Combination Immunotherapy Treatment for Glioblastoma Including BreakVax ImmunoTreatment (Designed and Manufactured Per Patient), Chemotherapy and Anti PD-1 Therapy
Phase 1/2 Trial of a Combination Immunotherapy Treatment for Solid Tumors Including BreakVax ImmunoTreatment (Designed and Manufactured Per Patient), Chemotherapy and Anti PD-1 Therapy
The objective of this study is to evaluate the safety, feasibility, and preliminary antitumor activity of this multi-component immunotherapy strategy in patients with advanced solid tumors. The study is designed to assess whether coordinated enhancement of antigen-specific T-cell priming and tumor microenvironment modulation can improve immune-mediated tumor control.
In this study, BreakVax with adjuvants (Montanide and Poly-ICLC) is administered in combination with:
- Low-dose subcutaneous ipilimumab at the injection site as a dendritic cell adjuvant to enhance local dendritic cell-mediated T-cell priming;
- Pembrolizumab to mitigate PD-1-mediated T-cell exhaustion and sustain effector function;
- Standard-of-care chemotherapy, which may promote immunogenic cell death, increase antigen release and modulate the tumor microenvironment; and
- Losartan and aspirin, which have been associated in preclinical and translational studies with modulation of stromal architecture, vascular normalization, and reduction of tumor-associated immunosuppressive signaling.
This is a Phase 1/2, multi-center, open-label single-arm clinical study in adult patients who have Glioblastoma (GBM).
The study consists of two components: a Safety Lead-in Cohort and a combination therapy cohort. The Safety Lead-in Cohort is designed to evaluate safety and tolerability of the study combination and to characterize dose-limiting toxicities (DLTs). The combination therapy cohort portion intends to evaluate the superiority of the study combination treatment vs the SOC chemotherapy, measured by OS at 12 months
研究概览
地位
条件
研究类型
注册 (估计的)
阶段
- 阶段2
- 阶段1
联系人和位置
学习地点
-
-
Florida
-
Miami、Florida、美国、33176
- Miami Herbert Wertheim Cancer Institute
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首席研究员:
- Manmeet Ahluwalia, MD, MBA, FASCO
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-
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:
- Age ≥ 18 years and < 72
- Patients with histologically confirmed GBM who have sent 200 mg (approximately) of tumor tissue (fresh tissue immersed in AllProtect then frozen) to BreakBio for analysis.
- KPS of 80 or greater on day of first dose
Patient has adequate organ function on day one of the trial as defined by:
- Neutrophil to Lymphocyte Ratio (NLR)1 at a healthy adult's normal level: ≤ 3.5
- Absolute Neutrophil Count (ANC) at the normal level: ≤ 7,000/mm3
- Absolute Lymphocyte Count in normal level: ≥ 1,000/mm3
- Monocytes at normal level: < 700/mm3
- Platelet count: ≥ 100 x 109/L (without transfusion support in the last two weeks)
- Hemoglobin: > 10.0 g/dL (without transfusion support in the last two weeks)
- AST and ALT: ≤ 3 X institutional upper limit of normal (ULN) in the absence of liver mets; AST and/or ALT may be ≤ 5 x ULN in the setting of liver metastases
- Total Bilirubin at the normal level: ≤ 1.2mg/dL
- Serum creatinine: ≤ 1.5 x institution's ULN
- Albumin at the normal level: ≥ 3.4 g/dL
- Serum Magnesium at normal level: ≥ 1.7 mg/dL
- Pulse oximetry ≥ 92% on room air.
- Provision of consent for on-treatment biopsy (compulsory) and post-treatment biopsy (optional).
- Both male and female patients enrolled in this trial must agree to use effective contraception during the course of the trial and for at least 3 months after discontinuing study treatment. Patients and/or partners who are surgically sterile or postmenopausal are exempt from this requirement.
- Negative pregnancy test ≤ 7 days prior to day one of cycle 1, for women of childbearing potential only.
- Life expectancy > 6 months.
- Willing and able to provide informed consent
Exclusion Criteria:
- Prior exposure to anti PD- 1/PD-L1 agents.
- Prior exposure to immunosuppressive therapy within the last 12 months prior to enrollment.
- Receiving or previously receiving oral or IV steroids within 6 weeks of starting study treatment. Currently using or previously used topical steroids within 6 weeks of starting study treatment. Expected to require steroid-containing pre-meds before chemotherapy doses.
- Patients with deficient mismatch repair (dMMR) or microsatellite instability (MSI-H) phenotype
- Any liver metastasis greater than 2 cm or greater than 5 liver metastases. Patients who have liver metastasis removed by surgery, and therefore meet this criterion at first dose, may enroll.
- Patients not recovered from all clinically significant toxic effects of previous therapies to ≤Grade 1 or baseline with the exception of peripheral neuropathy and alopecia.
- Patients not recovered adequately from the toxicity and/or complications from any major surgery prior to starting study treatment.
