Axelopran and Nivolumab in PD-1-Refractory Metastatic Melanoma (MORALE-MM)
A Phase II Trial of MOR Antagonism With Axelopran and Nivolumab in PD-1-Refractory Metastatic Melanoma (MORALE-MM)
研究概览
详细说明
Immune checkpoint inhibitors have substantially improved outcomes in melanoma; however, resistance to PD-1 blockade remains a major clinical limitation. Approximately 60% of patients with metastatic melanoma fail to achieve an objective response to anti-PD-1 therapy, and many initial responders ultimately develop acquired resistance. These limitations highlight the need to identify additional, biologically grounded mechanisms of immune escape that can be therapeutically targeted. Importantly, pharmacologic inhibition of MOR with axelopran, a peripherally acting MOR antagonist restores T-cell effector function and enhances antitumor responses to PD-1 inhibition in preclinical oral squamous cell carcinoma models exposed to exogenous opioids. Notably, endogenous μ-opioid signaling in melanoma suppresses antitumor immunity via MOR, providing a rationale for evaluating peripheral MOR antagonism to enhance checkpoint inhibitor efficacy in melanoma. These findings suggest that constitutive MOR signaling may represent a distinct and targetable pathway of immune resistance that has not been addressed in current immunotherapy strategies.
To date, the role of MOR antagonism has not been evaluated clinically in melanoma, and the OPRM1-MOR axis has not been studied as a mechanism of immune escape in humans. This first-in-human study will evaluate the safety, clinical activity, and immunologic effects of combining axelopran with anti-PD-1 therapy in patients with unresectable or metastatic melanoma who have progressed on prior PD-1-based treatment. Post hoc analyses incorporating exogenous opioid exposure and OPRM1 genotype will explore biologically relevant heterogeneity in treatment response and inform future precision-based approaches. Given the strong mechanistic rationale, consistent preclinical evidence of synergy, and the lack of effective options for patients with PD-1-refractory melanoma, this study addresses a critical unmet need and may establish MOR antagonism as a novel therapeutic strategy to overcome immune resistance in melanoma.
研究类型
注册 (估计的)
阶段
- 阶段2
- 阶段1
联系人和位置
学习联系方式
- 姓名:Danielle Bednarz, RN, BSN
- 电话号码:412-623-1191
- 邮箱:bednarzdl@upmc.edu
研究联系人备份
- 姓名:Amy Rose, RN, BSN
- 电话号码:412-647-8587
- 邮箱:kennaj@upmc.edu
学习地点
-
-
Pennsylvania
-
Pittsburgh、Pennsylvania、美国、15232
- UPMC Hillman Cancer Center
-
接触:
- Amy Rose, RN, BSN
- 电话号码:412-647-8587
- 邮箱:kennaj@upmc.edu
-
首席研究员:
- John M Kirkwood, MD
-
接触:
- Dannielle Bednarz, RN, BSN
- 电话号码:412-623-1191
- 邮箱:bednarzdl@upmc.edu
-
-
参与标准
资格标准
适合学习的年龄
- 成人
- 年长者
接受健康志愿者
描述
Inclusion Criteria:
Histologically confirmed, unresectable or metastatic cutaneous melanoma with documented PD-1-refractory disease, defined as disease progression during or after prior anti-PD-1-based therapy, meeting criteria for either primary or secondary (acquired) resistance, as follows57:
- Primary resistance: Disease progression as best response, or stable disease lasting <6 months, following at least 6 weeks (approximately two cycles) of anti-PD-1-based therapy, with progression confirmed by RECIST v1.1.
- Secondary (acquired) resistance: Disease progression occurring after an initial objective response (complete or partial response) or durable stable disease (≥6 months) per RECIST v1.1 while receiving anti-PD-1-based therapy, or within 12 weeks of discontinuation of anti-PD-1 therapy following prior clinical benefit.
- Note: Disease must have been histologically confirmed through prior pathology assessment conducted as per SOC.
- Note: Prior anti-PD-1-based therapy may have been administered as monotherapy or in combination with other agents.
- Age ≥ 18 years.
- Eastern Cooperative Oncology Group (ECOG) Performance Scale (PS) 0-2
- Measurable disease using Response Evaluation Criteria in Solid Tumors (RECIST) 1.1.
