Ignite Creation Date:
2025-12-24 @ 5:34 PM
Ignite Modification Date:
2026-01-03 @ 9:16 AM
Study NCT ID:
NCT05625568
Status:
UNKNOWN
Last Update Posted:
2022-11-23
First Post:
2022-11-07
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study of VYNT-0126 in the Treatment of Rett Syndrome in Adult Patients
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D015518', 'term': 'Rett Syndrome'}], 'ancestors': [{'id': 'D038901', 'term': 'X-Linked Intellectual Disability'}, {'id': 'D008607', 'term': 'Intellectual Disability'}, {'id': 'D019954', 'term': 'Neurobehavioral Manifestations'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 48}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2023-03', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2022-11', 'completionDateStruct': {'date': '2024-06', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2022-11-14', 'studyFirstSubmitDate': '2022-11-07', 'studyFirstSubmitQcDate': '2022-11-14', 'lastUpdatePostDateStruct': {'date': '2022-11-23', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2022-11-23', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2024-03', 'type': 'ESTIMATED'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Quantitative Electroencephalograms (EEGs)', 'timeFrame': 'Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).', 'description': 'Quantitative EEGs will be explored as potential biomarkers of intervention effects on brain function and clinical severity.'}, {'measure': 'Electrophysiological Evoked Potentials', 'timeFrame': 'Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).', 'description': 'Evoked potential following auditory and visual stimuli will be explored as potential biomarkers of intervention effects on brain function and clinical severity.'}], 'primaryOutcomes': [{'measure': 'Adverse Events', 'timeFrame': 'Through study completion, approximately 14 weeks', 'description': 'Incidence of adverse events (AEs), including serious adverse events (SAEs), will be compared across the two VYNT-0126 doses and placebo. SAEs and AEs will be examined throughout the study.'}], 'secondaryOutcomes': [{'measure': 'Change from Baseline in the Rett Syndrome Behavioral Questionnaire (RSBQ)', 'timeFrame': 'Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).'}, {'measure': 'Change from Baseline in the 24-Item Motor-Behavioral Assessment (MBA)', 'timeFrame': 'Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).'}, {'measure': 'Change from Baseline in Clinical Global Impression of Severity (CGI-S)', 'timeFrame': 'Obtained at baseline, end of dose titration, and end of treatment (approximately 14 weeks).'}, {'measure': 'Clinical Global Impression of Improvement (CGI-I)', 'timeFrame': 'Obtained at the end of dose titration and end of treatment (approximately 14 weeks).'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Rett Syndrome']}, 'descriptionModule': {'briefSummary': 'This is an exploratory, Phase 2, multicenter, double-blind, parallel-group, placebo-controlled study to assess the safety, tolerability, and efficacy of oral treatment with VYNT-0126 in female subjects 18-45 years of age with Rett syndrome.'}, 'eligibilityModule': {'sex': 'FEMALE', 'stdAges': ['ADULT'], 'maximumAge': '45 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Females age 18-45 (inclusive)\n* Diagnosis of classic/typical Rett syndrome with a documented mutation of the MeCP2 gene\n* Severity rating of between 10 and 36 (Rett Syndrome Natural History/Clinical Severity Scale)\n* Concomitant medications (including approved medications for treatment of Rett syndrome) must be stable for \\>4 weeks prior to enrollment\n* Able to receive liquid study drug orally or via gastrostomy tube (G-tube)\n\nExclusion Criteria:\n\n* Actively undergoing neurological regression;\n* Abnormal QT interval, prolongation or significant cardiovascular history\n* Excluded concomitant medications\n* Current clinically significant (as determined by the investigator). cardiovascular, endocrine, hepatic, renal, or respiratory disease\n* Gastrointestinal disease which may interfere with the absorption, distribution, metabolism or excretion of the study medication\n* History of, or current cerebrovascular disease or brain trauma\n* History of, or current, malignancy\n* Clinically significant abnormalities in safety laboratory tests, vital signs, or ECG, as measured at screening or baseline\n* Any condition which in the investigator's opinion would affect the ability of the subject to participate in the study\n* Allergy to VYNT-0126 or any ingredients of the liquid formulation"}, 'identificationModule': {'nctId': 'NCT05625568', 'briefTitle': 'Study of VYNT-0126 in the Treatment of Rett Syndrome in Adult Patients', 'organization': {'class': 'INDUSTRY', 'fullName': 'Vyant Bio'}, 'officialTitle': 'Phase 2, Double-Blind, Randomized, Placebo-Controlled Clinical Study of VYNT-0126 in the Treatment of Rett Syndrome in Adult Female Patients', 'orgStudyIdInfo': {'id': 'VYNT-0126-001'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'interventionNames': ['Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': '5 mg VYNT-0126', 'interventionNames': ['Drug: VYNT-0126']}, {'type': 'EXPERIMENTAL', 'label': '10 mg VYNT-0126', 'interventionNames': ['Drug: VYNT-0126']}], 'interventions': [{'name': 'VYNT-0126', 'type': 'DRUG', 'description': 'Liquid for oral administration once daily', 'armGroupLabels': ['10 mg VYNT-0126', '5 mg VYNT-0126']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Liquid for oral administration once daily', 'armGroupLabels': ['Placebo']}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Vyant Bio', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}