- Known or suspected hypersensitivity to any of the study drugs.
- Received an investigational agent within 28 days prior to the first dose of study drug.
- History of myocarditis or congestive heart failure (as defined by New York Heart Association Functional Classification III or IV), as well as unstable angina, serious uncontrolled cardiac arrhythmia, uncontrolled infection, or myocardial infarction within the past 6 months. Note: Prior to trial entry, any ECG abnormality at screening must be documented by the investigator as not medically relevant.
- LVEF <50%.
- Active interstitial lung disease (ILD)/pneumonitis or a history of ILD/pneumonitis requiring treatment with systemic steroids.
- Known history of Hepatitis B (defined as Hepatitis B surface antigen [HBsAg] reactive) or known active Hepatitis C virus (defined as HCV RNA [qualitative] is detected) infection. Note: no testing for Hepatitis B and Hepatitis C is required unless mandated by local health authority. Individuals who are hepatitis C antibody positive may be enrolled if negative viral load confirmed.
- History of autoimmune disease including inflammatory bowel disease (including ulcerative colitis and Crohn's Disease), rheumatoid arthritis, systemic progressive sclerosis (scleroderma), systemic lupus erythematosus, autoimmune vasculitis (e.g. Wegener's granulomatosis); central nervous system or motor neuropathy considered of autoimmune origin (e.g. Guillain-Barré syndrome, myasthenia gravis, multiple sclerosis). Individuals with vitiligo, Sjogren's Syndrome, interstitial cystitis, Graves' or Hashimoto's Disease, celiac disease, DM1, hypothyroidism stable on hormone replacement, or any autoimmune disease without symptoms and not requiring active therapy for at least 2 years will be allowed with Study Medical Monitor's approval.
- A serious local infection (e.g. cellulitis, abscess) or systemic infection (e.g. pneumonia, septicemia) which requires systemic antibiotic treatment within 4 weeks prior to the first dose of study medication.
- Receiving coumarin-derived anticoagulants.
- Unable or unwilling to withhold or discontinue any prohibited or restricted medications/procedures for the specified windows during the study.
- Inability or refusal to comply with the protocol or with the clinical trial procedures.
- If female, pregnant or breastfeeding. All female patients with reproductive potential must have a negative pregnancy test prior to starting treatment.
- Chronic intake of drugs that lead to known interference with metabolism through strong Cytochrome P450 3A4 (CYP3A4) interaction: e.g. Rifampicin, Rifabutin, Clarithromycin, Telithromycin, Ketoconazole, Itraconazole, Fluconazole, Hypericum perforatum (St. John's Wort /Johanniskraut) or any strong CYP3A4 inducing or inhibiting drug (Note: participants in this study should avoid consuming grapefruit or grapefruit-containing products during the duration of the study, including the screening phase, treatment period, and follow-up period).
- Uncontrolled hypertension defined as persistent systolic blood pressure ≥ 150 mmHg or diastolic blood pressure ≥ 100 mmHg despite current therapy.
- Arterial thrombotic or embolic events such as cerebrovascular accident (including transient ischemic attacks) within 6 months before the start of study medication. Active pulmonary emboli or deep vein thrombosis that are significant or not adequately controlled on anticoagulation regimen
- Any hemorrhage or bleeding event ≥ National Cancer Institute - Common terminology criteria for adverse events (NCI-CTCAE) Grade 3 within 28 days prior to the start of study medication
- History of other invasive cancer within 2 years prior to enrollment, except for treated non-melanoma skin cancer
- Known DNA Polymerase Epsilon (POLE) mutations
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:BreakVax Arm
A cycle for this study is defined as 28 days. BreakVax along with adjuvants and low-dose subcutaneous ipilimumab (as an adjuvant) will be administered on Day 1 and D15 of Cycle 1, and will then be administered on D1 of every cycle thereafter until disease progression per RANO criteria. In addition to BreakVax, patients in the BreakVax Arm will receive losartan daily and temozolomide - without steroid pre-medications (see disallowed concomitant treatment below). Patients will receive aspirin QD except the day BreakVax is administered and the 2 following days in cycles in which the patient is receiving BreakVax. Patients will begin pembrolizumab on Day 21 of Cycle 2 and it will then be administered every 6 weeks thereafter |
An investigational, personalized peptide-based cancer immunotherapy designed to stimulate patient-specific antitumor T-cell responses.
For each patient, tumor tissue undergoes integrated genomic, transcriptomic, and proteomic analysis to identify tumor-associated antigens (TAAs) and tumor-specific neoantigens (TSAs).
A proprietary selection algorithm prioritizes peptides predicted to be presented by the patient's HLA molecules and selectively expressed or overexpressed in tumor tissue.