Patients must have normal organ and marrow function as defined below:
- absolute neutrophil count ≥1,000/mcL
- platelets ≥75,000/mcL
- total bilirubin ≤1.5 × the institutional upper limit of normal (ULN)
- AST(SGOT)/ALT(SGPT) ≤2.5 × institutional ULN (≤5 × the institutional ULN for patients with liver metastasis)
- Creatinine clearance ≥40 mL/min/1.73 m2 for patients with a creatinine level above institutional normal.
- Sexually active fertile subjects and their partners must agree to use highly effective methods of contraception prior to study entry, during the course of the study, and for 5 months after the last dose of treatment (whichever is later). In addition, men must agree not to donate sperm and women must agree not to donate eggs (ova, oocyte) for the purpose of reproduction during these same periods.
Female subjects of childbearing potential must not be pregnant or breastfeeding at screening. Female subjects are considered to be of childbearing potential unless one of the following criteria is met:
a. Permanent sterilization (hysterectomy, bilateral salpingectomy, or bilateral oophorectomy) or documented postmenopausal status (defined as 12 months of amenorrhea in a woman > 45 years-of-age in the absence of other biological or physiological causes). Note: Documentation may include review of medical records, medical examination, or medical history interview by study site staff.
- Ability to understand and the willingness to sign a written informed consent document.
- If known to have prior brain metastases, must not have evidence of active (enlarging and/or symptomatic lesions) brain disease on MRI/CT evaluation done within 28 days of start of study treatment.
Exclusion Criteria:
- Participant is not a candidate to continue anti-PD-1 therapy due to unresolved toxicities resulting from previous treatment with anti-PD-1 therapy.
Has an active autoimmune disease requiring systemic treatment within the past 3 months, or a syndrome that requires ongoing systemic steroids or immunosuppressive agents. Subjects with vitiligo, Graves' disease, or psoriasis not requiring systemic therapy or resolved childhood asthma/atopy would be an exception to this rule.
i. Subjects that require intermittent use of bronchodilators or local steroid injections would not be excluded from the study. Subjects with stable hypothyroidism or Sjogren's syndrome will not be excluded from the study.
ii. Physiological replacement doses of steroids are permitted.
- Participant is taking a daily dose of opioids that is >30 morphine milligram equivalents (MME) i. Daily opioid doses ≤30 MME are allowed
- Has a history of non-infectious pneumonitis that required steroids, evidence of interstitial lung disease, or currently active non-infectious pneumonitis.
- Prior malignancy within 2 years that in the investigator's opinion would be likely to affect the outcomes of the patients with unresectable melanoma.
- Has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the trial, interfere with the subject's participation for the full duration of the trial, or is not in the best interest of the subject to participate, in the opinion of the treating investigator.
- Known HIV or active Hepatitis B or C. Checking viral serology will not be mandated.
- Patients with major gastrointestinal surgery within 12 weeks prior to the first dose of study treatment. Patients who have a colostomy will be excluded from the trial.
Are unable to swallow pills or have known active inflammatory gastrointestinal disease, chronic diarrhea, previous gastric resection or lap band dysphagia, short-gut syndrome, gastroparesis, or other condition that limits the ingestion or gastrointestinal absorption of drugs administered orally.
i. Note: Gastroesophageal reflux disease under medical treatment is allowed (assuming no drug interaction potential).
- Subjects taking moderate to strong CYP3A inhibitors or P-gp inhibitors
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 |
|---|---|
|
实验性的:Axelopran + Nivolumab
Run in (1 week): Days -6 to 0, Axelopran monotherapy with dose escalating to 15mg PO daily by day 0 Combination Phase: Axelopran: 15 mg PO daily Nivolumab: 480 mg IV q4w (until disease progression, up to 3 years) |
A peripherally restricted mu-opioid receptor antagonist (PAMORA) designed to mitigate the undesirable peripheral effects of opioids without compromising their central analgesic action.
其他名称:
An immune checkpoint inhibitor that helps the immune system attack cancer cells by blocking PD-1 receptors.
其他名称:
|
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 |
|---|---|---|
|
Adverse Events Related to Treatment
大体时间:Up to 30 days post end of treatment
|
Adverse events (AEs), serious adverse events (SAEs), AEs leading to discontinuation or death, and severity of AEs as assessed by the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events version 5.0 (CTCAE v5.0) determined to be possibly, probably or definitely related to study treatment.
|
Up to 30 days post end of treatment
|
|
Best objective response rate (BORR)
大体时间:Up to 3 years
|
The proportion of subjects with a confirmed best objective response of Complete Response (CR) or Partial Response (PR) based on RECIST v1.1.
CR: Disappearance of all target lesions.
Pathological lymph nodes must decrease to a short axis of < 10 mm.
