Ignite Creation Date:
2025-12-24 @ 3:38 PM
Ignite Modification Date:
2026-05-29 @ 4:17 PM
Study NCT ID:
NCT05419492
Status:
RECRUITING
Last Update Posted:
2025-06-12
First Post:
2022-06-10
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and 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'}, {'id': 'C565810', 'term': 'Generalized Epilepsy With Febrile Seizures Plus, Type 2'}], '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': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR'], 'maskingDescription': 'Part 1 is open-label with no blinding.\n\nPart 2 will be conducted in a double-blinded manner whereby all Primary Site Staff (including clinicians, research coordinators, neuropsychologists, pharmacists, and physical therapists), study participants and caregivers, Sponsor and Sponsor-designees will be blinded through the end of Week 52 following Day 1 for each participant. The Surgical Site Staff will be unblinded to treatment assignment.'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 22}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-05-14', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-01', 'completionDateStruct': {'date': '2031-04', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-06-10', 'studyFirstSubmitDate': '2022-06-10', 'studyFirstSubmitQcDate': '2022-06-10', 'lastUpdatePostDateStruct': {'date': '2025-06-12', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2022-06-15', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-04', '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 ≥ 3, AEs resulting in study discontinuation, and AEs with a fatal outcome.', 'timeFrame': 'Day 1 through Study Completion'}, {'measure': 'Percent change in monthly countable seizure frequency (MCSF) period, with countable seizures defined as generalized tonic-clonic/clonic, focal motor with clearly observable clinical signs, tonic, or atonic seizures.', 'timeFrame': 'Between the pre-dose period (Part 1: 30 days prior to consent and the collection period through Day -1; Part 2: collection period between Day -72 and Day -1) and the 48-week post dosing period (defined as Week 5 through Week 52)'}], 'secondaryOutcomes': [{'measure': 'Absolute change in monthly countable seizure frequency (MCSF)', 'timeFrame': 'Between the pre-dose period (Part 1: 30 days prior to consent and the collection period through Day -1; Part 2: collection period between Day -72 and Day -1) and the 48-week post dosing period (defined as Week 5 through Week 52)'}, {'measure': 'Proportion of participants with ≥ 90% reduction in monthly countable seizure frequency (MCSF).', 'timeFrame': 'Between the pre-dose period (Part 1: 30 days prior to consent and the collection period through Day -1; Part 2: collection period between Day -72 and Day -1) and the 48-week post dosing period (defined as Week 5 through Week 52)'}, {'measure': 'Change from baseline in the Vineland-3 Expressive Communication raw score at Week 52', 'timeFrame': 'Baseline through Week 52'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Dravet', 'SCN1A', 'DEE', 'developmental and epileptic encephalopathy', 'Dravet Syndrome', 'SCN1A-positive', 'SCN1A+'], 'conditions': ['Dravet Syndrome']}, 'descriptionModule': {'briefSummary': 'ENDEAVOR is a Phase 1/2, 2-part, multicenter study to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged ≥6 to \\<36 months (Part 1) and aged ≥6 to \\<48 months (Part 2). Part 1 follows an open-label, dose-escalation design, and Part 2 is a randomized, double-blind, sham delayed-treatment control, dose-selection study.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '47 Months', 'minimumAge': '6 Months', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Participant must be aged between ≥6 months and \\<36 months in Part 1 and \\<48 months in Part 2.\n* Participant must have a predicted loss of function pathogenic or likely pathogenic SCN1A variant.\n* Participant must have experienced their first seizure between the ages of 3 and 15 months.\n* Participant must have a clinical diagnosis of Dravet syndrome or the treating clinician must have a 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, including under an expanded access and/or compassionate use program.\n* Participant has clinically significant underlying liver disease.'}, 'identificationModule': {'nctId': 'NCT05419492', 'acronym': 'ENDEAVOR', 'briefTitle': 'A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome', 'organization': {'class': 'INDUSTRY', 'fullName': 'Encoded Therapeutics'}, 'officialTitle': 'ENDEAVOR: A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Infants and Children With SCN1A-Positive Dravet Syndrome', 'orgStudyIdInfo': {'id': 'ETX-DS-002'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Part 1', 'description': 'Part 1 will follow an open-label, rule-based, dose-escalation design and will evaluate up to 4 dose levels of ETX101.', 'interventionNames': ['Drug: ETX101']}, {'type': 'SHAM_COMPARATOR', 'label': 'Part 2', 'description': 'Part 2 is a dose-selection study, which will follow a double-blind (up through Week 52), randomized, sham delayed-treatment control design.\n\nThere will be up to 3 cohorts in Part 2. If it is determined two dose levels of ETX101 will be evaluated in Part 2, participants will be randomized 1:1:1 to study treatment or sham procedure with delayed treatment. If Part 2 proceeds with a single dose level of ETX101, participants will be randomized 2:1 to study treatment or sham procedure with delayed treatment.', 'interventionNames': ['Drug: ETX101']}], 'interventions': [{'name': 'ETX101', 'type': 'DRUG', 'description': 'ETX101 is a non-replicating, recombinant adeno-associated viral vector serotype 9 (rAAV9) comprising a GABAergic regulatory element (reGABA) and an engineered transcription factor that increases transcription of the SCN1A gene (eTFSCN1A). ETX101 is intended as a one-time intracerebroventricular (ICV) administration.', 'armGroupLabels': ['Part 1', 'Part 2']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94158', 'city': 'San Francisco', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Joseph Sullivan, MD', 'role': 'CONTACT', 'email': 'joseph.sullivan@ucsf.edu'}, {'name': 'Joseph Sullivan', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': "UCSF Benioff Children's Hospitals", 'geoPoint': {'lat': 37.77493, 'lon': -122.41942}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Enrique Rojas', 'role': 'CONTACT', 'email': 'erojas@luriechildrens.org', 'phone': '312-227-2532'}, {'name': 'Linda Laux', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': "Ann & Robert H. Lurie Children's Hospital of Chicago", 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '76104', 'city': 'Fort Worth', 'state': 'Texas', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Dianna Grado, RN, CCRC', 'role': 'CONTACT', 'email': 'Dianna.Grado@cookchildrens.org', 'phone': '(682) 885-2844'}, {'name': 'Scott Perry', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': "Cook Children's Medical Center", 'geoPoint': {'lat': 32.72541, 'lon': -97.32085}}], 'centralContacts': [{'name': 'Encoded Patient Advocacy', 'role': 'CONTACT', 'email': 'patientadvocacy@encoded.com', 'phone': '+1 (650) 398-4301'}], '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'}}}}