SKCD in Early Relapsed/Refractory Multiple Myeloma
A Prospective, Multicenter, Open-Label, Single-Arm Phase II Clinical Study Evaluating the Efficacy and Safety of Sonrotoclax, Carfilzomib, Aponermin, and Dexamethasone in Patients With Early Relapsed or Refractory Multiple Myeloma
Multiple myeloma (MM) is a malignant hematologic neoplasm arising from abnormal plasma cells within the bone marrow. Although substantial therapeutic progress has been achieved over the past twenty years, MM remains an incurable disease for the vast majority of patients. When the disease relapses or becomes refractory after 1 to 3 prior lines of therapy (termed early relapsed or refractory multiple myeloma [RRMM]), achieving deep and durable remission during early salvage therapy is critical to extending long-term survival.
Current guideline-recommended second-line therapies exhibit distinct clinical challenges: standard triplet regimens lack head-to-head comparisons, novel cellular therapies (such as CAR-T) are costly and difficult to access, and optimal combinations for early relapse remain undefined. Furthermore, novel targeted therapeutics have predominantly originated overseas, highlighting an urgent need for effective regimens based on innovative domestic agents.
Therapeutic Rationale of the SKCD Regimen: This study evaluates a novel quadruplet combination regimen termed SKCD, comprising four complementary therapeutics:
- 1. Sonrotoclax: Sonrotoclax (BGB-11417): A highly potent and selective oral BCL-2 inhibitor that restores the cell's internal (mitochondrial) apoptosis pathway.
- 2. Carfilzomib: Carfilzomib: A second-generation selective proteasome inhibitor that inhibits myeloma cell proliferation and downregulates anti-apoptotic proteins like MCL-1.
- 3. Aponermin (CPT): Aponermin (CPT): A first-in-class recombinant circularly permuted human TRAIL and DR4/DR5 agonist, independently developed in China and approved for RRMM, which selectively activates external (death receptor) apoptosis.
- 4. Dexamethasone: Dexamethasone: A synthetic corticosteroid with established anti-myeloma activity that synergizes with BCL-2 and proteasome inhibitors.
Preclinical studies demonstrate that simultaneously stimulating internal mitochondrial apoptosis and external death receptor apoptosis produces synergistic anti-myeloma cytotoxicity. The primary objective of this Phase II study is to evaluate the clinical efficacy (assessed by the best complete response or better rate [>= CR]) and the safety profile of the SKCD quadruplet regimen followed by carfilzomib and dexamethasone (Kd) maintenance therapy in early relapsed RRMM.
研究概览
详细说明
Sonrotoclax is being investigated in combination with carfilzomib, aponermin, and dexamethasone for the treatment of early-line relapsed or refractory multiple myeloma. This use is outside the approved indications in China and is investigational.
研究类型
注册 (估计的)
阶段
- 阶段2
联系人和位置
学习联系方式
- 姓名:Wen Gao, MD
- 电话号码:+86 13651275440
- 邮箱:gaowenpingping@mail.ccmu.edu.cn
学习地点
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Beijing Municipality
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Beijing、Beijing Municipality、中国、100020
- 招聘中
- Beijing Chao-Yang Hospital,Capital Medical University
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接触:
- Wen Gao, MD
- 电话号码:+86 010-85231572
- 邮箱:gaowenpingping@mail.ccmu.edu.cn
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-
参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
描述
Inclusion Criteria
Candidates must satisfy all of the following inclusion criteria to be enrolled in the study:
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1. Age: Age > 18 years, male or female.
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2. Measurable Disease: Diagnosed with multiple myeloma according to the 2014 IMWG diagnostic criteria, with measurable disease at screening defined by at least one of the following:
- Serum M-protein level > 10 g/L (1.0 g/dL); OR
- 24-hour urine M-protein excretion > 200 mg/24 hours; OR
- In light chain multiple myeloma without measurable serum or urine M-protein: serum involved free light chain (FLC) > 100 mg/L (10 mg/dL) with an abnormal serum FLC kappa/lambda ratio (normal reference range: 0.26-1.65).
