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| ID | Type | Description | Link |
|---|---|---|---|
| NCI-2014-01234 | Registry Identifier | CTRP (Clinical Trial Reporting Program) | |
| CCCWFU 99114 | |||
| P30CA012197 | U.S. NIH Grant/Contract | View source |
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| Name | Class |
|---|---|
| National Cancer Institute (NCI) | NIH |
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This phase I trial studies the side effects and best dose of small interfering ribonucleic acid (siRNA)-transfected peripheral blood mononuclear cells APN401 (APN401) in treating patients with melanoma, kidney, or pancreatic cancer, or other solid tumors that have spread to other parts of the body or that cannot be removed by surgery. There are factors in immune cells in the blood that inhibit their ability to kill cancers. Treating white blood cells with one of these factors in the laboratory may help the white blood cells kill more cancer cells when they are put back in the body.
PRIMARY OBJECTIVES:
I. To determine the toxicities and establish the maximal tolerated dose (MTD) of APN401.
II. To determine the effects of APN401 on immune response.
SECONDARY OBJECTIVES:
I. To document clinical response and survival.
OUTLINE: This is a dose-escalation study.
Patients receive autologous siRNA-transfected peripheral blood mononuclear cells APN401 intravenously (IV) over 30 minutes for 1 course in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up every 3 months for 2 years, every 6 months for 2 years, and then annually for 1 year.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Treatment (APN401) | Experimental | Patients receive autologous siRNA-transfected peripheral blood mononuclear cells APN401 IV over 30 minutes. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| siRNA-transfected peripheral blood mononuclear cells APN401 | Biological | Given IV |
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| Measure | Description | Time Frame |
|---|---|---|
| Maximum tolerated dose of siRNA-transfected peripheral blood mononuclear cells APN401, defined as the dose in which the number of patients with dose limiting toxicity is less than or equal to one out of six | 14 days | |
| Incidence of adverse events of APN401, graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0 | Adverse events will be categorized by organ system and severity and summarized as frequency counts and percentages. | Up to 2 years |
| Immune response, measured by change in blood Th1-associated cytokines production in response to anti-CD3/28 stimulation or >= tumor antigens post-therapy | Summarized as medians and ranges. The effects of treatment on these markers individually will be analyzed using paired t-tests (possibly after transformation, e.g., logarithmic) or the non-parametric counterpart. Differences between dose levels at particular time points will be analyzed using analysis of variance and 2-sample t-tests, and differences over time will be analyzed using longitudinal methods such as repeated measures. The association between the markers (baseline and changes) and objective response will also be examined. | Baseline to week 9 |
| Clinical response assessed by RECIST | Summarized as frequency counts and percentages. | Up to 5 years |
| Progression-free survival | Standard survival analysis techniques using Kaplan Meier methods will be used. | From the initial infusion to confirmation of progression or death, assessed up to 5 years |
| Overall survival | Standard survival analysis techniques using Kaplan Meier methods will be used. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Pierre Triozzi | Wake Forest University Health Sciences | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Comprehensive Cancer Center of Wake Forest University | Winston-Salem | North Carolina | 27157 | United States |
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| laboratory biomarker analysis | Other | Correlative studies |
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| From the initial infusion to confirmation of progression or death, assessed up to 5 years |
| ID | Term |
|---|---|
| D008545 | Melanoma |
| D010190 | Pancreatic Neoplasms |
| D002292 | Carcinoma, Renal Cell |
| ID | Term |
|---|---|
| D018358 | Neuroendocrine Tumors |
| D017599 | Neuroectodermal Tumors |
| D009373 | Neoplasms, Germ Cell and Embryonal |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D009380 | Neoplasms, Nerve Tissue |
| D018326 | Nevi and Melanomas |
| D012878 | Skin Neoplasms |
| D009371 | Neoplasms by Site |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D004067 | Digestive System Neoplasms |
| D004701 | Endocrine Gland Neoplasms |
| D004066 | Digestive System Diseases |
| D010182 | Pancreatic Diseases |
| D004700 | Endocrine System Diseases |
| D000230 | Adenocarcinoma |
| D002277 | Carcinoma |
| D009375 | Neoplasms, Glandular and Epithelial |
| D007680 | Kidney Neoplasms |
| D014571 | Urologic Neoplasms |
| D014565 | Urogenital Neoplasms |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052801 | Male Urogenital Diseases |
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