Ignite Creation Date:
2025-12-24 @ 9:30 PM
Ignite Modification Date:
2026-02-20 @ 6:41 PM
Study NCT ID:
NCT03790332
Status:
COMPLETED
Last Update Posted:
2025-10-21
First Post:
2018-12-12
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Phase 1/2 Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects With Chronic Graft Versus Host Disease (cGVHD)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000092122', 'term': 'Bronchiolitis Obliterans Syndrome'}, {'id': 'D006086', 'term': 'Graft vs Host Disease'}], 'ancestors': [{'id': 'D000092124', 'term': 'Organizing Pneumonia'}, {'id': 'D001989', 'term': 'Bronchiolitis Obliterans'}, {'id': 'D001988', 'term': 'Bronchiolitis'}, {'id': 'D001991', 'term': 'Bronchitis'}, {'id': 'D001982', 'term': 'Bronchial Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D008173', 'term': 'Lung Diseases, Obstructive'}, {'id': 'D008171', 'term': 'Lung Diseases'}, {'id': 'D007154', 'term': 'Immune System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C551803', 'term': 'ibrutinib'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 59}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2018-11-19', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-05', 'completionDateStruct': {'date': '2025-10-09', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-10-17', 'studyFirstSubmitDate': '2018-12-12', 'studyFirstSubmitQcDate': '2018-12-27', 'lastUpdatePostDateStruct': {'date': '2025-10-21', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2018-12-31', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-10-09', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Part A- PK (measured by AUC) will be reported descriptively', 'timeFrame': 'Approximately 24 months'}, {'measure': 'Part B- PK (measured by AUC) will be reported descriptively', 'timeFrame': 'Approximately 7 years'}, {'measure': 'Number of patients with adverse events as a measure of safety and tolerability', 'timeFrame': 'Approximately 7 years'}], 'secondaryOutcomes': [{'measure': 'Part A- Number of patients with adverse events as a measure of safety and tolerability', 'timeFrame': 'Approximately 24 months'}, {'measure': 'Part A- Pharmacodynamic effects as measured by in vitro BTK occupancy will be reported descriptively', 'timeFrame': 'Approximately 24 months'}, {'measure': 'Part A continuation cohort and Part B-Response rate at 24 weeks', 'timeFrame': 'Approximately 6 months after last subject in enrolled'}, {'measure': 'Part A continuation cohort and Part B- Duration of response (DOR)', 'timeFrame': 'Up to 48 weeks'}, {'measure': 'Part A continuation cohort and Part B-Overall survival (OS)', 'timeFrame': 'Approximately 5 years after last subject enrolled'}, {'measure': 'Part A continuation cohort and Part B-Late Effects Surveillance', 'timeFrame': 'Up to 5 years post enrollment'}, {'measure': 'Growth Parameter height in meters will be reported descriptively', 'timeFrame': 'Up to 5 years post enrollment', 'description': 'Subjects will be monitored for growth and development'}, {'measure': 'Growth Parameter weight in kilograms will be reported descriptively.', 'timeFrame': 'Up to 5 years post enrollment', 'description': 'Subjects will be monitored for growth and development'}, {'measure': 'Available immune reconstitution laboratory parameters will be reported descriptively', 'timeFrame': 'Up to 5 years post enrollment', 'description': 'Subjects will be monitored for immune reconstitution'}, {'measure': 'Late effects (Adverse events suspected to be related to treatment) will be quantified and reported descriptively', 'timeFrame': 'Up to 5 years post enrollment'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['cGVHD', 'chronic graft versus host disease', 'PCYC1146IM', 'Pediatric', 'GVHD', 'chronic', '1146', 'Pharmacyclics', 'PCYC', 'Imbruvica', 'Ibrutinib', 'Immunology', 'graft versus host disease', 'corticosteroids', 'prednisone', 'PCI32765', 'PCYC1146', 'refractory', 