Ignite Creation Date:
2025-12-25 @ 12:32 AM
Ignite Modification Date:
2026-02-13 @ 3:55 AM
Study NCT ID:
NCT01640067
Status:
COMPLETED
Last Update Posted:
2015-12-30
First Post:
2012-07-09
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Human Neural Stem Cell Transplantation in Amyotrophic Lateral Sclerosis (ALS)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000690', 'term': 'Amyotrophic Lateral Sclerosis'}, {'id': 'D016472', 'term': 'Motor Neuron Disease'}], 'ancestors': [{'id': 'D013118', 'term': 'Spinal Cord Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D057177', 'term': 'TDP-43 Proteinopathies'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D057165', 'term': 'Proteostasis Deficiencies'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 18}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2011-12'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2015-12', 'completionDateStruct': {'date': '2015-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2015-12-29', 'studyFirstSubmitDate': '2012-07-09', 'studyFirstSubmitQcDate': '2012-07-11', 'lastUpdatePostDateStruct': {'date': '2015-12-30', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2012-07-13', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2015-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'safety of a microsurgery human neural stem cells transplantation into spinal cord of ALS patients', 'timeFrame': 'Participants will be followed for at least 3 years. Monthly in the first year and every three months the following two years', 'description': '* Percentage of subjects (%) with treatment-related mortality defined as death due to procedure and not to the course of the disease\n* Number of adverse events related to the procedure\n* Changes in neuroradiological (MRI) and neurophysiological variables (SEP, MEP)\n* Changes in neuropsychological variables'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'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'], 'conditions': ['Amyotrophic Lateral Sclerosis']}, 'referencesModule': {'references': [{'pmid': '23934834', 'type': 'BACKGROUND', 'citation': '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': '25889343', 'type': 'RESULT', '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, 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': '34558001', 'type': 'DERIVED', 'citation': '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': '23463272', 'type': 'DERIVED', 'citation': '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.'}], 'seeAlsoLinks': [{'url': 'http://www.revertonlus.org/', 'label': 'No profit Association that grant this study'}]}, 'descriptionModule': {'briefSummary': "Primary aim of the trial\n\n1. to verify safety and tolerability of expanded human fetal neural stem cells\n2. to verify safety and tolerability of a microsurgery human fetal neural stem cells transplantation model\n3. to recognize each change in patient's quality of life\n\nSecondary aim of the trial\n\n1. assessment of the impact of human neural stem cells transplantation on the disability of ALS in a clinically significant and measurable way\n2. Assessment of the impact of human neural stem cells transplantation on mortality (all causes)", 'detailedDescription': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '75 Years', 'minimumAge': '20 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Diagnosis of definite or possible ALS according to revised EL Escorial criteria\n* Age: 20-75 years\n* documented progression of disease during the last 6 months.\n* Absence of concomitant diseases\n* Adequate assurances of adherence to protocol\n* The patient must be able to communicate verbally or with the use of non-verbal communication system\n\nGroup 1\n\n* maximum score of 1 on walking item of ALS functional rating scale\n* forced vital capacity \\> or equal to 60\n\nGroup 2\n\n* forced vital capacity \\> or equal to 60\n* ambulation difficulties (maximum score of 2 on walking item of ALS functional rating scale)\n\nGroup 3\n\n* Independent ambulation (score 4 on a scale ALS-FRS)\n* forced vital capacity \\> or equal to 70\n\nExclusion Criteria:\n\nThe presence of at least one of the following will lead to patient exclusion.\n\n1. psychiatric diseases or other neurological diseases different from ALS\n2. other systemic diseases\n3. Neoplasms or other diseases reducing life expectancy\n4. Antecedent polio infection\n5. corticosteroids, immunoglobulin or immunosuppressive treatment\n6. involvement in other clinical trials\n7. 2 or more critical items in MMPI\n8. neuropsychological evaluation suggestive of mental deterioration or cognitive sphere disturbance.\n9. Impediments to MRI\n10. patients unable to understand informed consent form and study aims.\n11. 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'}, 'identificationModule': {'nctId': 'NCT01640067', 'acronym': 'hNSCALS', 'briefTitle': 'Human Neural Stem Cell Transplantation in Amyotrophic Lateral Sclerosis (ALS)', 'organization': {'class': 'OTHER', 'fullName': 'Azienda Ospedaliera Santa Maria, Terni, Italy'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'EudraCT: 2009-014484-39'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Human Neural Stem Cells Suspension', 'description': '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.', 'interventionNames': ['Biological: Human Neural Stem Cells']}], 'interventions': [{'name': 'Human Neural Stem Cells', 'type': 'BIOLOGICAL', 'otherNames': ['Human Neural Stem Cells Suspension'], 'description': 'Surgical microinjection of human neural stem cells on spinal cord of ALS patients', 'armGroupLabels': ['Human Neural Stem Cells Suspension']}]}, 'contactsLocationsModule': {'locations': [{'zip': '05100', 'city': 'Terni', 'state': 'Terni', 'country': 'Italy', 'facility': 'Azienda Ospedaliera Santa Maria', 'geoPoint': {'lat': 42.56335, 'lon': 12.64329}}], 'overallOfficials': [{'name': 'Angelo L Vescovi, Professor', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'IRCCS Casa Sollievo della Soffernza and AOSP Terni, Italy'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Azienda Ospedaliera Santa Maria, Terni, Italy', 'class': 'OTHER'}, 'collaborators': [{'name': 'Azienda Ospedaliero Universitaria Maggiore della Carita', 'class': 'OTHER'}, {'name': 'Università di Padova Italy', 'class': 'UNKNOWN'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Scientific Director of Laboratorio Cellule Staminali, Cell Factory e Biobanca AOSP di Terni, Scientific Director IRCCS Casa Sollievo della Sofferenza S.Giovanni Rotondo Italy', 'investigatorFullName': 'Angelo Luigi Vescovi', 'investigatorAffiliation': 'Azienda Ospedaliera Santa Maria, Terni, Italy'}}}}