EUCTR2021-001010-10-ES进行中（未招募）1 期
A Phase 2 Open-Label, Multicenter Clinical Study of the Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Profiles of CGT9486 as a Single Agent in Patients With Advanced Systemic Mastocytosis
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 140
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Diagnosed with 1 of the following advanced mastocytosis diagnoses based on WHO diagnostic criteria (Appendix A of the protocol):
- •SM-AHN (The associated hematologic neoplasm must be myeloid, with the following diagnoses excluded from study entry: acute myeloid leukemia, myelodysplastic syndrome that is very high- or high-risk as defined by the Revised International Prognostic Scoring System for Myelodysplastic Syndromes, Philadelphia Chromosome positive malignancies, and patients with =10% blast cells in bone marrow)
- •2. Measurable disease according to modified IWG-MRT-ECNM consensus eligibility and response criteria for CI (Appendix A of the protocol)
- •NOTE: Diagnosis and evidence of measurable disease must be evaluable according to response criteria and confirmed by the Eligibility Committee prior to the first dose of study drug.
- •NOTE: Approximately 20 patients with AdvSM may be enrolled in Part 2 who are inevaluable per modified IWG-MRT-ECNM response criteria based on lack of evaluable organ damage per modified IWG-MRT-ECNM.
- •3. Able to provide written informed consent
- •4. Able and willing to commit to study assessments and visit schedule
- •5. Age =18 years of age
- •6. Eastern Cooperative Oncology Group (ECOG) Performance Status 0 to 3
- •7. Have clinically acceptable local laboratory screening results (clinical chemistry, hematology) within certain limits specified below:
- •a. Absolute neutrophil count >500/µL (subjects enrolled in Part 1 only)
- •b. Platelet count =50,000/µL for 2 weeks prior to the first dose of study drug
- •Note: Platelet transfusion to meet enrollment criteria is not allowed.
- •c. AST and ALT =2.5× upper limit of normal (ULN) or =5×ULN if there is liver involvement by AdvSM
- •d. Direct bilirubin =1.5×ULN; if related to AdvSM may be =3×ULN
- •e. Calculated creatinine clearance (Cockcroft-Gault) >40 mL/min
- •f. Serum tryptase =20 ng/mL
- •8. For women of childbearing potential (defined as physiologically and anatomically capable of becoming pregnant), confirmation of a negative serum pregnancy test and agreement to the use of a highly effective method of contraception or at least 2 effective methods at the same time during the study treatment period and for up to 6 months after the last dose of CGT9486; for male subjects, agreement to use effective barrier contraception (ie, condoms) during the study treatment period and for up to 6 months after the last dose of CGT9486
- •9. Able to swallow pills
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 70
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 70
排除标准
- •1. Persistent toxicity from previous therapy for AdvSM that has not resolved to = Grade 1.
- •2. Patients presenting with an associated hematologic neoplasm who require immediate antineoplastic therapy.
- •3. Clinically significant cardiac disease, defined by any of the following:
- •g. Clinically significant cardiac arrhythmias, and/or the need for anti-arrhythmic therapy (excluding beta blockers or digoxin). (Subjects with controlled atrial fibrillation are not excluded.)
- •h. Congenital long QT syndrome or concomitant medications known to prolong the QT interval except those required for infections that carry a low risk of QTc prolongation.
- •i. A marked baseline prolongation of QT/QTc interval (eg, repeated demonstration of a QTcF interval >480 ms).
- •j. History of clinically significant cardiac disease or congestive heart failure > New York Heart Association Class II. (Patients must not have unstable angina [anginal symptoms at rest] or new-onset angina within the last 3 months or myocardial infarction within the past 6 months.)
- •k. Arterial or venous thrombotic or embolic events such as cerebrovascular accident (including transient ischemic attacks), deep vein thrombosis, or pulmonary embolism within the 6 months before study drug initiation (except for adequately treated catheter-related venous thrombosis occurring more than 1 month before the first dose of study drug).
