Ignite Creation Date:
2025-12-26 @ 11:00 AM
Ignite Modification Date:
2026-01-13 @ 10:54 AM
Study NCT ID:
NCT04901806
Status:
TERMINATED
Last Update Posted:
2024-03-28
First Post:
2021-05-20
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study of PBI-200 in Subjects With NTRK-Fusion-Positive Solid Tumors
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D001932', 'term': 'Brain Neoplasms'}, {'id': 'D058405', 'term': 'Desmoplastic Small Round Cell Tumor'}], 'ancestors': [{'id': 'D016543', 'term': 'Central Nervous System Neoplasms'}, {'id': 'D009423', 'term': 'Nervous System Neoplasms'}, {'id': 'D009371', 'term': 'Neoplasms by Site'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D012509', 'term': 'Sarcoma'}, {'id': 'D018204', 'term': 'Neoplasms, Connective and Soft Tissue'}, {'id': 'D009370', 'term': 'Neoplasms by Histologic Type'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL', 'interventionModelDescription': 'Dose Escalation: Single-subject cohorts will be enrolled initially, until a subject has a Grade 2 or greater adverse event (AE), at which time a 3+3 design will be utilized. Dose escalation will continue until the maximum-tolerated dose (MTD) is reached, or the Recommended Phase 2 Dose (RP2D) is established.\n\nCohort Expansion: Two cohorts will be opened to accrual. Cohort A will enroll subjects with a non-brain primary tumor and Cohort B will enroll subjects with a primary brain tumor.'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 29}}, 'statusModule': {'whyStopped': 'Sponsor terminated development of PBI-200', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2021-07-20', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-03', 'completionDateStruct': {'date': '2023-07-26', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-03-26', 'studyFirstSubmitDate': '2021-05-20', 'studyFirstSubmitQcDate': '2021-05-20', 'lastUpdatePostDateStruct': {'date': '2024-03-28', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2021-05-26', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2023-07-26', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Phase 1: Number of patients with AEs', 'timeFrame': 'Through study completion, estimated as an average of 36 months', 'description': 'Severity of AEs will be assessed according to the NCI CTCAE v5.0'}, {'measure': 'Phase 1: Recommended Phase 2 Dose', 'timeFrame': 'Approximately 12 months'}, {'measure': 'Phase 2: Cohort A - Overall Response Rate (ORR)', 'timeFrame': 'Through study completion, estimated as an average of 36 months', 'description': 'Assessed using Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1'}, {'measure': 'Phase 2: Cohort B - ORR', 'timeFrame': 'Through study completion, estimated as an average of 36 months', 'description': 'Assessed using Response Assessment in Neuro-Oncology (RANO) criteria'}], 'secondaryOutcomes': [{'measure': 'Phase 1: Area under the plasma drug concentration-time curve from 0 to 24 hours after one dose and after 28 doses', 'timeFrame': '29 days'}, {'measure': 'Phase 1: ORR', 'timeFrame': 'Through study completion, estimated as an average of 36 months', 'description': 'Assessed by RECIST for subjects with non-brain primary tumors and by RANO for subjects with primary brain tumors'}, {'measure': 'Duration of Response (DoR)', 'timeFrame': 'Through study completion, estimated as an average of 36 months', 'description': 'Assessed by RECIST for subjects with non-brain primary tumors and by RANO for subjects with primary brain tumors'}, {'measure': 'Progression-free Survival', 'timeFrame': 'Through study completion, estimated as an average of 36 months', 'description': 'Assessed by RECIST for subjects with non-brain primary tumors and by RANO for subjects with primary brain tumors'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['NTRK', 