The resulting individualized peptide pools are manufactured, formulated with adjuvants (Montanide ISA 51 and Poly-ICLC), and administered subcutaneously
Low-dose subcutaneous ipilimumab at the injection site as a dendritic cell adjuvant to enhance local dendritic cell-mediated T-cell priming
To mitigate PD-1-mediated T-cell exhaustion and sustain effector function
May promote immunogenic cell death, increase antigen release and modulate the tumor microenvironment
Have been associated in preclinical and translational studies with modulation of stromal architecture, vascular normalization, and reduction of tumor-associated immunosuppressive signaling
|
研究衡量的是什么?
主要结果指标
结果测量 |
大体时间 |
|---|---|
|
Overall Survival (OS)
大体时间:12 Months
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12 Months
|
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Percentage of patients for whom BreakVax is successfully manufactured
大体时间:up to 24 Months
|
up to 24 Months
|
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Adverse events
大体时间:up to 24 Months
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up to 24 Months
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
T-cell infiltration
大体时间:From pre-treatment biopsy to on-treatment biopsy (Cycle 2 after imaging)
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Change in tumor-infiltrating T-cells between the pre-treatment biopsy and the on-treatment biopsy
|
From pre-treatment biopsy to on-treatment biopsy (Cycle 2 after imaging)
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|
Immune-related Objective Response Rate (irORR) per iRANO
大体时间:From start of study treatment through disease progression or end of treatment, up to 2 years.
|
Percentage of participants with measurable enhancing disease at baseline who achieve an immune-related complete response or partial response according to iRANO criteria, as assessed by a central independent review committee (IRC).
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From start of study treatment through disease progression or end of treatment, up to 2 years.
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Progression-Free Survival (PFS) per RANO
大体时间:From start of study treatment through progression or death, up to 2 years.
|
Time from the start of study treatment to the first documentation of progressive disease according to RANO criteria or death from any cause, whichever occurs first, as assessed by a central independent review committee (IRC).
Participants without documented progression or death will be censored according to the Statistical Analysis Plan.
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From start of study treatment through progression or death, up to 2 years.
|
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Duration of Response (DoR) per RANO
大体时间:From first documented CR or PR through progression or death, up to 2 years.
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Among participants who achieve a complete response or partial response according to RANO criteria, duration of response is measured from the first date on which criteria for complete response or partial response are met until the first date on which recurrent or progressive disease is objectively documented or death occurs, as assessed by a central independent review committee (IRC).
|
From first documented CR or PR through progression or death, up to 2 years.
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Clinical Benefit Rate (CBR) per RANO
大体时间:From start of study treatment through disease progression or end of treatment, up to 2 years.
|
Percentage of participants who achieve complete response (CR), partial response (PR), or stable disease (SD) lasting at least 12 weeks according to RANO criteria, as assessed by a central independent review committee (IRC).
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From start of study treatment through disease progression or end of treatment, up to 2 years.
|
|
Objective Response Rate (ORR) per RANO
大体时间:From start of study treatment through disease progression or end of treatment, up to 2 years.
|
Percentage of participants with measurable enhancing disease at baseline who achieve a complete response (CR) or partial response (PR) according to RANO criteria, as assessed by a central independent review committee (IRC).
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From start of study treatment through disease progression or end of treatment, up to 2 years.
|
|
Immune-related Progression-Free Survival (irPFS) per iRANO
大体时间:From start of study treatment through confirmed progression or death, up to 2 years.
|
Time from the start of study treatment to confirmed disease progression according to iRANO criteria or death from any cause, whichever occurs first.
Unconfirmed progression does not constitute a progression event until confirmation according to iRANO criteria.
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From start of study treatment through confirmed progression or death, up to 2 years.
|
|
Incidence of Pseudoprogression per iRANO
大体时间:From start of study treatment through disease progression or end of treatment, up to 2 years.
|
Percentage of participants who experience apparent radiographic progression that is subsequently determined not to represent true disease progression according to iRANO criteria.
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From start of study treatment through disease progression or end of treatment, up to 2 years.
|
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Stable Disease for at Least 12 Weeks per iRANO
大体时间:From start of study treatment through disease progression or end of treatment, up to 2 years.
|
Percentage of participants whose best response is stable disease according to iRANO criteria for at least 12 weeks, using the protocol-specified assessment window of 12 weeks ±1 week.
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From start of study treatment through disease progression or end of treatment, up to 2 years.
|
|
Overall Survival (OS)
大体时间:Up to 5 years.
|
Time from the start of study treatment to death from any cause.
|
Up to 5 years.
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其他结果措施
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
T-cell priming
大体时间:24 Months
|
Rate and magnitude of antigen-specific T-cell responses induced by BreakVax in peripheral blood
|
24 Months
|
|
Tumor T-cell infiltration
大体时间:24 Months
|
On-treatment tumor biopsy intratumoral and stromal T-cell infiltration
|
24 Months
|
|
Tumor microenvironment modulation
大体时间:24 Months
|
Change in tumor-associated immune cell populations, including macrophage polarization (e.g., M1/M2 markers), and other immunosuppressive or activation signatures
|
24 Months
|
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
其他相关的 MeSH 术语
其他研究编号
- BreakBio-3 Substudy B
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
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