.7x10^6 ± 10% cells/kg of body weight.
The intrathecal injection of hNSCs will be performed in a hospitalized setting. The trial will start with TC-A and will go through the subsequent enrolment of patients to be included in TCs from B to D.
Each cohort will consist of three patients at minimum. In case of safety issue the number in each TC will be increased to six patients.
After the inclusion of the first patient of the TC the investigators will wait at least 14 days to treat the second patient. The same interval time will be used for all the following patient within the same TC. After the inclusion of all the planned patients within a TC and with no Dose Limiting Toxicity (DLT) within the TC, the investigators will wait at least 3 months before switching to the upper TC. In case of 1 DLT within the TC, the cohort will be extended to six patients. If another DLT will be observed, the current dosage will be considered excessive and the immediate lower dosage will be considered the Maximum Tolerated Dose (MTD).
The safety monitoring board will review all safety date in the case of evaluated Adverse Event (AE) ≥ 3 "possibly related to human Neural Stem Cells" by investigators and in any case before the shifts between TCs.
This approach will be repeated for every TC up to the end of the study. At the end of the total follow-up, a long term follow up is planned for all enrolled and treated patients in the study, in accordance with the national applicable laws and the international guidelines.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Treatment Cohort A | Experimental | Drug: human fetal-derived Neural Stem Cells (hNSCs)See Study Description TC-A: 0.7 x 10^6 ± 10% human fetal-derived Neural Stem Cells (hNSCs) / kg of body weight |
| Treatment Cohort B | Experimental | Drug: human fetal-derived Neural Stem Cells (hNSCs)See Study Description TC-B: 1.4 x 10^6 ± 10% human fetal-derived Neural Stem Cells (hNSCs) / kg of body weight |
| Treatment Cohort C | Experimental | Drug: human fetal-derived Neural Stem Cells (hNSCs)See Study Description TC-C: 2.8 x 10^6 ± 10% human fetal-derived Neural Stem Cells (hNSCs) / kg of body weight |
| Treatment Cohort D | Experimental | Drug: human fetal-derived Neural Stem Cells (hNSCs)See Study Description TC-D: 5.7 x 10^6 ± 10% human fetal-derived Neural Stem Cells (hNSCs) / kg of body weight |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- Signature of the informed consent by the patient or patients' legal tutors
- Age 18 to 55 years
- Diagnosis of a. Progressive MS as per the revised MC Donald 2010 criteria with a progressive course according to 2013 Lublin phenotypes classification (PMS) with failure or intolerance to all approved therapies according to the disease course or without any alternative approved therapy
- Evidence of progression of disease defined by an increase of ≥ 0.5 Expanded Disability Status Scale (EDSS) points in the last 12 months
- Disease duration 2 to 20 years (included)
Key exclusion criteria (10 listed)
- with any active or chronic infection or diseases other than MS including but not limited to infection with HIV1-2, Hepatitis B or Hepatitis C and tuberculosis or immune deficiency syndromes;
- treated with any immunosuppressive therapy, including but not limited to natalizumab and fingolimod, within the 3 months prior to screening;
- treated with interferon-beta or glatiramer acetate within the 30 days prior to screening;
- treated with corticosteroids within the 30 days prior to screening;
- if relapse occurred during the 30 days prior to screening;
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 fetal-derived Neural Stem Cells (hNSCs)Also: human Neural Stem Cells (hNCSs)The Drug Product (DP) can be classified as an ATIMP belonging to the class of Cell Therapy Medicinal Products (EU law). The ATIMP consists of human fetal-derived Neural Stem Cells (hNSCs) re-suspended in their final formulation medium as defined in the Investigational Medical Product Dossier (IMPD). For dosage indications, see specific Treatment Cohorts (TC), from A to D. The product will be administered intrathecally through lumbar puncture. | Drug | Treatment Cohort A, Treatment Cohort B, Treatment Cohort C, Treatment Cohort D |
Primary outcome
SHORT TERM (0-24 hours) Overall survival
Time frame: 0-24 hours after intrathecal injection of human Neural Stem Cells (hNSCs)
Number of patients alive all over the trial
SHORT TERM (0-24 hours) Overall safety and tolerability measured by Adverse Event (AE) recording
Time frame: 0-24 hours after intrathecal injection of human Neural Stem Cells (hNSCs)
Number of AEs in alive patients all over the trial
SHORT TERM (0-24 hours) Changes in neurological conditions not related to disease
Time frame: 0-24 hours after intrathecal injection of human Neural Stem Cells (hNSCs)
Number of changes in neurological conditions not related to disease of alive patients all over the trial
SHORT TERM (0-24 hours) Proportion of successful intrathecal administration procedure (feasibility)
Time frame: 0-24 hours after intrathecal injection of human Neural Stem Cells (hNSCs)
