A Phase 1 interventional study of siRNA-transfected peripheral blood mononuclear cells APN401 and laboratory biomarker analysis in Recurrent Melanoma, Recurrent Pancreatic Cancer and Recurrent Renal Cell Cancer, sponsored by Wake Forest University Health Sciences. Completed at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2018-07-03.
Sponsored by Wake Forest University Health Sciences · Phase 1, Interventional, and Treatment
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.
3,006 studies on the registry are indexed under Melanoma; 520 are open to participants now.
This study's enrollment of 17 is below the median of 38 across 2,351 interventional studies indexed under Melanoma.
Browse Melanoma studies →Wake Forest University Health Sciences is the lead sponsor of 1,320 studies on the registry; 199 are open to participants now.
Of its 323 completed or terminated interventional studies of FDA-regulated products, 243 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Patients receive autologous siRNA-transfected peripheral blood mononuclear cells APN401 IV over 30 minutes.
Biological: siRNA-transfected peripheral blood mononuclear cells APN401 · Other: laboratory biomarker analysis
Given IV
Also known as: APN401
Correlative studies
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
Time frame: 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.
Time frame: 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.
Time frame: Baseline to week 9
Clinical response assessed by RECIST
Summarized as frequency counts and percentages.
Time frame: Up to 5 years
Progression-free survival
Standard survival analysis techniques using Kaplan Meier methods will be used.
Time frame: 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.
Time frame: From the initial infusion to confirmation of progression or death, assessed up to 5 years
This study is completed, as verified in Jul 2018. You cannot join it, but the record below documents what was studied.
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Wake Forest University Health Sciences