Ignite Creation Date:
2025-12-26 @ 10:44 PM
Ignite Modification Date:
2025-12-30 @ 11:05 PM
Study NCT ID:
NCT06283212
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-12-12
First Post:
2024-02-22
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D004831', 'term': 'Epilepsies, Myoclonic'}], 'ancestors': [{'id': 'D004829', 'term': 'Epilepsy, Generalized'}, {'id': 'D004827', 'term': 'Epilepsy'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D000073376', 'term': 'Epileptic Syndromes'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 5}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2024-05-09', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-11', 'completionDateStruct': {'date': '2030-10', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-12-10', 'studyFirstSubmitDate': '2024-02-22', 'studyFirstSubmitQcDate': '2024-02-22', 'lastUpdatePostDateStruct': {'date': '2025-12-12', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2024-02-28', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2030-10', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Proportions of participants experiencing any treatment-emergent adverse events (AEs), serious adverse events (SAEs), related AEs, AEs with severity Grade ā„ 3, AEs resulting in study discontinuation, and AEs with fatal outcome.', 'timeFrame': 'Day 1 through Study Completion, an average of 5 years'}, {'measure': 'Change from baseline in the standard score of the Vineland Adaptive Behavior Scales - Third Edition Adaptive Behavior Composite at Week 52.', 'timeFrame': 'Baseline to Week 52. Standard scores are normalized to a mean and SD of 100 and 15, respectively, and are not bounded by a range. Higher scores correspond to better outcomes.'}], 'secondaryOutcomes': [{'measure': 'Percent change in monthly countable seizure frequency (MCSF) to Week 52, with countable seizures defined as generalized tonic-clonic/clonic, focal motor with clearly observable clinical signs, tonic bilateral, and atonic seizures.', 'timeFrame': 'Between the 8-week baseline period and the 48-week post-dosing assessment period (defined as Week 5 to Week 52 following administration of ETX101)'}, {'measure': 'Change from baseline in the raw score of the Bayley Scales of Infant and Toddler DevelopmentĀ® 4th Edition receptive language sub-domain at Week 52.', 'timeFrame': 'Baseline to Week 52. Raw scores range from 0 to 49 and higher scores correspond to better outcomes.'}]}, 'oversightModule': {'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Dravet Syndrome']}, 'descriptionModule': {'briefSummary': 'EXPEDITION is a Phase 1/2 study in the UK to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet Syndrome aged 6 to \\< 48 months. The study follows and open-label, dose-escalation design.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '47 Months', 'minimumAge': '6 Months', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Participant has a predicted loss of function pathogenic or likely pathogenic SCN1A variant\n* Participant must have experienced their first seizure between the age of 3 and 15 months\n* Participant must have a clinical diagnosis of Dravet syndrome or the treating clinician must have high clinical suspicion of a diagnosis of Dravet syndrome\n* Participant is receiving at least one prophylactic antiseizure medication\n\nExclusion Criteria:\n\n* Participant has another genetic mutation or clinical comorbidity which could potentially confound the typical Dravet phenotype\n* Participant has a known central nervous system structural and/or vascular abnormality (indicated by an MRI or CT scan of the brain).\n* Participant has an abnormality that may interfere with CSF distribution and/or has an existing ventriculoperitoneal shunt.\n* Participant is currently taking or has taken antiseizure medications (ASMs) at a therapeutic dose that are contraindicated in Dravet syndrome, including sodium channel blockers.\n* Participant has experienced seizure freedom for a period of 4 consecutive weeks within the 90-day period prior to informed consent.\n* Participant has previously received gene or cell therapy.\n* Participant is currently enrolled in a clinical trial or receiving an investigational therapy.\n* Participant has clinically significant underlying liver disease.'}, 'identificationModule': {'nctId': 'NCT06283212', 'acronym': 'UK Only', 'briefTitle': 'A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome', 'organization': {'class': 'INDUSTRY', 'fullName': 'Encoded Therapeutics'}, 'officialTitle': 'EXPEDITION: A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome', 'orgStudyIdInfo': {'id': 'ETX-DS-005'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cohort A', 'description': 'Cohort A will evaluate ETX101 dose level 1.', 'interventionNames': ['Drug: ETX101']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort B', 'description': 'Cohort B will evaluate ETX101 dose level 2.', 'interventionNames': ['Drug: ETX101']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort C', 'description': 'Cohort C will evaluate ETX101 dose level 3.', 'interventionNames': ['Drug: ETX101']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort D', 'description': 'Cohort D will evaluate ETX101 dose level 4.', 'interventionNames': ['Drug: ETX101']}], 'interventions': [{'name': 'ETX101', 'type': 'DRUG', 'description': 'ETX101 is composed of a non-replicating, recombinant adeno-associated viral serotype 9 (rAAV9) vector used to deliver a GABAergic regulatory element (reGABA) and an engineered transcription factor that increases transcription of the SCN1A gene (eTFSCN1A)', 'armGroupLabels': ['Cohort A', 'Cohort B', 'Cohort C', 'Cohort D']}]}, 'contactsLocationsModule': {'locations': [{'zip': 'G51 4TF', 'city': 'Glasgow', 'country': 'United Kingdom', 'facility': 'Queen Elizabeth Hospital', 'geoPoint': {'lat': 55.86515, 'lon': -4.25763}}, {'zip': 'WC1N3JH', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Great Ormond Street Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'S10 2TH', 'city': 'Sheffield', 'country': 'United Kingdom', 'facility': "Sheffield Children's Hospital", 'geoPoint': {'lat': 53.38297, 'lon': -1.4659}}], 'overallOfficials': [{'name': 'Salvador Rico, M.D., Ph. D', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Encoded Therapeutics'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Encoded Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}