Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

Safety Study of Human Neural Stem Cells Injections for Secondary Progressive Multiple Sclerosis Patients (NSC-SPMS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03282760
Recruitment Status : Active, not recruiting
First Posted : September 14, 2017
Last Update Posted : January 14, 2019
Sponsor:
Collaborators:
Associazione Revert ONLUS
Neurocenter of Southern Switzerland
Fondazione Cellule Staminali
Information provided by (Responsible Party):
Casa Sollievo della Sofferenza IRCCS

Brief Summary:

This will be a phase I, open, multicenter, international study performed by 3 participating centres across two countries (Italy and Switzerland). Fifteen to 24 patients affected by SPMS will be enrolled, according to a "standard" phase I design over 18 months. All patients will enter a 3 months run in phase. Thereafter they will receive one of four different doses of allogenic hNSCs (dose A=5 millions hNSCs; dose B=10 millions hNSCs; dose C=16 millions hNSCs; dose D=24 millions hNSCs). Following hNSCs injection, all SPMS patients will receive immunosuppression with tacrolimus for 6 months. Patients will be clinically followed monthly for 1 year and then every 6 months for the 5 years following the study completion (possibly all life long).

MRI assessments will be performed monthly for the first 6 months and then every 3 months for 5 years following the study completion.


Condition or disease Intervention/treatment Phase
Secondary-progressive Multiple Sclerosis Biological: Human Neural Stem Cells Phase 1

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 24 participants
Intervention Model: Sequential Assignment
Intervention Model Description: Fifteen to 24 patients affected by SPMS will be enrolled, according to a "standard" phase I design over 18 months. All patients will enter a 3 months run in phase. Thereafter they will receive one of four different doses of allogenic hNSCs (dose A=5 millions hNSCs; dose B=10 millionshNSCs; dose C=16 millions hNSCs; dose D=24 millions hNSCs).
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I Multicenter Study of Allogenic, Intracerebroventricular Human Neural Stem Cells Transplantation for the Experimental Treatment of Secondary Progressive Multiple Sclerosis Patients
Actual Study Start Date : September 9, 2017
Estimated Primary Completion Date : April 30, 2020
Estimated Study Completion Date : February 28, 2021

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Human Neural Stem Cells Suspension
Intraventricular injections of Allogenic human Neural Stem Cells (hNSCs) in four different dosages (5, 10,16 or 24 millions)
Biological: Human Neural Stem Cells
Allogenic human Neural Stem Cells (hNSCs) in four different dosages (5, 10, 16 or 24 millions). hNSCs are produced by the Laboratorio Cellule Staminali of Terni according to GMP guidelines and are obtained from brain specimens of several fetal human donors from spontaneous miscarriages occurred after the 8th week after conception.




Primary Outcome Measures :
  1. Incidence of Treatment Emergent AE [ Time Frame: 1 year ]
    To Evaluate the Feasibility, Safety and Tolerability of intracerebroventricular injection of allogenic hNSCs

  2. Percentage of Mortality in treated patients [ Time Frame: 1 year ]
    Percentage of subjects (%) with death due to procedure (mortality correlated to treatment)


Secondary Outcome Measures :
  1. Change in Functional disability [ Time Frame: Up to 1 year ]
    this will be measured by the change of the Expanded Disability Scale (EDSS-disability score about pyramidal, cereberral, brainstem, sensory, bowel and bladder, visual, cerebral Functional Systems) during the study period.

  2. Change in Functional disability [ Time Frame: Up to 1 year ]
    this will be measured by the change of the the Multiple Sclerosis Functional Composite (MSFC-scores about upper extremity function, ambulation and cognitive function) during the study period.

  3. Activity of Cognitive function [ Time Frame: Up to 1 year ]
    This will be measured as the mean change of the score of the RAO Brief Repeatable Battery of Neuropsychological Test, during the study period.

  4. Relapses Rate [ Time Frame: Up to 1 year ]
    Relapses will be measured by the change in EDSS scale

  5. Relapses Rate [ Time Frame: Up to 1 year ]
    Relapses will be measured by the Imaging evaluations

  6. MS Biomarkers [ Time Frame: Up to 1 year ]
    Investigation of potential candidate biomarkers able to monitor disease activity and predict clinical course in MS (Neurofilaments)

  7. Alteration in Neurophysiological parameters [ Time Frame: Up to 1 year ]
    Assessed by Evoked Potentials.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. SPMS with progressive accumulation of disability after initial relapsing course, with or without disease activity (Lublin et al. 2014).
  2. EDSS ≥ 6.5 and ≤ 8
  3. EDSS progression over the 2 years prior to study start of ≥ 1.0 point for patients with EDSS =6.5 at the time of inclusion , and of ≥ 0.5 points for patients with EDSS > 6.5 at the time of inclusion
  4. Age ≥ 18 and ≤ 60 years
  5. Failure of best medical treatment as judged by the treating neurologist and declared absence of therapeutic alternatives

Exclusion Criteria:

  1. Neurological conditions other than MS.
  2. Psychiatric disorders, severe cognitive decline and personality and relational disorders.
  3. History or known presence of significant systemic, infectious, oncologic or metabolic disorders.
  4. Presence of any other autoimmune disease.
  5. Chronic infections (HBV, HCV, HIV, tuberculosis).
  6. Inability to perform MRI scans.
  7. Immunomodulant/immunosuppressive treatments in the last 6 months before inclusion.
  8. Current participation to other experimental studies.
  9. Inability to provide informed consent.
  10. Any contra-indication to lumbar puncture and the surgical procedure (e.g. use of anticoagulants)
  11. Pregnancy and breast feeding.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03282760


Locations
Layout table for location information
Italy
Casa Sollievo della Sofferenza - IRCCS
San Giovanni Rotondo, Foggia, Italy, 71013
Azienda Ospedaliera Santa Maria di Terni
Terni, Italy, 05100
Switzerland
Neurocentro della Svizzera Italiana, Istituto di Neurosienze cliniche della svizzera italiana, Centro sclerosi Multipla
Lugano, Switzerland, 6900
Sponsors and Collaborators
Casa Sollievo della Sofferenza IRCCS
Associazione Revert ONLUS
Neurocenter of Southern Switzerland
Fondazione Cellule Staminali
Investigators
Layout table for investigator information
Study Director: Angelo L Vescovi, PhD Casa Sollievo della Sofferenza IRCCS

Publications:

Layout table for additonal information
Responsible Party: Casa Sollievo della Sofferenza IRCCS
ClinicalTrials.gov Identifier: NCT03282760     History of Changes
Other Study ID Numbers: NSC SPMS
2015-004855-37 ( EudraCT Number )
First Posted: September 14, 2017    Key Record Dates
Last Update Posted: January 14, 2019
Last Verified: January 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: All IPD that underlie results in a publication
Supporting Materials: Clinical Study Report (CSR)

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Casa Sollievo della Sofferenza IRCCS:
Cell Transplantation
Neural Stem Cells

Additional relevant MeSH terms:
Layout table for MeSH terms
Sclerosis
Multiple Sclerosis
Multiple Sclerosis, Chronic Progressive
Pathologic Processes
Neoplastic Processes
Neoplasm Metastasis
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Autoimmune Diseases
Immune System Diseases
Neoplasms