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3. Disease Status & Life Expectancy: Patients with early relapsed or refractory multiple myeloma, with an anticipated life expectancy of >= 6 months.
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4. Prior Therapy Lines: Must have received 1 to 3 prior systemic treatment lines. Induction therapy followed by autologous stem cell transplantation (ASCT) with or without maintenance therapy is counted as a single line of therapy. Participants must have received at least one regimen containing a proteasome inhibitor (e.g., bortezomib, ixazomib, carfilzomib) and/or an immunomodulatory drug (e.g., lenalidomide, pomalidomide; excluding thalidomide monotherapy), and demonstrated disease progression or relapse during or after such therapy (single-agent or combination). Prior anti-CD38 monoclonal antibody exposure is permitted but not mandatory. Radiotherapy, bisphosphonate therapy, or a single brief course of corticosteroids (equivalent dose not exceeding dexamethasone 20 mg/day for <= 4 days) are not counted as prior systemic treatment lines.
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5. Documented Disease Progression: Documented evidence of progressive disease (PD) during or following the most recent anti-myeloma regimen based on IMWG consensus criteria. Progressive disease requires at least one of the following compared with the nadir value:
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An increase of >= 25% in serum M-protein (with an absolute increase of >= 5 g/L); or an absolute increase of >= 10 g/L if baseline serum M-protein was >= 50 g/L;
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An increase of >= 25% in 24-hour urine M-protein (with an absolute increase of >= 200 mg/24 h);
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In patients without measurable serum and urine M-protein, an increase of >= 25% in the difference between involved and uninvolved FLC levels (dFLC, with an absolute increase > 100 mg/L);
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In patients without measurable serum/urine M-protein and involved FLC, an increase of >= 25% in bone marrow plasma cell percentage (with an absolute increase of >= 10%);
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Development of new extramedullary plasmacytomas: an increase of >= 50% in the sum of the products of the perpendicular diameters (SPD) of > 1 existing measurable lesion compared to nadir, or an increase of >= 50% in the longest diameter of a pre-existing lesion >= 1 cm;
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When circulating plasma cells represent the only measurable parameter, an increase of > 50% in circulating plasma cells (with an absolute count of at least 200 cells/uL).
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6. Performance Status: Eastern Cooperative Oncology Group (ECOG) performance status score of 0 to 2 at screening and prior to initiation of study treatment.
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7. Organ and Laboratory Function: Adequate hematologic, hepatic, renal, and metabolic organ function prior to enrollment, satisfying:
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Hemoglobin >= 80 g/L (without red blood cell transfusions or erythropoietin within 7 days prior to laboratory assessment);
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Platelet count: > 75 x 10⁹/L in patients with < 50% bone marrow nucleated cells composed of plasma cells; > 50 x 10⁹/L in patients with >= 50% bone marrow plasma cells (without platelet transfusions or thrombopoietin receptor agonists within 7 days prior to assessment);
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Absolute Neutrophil Count (ANC) > 0.5 x 10⁹/L (growth factor support permitted, but G-CSF or GM-CSF must be discontinued for >= 7 days, and pegylated G-CSF for >= 14 days prior to assessment);
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Hepatic function: AST and ALT <= 2.5 x ULN; Total bilirubin <= 2.5 x ULN, except in patients with congenital hyperbilirubinemia such as Gilbert syndrome (in which direct bilirubin must be < 1.5 x ULN);
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Renal function: Estimated glomerular filtration rate (eGFR) > 30 mL/min, calculated using the Modification of Diet in Renal Disease (MDRD) equation or measured by 24-hour urinary creatinine clearance;
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Serum calcium: Albumin-corrected serum calcium <= 14 mg/dL (< 3.5 mmol/L) or ionized calcium <= 6.5 mg/dL.
