A Phase 1 interventional study of human fetal-derived Neural Stem Cells (hNSCs) in Progressive Multiple Sclerosis, sponsored by IRCCS San Raffaele. Completed at 1 site in Italy. Open to participants aged 18 Years to 55 Years. Per ClinicalTrials.gov, last updated 2021-09-05.
Sponsored by IRCCS San Raffaele · Phase 1, Interventional, and Treatment
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
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:
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.
244 studies on the registry are indexed under Multiple Sclerosis, Chronic Progressive; 53 are open to participants now.
This study's enrollment of 4 is below the median of 54 across 189 interventional studies indexed under Multiple Sclerosis, Chronic Progressive.
Browse Multiple Sclerosis, Chronic Progressive studies →IRCCS San Raffaele is the lead sponsor of 453 studies on the registry; 234 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
They will be excluded from the study patients:
who, in the opinion of the investigator, showing any condition that would preclude study participation.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
See Study Description TC-A: 0.7 x 10\^6 ± 10% human fetal-derived Neural Stem Cells (hNSCs) / kg of body weight
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
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
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
Drug: human fetal-derived Neural Stem Cells (hNSCs)
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.
Also known as: human Neural Stem Cells (hNCSs)
SHORT TERM (0-24 hours) Overall survival
Number of patients alive all over the trial
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: from day 15 to week 96 after intrathecal injection of human Neural Stem Cells (hNSCs)
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No — It is not yet known the best way to exploit the Individual Participant data (IPD) that will become available
Ran from May 2017 to primary completion in Jul 2021, 50 months.
Of 20 phase 1 interventional studies under Multiple Sclerosis, Chronic Progressive, half reached primary completion within 44 months of starting (the median), counting those still running; a quarter within 17 and three quarters within 56. This study's 50 months is above that.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Sep 2021. You cannot join it, but the record below documents what was studied.
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Multiple Sclerosis, Chronic Progressive →