Ignite Creation Date:
2025-12-25 @ 1:10 AM
Ignite Modification Date:
2025-12-31 @ 4:01 AM
Study NCT ID:
NCT04644393
Status:
UNKNOWN
Last Update Posted:
2020-11-25
First Post:
2020-11-20
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Responsiveness and Validation Study of MFM-20 in SMA Patients Treated With Nusinersen
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009134', 'term': 'Muscular Atrophy, Spinal'}], 'ancestors': [{'id': 'D013118', 'term': 'Spinal Cord Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D016472', 'term': 'Motor Neuron Disease'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'RETROSPECTIVE', 'observationalModel': 'OTHER'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 40}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2020-11', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2020-11', 'completionDateStruct': {'date': '2021-03', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2020-11-20', 'studyFirstSubmitDate': '2020-11-20', 'studyFirstSubmitQcDate': '2020-11-20', 'lastUpdatePostDateStruct': {'date': '2020-11-25', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2020-11-25', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2021-03', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'MFM-20 responsiveness', 'timeFrame': 'At least 6 Months after the first MFM-20 assessment, and at maximum 24 months', 'description': 'The MFM-20 sensitivity to change indexes considered will be the effect amplitude and the Standardized Response Mean (SRM).'}]}, 'oversightModule': {'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Spinal Muscular Atrophy', 'Motor Function Measure', 'Outcome Assessment', 'Nusinersen'], 'conditions': ['Spinal Muscular Atrophy']}, 'descriptionModule': {'briefSummary': 'The Motor Function Measure (MFM), a reliable tool assessing motor function and its progression in most neuromuscular diseases, is widely used in France in many teams. It can be used regardless of the severity of the motor impairment or the ambulatory status of the patient, allowing its use throughout the whole follow-up period of the patient, even in case of the loss of walking. Two versions of the MFM exist, one composed of 32 items validated for patients from 6 years old (MFM-32) and a shorter version composed of 20 items validated for patients between 2 and 6 years old (MFM-20).\n\nIn order to show the possible use of MFM-20 as early as the age of 2 years to validly and reliably monitor the evolution of the motor function of children treated with Nusinersen, we propose in this project to study the sensitivity to treatment-induced change of MFM-20 and the validity of the scale in this population.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '6 Years', 'minimumAge': '2 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': '* Boys and girls with Type 1 or 2 Spinal Muscular Atrophy, genetically confirmed\n* Aged 2 to 6 years old\n* Treated by Nusinersen for at least two months\n* With at least 3 MFM-20 repeated measures, and with adelay minimum between the first and the last MFM-20 of 6 months', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Boys and girls with Type 1 or 2 Spinal Muscular Atrophy, genetically confirmed\n* Aged 2 to 6 years old\n* Treated by Nusinersen for at least two months\n* With at least 3 MFM-20 repeated measures, and with adelay minimum between the first and the last MFM-20 of 6 months\n* With parental assent\n\nExclusion Criteria:\n\n* \\- Patients with associated cognitive impairment making impossible evaluation of motor function\n* Patients participating to a clinical study with a potential effect on their motor function.'}, 'identificationModule': {'nctId': 'NCT04644393', 'acronym': 'RetroNusiMFM', 'briefTitle': 'Responsiveness and Validation Study of MFM-20 in SMA Patients Treated With Nusinersen', 'organization': {'class': 'OTHER', 'fullName': 'Hospices Civils de Lyon'}, 'officialTitle': 'Responsiveness and Validation Study of MFM-20 in SMA Patients Treated With Nusinersen: Retro NusiMFM', 'orgStudyIdInfo': {'id': '69HCL20_1086'}}, 'contactsLocationsModule': {'locations': [{'city': 'Angers', 'country': 'France', 'contacts': [{'name': 'Julien DURIGNEUX, MD', 'role': 'CONTACT', 'email': 'judurigneux@chu-angers.fr', 'phone': '02 41 35 44 45'}], 'facility': "Service de neuropédiatrie et neurochirurgie de l'enfant Centre de Référence maladies neuromusculaires AOC CHU d'Angers", 'geoPoint': {'lat': 47.47156, 'lon': -0.55202}}, {'city': 'Clermont-Ferrand', 'country': 'France', 'contacts': [{'name': 'Catherine SARRET, MD', 'role': 'CONTACT', 'email': 'csarret@chu-clermontferrand.fr', 'phone': '04.73.75.06.53;'}], 'facility': 'Service de génétique médicale Pôle de Pédiatrie CHU Estaing', 'geoPoint': {'lat': 45.77969, 'lon': 3.08682}}, {'city': 'Garche', 'country': 'France', 'contacts': [{'name': 'Susana QUIJANO-ROY, MD', 'role': 'CONTACT', 'email': 'susana.quijano-y@aphp.fr', 'phone': '01.47.41.78.90;'}], 'facility': 'Service de Neurologie et réanimation pédiatriques CHU Paris IdF Ouest - Hôpital Raymond Poincaré', 'geoPoint': {'lat': 49.39455, 'lon': 6.19775}}, {'city': 'Lyon', 'country': 'France', 'contacts': [{'name': 'Laure LE GOFF, MD', 'role': 'CONTACT', 'email': 'laure.le-goff@chu-lyon.fr', 'phone': '+33 4 72 12 95 04'}, {'name': 'Laure LE GOFF, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Hospices Civils de Lyon, Hôpital Femme Mère Enfant', 'geoPoint': {'lat': 45.74906, 'lon': 4.84789}}, {'city': 'Nantes', 'country': 'France', 'contacts': [{'name': 'Capucine de LATTRE, MD', 'role': 'CONTACT', 'email': 'c.delattre@esean.fr', 'phone': '02 40 34 76 74'}], 'facility': 'Apf Esean', 'geoPoint': {'lat': 47.21725, 'lon': -1.55336}}, {'city': 'Paris', 'country': 'France', 'contacts': [{'name': 'Andreea SEFERIAN, MD', 'role': 'CONTACT', 'email': 'a.seferian@institut-myologie.org', 'phone': '01 44 73 65 37'}], 'facility': "Institut I-Motion - Centre de recherche pédiatrique en pathologies neuromusculaires CHU Paris Est - Hôpital d'Enfants Armand-Trousseau", 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'city': 'Saint-Priest-en-Jarez', 'country': 'France', 'contacts': [{'name': 'Gaelle Gousse, MD', 'role': 'CONTACT', 'email': 'gaelle.gousse@chu-st-etienne.fr', 'phone': '04 77 82 87 29'}], 'facility': 'Service de pédiatrie CHU de Saint-Etienne', 'geoPoint': {'lat': 45.4739, 'lon': 4.37678}}, {'city': 'Toulouse', 'country': 'France', 'contacts': [{'name': 'Claude CANCES, MD', 'role': 'CONTACT', 'email': 'cances.c@chu-toulouse.fr', 'phone': '05.34.55.85.77;'}], 'facility': 'Unité de Neurologie Pédiatrique Centre de Référence Maladies NeuroMusculaires Hôpital des Enfants CHU Toulouse', 'geoPoint': {'lat': 43.60426, 'lon': 1.44367}}], 'centralContacts': [{'name': 'Laure LE GOFF, MD', 'role': 'CONTACT', 'email': 'laure.le-goff@chu-lyon.fr', 'phone': '+33 4 72 12 95 04'}, {'name': 'Pascal Ripeprt, RCA', 'role': 'CONTACT', 'email': 'pascal.rippert@chu-lyon.fr', 'phone': '+33 4 27 85 63 08'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Hospices Civils de Lyon', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}