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8. Disease Subtype Definitions: Relapsed, refractory, or clinical relapse defined as follows:
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Relapsed Disease: Patients who initially achieved a response to prior therapy and subsequently developed confirmed disease progression per IMWG criteria > 60 days after therapy cessation.
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Refractory Disease: Patients who experienced disease progression per IMWG criteria during prior therapy or within < 60 days of discontinuing prior therapy, or had an M-protein increase of >= 25%.
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Clinical Relapse: Defined by >= 1 of the following: (1) Development of new bone lesions or soft-tissue plasmacytomas (excluding osteoporotic fractures); (2) Definite increase in the size of existing plasmacytomas or bone lesions (>= 50% increase in SPD of measurable lesions and absolute increase >= 1 cm); (3) Hypercalcemia (> 2.75 mmol/L); (4) Decrease in hemoglobin concentration >= 20 g/L (unrelated to therapy or non-MM causes); (5) Increase in serum creatinine >= 176.8 umol/L (2 mg/dL) from initiation of MM therapy, attributable to myeloma; (6) Hyperviscosity related to serum M-protein.
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9. Informed Consent: Voluntary written informed consent signed by the patient or legally authorized representative.
Exclusion Criteria
Candidates meeting any of the following exclusion criteria shall be excluded from study participation:
- 1. Hypersensitivity: Known contraindication, life-threatening allergy, severe hypersensitivity, or intolerance to any study drug active ingredients or excipients.
- 2. Prior Antineoplastic Therapy Washout: Prior therapy washout requirements:
- Targeted therapy, epigenetic therapy, or investigational drug treatment within 21 days or > 5 elimination half-lives (whichever is shorter) prior to first study dose; or use of an invasive investigational medical device;
- Administration of an approved or emergency-use investigational vaccine (other than SARS-CoV-2 vaccines) within 4 weeks; administration of live attenuated vaccines within 4 weeks;
- Monoclonal antibody therapy within 21 days;
- Cytotoxic chemotherapy within 21 days;
- Proteasome inhibitor (PI) therapy within 14 days;
- Immunomodulatory drug (IMiD) therapy within 14 days;
- Radiotherapy within 14 days, or focal palliative radiotherapy within 7 days.
- 3. CNS Involvement: Known active central nervous system (CNS) involvement or clinical signs of meningeal myeloma. In suspected cases, brain MRI and lumbar puncture cytology must be negative.
- 4. Specific Hematologic Conditions: Diagnosis of plasma cell leukemia, Waldenstrom macroglobulinemia, POEMS syndrome (polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, skin changes), or primary light chain (AL) amyloidosis at screening.
- 5. Second Malignancies: Non-hematologic malignancy diagnosed within 3 years prior to enrollment (unless curatively treated), or presence of therapy-related myelodysplastic syndrome (t-MDS).
- 6. Cardiovascular Diseases: Cardiovascular diseases including:
- New York Heart Association (NYHA) Class III or IV congestive heart failure;
- Myocardial infarction or coronary artery bypass grafting (CABG) within 6 months prior to enrollment;
- Clinically significant ventricular arrhythmias, or history of unexplained syncope;
- History of severe non-ischemic cardiomyopathy.
- 7. Infectious & Pulmonary Diseases: Infectious, pulmonary, or neurologic conditions including:
- Uncontrolled hepatitis B virus infection (positive HBsAg or HBV-DNA): if infection status is indeterminate, HBV-DNA viral quantification must be performed. Active hepatitis C virus (HCV) infection, defined as positive HCV-RNA. Patients with a history of HCV antibody positivity must undergo HCV-RNA testing; patients with resolved chronic hepatitis C who completed antiviral therapy and maintained undetectable HCV-RNA for >= 12 weeks post-treatment are eligible;
- Chronic obstructive pulmonary disease (COPD) with FEV1 < 50% of predicted normal value;
- Moderate or severe persistent asthma within the past 2 years, or uncontrolled asthma of any type;
- Significant peripheral neuropathy at baseline (Grade 3, Grade 4, or Grade 2 with associated pain).
