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

Phase 1

Completed

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Biological: Human Neural Stem Cells

• Registration Number: NCT01640067
• Lead Sponsor: 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)

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 Timeline

• Study Start: 2011-12
• Study First Submitted: 2012-07-09
• Study First Submitted QC: 2012-07-11
• Study First Posted: 2012-07-13
• Status Verified: 2015-12
• Primary Completion: 2015-12
• Study Completion: 2015-12
• Last Update Submitted: 2015-12-29
• Last Update Posted: 2015-12-30

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 18

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Human Neural Stem Cells Suspension	Human Neural Stem Cells	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.

Primary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (1)

Azienda Ospedaliera Santa Maria; 🇮🇹 Terni, Italy