NCT01640067

Brief Summary

Primary aim of the trial

  1. 1.to verify safety and tolerability of expanded human fetal neural stem cells
  2. 2.to verify safety and tolerability of a microsurgery human fetal neural stem cells transplantation model
  3. 3.to recognize each change in patient's quality of life
  4. 4.assessment of the impact of human neural stem cells transplantation on the disability of ALS in a clinically significant and measurable way
  5. 5.Assessment of the impact of human neural stem cells transplantation on mortality (all causes)

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
18

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Dec 2011

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 1, 2011

Completed
7 months until next milestone

First Submitted

Initial submission to the registry

July 9, 2012

Completed
4 days until next milestone

First Posted

Study publicly available on registry

July 13, 2012

Completed
3.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2015

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2015

Completed
Last Updated

December 30, 2015

Status Verified

December 1, 2015

Enrollment Period

4 years

First QC Date

July 9, 2012

Last Update Submit

December 29, 2015

Conditions

Amyotrophic Lateral Sclerosis

Keywords

amyotrophic lateral sclerosismotor neuron diseasestem cellssafetyALS-FRS-Functional scale R and FVC (%) variationSurvival analysisChanges in quality of life scales

Outcome Measures

Primary Outcomes (1)

  • 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

Study Arms (1)

Human Neural Stem Cells Suspension

EXPERIMENTAL

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

Interventions

Human Neural Stem CellsBIOLOGICAL

Surgical microinjection of human neural stem cells on spinal cord of ALS patients

Also known as: Human Neural Stem Cells Suspension
Human Neural Stem Cells Suspension

Eligibility Criteria

Age20 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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
  • or more critical items in MMPI
  • neuropsychological evaluation suggestive of mental deterioration or cognitive sphere disturbance.
  • Impediments to MRI
  • patients unable to understand informed consent form and study aims.
  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Azienda Ospedaliera Santa Maria

Terni, Terni, 05100, Italy

Location

Related Publications (4)

  • 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.

    PMID: 23934834BACKGROUND

  • 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, Ferrari D, Zalfa C, Binda E, Visioli A, Trombetta D, Novelli A, Torres B, Bernardini L, Carriero A, Prandi P, Servo S, Cerino A, Cima V, Gaiani A, Nasuelli N, Massara M, Glass J, Soraru G, Boulis NM, Vescovi AL. Human neural stem cell transplantation in ALS: initial results from a phase I trial. J Transl Med. 2015 Jan 27;13:17. doi: 10.1186/s12967-014-0371-2.

    PMID: 25889343RESULT

  • Gelati M, Profico DC, Ferrari D, Vescovi AL. Culturing and Expansion of "Clinical Grade" Neural Stem Cells from the Fetal Human Central Nervous System. Methods Mol Biol. 2022;2389:57-66. doi: 10.1007/978-1-0716-1783-0_5.

    PMID: 34558001DERIVED

  • Robberecht W, Philips T. The changing scene of amyotrophic lateral sclerosis. Nat Rev Neurosci. 2013 Apr;14(4):248-64. doi: 10.1038/nrn3430. Epub 2013 Mar 6.

    PMID: 23463272DERIVED

Related Links

MeSH Terms

Conditions

Amyotrophic Lateral SclerosisMotor Neuron Disease

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesNeurodegenerative DiseasesTDP-43 ProteinopathiesNeuromuscular DiseasesProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Angelo L Vescovi, Professor

    IRCCS Casa Sollievo della Soffernza and AOSP Terni, Italy

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Scientific Director of Laboratorio Cellule Staminali, Cell Factory e Biobanca AOSP di Terni, Scientific Director IRCCS Casa Sollievo della Sofferenza S.Giovanni Rotondo Italy

Study Record Dates

First Submitted

July 9, 2012

First Posted

July 13, 2012

Study Start

December 1, 2011

Primary Completion

December 1, 2015

Study Completion

December 1, 2015

Last Updated

December 30, 2015

Record last verified: 2015-12

Locations

IT(1)