'new onset graft versus host disease', 'refractory graft versus host disease', 'moderate cGVHD', 'severe cGVHD', 'moderate chronic graft versus host disease', 'severe chronic graft versus host disease'], 'conditions': ['Chronic Graft Versus Host Disease']}, 'descriptionModule': {'briefSummary': 'Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects with Chronic Graft Versus Host Disease (cGVHD)'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '21 Years', 'minimumAge': '1 Year', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Eligibility:\n\nInclusion Criteria:\n\n1. Part A: Subjects with moderate or severe cGVHD after failure of 1 or more lines of systemic therapy\n2. Part B: Subjects with moderate or severe cGVHD after failure of 1 or more lines of systemic therapy, or subjects with new onset moderate or severe cGVHD and in need of systemic immunosuppression\n3. History of allogeneic stem cell transplantation\n4. Age\n\n * Part A: ≥1 to \\<12 years of age at the time of enrollment\n * Part B: ≥1 to \\<22 years of age at the time of enrollment\n5. Karnofsky or Lansky (subjects \\<16 years of age) performance status ≥60\n\nKey Eligibility:\n\nExclusion Criteria:\n\n1. Presence of single organ genito-urinary involvement as the only manifestation of cGVHD\n2. Received an investigational agent within 28 days before enrollment.\n3. Received donor lymphocyte infusion (DLI) within 56 days before enrollment\n4. Progressive underlying malignant disease or active post-transplant lymphoproliferative disease\n5. Any uncontrolled infection or active infection requiring ongoing systemic treatment\n6. Known bleeding disorders\n7. Active hepatitis C virus (HCV) or hepatitis B virus (HBV)'}, 'identificationModule': {'nctId': 'NCT03790332', 'briefTitle': 'Phase 1/2 Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects With Chronic Graft Versus Host Disease (cGVHD)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Pharmacyclics LLC.'}, 'officialTitle': 'Phase 1/2 Dose Finding and Safety Study of Ibrutinib in Pediatric Subjects With Chronic Graft Versus Host Disease (cGVHD)', 'orgStudyIdInfo': {'id': 'PCYC-1146-IM'}, 'secondaryIdInfos': [{'id': '2017-004558-41', 'type': 'EUDRACT_NUMBER'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Phase 1/2', 'description': 'Part A: Subjects ≥1 to \\<12 years of age with moderate or severe cGVHD after failure of 1 or more lines of systemic therapy, will receive oral ibrutinib once daily to determine Recommended Pediatric Equivalent Dose (RPED).\n\nPart A Continuation: Subjects participating in Part A may continue receiving daily ibrutinib until the RPED is determined, at which time their dose may be adjusted to the RPED.\n\nPart B: Subjects ≥1 to \\<12 years of age( upper age limit is \\< 22 years) with moderate or severe cGVHD after failure of 1 or more lines of systemic therapy or with newly diagnosed moderate or severe cGVHD will be dosed at the RPED. Subjects ≥12 will be given 420mg orally ibrutinib once daily.', 'interventionNames': ['Drug: Ibrutinib']}], 'interventions': [{'name': 'Ibrutinib', 'type': 'DRUG', 'otherNames': ['IMBRUVICA®', 'PCI-32765'], 'description': 'Ibrutinib capsule, tablet, or suspension administered orally once daily', 'armGroupLabels': ['Phase 1/2']}]}, 'contactsLocationsModule': {'locations': [{'zip': '91010', 'city': 'Duarte', 'state': 'California', 'country': 'United States', 'facility': 'City of Hope', 'geoPoint': {'lat': 34.13945, 'lon': -117.97729}}, {'zip': '92123', 'city': 'San Diego', 'state': 'California', 'country': 'United States', 'facility': "Rady Children's Hospital", 'geoPoint': {'lat': 32.71571, 'lon': -117.16472}}, {'zip': '94158', 'city': 'San Francisco', 'state': 'California', 'country': 'United States', 'facility': 'University of California', 'geoPoint': {'lat': 37.77493, 'lon': -122.41942}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': "Children's