- •4. Known positivity for the FIP1L1 PDGFRA fusion (except for patients who demonstrated relapse or disease progression on prior imatinib therapy). Patients with eosinophilia (eosinophil count >1.5 × 109/L) who do not have a detectable KIT D816V mutation must provide documentation of the lack of a PDGFRA fusion mutation by fluorescence in situ hybridization or polymerase chain reaction prior to enrollment.
- •5. Any other concurrent severe known disease or concurrent severe and/or uncontrolled medical condition (eg, uncontrolled diabetes or active uncontrolled infection), either of which could compromise participation in the study.
- •6. Seropositive for human immunodeficiency virus (HIV) 1 or 2, or positive for hepatitis B surface antigen or hepatitis C virus (HCV) antibody. (Subjects with a positive HCV antibody may be eligible if HCV RNA is undetectable on a quantitative HCV RNA assay, following discussion with the Medical Monitor.)
- •7. Active, uncontrolled, systemic bacterial, fungal, or viral infections at Screening.
- •NOTE: Oral antibiotics for a controlled infection are permitted with Medical Monitor approval. Patients on antimicrobial, antifungal, or antiviral prophylaxis are not specifically excluded if all other inclusion/exclusion criteria are met.
- •8. History of clinically significant bleeding event within 30 days before the first dose of study drug or need for therapeutic anticoagulation on study.
- •9. Diagnosed with or treated for malignancy other than the disease under study within the prior 3 years before enrollment, or expected to need treatment for an active malignancy. (The following are allowed within 3 years of study enrollment if the subject has received definitive local therapy [eg, surgical excision, external beam radiation, or other local therapy with curative intent]: non-melanoma skin cancers, localized prostate cancer, or carcinoma in situ.)
- •10. Any condition that could hamper compliance with the study protocol in the judgment of the Investigator.
- •11. Pregnant or currently breastfeeding.
- •12. Received any cytoreductive therapy (including midostaurin and other tyrosi
研究者
相似试验
进行中（未招募）
1 期
A Study of CGT9486 in Patients with Advanced Systemic MastocytosisAdvanced Systemic Mastocytosis (AdvSM)MedDRA version: 27.0Level: LLTClassification code 10056453Term: Aggressive systemic mastocytosisSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)EUCTR2021-001010-10-FRCogent Biosciences, Inc.140
进行中（未招募）
1 期
A Study of CGT9486 in Patients with Advanced Systemic MastocytosisAdvanced Systemic Mastocytosis (AdvSM)MedDRA version: 20.0Level: LLTClassification code 10056453Term: Aggressive systemic mastocytosisSystem Organ Class: 100000004864EUCTR2021-001010-10-PLCogent Biosciences, Inc.120
进行中（未招募）
1 期
A Study of CGT9486 in Patients with Advanced Systemic MastocytosisAdvanced Systemic Mastocytosis (AdvSM)MedDRA version: 20.0Level: LLTClassification code 10056453Term: Aggressive systemic mastocytosisSystem Organ Class: 100000004864EUCTR2021-001010-10-NOCogent Biosciences, Inc.120
尚未招募
2 期
A Phase 2 Open-Label, Multicenter Clinical Study of the Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Profiles of CGT9486 as a Single Agent in Patients With Advanced Systemic MastocytosisAdvanced Mastocytosismast cell accumulationmyeloid neoplasm10047954NL-OMON53808Cogent Biosciences, Inc.5
招募中
1 期
A Study to Evaluate the Safety, Efficacy, Pharmacokinetic and Pharmacodynamic Profiles of CGT9486 in subjects with Advanced Systemic MastocytosisAdvanced Systemic MastocytosisMedDRA version: 26.1Level: LLTClassification code: 10056453Term: Aggressive systemic mastocytosis Class: 10005329CTIS2024-511407-42-00Cogent Biosciences Inc.140