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

Amyotrophic Lateral Sclerosis Clinical Trial

— hNSCALS  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01640067
• Other study ID #: EudraCT: 2009-014484-39
• Status: Completed
• Phase: Phase 1
• First received: July 9, 2012
• Last updated: December 29, 2015
• Start date: December 2011
• Est. completion date: December 2015

Study information

• Verified date: December 2015
• Source: Azienda Ospedaliera Santa Maria, Terni, Italy
• Contact: n/a
• Is FDA regulated: No
• Health authority: Italy: Ethics CommitteeItaly: Ministry of HealthItaly: National Institute of HealthItaly: National Monitoring Centre for Clinical Trials - Ministry of Health
• Study type: Interventional

Clinical Trial 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)

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 18
• Est. completion date: December 2015
• Est. primary completion date: December 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 20 Years to 75 Years

Eligibility

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

Study Design

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease
• Sclerosis

Intervention

Biological:
• Human Neural Stem Cells
Surgical microinjection of human neural stem cells on spinal cord of ALS patients

Locations

Country	Name	City	State
Italy	Azienda Ospedaliera Santa Maria	Terni

Sponsors (3)

Lead Sponsor	Collaborator
Azienda Ospedaliera Santa Maria, Terni, Italy	Azienda Ospedaliero Universitaria Maggiore della Carita, Università di Padova Italy

Country where clinical trial is conducted

Italy, 

References & Publications (2)

Gelati M, Profico D, Projetti-Pensi M, Muzi G, Sgaravizzi G, Vescovi AL. Culturing and expansion of "clinical grade" precursors cells from the fetal human central nervous system. Methods Mol Biol. 2013;1059:65-77. doi: 10.1007/978-1-62703-574-3_6. — View Citation

Mazzini L, Gelati M, Profico DC, Sgaravizzi G, Projetti Pensi M, Muzi G, Ricciolini C, Rota Nodari L, Carletti S, Giorgi C, Spera C, Domenico F, Bersano E, Petruzzelli F, Cisari C, Maglione A, Sarnelli MF, Stecco A, Querin G, Masiero S, Cantello R, Ferrar — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	safety of a microsurgery human neural stem cells transplantation into spinal cord of ALS patients	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	Participants will be followed for at least 3 years. Monthly in the first year and every three months the following two years	Yes