Hospital", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '20010', 'city': 'Washington D.C.', 'state': 'District of Columbia', 'country': 'United States', 'facility': "Children's National Medical Center", 'geoPoint': {'lat': 38.89511, 'lon': -77.03637}}, {'zip': '33701', 'city': 'St. Petersburg', 'state': 'Florida', 'country': 'United States', 'facility': "Johns Hopkins All Children's Hospital", 'geoPoint': {'lat': 27.77086, 'lon': -82.67927}}, {'zip': '60611', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': "Ann & Robert H Lurie Children's Hospital of Chicago", 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '21287', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Johns Hopkins University', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '02215', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Dana-Farber Cancer Institute', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '55454', 'city': 'Minneapolis', 'state': 'Minnesota', 'country': 'United States', 'facility': "University of Minnesota Masonic Children's Hospital", 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}, {'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Washington University', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '07601', 'city': 'Hackensack', 'state': 'New Jersey', 'country': 'United States', 'facility': 'Hackensack University Medical Center', 'geoPoint': {'lat': 40.88593, 'lon': -74.04347}}, {'zip': '10065', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Memorial Sloan Kettering Cancer Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': "Cincinnati Children's Hospital Medical Center", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '97239', 'city': 'Portland', 'state': 'Oregon', 'country': 'United States', 'facility': 'Oregon Health and Science University', 'geoPoint': {'lat': 45.52345, 'lon': -122.67621}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "The Children's Hospital of Philadelphia", 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '38105-2729', 'city': 'Memphis', 'state': 'Tennessee', 'country': 'United States', 'facility': "St. Jude Children's Research Hospital", 'geoPoint': {'lat': 35.14953, 'lon': -90.04898}}, {'zip': '84113', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': "Primary Children's Hospital", 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '98109', 'city': 'Seattle', 'state': 'Washington', 'country': 'United States', 'facility': 'Fred Hutchinson Cancer Research Center', 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}, {'zip': '53226', 'city': 'Milwaukee', 'state': 'Wisconsin', 'country': 'United States', 'facility': 'Medical College of Wisconsin', 'geoPoint': {'lat': 43.0389, 'lon': -87.90647}}, {'zip': '2145', 'city': 'Westmead', 'state': 'New South Wales', 'country': 'Australia', 'facility': "Cancer Center for Children. The Children's Hospital at Westmead", 'geoPoint': {'lat': -33.80383, 'lon': 150.98768}}, {'zip': '4101', 'city': 'South Brisbane', 'state': 'Queensland', 'country': 'Australia', 'facility': "Queensland Children's Hospital", 'geoPoint': {'lat': -27.48034, 'lon': 153.02049}}, {'zip': '3052', 'city': 'Parkville', 'state': 'Victoria', 'country': 'Australia', 'facility': "The Royal Children's Hospital", 'geoPoint': {'lat': -37.78333, 'lon': 144.95}}, {'zip': '2031', 'city': 'Randwick', 'country': 'Australia', 'facility': "Sydney Children's Hospital", 'geoPoint': {'lat': -33.91439, 'lon': 151.24895}}, {'zip': '1090', 'city': 'Vienna', 'country': 'Austria', 'facility': 'St. Anna Kinderspital', 'geoPoint': {'lat': 48.20849, 'lon': 16.37208}}, {'zip': 'V6H 3V4', 'city': 'Vancouver', 'state': 'British Columbia', 'country': 'Canada', 'facility': "children's and Women's Health Centre of British Columbia", 'geoPoint': {'lat': 49.24966, 'lon': -123.11934}}, {'zip': 'H3T1C5', 'city': 'Montreal', 'state': 'Quebec', 'country': 'Canada', 'facility': 'CHU Sainte-Justine', 'geoPoint': {'lat': 45.50884, 