All non-target lesions must have disappeared, and tumor markers must have normalized.
PR: At least a 30% decrease in the sum of diameters of target lesions, using baseline measurements as a reference.
|
Up to 3 years
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Duration of response (DOR)
大体时间:Up to 3 years
|
Time from date of first documented response Complete Response (CR) or Partial Response (PR) to date of documented Progressive Disease (PD) or death from any cause, based on RECIST v1.1.
CR: Disappearance of all target lesions.
Pathological lymph nodes must decrease to a short axis of < 10 mm.
All non-target lesions must have disappeared, and tumor markers must have normalized.
PR: At least a 30% decrease in the sum of diameters of target lesions, using baseline measurements as a reference.
PD: At least 20% increase in the sum of diameters of target lesions, taking the smallest sum on study (nadir) as the reference.
In addition to the 20% relative increase, the sum must also show an absolute increase of at least 5 mm.
The unequivocal progression of non-target lesions or the appearance of any new lesion also classifies as PD.
|
Up to 3 years
|
|
Best Treatment Response in Target lesions
大体时间:Up to 3 years
|
Best treatment response in target lesions based on RECIST v1.1.
CR: Disappearance of all target lesions.
Pathological lymph nodes must decrease to a short axis of < 10 mm.
All non-target lesions must have disappeared, and tumor markers must have normalized.
PR: At least a 30% decrease in the sum of diameters of target lesions, using baseline measurements as a reference.
|
Up to 3 years
|
|
Progression-free Survival (PFS)
大体时间:Up to 3 years
|
Time from date of first dose of study treatment to date of first documented Progressive Disease (PD) based on RECIST v1.1, or death from any cause, whichever occurs first.
PD: At least 20% increase in the sum of diameters of target lesions, taking the smallest sum on study (nadir) as the reference.
In addition to the 20% relative increase, the sum must also show an absolute increase of at least 5 mm.
The unequivocal progression of non-target lesions or the appearance of any new lesion also classifies as PD.
iUPD (Unconfirmed Progressive Disease) requires a follow-up scan to confirm whether it is true tumor growth or temporary.
|
Up to 3 years
|
|
Overall survival (OS)
大体时间:Up to 4.5 years
|
Overall survival, defined as the time from date of first dose of study treatment to date of death from any cause.
|
Up to 4.5 years
|
|
Immune best objective response rate (iBORR)
大体时间:Up to 3 years
|
Proportion of subjects with a confirmed best objective response to immunotherapy of CR or PR based on iRECIST .
CR: Disappearance of all target lesions.
Pathological lymph nodes must decrease to a short axis of < 10 mm.
All non-target lesions must have disappeared, and tumor markers must have normalized.
PR: At least a 30% decrease in the sum of diameters of target lesions, using baseline measurements as a reference.
CR or PR iUPD (one or more instances), but not iCPD, before iCR, iPR.
|
Up to 3 years
|
|
Immune duration of response (iDOR)
大体时间:Up to 3 years
|
Time from date of first documented response (CR or PR) to date of documented PD or death from any cause, based on iRECIST.
CR: Disappearance of all target lesions.
Pathological lymph nodes must decrease to a short axis of < 10 mm.
All non-target lesions must have disappeared, and tumor markers must have normalized.
PR: At least a 30% decrease in the sum of diameters of target lesions, using baseline measurements as a reference.
CR or PR iUPD (one or more instances), but not iCPD, before iCR, iPR.
|
Up to 3 years
|
|
Immune progression-free survival (iPFS)
大体时间:Up to 3 years
|
Time from date of first dose of study treatment to date of first documented PD based on iRECIST, or death from any cause, whichever occurs first.
PD: At least 20% increase in the sum of diameters of target lesions, taking the smallest sum on study (nadir) as the reference.
In addition to the 20% relative increase, the sum must also show an absolute increase of at least 5 mm.
The unequivocal progression of non-target lesions or the appearance of any new lesion also classifies as PD.
iUPD (Unconfirmed Progressive Disease) requires a follow-up scan to confirm whether it is true tumor growth or temporary.
|
Up to 3 years
|
合作者和调查者
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
其他研究编号
- HCC 26-041
计划个人参与者数据 (IPD)
计划共享个人参与者数据 (IPD)?
药物和器械信息、研究文件
研究美国 FDA 监管的药品
研究美国 FDA 监管的设备产品
此信息直接从 clinicaltrials.gov 网站检索,没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求,请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改,我们的网站上也会自动更新.