- 8. Major Surgery: Major surgery within 2 weeks prior to initiating study treatment, incomplete surgical recovery, or major elective surgery planned during the study treatment period or within 2 weeks following the last study dose.
- 9. Investigator Discretion: Any medical, psychiatric, or other condition that, in the opinion of the investigator, makes the patient unsuitable for trial participation.
学习计划
研究是如何设计的?
设计细节
- 主要用途:治疗
- 分配:不适用
- 介入模型:单组作业
- 屏蔽:无(打开标签)
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
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实验性的:SKCD:Sonrotoclax+Carfilzomib+Aponermin+Dexamethasone
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Eligible patients will receive the SKCD regimen for up to 9 cycles (28 days per cycle). Patients who do not develop disease progression or meet withdrawal criteria proceed to Kd maintenance therapy:
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研究衡量的是什么?
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Best Complete Response or Better Rate (≥CR)
大体时间:From enrollment to the end of treatment at 9 cycles (28 days per cycle)
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The proportion of participants who achieve a confirmed complete response (CR) or stringent complete response (sCR), according to the International Myeloma Working Group criteria, during the first nine cycles of SKCD treatment and before initiation of Kd maintenance therapy. |
From enrollment to the end of treatment at 9 cycles (28 days per cycle)
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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Best Complete Response or Better Rate During SKCD and Kd Maintenance
大体时间:From enrollment to the end of treatment at 9 cycles (28 days per cycle)].
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The proportion of participants whose best confirmed response is complete response (CR) or stringent complete response (sCR), assessed according to the International Myeloma Working Group criteria from the first dose of study treatment through SKCD treatment and subsequent Kd maintenance, before disease progression or initiation of a new anti-myeloma treatment, whichever occurs first. |
From enrollment to the end of treatment at 9 cycles (28 days per cycle)].
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Overall Response Rate (ORR)
大体时间:From enrollment to the end of treatment at 9 cycles (28 days per cycle)
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The proportion of participants who achieve a partial response (PR) or better as their best response, assessed according to the International Myeloma Working Group (IMWG) response criteria. |
From enrollment to the end of treatment at 9 cycles (28 days per cycle)
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Very Good Partial Response or Better Rate (≥VGPR)
大体时间:From enrollment to the end of treatment at 9 cycles (28 days per cycle)
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The proportion of participants who achieve a very good partial response (VGPR), complete response (CR), or stringent complete response (sCR) as their best response, assessed according to the International Myeloma Working Group (IMWG) response criteria. |
From enrollment to the end of treatment at 9 cycles (28 days per cycle)
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Minimal Residual Disease (MRD) Negativity Rate by Next-Generation Flow Cytometry
大体时间:From enrollment to the end of treatment at 9 cycles (28 days per cycle), during maintenance treatment
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The proportion of participants who achieve minimal residual disease (MRD) negativity in bone marrow, assessed by next-generation flow cytometry (NGF) at a sensitivity of 10^-5. Bone marrow MRD is assessed when a participant achieves a very good partial response (VGPR) or better. The proportion of participants who achieve MRD negativity among participants who undergo bone marrow MRD testing by next-generation flow cytometry at a sensitivity of 10^-5. |
From enrollment to the end of treatment at 9 cycles (28 days per cycle), during maintenance treatment
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Progression-Free Survival (PFS)
大体时间:From the first dose of study treatment until disease progression or death from any cause, assessed up to 36 months.
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The time from the first dose of study treatment to the first documented disease progression, assessed according to the International Myeloma Working Group (IMWG) response criteria, or death from any cause, whichever occurs first. |
From the first dose of study treatment until disease progression or death from any cause, assessed up to 36 months.