'lon': -73.58781}}, {'zip': '44093', 'city': 'Nantes', 'country': 'France', 'facility': 'CHU Nantes - Hopital Enfant Adolescent', 'geoPoint': {'lat': 47.21725, 'lon': -1.55336}}, {'zip': '75019', 'city': 'Paris', 'country': 'France', 'facility': 'Hôpital Robert-Debré Ap-Hp', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '13353', 'city': 'Berlin', 'country': 'Germany', 'facility': 'Charite-Universitaetsmedizin Berlin', 'geoPoint': {'lat': 52.52437, 'lon': 13.41053}}, {'zip': '9112001', 'city': 'Jerusalem', 'country': 'Israel', 'facility': 'Hadassah Medical Centre', 'geoPoint': {'lat': 31.76904, 'lon': 35.21633}}, {'zip': '4920235', 'city': 'Petah Tikva', 'country': 'Israel', 'facility': "Schneider Children's Medical Center in Israel", 'geoPoint': {'lat': 32.08707, 'lon': 34.88747}}, {'zip': '5262100', 'city': 'Ramat Gan', 'country': 'Israel', 'facility': "The Edmond and Lily Safra Children's Hospital", 'geoPoint': {'lat': 32.08227, 'lon': 34.81065}}, {'zip': '27100', 'city': 'Pavia', 'state': 'PV', 'country': 'Italy', 'facility': 'U.O.C. Ematologia Oncoematologia Pediatrica', 'geoPoint': {'lat': 45.19205, 'lon': 9.15917}}, {'zip': '20900', 'city': 'Monza', 'country': 'Italy', 'facility': 'Fondazione MBBM-Clinica Pediatrica', 'geoPoint': {'lat': 45.58005, 'lon': 9.27246}}, {'zip': '00165', 'city': 'Roma', 'country': 'Italy', 'facility': 'Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica', 'geoPoint': {'lat': 44.99364, 'lon': 11.10642}}, {'zip': '10126', 'city': 'Turin', 'country': 'Italy', 'facility': 'S.C. Farmacia Pediatrica - Presidio Ospedaliero Infantile Regina Margherita', 'geoPoint': {'lat': 45.07049, 'lon': 7.68682}}, {'zip': '3584 CS', 'city': 'Utrecht', 'country': 'Netherlands', 'facility': 'Princess Maxima Center', 'geoPoint': {'lat': 52.09083, 'lon': 5.12222}}, {'zip': '117997', 'city': 'Moscow', 'country': 'Russia', 'facility': 'Federal State Budgetary Institution "Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology', 'geoPoint': {'lat': 55.75204, 'lon': 37.61781}}, {'zip': '197022', 'city': 'Saint Petersburg', 'country': 'Russia', 'facility': 'Federal State Budgetary Educational Institution of Higher Professional Education "LP.Pavlov First Saint Petersburg State Medical', 'geoPoint': {'lat': 59.93863, 'lon': 30.31413}}, {'zip': '03080', 'city': 'Seoul', 'country': 'South Korea', 'facility': 'Seoul National University Hospital', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}, {'zip': '05505', 'city': 'Seoul', 'country': 'South Korea', 'facility': 'Asan Medical Center', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}, {'zip': '06351', 'city': 'Seoul', 'country': 'South Korea', 'facility': 'Samsung Medical Center', 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}, {'zip': '06591', 'city': 'Seoul', 'country': 'South Korea', 'facility': "Seoul Saint Mary's Hospital", 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}, {'zip': '08035', 'city': 'Barcelona', 'country': 'Spain', 'facility': "Hospital Universitario Vall d'Hebron", 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'zip': '28009', 'city': 'Madrid', 'country': 'Spain', 'facility': 'Hospital Infantil Universitario Nino Jesus', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}, {'zip': '28046', 'city': 'Madrid', 'country': 'Spain', 'facility': 'Hospital Universitario Universitario La Paz', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}, {'zip': 'WC1N 3JH', 'city': 'London', 'state': 'Greater London', 'country': 'United Kingdom', 'facility': 'Safari Day Care, Great Ormond Street Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'overallOfficials': [{'name': 'Gauri Sunkersett', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Pharmacyclics LLC.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Pharmacyclics LLC.', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Janssen Research & Development, LLC', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}