Amyotrophic Lateral Sclerosis
Conditions
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
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)
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.
Interventions
BIOLOGICALHuman Neural Stem Cells
Surgical microinjection of human neural stem cells on spinal cord of ALS patients
Sponsors
Azienda Ospedaliero Universitaria Maggiore della Carita
Università di Padova Italy
Azienda Ospedaliera Santa Maria, Terni, Italy
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
20 Years to 75 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| safety of a microsurgery human neural stem cells transplantation into spinal cord of ALS patients | 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 |
Countries
Italy
Outcome results
None listed