Ignite Creation Date:
2025-12-25 @ 12:21 AM
Ignite Modification Date:
2025-12-25 @ 10:24 PM
Study NCT ID:
NCT06983158
Status:
SUSPENDED
Last Update Posted:
2025-10-01
First Post:
2025-05-05
Is Possible Gene Therapy:
True
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Clinical Trial of CAP-002 Gene Therapy in Pediatric Patients With Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'interventionBrowseModule': {'meshes': [{'id': 'D015316', 'term': 'Genetic Therapy'}], 'ancestors': [{'id': 'D001691', 'term': 'Biological Therapy'}, {'id': 'D013812', 'term': 'Therapeutics'}, {'id': 'D005818', 'term': 'Genetic Engineering'}, {'id': 'D005821', 'term': 'Genetic Techniques'}, {'id': 'D008919', 'term': 'Investigative Techniques'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 12}}, 'statusModule': {'whyStopped': 'Per protocol stopping rule was met. Comprehensive review is needed prior to resuming enrollment.', 'overallStatus': 'SUSPENDED', 'startDateStruct': {'date': '2025-07-03', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-07', 'completionDateStruct': {'date': '2028-12-20', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-09-25', 'studyFirstSubmitDate': '2025-05-05', 'studyFirstSubmitQcDate': '2025-05-13', 'lastUpdatePostDateStruct': {'date': '2025-10-01', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2025-05-21', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2028-12-20', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Primary Safety', 'timeFrame': '2 years', 'description': 'Incidence of Adverse Events and Serious Adverse Events assessed through Clinical safety laboratory tests (hematology, chemistry, liver function, and urinalysis), ECG, vital sign measurements, and Physical Examinations'}], 'secondaryOutcomes': [{'measure': 'Bayley Scales of Infant and Toddler Development- Fourth Edition', 'timeFrame': 'Baseline, Month 6, Month 12, Month 18 and Month 24', 'description': 'Clinician Scores: 0 to 2; Total Scores: Cognitive 0-162; Receptive 0-84; Expressive 0-74; Fine Motor 0-92; Gross Motor 0-116; High score is a better outcome'}, {'measure': 'Gross Motor Function Measure', 'timeFrame': 'Baseline, Month 6, Month 12, Month 18 and Month 24', 'description': 'Clinician Scores: 0 to 3; Total Score: 0 to 264; High score is a better outcome'}, {'measure': 'Peabody Developmental Motor Scales-Third Edition', 'timeFrame': 'Baseline, Month 6, Month 12, Month 18 and Month 24', 'description': 'Clinician Scores: 0 to 2; High score is a better outcome'}, {'measure': 'STXBP1-Clinical Severity Assessment-Clinician', 'timeFrame': 'Baseline, Month 6, Month 12, Month 18 and Month 24', 'description': 'Clinician Scores: 0 to 100; High score is a worse outcome'}, {'measure': 'Vineland Adaptive Behavior Scales | Third Edition', 'timeFrame': 'Baseline, Month 6, Month 12, Month 18 and Month 24', 'description': 'Caregiver Scores: 0 to 2; Total Scores: Receptive 0-78; Expressive 0-98, Written 0-76, Personal 0-110, Domestic 0-60, Community 0-116, Interpersonal 0- 86, Play and Leisure 0-72, Coping 0-66, Fine Motor 0- 68; Gross Motor 0-86: High score is a better outcome'}, {'measure': 'Observer-Reported Communication Ability', 'timeFrame': 'Baseline, Month 6, Month 12, Month 18 and Month 24', 'description': 'Caregiver Scores: No or only once; Sometimes, Yes, almost all the time; Yes, almost all the time is better outcome'}, {'measure': 'Sensory Profile-2', 'timeFrame': 'Baseline, Month 6, Month 12, Month 18 and Month 24', 'description': 'Caregiver Scores: 1 to 5; Total Score: 0 to 625; High score is a worse outcome'}, {'measure': 'Quality of Life Inventory-Disability', 'timeFrame': 'Baseline, Week 6, Month 6, Month 12, Month 18 and Month 24', 'description': 'Caregiver Scores: Never, Rarely, Sometimes, Often, Very often; Very Often is better outcome'}, {'measure': 'STXBP1-Clinical Severity Assessment-Caregiver', 'timeFrame': 'Baseline, Week 4, Week 8, Week 12, Month 4, Month 6, Month 12, Month 18 and Month 24', 'description': 'Caregiver Scores: 0 to 100; High score is a worse outcome'}, {'measure': 'Aberrant Behavior Checklist', 'timeFrame': 'Baseline, Month 6, Month 12, Month 18 and Month 24', 'description': 'Caregiver Scores: 0 to 3; Total Score: 0-174; High score is a worse outcome'}, {'measure': 'Child Behavior Checklist', 'timeFrame': 'Baseline, Month 6, Month 12, Month 18 and Month 24', 'description': 'Caregiver Scores: 0 to 2; Total Behavior: 0 to 224; High score is a worse outcome'}, {'measure': "Child's Sleep Habits", 'timeFrame': 'Baseline, Month 6, Month 12, Month 18 and Month 24', 'description': 'Caregiver Scores: 1 to 3; High score is a worse outcome'}, {'measure': 'Seizure frequency', 'timeFrame': 'Baseline and daily though Month 24', 'description': 'Caregiver assessment: Change from baseline in seizure experience as recorded in a seizure diary'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['STXBP1', 'Gene therapy'], 'conditions': ['Developmental and Epileptic Encephalopathy']}, 'descriptionModule': {'briefSummary': 'The goal of this clinical trial is to learn about the safety of CAP-002 gene therapy in children with Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy. It will also provide information about whether CAP-002 demonstrates efficacy in treating children with STXBP1 with and without seizures.