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Overall Survival (OS)
大体时间:From the first dose of study treatment until death for any cause, assessed up to 36 months.
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The time from the first dose of study treatment to death from any cause. |
From the first dose of study treatment until death for any cause, assessed up to 36 months.
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Incidence and Severity of Adverse Events
大体时间:From signing of informed consent through the post-treatment safety visit 60 days after the final dose of study treatment.
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Number and proportion of participants experiencing adverse events, serious adverse events, and adverse events leading to dose modification or treatment discontinuation. Adverse events will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), version 6.0. |
From signing of informed consent through the post-treatment safety visit 60 days after the final dose of study treatment.
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合作者和调查者
调查人员
- 首席研究员:Wen Gao, MD、Beijing Chao Yang Hospital
出版物和有用的链接
一般刊物
- Quach H, Popat R, Chen W, et al. Initial phase 1b/2 study results with sonrotoclax (BGB-11417) in combination with carfilzomib and dexamethasone in patients with t(11;14)-positive relapsed/refractory multiple myeloma. Blood. 2025;146(Suppl 1):102-103. doi:10.1182/blood-2025-102.
- Kumar SK, Harrison SJ, Cavo M, de la Rubia J, Popat R, Gasparetto C, Hungria V, Salwender H, Suzuki K, Kim I, Punnoose EA, Hong WJ, Freise KJ, Yang X, Sood A, Jalaluddin M, Ross JA, Ward JE, Maciag PC, Moreau P. Venetoclax or placebo in combination with bortezomib and dexamethasone in patients with relapsed or refractory multiple myeloma (BELLINI): a randomised, double-blind, multicentre, phase 3 trial. Lancet Oncol. 2020 Dec;21(12):1630-1642. doi: 10.1016/S1470-2045(20)30525-8. Epub 2020 Oct 29.
- Costa LJ, Davies FE, Monohan GP, Kovacsovics T, Burwick N, Jakubowiak A, Kaufman JL, Hong WJ, Dail M, Salem AH, Yang X, Masud AA, Munasinghe W, Ross JA, Bueno OF, Kumar SK, Stadtmauer EA. Phase 2 study of venetoclax plus carfilzomib and dexamethasone in patients with relapsed/refractory multiple myeloma. Blood Adv. 2021 Oct 12;5(19):3748-3759. doi: 10.1182/bloodadvances.2020004146.
- Xia Z, Leng Y, Fang B, Liang Y, Li W, Fu C, Yang L, Ke X, Jiang H, Weng J, Liu L, Zhao Y, Zhang X, Huang Z, Liu A, Shi Q, Gao Y, Chen X, Pan L, Cai Z, Wang Z, Wang Y, Fan Y, Hou M, Ma Y, Hu J, Liu J, Zhou J, Zhang X, Meng H, Lu X, Li F, Ren H, Huang B, Shao Z, Zhou H, Hu Y, Yang S, Zheng X, Wei P, Pang H, Yu W, Liu Y, Gao S, Yan L, Ma Y, Jing H, Du J, Ling W, Zhang J, Sui W, Wang F, Li X, Chen W. Aponermin or placebo in combination with thalidomide and dexamethasone in the treatment of relapsed or refractory multiple myeloma (CPT-MM301): a randomised, double-blinded, placebo-controlled, phase 3 trial. BMC Cancer. 2023 Oct 14;23(1):980. doi: 10.1186/s12885-023-11489-8.
研究记录日期
研究主要日期
学习开始 (估计的)
初级完成 (估计的)
研究完成 (估计的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
与本研究相关的术语
其他相关的 MeSH 术语
其他研究编号
- LGH-2026-LI-104
计划个人参与者数据 (IPD)
计划共享个人参与者数据 (IPD)?
IPD 计划说明
Individual participant data (IPD) will not be made publicly available due to participant privacy and confidentiality considerations. Aggregate study results may be reported in scientific publications.
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