Number of successful intrathecal administration procedures versus all intrathecal administration procedures in the whole trial
MID TERM (day 1- day 14) Overall survival
Time frame: from 1 to 14 days after intrathecal injection of human Neural Stem Cells (hNSCs)
Number of alive patients in the whole trial
MID TERM (day 1- day 14) Overall safety and tolerability measured by AE recording
Time frame: from 1 to 14 days after intrathecal injection of human Neural Stem Cells (hNSCs)
Number of AEs of alive patients in the whole trial
MID TERM (day 1- day 14) Changes in neurological conditions not related to disease
Time frame: from 1 to 14 days after intrathecal injection of human Neural Stem Cells (hNSCs)
Number of changes in neurological conditions not related to disease in alive patients of the whole trial
LONG TERM (day 15 - week 96) Overall survival
Time frame: from day 15 to week 96 after intrathecal injection of human Neural Stem Cells (hNSCs)
Number of alive patients in the whole trial
LONG TERM (day 15 - week 96) Overall safety and tolerability measured by AE recording
Time frame: from day 15 to week 96 after intrathecal injection of human Neural Stem Cells (hNSCs)
Number of AEs in alive patients of the whole trial
Long term incidence of malignancies
Time frame: from day 0 to week 96 after intrathecal injection of human Neural Stem Cells (hNSCs)
Incidence of malignancies in alive patients of the whole trial
Evaluation of changes in quality of life measures
Time frame: 12, 24, 48, 72 and 96 weeks
Health-related quality of life will be assessed by standardized questionnaires
LONG TERM (day 15 - week 96) Changes in neurological conditions not related to the disease
Time frame: from day 15 to week 96 after intrathecal injection of human Neural Stem Cells (hNSCs)
Number of changes in the neurological conditions not related to disease in alive patients of the whole trial
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 5 Sep 2021.
- Results publication: PubMed 22837038Kokaia Z, Martino G, Schwartz M, Lindvall O. Cross-talk between neural stem cells and immune cells: the key to better brain repair? Nat Neurosci. 2012 Jul 26;15(8):1078-87. doi: 10.1038/nn.3163.
- Results publication: PubMed 20404843Martino G, Franklin RJ, Baron Van Evercooren A, Kerr DA; Stem Cells in Multiple Sclerosis (STEMS) Consensus Group. Stem cell transplantation in multiple sclerosis: current status and future prospects. Nat Rev Neurol. 2010 May;6(5):247-55. doi: 10.1038/nrneurol.2010.35. Epub 2010 Apr 20.
- Results publication: PubMed 19798728Pluchino S, Gritti A, Blezer E, Amadio S, Brambilla E, Borsellino G, Cossetti C, Del Carro U, Comi G, 't Hart B, Vescovi A, Martino G. Human neural stem cells ameliorate autoimmune encephalomyelitis in non-human primates. Ann Neurol. 2009 Sep;66(3):343-54. doi: 10.1002/ana.21745.
- Results publication: PubMed 12700753Pluchino S, Quattrini A, Brambilla E, Gritti A, Salani G, Dina G, Galli R, Del Carro U, Amadio S, Bergami A, Furlan R, Comi G, Vescovi AL, Martino G. Injection of adult neurospheres induces recovery in a chronic model of multiple sclerosis. Nature. 2003 Apr 17;422(6933):688-94. doi: 10.1038/nature01552.
- Results publication: PubMed 16015332Pluchino S, Zanotti L, Rossi B, Brambilla E, Ottoboni L, Salani G, Martinello M, Cattalini A, Bergami A, Furlan R, Comi G, Constantin G, Martino G. Neurosphere-derived multipotent precursors promote neuroprotection by an immunomodulatory mechanism. Nature. 2005 Jul 14;436(7048):266-71. doi: 10.1038/nature03889.
- Derived from this study: PubMed 36624312Genchi A, Brambilla E, Sangalli F, Radaelli M, Bacigaluppi M, Furlan R, Andolfo A, Drago D, Magagnotti C, Scotti GM, Greco R, Vezzulli P, Ottoboni L, Bonopane M, Capilupo D, Ruffini F, Belotti D, Cabiati B, Cesana S, Matera G, Leocani L, Martinelli V, Moiola L, Vago L, Panina-Bordignon P, Falini A, Ciceri F, Uglietti A, Sormani MP, Comi G, Battaglia MA, Rocca MA, Storelli L, Pagani E, Gaipa G, Martino G. Neural stem cell transplantation in patients with progressive multiple sclerosis: an open-label, phase 1 study. Nat Med. 2023 Jan;29(1):75-85. doi: 10.1038/s41591-022-02097-3. Epub 2023 Jan 9.
- Individual participant dataSharing planned: No. It is not yet known the best way to exploit the Individual Participant data (IPD) that will become available
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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.
Gianvito Martino principal investigator
IRCCS San Raffaele
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 fetal-derived Neural Stem Cells (hNSCs) in people with Progressive Multiple Sclerosis, run by IRCCS San Raffaele 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 7 inclusion and 10 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 5 Sep 2021. 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?
SHORT TERM (0-24 hours) Overall survival (time frame: 0-24 hours after intrathecal injection of human Neural Stem Cells (hNSCs)). The study lists 12 primary outcomes in all. 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 NCT03269071, which also lists 6 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 5 Sep 2021.