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
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Amyotrophic Lateral Sclerosis
- Sponsor
- Azienda Ospedaliera Santa Maria, Terni, Italy
- Enrollment
- 18
- Locations
- 1
- Primary Endpoint
- safety of a microsurgery human neural stem cells transplantation into spinal cord of ALS patients
- Status
- Completed
- Last Updated
- 10 years ago
Overview
Brief Summary
Primary aim of the trial
- to verify safety and tolerability of expanded human fetal neural stem cells
- to verify safety and tolerability of a microsurgery human fetal neural stem cells transplantation model
- to recognize each change in patient's quality of life
Secondary aim of the trial
- assessment of the impact of human neural stem cells transplantation on the disability of ALS in a clinically significant and measurable way
- 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.
Investigators
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
Eligibility 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
- •maximum score of 1 on walking item of ALS functional rating scale
- •forced vital capacity \> or equal to 60
- •forced vital capacity \> or equal to 60
- •ambulation difficulties (maximum score of 2 on walking item of ALS functional rating scale)
Exclusion Criteria
- •The presence of at least one of the following will lead to patient exclusion.
- •psychiatric diseases or other neurological diseases different from ALS
- •other systemic diseases
- •Neoplasms or other diseases reducing life expectancy
- •Antecedent polio infection
- •corticosteroids, immunoglobulin or immunosuppressive treatment
- •involvement in other clinical trials
- •2 or more critical items in MMPI
- •neuropsychological evaluation suggestive of mental deterioration or cognitive sphere disturbance.
- •Impediments to MRI
Outcomes
Primary Outcomes
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