'NTRK Fusion', 'Resistance Mutation'], 'conditions': ['Solid Tumor, Adult', 'Brain Tumor, Primary', 'Desmoplastic Small Round Cell Tumor']}, 'descriptionModule': {'briefSummary': 'This is a first-in-human, open-label, multicenter, dose-escalation, safety, PK, and biomarker study of PBI-200 in subjects with NTRK-fusion-positive advanced or metastatic solid tumors.', 'detailedDescription': 'This is a first-in-human, open-label, multicenter, dose-escalation, safety, PK, and biomarker study of PBI-200 in subjects with NTRK-fusion-positive advanced or metastatic solid tumors. Phase 1 will also include subjects with NTRK-amplified advanced or metastatic solid tumors or refractory EWSR1-WT1-fusion-positive desmoplastic small round cell tumors (DSRCTs).\n\nPhase 1 is the dose-escalation portion of the study in which the evaluation of safety and tolerability and establishing the RP2D are primary objectives. Once the RP2D has been established, two expansion cohorts will open to accrual, a Non-Brain Primary Tumor cohort and a Primary Brian Tumor cohort.\n\nAlthough this was intended to be a Phase 1/2 trial, the trial was terminated without proceeding to Phase 2.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Key Inclusion Criteria:\n\n* Subject has one of the following solid tumors which has progressed on or following at least one systemic therapy regimen administered for advanced or metastatic disease or for which no approved therapy exists:\n\n * NTRK-fusion-positive, locally advanced (i.e., not amenable to surgical resection) or metastatic solid tumor Note: Subjects with any grade of malignant glioma previously treated with systemic therapy are eligible.\n\nPhase 1\n\n* NTRK-gene amplified, locally advanced or metastatic solid tumor\n* EWSR1-WT1-positive DSRCTs.\n* Subjects with NTRK-fusion-positive solid tumors other than primary brain tumors must have previously received treatment with a TRK inhibitor, unless the subject does not have access to TRK-inhibitor therapy (e.g., no TRK inhibitor is marketed and available to the subject in the subject's country) or the subject has declined treatment with available marketed TRK inhibitors.\n* Subjects with NTRK-gene amplified solid tumors, primary brain tumors or EWSR1-WT1-positive DSRCTs may have received prior treatment with a TRK inhibitor but this is not required.\n\nPhase 2\n\n* Has measurable disease by RECIST v1.1 for subjects with non-brain primary tumors or RANO criteria for subjects with primary brain tumors.\n* Subjects with non-brain primary tumors must have previously received treatment with a TRK inhibitor and a documented resistance mutation(s) (e.g., solvent front, gatekeeper or xDFG mutation). Archival tissue from a prior biopsy taken after the subject completed TRK inhibitor treatment but prior to additional systemic therapy may be used to meet this eligibility criterion with Medical Monitor approval.\n* Subjects with primary brain tumors may have received prior treatment with a TRK inhibitor but this is not required. Biopsies of brain tumors are not required for eligibility.\n\nKey Exclusion Criteria:\n\n* Cytotoxic chemotherapy, biologic agent, investigational agent, or radiation therapy ≤ 3 weeks prior to the first dose of PBI-200 (6 weeks for nitrosoureas).\n\n * Subjects with either primary brain tumors or brain metastasis must have completed brain radiation therapy 12 weeks prior to the brain MRI obtained within 4 weeks of the first dose of PBI-200.\n* Small-molecule kinase inhibitors or hormonal agents ≤ 14 days and within 5 half-lives prior to the first dose of PBI-200."}, 'identificationModule': {'nctId': 'NCT04901806', 'briefTitle': 'Study of PBI-200 in Subjects With NTRK-Fusion-Positive Solid Tumors', 'organization': {'class': 'INDUSTRY', 'fullName': 'Pyramid Biosciences'}, 'officialTitle': 'A Phase 1/2 Study of PBI-200 in Subjects With NTRK-Fusion-Positive Advanced or Metastatic Solid Tumors', 'orgStudyIdInfo': {'id': 'PBI-200-101'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Phase 1 Dose Escalation', 'interventionNames': ['Drug: PBI-200']}, {'type': 'EXPERIMENTAL', 'label': 'Phase 2 Cohort Expansion', 'interventionNames': ['Drug: PBI-200']}], 'interventions': [{'name': 'PBI-200', 'type': 'DRUG', 'description': 'PBI-200 will be administered orally over continuous 28-day cycles', 'armGroupLabels': ['Phase 1 Dose Escalation', 'Phase 2 Cohort Expansion']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90404', 'city': 'Santa Monica', 'state': 'California', 'country': 'United States', 'facility': 'John Wayne Cancer Institute at St. Johns Health Center', 'geoPoint': {'lat': 34.01949, 'lon': -118.49138}}, {'zip': '94305', 'city': 'Stanford', 'state': 'California', 'country': 'United States', 'facility': 'Stanford Hospital and Clinics', 'geoPoint': {'lat': 37.42411, 'lon': -122.16608}}, {'zip': '80218', 'city': 'Denver', 'state': 'Colorado', 'country': 'United States', 'facility': 'Sarah Cannon Research Institute at HealthONE', 'geoPoint': {'lat': 39.73915, 'lon': -104.9847}}, {'zip': '32746', 'city': 'Lake Mary', 'state': 'Florida', 'country': 'United States', 'facility': 'Florida Cancer Specialists', 'geoPoint': {'lat': 28.75888, 'lon': -81.31784}}, {'zip': '33136', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': 'Sylvester Comprehensive Cancer Center (University of Miami)', 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '33176', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': 'Miami Cancer Institute', 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '02215', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Dana-Farber Cancer Institute', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '10532', 'city': 'Hawthorne', 'state': 'New York', 'country': 'United States', 'facility': 'Westchester Medical Center', 'geoPoint': {'lat': 41.10732, 'lon': -73.79597}}, {'zip': '19107', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': 'Thomas Jefferson University Hospital', 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '37203', 'city': 'Nashville', 'state': 'Tennessee', 'country': 'United States', 'facility': 'Tennessee Oncology, PLLC', 'geoPoint': {'lat': 36.16589, 'lon': -86.78444}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'country': 'United States', 'facility': 'The University of Texas MD Anderson Cancer Center', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '3000', 'city': 'Melbourne', 'state': 'Victoria', 'country': 'Australia', 'facility': 'Peter MacCallum Cancer Centre', 'geoPoint': {'lat': -37.814, 'lon': 144.96332}}, {'zip': '2100', 'city': 'Copenhagen', 'country': 'Denmark', 'facility': 'Rigshospitalet, University Hospital of Copenhagen', 'geoPoint': {'lat': 55.67594, 'lon': 12.56553}}, {'zip': '33076', 'city': 'Bordeaux', 'country': 'France', 'facility': 'Institut Bergonie', 'geoPoint': {'lat': 44.84124, 'lon': -0.58046}}, {'zip': '69008', 'city': 'Lyon', 'country': 'France', 'facility': 'Centre Léon Bérard', 'geoPoint': {'lat': 45.74906, 'lon': 4.84789}}, {'zip': '75015', 'city': 'Paris', 'country': 'France', 'facility': 'Hopital Europeen Georges Pompidou', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '86000', 'city': 'Poitiers', 'country': 'France', 'facility': 'CHU Poitiers - Hopital la Miletrie', 'geoPoint': {'lat': 46.58261, 'lon': 0.34348}}, {'zip': '94805', 'city': 'Villejuif', 'country': 'France', 'facility': 'Institut Gustave Roussy', 'geoPoint': {'lat': 48.7939, 'lon': 2.35992}}, {'zip': '60528', 'city': 'Frankfurt am Main', 'country': 'Germany', 'facility': 'Dr. Senckenberg Institute of Neurooncology', 'geoPoint': {'lat': 50.11552, 'lon': 8.68417}}, {'zip': '69120', 'city': 'Heidelberg', 'country': 'Germany', 'facility': 'Universitaetsklinikum Heidelberg', 'geoPoint': {'lat': 49.40768, 'lon': 8.69079}}, {'zip': '44625', 'city': 'Herne', 'country': 'Germany', 'facility': 'Marienhospital Herne', 'geoPoint': {'lat': 51.5388, 'lon': 7.22572}}, {'city': 'Pok Fu Lam', 'country': 'Hong Kong', 'facility': 'Queen Mary Hospital', 'geoPoint': {'lat': 22.26861, 'lon': 114.12924}}, {'city': 'Shatin', 'country': 'Hong Kong', 'facility': 'Prince of Wales Hospital', 'geoPoint': {'lat': 22.38333, 'lon': 114.18333}}, {'zip': '60126', 'city': 'Ancona', 'country': 'Italy', 'facility': 'Azienda Ospedaliero Universitaria delle Marche', 'geoPoint': {'lat': 43.60717, 'lon': 13.5103}}, {'zip': '20132', 'city': 'Milan', 'country': 'Italy', 'facility': 'IRCCS Ospedale San Raffaele', 'geoPoint': {'lat': 42.78235, 'lon': 12.59836}}, {'zip': '20133', 'city': 'Milan', 'country': 'Italy', 'facility': 'Fondazione IRCCS Istituto Nazionale Tumori', 'geoPoint': {'lat': 42.78235, 'lon': 12.59836}}, {'zip': '20141', 'city': 'Milan', 'country': 'Italy', 'facility': 'IRCCS (IEO) Istituto Europeo di Oncologia', 'geoPoint': {'lat': 42.78235, 'lon': 12.59836}}, {'zip': '80131', 'city': 'Napoli', 'country': 'Italy', 'facility': 'IRCCS Istituto Nazionale Tumori Fondazione Pascale', 'geoPoint': {'lat': 40.87618, 'lon': 14.5195}}, {'zip': '37126', 'city': 'Verona', 'country': 'Italy', 'facility': 'Azienda Ospedaliera Universitaria Integrata Verona', 'geoPoint': {'lat': 45.43854, 'lon': 10.9938}}, {'zip': '169610', 'city': 'Singapore', 'country': 'Singapore', 'facility': 'National Cancer Centre Singapore', 'geoPoint': {'lat': 1.28967, 'lon': 103.85007}}, {'zip': '13620', 'city': 'Seongnam-si', 'state': 'Gyeonggi-do', 'country': 'South Korea', 'facility': 'Seoul National University Bundang Hosptial', 'geoPoint': {'lat': 37.43861, 'lon': 127.13778}}, {'zip': '16247', 'city': 'Suwon', 'state': 'Gyeonggi-do', 'country': 'South Korea', 'facility': 'The Catholic University of Korea St. Vincent Hosptial', 'geoPoint': {'lat': 37.29111, 'lon': 127.00889}}, {'zip': '03722', 'city': 'Seoul', 'country': 'South Korea', 'facility': 'Severance Hosptial, Yonsei University Health System', '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': "The Catholic University of Korea Soul St. Mary's Hosptial", 'geoPoint': {'lat': 37.566, 'lon': 126.9784}}, {'zip': '08035', 'city': 'Barcelona', 'country': 'Spain', 'facility': 'Hospital Universitari Vall d Hebron', 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'zip': '08908', 'city': "L'Hospitalet de Llobregat", 'country': 'Spain', 'facility': 'ICO l Hospitalet', 'geoPoint': {'lat': 41.35967, 'lon': 2.10028}}, {'zip': '28034', 'city': 'Madrid', 'country': 'Spain', 'facility': 'Hospital Universitario Ramon y Cajal', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}, {'zip': '28046', 'city': 'Madrid', 'country': 'Spain', 'facility': 'Hospital Universitario La Paz', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}, {'zip': '08195', 'city': 'Sant Cugat del Vallès', 'country': 'Spain', 'facility': 'Hospital General de Catalunya', 'geoPoint': {'lat': 41.47063, 'lon': 2.08611}}, {'zip': 'M20 4BX', 'city': 'Manchester', 'country': 'United Kingdom', 'facility': 'The Christie', 'geoPoint': {'lat': 53.48095, 'lon': -2.23743}}, {'zip': 'SM2 5PT', 'city': 'Sutton', 'country': 'United Kingdom', 'facility': 'Royal Marsden Hospital Institute Cancer Research', 'geoPoint': {'lat': 51.35, 'lon': -0.2}}], 'overallOfficials': [{'name': 'Chief Medical Officer', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Pyramid Biosciences'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Pyramid Biosciences', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}