Ignite Creation Date:
2025-12-24 @ 11:28 PM
Ignite Modification Date:
2025-12-25 @ 9:15 PM
Study NCT ID:
NCT07146256
Status:
RECRUITING
Last Update Posted:
2025-08-28
First Post:
2025-08-05
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Natural History of Oculo-Pharyngeal Muscular Dystrophy (OPMD) - Israel National OPMD Registry
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D039141', 'term': 'Muscular Dystrophy, Oculopharyngeal'}, {'id': 'D003680', 'term': 'Deglutition Disorders'}, {'id': 'D001763', 'term': 'Blepharoptosis'}, {'id': 'D018908', 'term': 'Muscle Weakness'}], 'ancestors': [{'id': 'D009136', 'term': 'Muscular Dystrophies'}, {'id': 'D020966', 'term': 'Muscular Disorders, Atrophic'}, {'id': 'D009135', 'term': 'Muscular Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D004935', 'term': 'Esophageal Diseases'}, {'id': 'D005767', 'term': 'Gastrointestinal Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D010608', 'term': 'Pharyngeal Diseases'}, {'id': 'D010038', 'term': 'Otorhinolaryngologic Diseases'}, {'id': 'D005141', 'term': 'Eyelid Diseases'}, {'id': 'D005128', 'term': 'Eye Diseases'}, {'id': 'D020879', 'term': 'Neuromuscular Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 300}, 'targetDuration': '5 Years', 'patientRegistry': True}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2022-02-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-08', 'completionDateStruct': {'date': '2030-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-08-21', 'studyFirstSubmitDate': '2025-08-05', 'studyFirstSubmitQcDate': '2025-08-21', 'lastUpdatePostDateStruct': {'date': '2025-08-28', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2025-08-28', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2030-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Severity of muscle strength decline over time', 'timeFrame': '1 year', 'description': '10-meter walk test'}, {'measure': 'Penetration/Aspiration Scale', 'timeFrame': '1 year', 'description': 'Penetration/Aspiration Scale (PAS)'}, {'measure': 'Upper Margin Reflex Distance', 'timeFrame': '1 year', 'description': 'Upper Margin Reflex Distance'}, {'measure': 'Lower Margin Reflex Distance', 'timeFrame': '1 year', 'description': 'Lower Margin Reflex Distance'}], 'secondaryOutcomes': [{'measure': 'Maximum Expiratory Pressure', 'timeFrame': '2 years', 'description': 'Maximum Expiratory Pressure (MEP)'}, {'measure': 'Cough Peak Flow', 'timeFrame': '2 years', 'description': 'Cough Peak Flow (CPF)'}, {'measure': 'Oral health', 'timeFrame': '2 years', 'description': 'Decayed, Missing, and Filled Teeth (DMFT) index'}, {'measure': 'Dysphagia severity', 'timeFrame': '1 year', 'description': 'Yale Pharyngeal Residue Severity Rating Scale'}, {'measure': 'Functional Oral Intake Scale', 'timeFrame': '1 year', 'description': 'Functional Oral Intake Scale (FOIS)'}, {'measure': 'Swallowing Disturbance', 'timeFrame': '1 year', 'description': 'Swallowing Disturbance Questionnaire (SDQ)'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Oculopharyngeal muscular dystrophy (OPMD)', 'Natural history', 'Dysphagia', 'Eyelid ptosis', 'Muscle weakness', 'Oral health'], 'conditions': ['Oculopharyngeal Muscular Dystrophy (OPMD)']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'https://doi.org/10.1007/s44162-023-00020-z', 'label': 'The National Israeli Registry for Oculo-Pharyngeal Muscular Dystrophy (IsrO-PMD): rationale and design'}]}, 'descriptionModule': {'briefSummary': 'The goal of this open prospective multi-disciplinary observational study of patients with OPMD at various stages of clinical manifestations is to explore the natural history of the disease in the Israeli population.', 'detailedDescription': 'The the following procedures will be performed by a multidisciplinary team:\n\nNeurological and neurocognitive examination; Clinical assessment of of swallowing and severity of dysphagia; Clinical assessment of eyelid ptosis severity; Clinical assessment of respiratory muscles function; Clinical assessment of oral health'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Genetically Confirmed Oculopharyngeal Muscular Dystrophy', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Patients with clinical diagnosis of OPMD or patients with signs /symptoms suggestive of OPMD\n\nExclusion Criteria:\n\n* Age \\<18 years and Pregnancy'}, 'identificationModule': {'nctId': 'NCT07146256', 'acronym': 'Isr-OPMD', 'briefTitle': 'Natural History of Oculo-Pharyngeal Muscular Dystrophy (OPMD) - Israel National OPMD Registry', 'organization': {'class': 'OTHER_GOV', 'fullName': 'Sheba Medical Center'}, 'officialTitle': 'Natural History of Oculo-Pharyngeal Muscular Dystrophy (OPMD) - Israel National OPMD Registry', 'orgStudyIdInfo': {'id': 'SHEBA-21-8801-AD-CTIL'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Subjects with genetically diagnosed OPMD', 'interventionNames': ['Other: Non-interventional observational study']}], 'interventions': [{'name': 'Non-interventional observational study', 'type': 'OTHER', 'description': 'No intervention will be studied', 'armGroupLabels': ['Subjects with genetically diagnosed OPMD']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Ramat Gan', 'status': 'RECRUITING', 'country': 'Israel', 'contacts': [{'name': 'Naama Study coordinator', 'role': 'CONTACT', 'email': 'OPMD@sheba.health.gov.il', 'phone': '+972552929006'}, {'name': 'Amir Dori, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR'}, {'name': 'Alex Zvulunov, MD, MHA', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Merav Ben-David, MD', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Limor Benyamini, MD', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Lior Greenbaum, MD, PhD', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Silvina Friedlander-Barenboim, DMD', 'role': 'SUB_INVESTIGATOR'}], 'facility': 'Sheba Medical Center', 'geoPoint': {'lat': 32.08227, 'lon': 34.81065}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Sheba Medical Center', 'class': 'OTHER_GOV'}, 'collaborators': [{'name': 'The non-profit organization for promotion of health and cure of OPMD', 'class': 'UNKNOWN'}], 'responsibleParty': {'type': 'SPONSOR'}}}}