\n\nParticipants will have a single infusion of CAP-002, visit the clinic regularly for 2 years for checkups and tests and have seizures recorded in a diary by their caregiver.', 'detailedDescription': "This is a Phase 1/2a, FIH, open-label, multi-center, dose-escalation trial to assess the safety, tolerability, and efficacy of a single intravenous (IV) dose of CAP-002 administered to participants with syntaxin-binding protein#1 (STXBP1) encephalopathy ages ≥18 months to \\<8 years of age.\n\nApproximately 12 participants will be dosed in this trial. Phase 1 is a dose escalation phase that will dose approximately 6 participants divided into 2 cohorts (Cohort 1 and Cohort 2) while Phase 2a will have 1 dose cohort and dose approximately 6 participants. Participants in Phase 1 will be dosed sequentially in each cohort. Phase 2a will allow participants to be dosed concurrently if safety and tolerability data from Phase 1 are deemed acceptable.\n\nParticipants will receive a single intravenous infusion of CAP-002 and will then be followed for 2 years with safety measures, assessments to measure changes from Baseline in development, language, cognition, motor skills and behavior, a seizure diary and structured caregiver interviews.\n\nUpon completion of the study or at the participant's final visit they will be invited to participate in a 3 year safety follow up study."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '7 Years', 'minimumAge': '18 Months', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\nMale or female, ≥18 months to \\<8 years of age;\n\nHas diagnosis of developmental encephalopathy due to an STXBP1 mutation with confirmation of a pathogenic or likely pathogenic STXBP1 gene mutation.\n\nHas a legally authorized representative (LAR) willing and able to complete the informed consent process, willing to comply with trial procedures, and able to travel for repeat visits.\n\nIs stable on any medication regimens (if being administered to control the signs and symptoms of underlying disease) for at least 6 weeks prior to trial entry and expected to be stable for at least 12 weeks post-CAP-002 administration.\n\nExclusion Criteria:\n\nHistory of prior gene therapy;\n\nTreatment with antisense oligonucleotide therapy within 6 months;\n\nPresence of a confirmed mutation in a gene other than STXBP1 that is known to contribute to a neurodevelopmental disability or epilepsy;\n\nHas presence of a significant non-STXBP1-related central nervous impairment/behavioral disturbance that would confound the scientific rigor or interpretation of results of the trial;\n\nHistory of prematurity (defined as gestational age \\<35 weeks), history of low birth weight (\\<2.5 kg) and/or intra-uterine growth restriction, significant interventricular hemorrhage, structural brain deficit, or congenital heart disease;\n\nKnown contraindication to immunosuppression or other protocol-defined medications, including but not limited to corticosteroids or PPIs;\n\nClinically significant abnormalities in safety lab tests, vital signs;\n\nOther illnesses or medications that may affect the interpretation of the study results;\n\nPositive anti-capsid antibody test result.'}, 'identificationModule': {'nctId': 'NCT06983158', 'briefTitle': 'A Clinical Trial of CAP-002 Gene Therapy in Pediatric Patients With Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy', 'organization': {'class': 'INDUSTRY', 'fullName': 'Capsida Biotherapeutics, Inc.'}, 'officialTitle': 'A Phase 1/2a, Open-Label, Multi-Center, Dose-Escalation Trial to Assess Safety, Tolerability, and Efficacy of a Single Dose of CAP-002 Gene Therapy Administered to Pediatric Patients With Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy', 'orgStudyIdInfo': {'id': 'CAP-002-101'}, 'secondaryIdInfos': [{'id': 'SYNRGY', 'type': 'OTHER', 'domain': 'Capsida Biotherapeutics'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Dose Level 1', 'description': 'Participants will receive a single dose of STXBP1, administered IV', 'interventionNames': ['Drug: gene therapy']}, {'type': 'EXPERIMENTAL', 'label': 'Dose Level 2', 'description': 'Participants will receive a single dose of STXBP1, administered IV', 'interventionNames': ['Drug: gene therapy']}], 'interventions': [{'name': 'gene therapy', 'type': 'DRUG', 'description': 'Intra-venous gene therapy', 'armGroupLabels': ['Dose Level 1', 'Dose Level 2']}]}, 'contactsLocationsModule': {'locations': [{'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': 'Colorado Child Health Research Institute', 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '10021', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Weill Cornell Medicine', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '19146', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "Buerger Center for Advanced Pediatric Care, Children's Hospital of Philadelphia", 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': "Texas Children's Hospital", 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}], 'overallOfficials': [{'name': 'Melanie Brandabur, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Capsida Biotherapeutics'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Capsida Biotherapeutics, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}