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Human Neural Stem Cell Transplantation in Amyotrophic Lateral Sclerosis (ALS) (hNSCALS)

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ClinicalTrials.gov Identifier: NCT01640067
Recruitment Status : Completed
First Posted : July 13, 2012
Last Update Posted : December 30, 2015
Sponsor:
Collaborators:
Azienda Ospedaliero Universitaria Maggiore della Carita
Università di Padova Italy
Information provided by (Responsible Party):
Angelo Luigi Vescovi, Azienda Ospedaliera Santa Maria, Terni, Italy

Brief 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. assessment of the impact of human neural stem cells transplantation on the disability of ALS in a clinically significant and measurable way
  2. Assessment of the impact of human neural stem cells transplantation on mortality (all causes)

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Biological: Human Neural Stem Cells Phase 1

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 Type : Interventional  (Clinical Trial)
Actual Enrollment : 18 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
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
Study Start Date : December 2011
Actual Primary Completion Date : December 2015
Actual Study Completion Date : December 2015


Arm Intervention/treatment
Experimental: Human Neural Stem Cells Suspension
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.
Biological: Human Neural Stem Cells
Surgical microinjection of human neural stem cells on spinal cord of ALS patients
Other Name: Human Neural Stem Cells Suspension



Primary Outcome Measures :
  1. 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



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Ages Eligible for Study:   20 Years to 75 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

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

Exclusion Criteria:

The presence of at least one of the following will lead to patient exclusion.

  1. psychiatric diseases or other neurological diseases different from ALS
  2. other systemic diseases
  3. Neoplasms or other diseases reducing life expectancy
  4. Antecedent polio infection
  5. corticosteroids, immunoglobulin or immunosuppressive treatment
  6. involvement in other clinical trials
  7. 2 or more critical items in MMPI
  8. neuropsychological evaluation suggestive of mental deterioration or cognitive sphere disturbance.
  9. Impediments to MRI
  10. patients unable to understand informed consent form and study aims.
  11. 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

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): NCT01640067


Locations
Italy
Azienda Ospedaliera Santa Maria
Terni, Italy, 05100
Sponsors and Collaborators
Azienda Ospedaliera Santa Maria, Terni, Italy
Azienda Ospedaliero Universitaria Maggiore della Carita
Università di Padova Italy
Investigators
Principal Investigator: Angelo L Vescovi, Professor IRCCS Casa Sollievo della Soffernza and AOSP Terni, Italy

Additional Information:
Publications of Results:
Other Publications:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Angelo Luigi Vescovi, Scientific Director of Laboratorio Cellule Staminali, Cell Factory e Biobanca AOSP di Terni, Scientific Director IRCCS Casa Sollievo della Sofferenza S.Giovanni Rotondo Italy, Azienda Ospedaliera Santa Maria, Terni, Italy
ClinicalTrials.gov Identifier: NCT01640067     History of Changes
Other Study ID Numbers: EudraCT: 2009-014484-39
First Posted: July 13, 2012    Key Record Dates
Last Update Posted: December 30, 2015
Last Verified: December 2015

Keywords provided by Angelo Luigi Vescovi, Azienda Ospedaliera Santa Maria, Terni, Italy:
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

Additional relevant MeSH terms:
Pathologic Processes
Sclerosis
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases