Ignite Creation Date:
2025-12-25 @ 2:07 AM
Ignite Modification Date:
2026-01-22 @ 6:35 AM
Study NCT ID:
NCT06316960
Status:
RECRUITING
Last Update Posted:
2024-08-22
First Post:
2024-03-12
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Safety and Efficacy of Avapritinib in Relapsed or Refractory Pediatric CBF-AML With KIT Mutation
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D015470', 'term': 'Leukemia, Myeloid, Acute'}, {'id': 'D012008', 'term': 'Recurrence'}], 'ancestors': [{'id': 'D007951', 'term': 'Leukemia, Myeloid'}, {'id': 'D007938', 'term': 'Leukemia'}, {'id': 'D009370', 'term': 'Neoplasms by Histologic Type'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D020969', 'term': 'Disease Attributes'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000707147', 'term': 'avapritinib'}, {'id': 'D001374', 'term': 'Azacitidine'}, {'id': 'D000077209', 'term': 'Decitabine'}, {'id': 'D015255', 'term': 'Idarubicin'}, {'id': 'D003561', 'term': 'Cytarabine'}, {'id': 'D016179', 'term': 'Granulocyte Colony-Stimulating Factor'}], 'ancestors': [{'id': 'D001372', 'term': 'Aza Compounds'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D003562', 'term': 'Cytidine'}, {'id': 'D011741', 'term': 'Pyrimidine Nucleosides'}, {'id': 'D011743', 'term': 'Pyrimidines'}, {'id': 'D006573', 'term': 'Heterocyclic Compounds, 1-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}, {'id': 'D009705', 'term': 'Nucleosides'}, {'id': 'D009706', 'term': 'Nucleic Acids, Nucleotides, and Nucleosides'}, {'id': 'D012263', 'term': 'Ribonucleosides'}, {'id': 'D003630', 'term': 'Daunorubicin'}, {'id': 'D018943', 'term': 'Anthracyclines'}, {'id': 'D009279', 'term': 'Naphthacenes'}, {'id': 'D011084', 'term': 'Polycyclic Aromatic Hydrocarbons'}, {'id': 'D006841', 'term': 'Hydrocarbons, Aromatic'}, {'id': 'D006844', 'term': 'Hydrocarbons, Cyclic'}, {'id': 'D006838', 'term': 'Hydrocarbons'}, {'id': 'D011083', 'term': 'Polycyclic Compounds'}, {'id': 'D000617', 'term': 'Aminoglycosides'}, {'id': 'D006027', 'term': 'Glycosides'}, {'id': 'D002241', 'term': 'Carbohydrates'}, {'id': 'D001087', 'term': 'Arabinonucleosides'}, {'id': 'D003115', 'term': 'Colony-Stimulating Factors'}, {'id': 'D006023', 'term': 'Glycoproteins'}, {'id': 'D006001', 'term': 'Glycoconjugates'}, {'id': 'D016298', 'term': 'Hematopoietic Cell Growth Factors'}, {'id': 'D016207', 'term': 'Cytokines'}, {'id': 'D036341', 'term': 'Intercellular Signaling Peptides and Proteins'}, {'id': 'D010455', 'term': 'Peptides'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D001685', 'term': 'Biological Factors'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 50}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-03-01', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-08', 'completionDateStruct': {'date': '2027-03', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-08-21', 'studyFirstSubmitDate': '2024-03-12', 'studyFirstSubmitQcDate': '2024-03-12', 'lastUpdatePostDateStruct': {'date': '2024-08-22', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-03-19', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-03', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Composite remission rate (CRc)', 'timeFrame': 'The evaluation time point is day28-day35 from the start of regimen.', 'description': 'Composite remission rate (CRc), including the sum of the number of patients with complete remission (CR), complete remission with partial hematologic recovery (CRh), complete remission with incomplete blood count recovery (CRi), and morphologically leukemia-free (MLFS) as a percentage of the total number of patients who participated in the efficacy analysis.'}], 'secondaryOutcomes': [{'measure': 'Overall survival', 'timeFrame': 'From date of enrollment until the date of the occurrence of death or last follow-up, assessed up to 60 months.', 'description': 'Overall survival (OS) was defined as the date from enrollment to the date of death or last follow-up for surviving patients.'}, {'measure': 'Progression-free survival', 'timeFrame': 'From date of enrollment until the date of disease progression, confirmed relapse, or death, whichever occurred first, assessed up to 60 months.', 'description': 'Progression-free survival (PFS) was defined as the date from enrollment to the date of disease progression, confirmed relapse, or death, whichever occurred first.'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Avapritinib', 'KIT', 'CBF AML', 'Childhood', 'Relapsed/Refractory'], 'conditions': ['AML, Childhood', 'Relapse/Recurrence', 'Refractory AML', 'Core Binding Factor Acute Myeloid Leukemia', 'C-KIT Mutation']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to evaluate the efficacy and safety of avapritinib in relapsed or refractory pediatric core binding factor acute myeloid leukemia with KIT mutation.', 'detailedDescription': 'This is a multicenter, single-arm, prospective, and intervention trial. About 30% of core binding factor acute myeloid leukemia (CBF-AML) patients still relapse under current treatment. Some studies have found that KIT mutations, especially the D816V mutation, may predict relapse and decrease overall survival (OS) in CBF-AML. Avapritinib has been approved for the treatment of gastrointestinal stromal tumors with KIT or PDGFRA mutations. Avapritinib was also effective for the treatment of minimal residual disease in acute myeloid leukemia with t (8;21) and KIT mutation failing to immunotherapy after allogeneic hematopoietic stem cell transplantation in a single-center, retrospective report. 11 centers from China carry out the AVACBFKIT regimen including Avapritinib, hypomethylating agents and low dose chemotherapy for the treatment of relapsed or refractory pediatric CBF-AML with KIT mutation. The main focus of this study is to evaluate the efficacy and safety of avapritinib in the regimen.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Gender unlimited;\n2. Under 18 years;\n3. Diagnosis of acute myeloid leukemia (according to the 2022 WHO classification).\n4. Presence of t(8;21)/RUNX1::RUNX1T1 or inv(16)/t(16;16)/CBFβ::MYH11;\n5. KIT mutation;\n6. Refractory AML: AML patients who do not achieve CR or CRi after induction therapy;\n7. Relapsed AML: patients who achieved remission after consolidation therapy or transplantation, FISH confirmed that the fusion gene turned positive, or extramedullary leukemia infiltration;\n8. No active infections;\n9. Liver function: Tbil ≤2×ULN, ALT/AST ≤3×ULN, creatinine clearance ≥50ml/min;\n10. ECOG score \\<2;\n11. Expected survival time \\>12 weeks;\n12. Participants must have the ability to understand and be willing to participate in this study and must sign an informed consent form.\n\nExclusion Criteria:\n\n1. Have received prior treatment with avapritinib;\n2. Receiving other targeted therapies for AML at the same time, such as dasatinib, sorafenib, gilteritinib, venetoclax, etc;\n3. Presence of active uncontrolled infection (including bacterial, fungal, or viral infection);\n4. Present of significant underlying organ diseases: such as myocardial infarction, chronic heart failure, decompensated liver or kidney dysfunction；\n5. With other malignancies requiring treatment；\n6. Already enrolled in another interventional clinical study；\n7. The researchers determined that the individual is not suitable to participate in this trial.'}, 'identificationModule': {'nctId': 'NCT06316960', 'briefTitle': 'Safety and Efficacy of Avapritinib in Relapsed or Refractory Pediatric CBF-AML With KIT Mutation', 'organization': {'class': 'OTHER', 'fullName': "Children's Hospital of Soochow University"}, 'officialTitle': 'A Prospective, Multicenter Clinical Study on The Safety and Efficacy of Avapritinib in The Treatment of Relapsed/Refractory Pediatric Core Binding Factor Acute Myeloid Leukemia (CBF-AML) With KIT Mutation', 'orgStudyIdInfo': {'id': 'AVACBFKIT'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Relapsed/Refractory CBF-AML with KIT mutation', 'description': 'The relapsed/refractory patients will receive a combination treatment of decitabine/azacitidine+ IdAG (idarubicine + cytarabine + granulocyte stimulating factor)regimen along with avapritinib. CBF-AML with KIT mutated patients with molecular relapse after hematopoietic stem cell transplantation may receive avapritinib combined with demethylating agents or interferon or donor lymphocyte infusion without low-dose chemotherapy. The dose of avapritinib will start at 50mg/m2/d, and if platelets stabilize at 50 ×10\\^9/L and neutrophils stabilize above 1.0 ×10\\^9/L after one week, the dose can be increased to 100mg/m2/d, with a maximum daily dose of 100mg. Avapritinib should be discontinued in the presence of febrile neutropenia or active infection, and avapritinib can be resumed once the infection is controlled, with each treatment cycle not exceeding 28 days.', 'interventionNames': ['Drug: Avapritinib', 'Drug: Azacitidine Injection', 'Drug: Decitabine Injection', 'Drug: Idarubicin Hydrochloride', 'Drug: Cytarabine', 'Drug: Granulocyte Colony-Stimulating Factor']}], 'interventions': [{'name': 'Avapritinib', 'type': 'DRUG', 'otherNames': ['AYVAKIT'], 'description': '50mg/m2/day for weighing bodyweight \\>10kg, 1.65mg/kg/day for weighing ≤ 10kg, po, qd, d1-28.', 'armGroupLabels': ['Relapsed/Refractory CBF-AML with KIT mutation']}, {'name': 'Azacitidine Injection', 'type': 'DRUG', 'otherNames': ['Azacitidine'], 'description': '75mg/m2/d for weighing \\>10kg, 2.5mg/kg/d for weighing ≤ 10kg, d1-7, ivgtt, qd, more than 3 hours. Azacitidine and decitabine cannot be used simultaneously.', 'armGroupLabels': ['Relapsed/Refractory CBF-AML with KIT mutation']}, {'name': 'Decitabine Injection', 'type': 'DRUG', 'otherNames': ['Decitabine'], 'description': '20mg/m2/d for weighing \\>10kg, 0.67mg/kg/d for weighing ≤ 10kg, d1-5, ivgtt, qd, more than 3 hours. Azacitidine and decitabine cannot be used simultaneously.', 'armGroupLabels': ['Relapsed/Refractory CBF-AML with KIT mutation']}, {'name': 'Idarubicin Hydrochloride', 'type': 'DRUG', 'otherNames': ['Idarubicine'], 'description': '5mg/m2/day for weighing \\>10kg, 0.17mg/kg/day for weighing ≤ 10kg, d 6, 8, 10 (d 8, 10, 12 for azacitidine) ivgtt, qod, more than 1 hour at 10 am.', 'armGroupLabels': ['Relapsed/Refractory CBF-AML with KIT mutation']}, {'name': 'Cytarabine', 'type': 'DRUG', 'otherNames': ['CYTOSAR'], 'description': '10mg/m2/day for weighing \\>10kg, 0.33mg/kg/day for weighing ≤ 10kg, d6-15 （d8-17 for azacitidine ）, s.c., q12h.', 'armGroupLabels': ['Relapsed/Refractory CBF-AML with KIT mutation']}, {'name': 'Granulocyte Colony-Stimulating Factor', 'type': 'DRUG', 'otherNames': ['Recombinant Human Granulocyte Colony-Stimulating Factor Injection'], 'description': '300ug/day for weighing \\>10kg, 10ug/kg/day for weighing ≤10kg, d0-5, s.c., qd.', 'armGroupLabels': ['Relapsed/Refractory CBF-AML with KIT mutation']}]}, 'contactsLocationsModule': {'locations': [{'zip': '230000', 'city': 'Hefei', 'state': 'Anhui', 'status': 'NOT_YET_RECRUITING', 'country': 'China', 'contacts': [{'name': 'Chun Li, MD,PhD', 'role': 'CONTACT', 'email': 'lichun1230@sohu.com', 'phone': '+86-13865992676'}], 'facility': 'First Affiliated Hospital Of University of Science and Technology of China', 'geoPoint': {'lat': 31.86389, 'lon': 117.28083}}, {'zip': '230000', 'city': 'Hefei', 'state': 'Anhui', 'status': 'NOT_YET_RECRUITING', 'country': 'China', 'contacts': [{'name': 'Ninglin Wang, MD', 'role': 'CONTACT', 'email': 'zwnltt@126.com', 'phone': '+86-13721113063'}], 'facility': 'The Second Hospital of Anhui Medical University', 'geoPoint': {'lat': 31.86389, 'lon': 117.28083}}, {'zip': '510000', 'city': 'Guangzhou', 'state': 'Guangdong', 'status': 'NOT_YET_RECRUITING', 'country': 'China', 'contacts': [{'name': 'Hua Jiang, MD, PhD', 'role': 'CONTACT', 'email': 'jiang_hua18@sina.cn', 'phone': '+86-13533330985'}], 'facility': 'Guangzhou Women and Children Medical Center', 'geoPoint': {'lat': 23.11667, 'lon': 113.25}}, {'zip': '530000', 'city': 'Nanning', 'state': 'Guangxi', 'status': 'NOT_YET_RECRUITING', 'country': 'China', 'contacts': [{'name': 'Ning Liao, MD', 'role': 'CONTACT', 'email': 'ln12808@163.com', 'phone': '+86-13978812808'}], 'facility': 'The First Affiliated Hospital of Guangxi Medical University', 'geoPoint': {'lat': 22.81667, 'lon': 108.31667}}, {'zip': '475000', 'city': 'Kaifeng', 'state': 'Henan', 'status': 'NOT_YET_RECRUITING', 'country': 'China', 'contacts': [{'name': 'Jixia Luo, MD', 'role': 'CONTACT', 'email': 'luojixia@163.com', 'phone': '+86-13592146539'}], 'facility': "Kaifeng Children's Hospital", 'geoPoint': {'lat': 34.7986, 'lon': 114.30742}}, {'zip': '450052', 'city': 'Zhengzhou', 'state': 'Henan', 'status': 'NOT_YET_RECRUITING', 'country': 'China', 'contacts': [{'name': 'Yufeng Liu, MD,PhD', 'role': 'CONTACT', 'email': 'lyf6012@126.com', 'phone': '+86-13673666608'}], 'facility': 'The First Affiliated Hospital of Zhengzhou University', 'geoPoint': {'lat': 34.75778, 'lon': 113.64861}}, {'zip': '410000', 'city': 'Changsha', 'state': 'Hunan', 'status': 'NOT_YET_RECRUITING', 'country': 'China', 'contacts': [{'name': 'Minghua Yang, MD', 'role': 'CONTACT', 'email': 'yamahua123@163.com', 'phone': '+86-13973135843'}], 'facility': 'Third Xiangya Hospital of Central South University', 'geoPoint': {'lat': 28.19874, 'lon': 112.97087}}, {'zip': '410008', 'city': 'Changsha', 'state': 'Hunan', 'status': 'NOT_YET_RECRUITING', 'country': 'China', 'contacts': [{'name': 'Liangchun Yang, MD,PhD', 'role': 'CONTACT', 'email': 'yangliangchung@163.com', 'phone': '+86-13974927514'}], 'facility': 'XiangYa Hospital Central South University', 'geoPoint': {'lat': 28.19874, 'lon': 112.97087}}, {'zip': '215000', 'city': 'Suzhou', 'state': 'Jiangsu', 'status': 'RECRUITING', 'country': 'China', 'contacts': [{'name': 'Shaoyan Hu, MD, PhD', 'role': 'CONTACT', 'email': 'hsy139@126.com', 'phone': '+86-13771835430'}, {'name': 'Li Gao, MD', 'role': 'CONTACT', 'email': 'joygaoli@163.com', 'phone': '+86-15821963190'}, {'name': 'Shaoyan Hu, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR'}, {'name': 'Peifang Xiao, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}, {'name': 'Li Gao, MD', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Jun Lu, MD', 'role': 'SUB_INVESTIGATOR'}, {'name': 'Jie Li, MD', 'role': 'SUB_INVESTIGATOR'}], 'facility': "Children's Hospital of Soochow University", 'geoPoint': {'lat': 31.30408, 'lon': 120.59538}}, {'zip': '221000', 'city': 'Xuzhou', 'state': 'Jiangsu', 'status': 'NOT_YET_RECRUITING', 'country': 'China', 'contacts': [{'name': 'Qi An, MD', 'role': 'CONTACT', 'email': 'anqi1974@sina.com', 'phone': '+86-13814422329'}], 'facility': "Xuzhou Children's Hospital", 'geoPoint': {'lat': 34.20442, 'lon': 117.28386}}, {'zip': '250000', 'city': 'Jinan', 'state': 'Shandong', 'status': 'NOT_YET_RECRUITING', 'country': 'China', 'contacts': [{'name': 'Xiuli Ju, MD,PhD', 'role': 'CONTACT', 'email': 'shellysdcn@hotmail.com', 'phone': '+86-13869192944'}], 'facility': 'Qilu Hospital of Shandong University', 'geoPoint': {'lat': 36.66833, 'lon': 116.99722}}, {'zip': '200000', 'city': 'Shanghai', 'state': 'Shanghai Municipality', 'status': 'NOT_YET_RECRUITING', 'country': 'China', 'contacts': [{'name': 'Xiaowen Zhai, MD, PhD', 'role': 'CONTACT', 'email': 'zhaixiaowendy@163.com', 'phone': '+86-18017590808'}], 'facility': "Children's Hospital Of Fudan University", 'geoPoint': {'lat': 31.22222, 'lon': 121.45806}}], 'centralContacts': [{'name': 'Shaoyan Hu, MD, PhD', 'role': 'CONTACT', 'email': 'hsy139@126.com', 'phone': '+86-13771870462'}, {'name': 'Li Gao, MD', 'role': 'CONTACT', 'email': 'joygaoli@163.com', 'phone': '+86-15821963190'}], 'overallOfficials': [{'name': 'Shaoyan Hu, MD, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "Children 's Hospital of Soochow University"}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': "Children's Hospital of Soochow University", 'class': 'OTHER'}, 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Study Chair', 'investigatorFullName': 'Shaoyan Hu', 'investigatorAffiliation': "Children's Hospital of Soochow University"}}}}