Clinical trial NCT02030067: Dose-Finding and Safety Study for Oral Single-Agent to Treat Advanced Malignancies
Official title: A Phase 1, Open-Label, Dose-Ranging, Safety and Pharmacokinetic Study to Determine the Maximum Tolerated Dose of RX-3117 Administered Orally as a Single-Agent to Subjects With Advanced Malignancies
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Solid Tumor
Metastatic Bladder Cancer
Keywords: oncology
tumor
metastatic
bladder
Lead sponsor: Processa Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2014-01-08
Enrollment: 127
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine the maximum tolerated dose of RX-3117 in subjects with advanced or metastatic solid tumors (Phase 1). The purpose of the Phase 2 portion is to estimate anti-tumor activity in subjects with advanced malignancies (relapsed or refractory pancreatic or advanced bladder cancer).

Detailed description: This is a dose-finding, open-label, single agent study of RX-3117. Once the maximum tolerated dose is identified additional subjects will be treated in a dose expansion followed by a 2-stage Phase 2 study. Subjects will be treated for up to 8 cycles of therapy. A cycle will be 4 weeks. RX-3117 dosing will be 3 times each week for 3 weeks follow by 1 week off treatment. All subjects will be followed for at least 30 days after the last dose of RX-3117.

Eligibility criteria: Inclusion Criteria:

* Males or females who are 18 years or older
* Able to swallow capsules
* Histological or cytological evidence of confirmed metastatic pancreatic or advanced bladder cancer
* Able to discontinue all anticancer therapies 2 weeks prior to study start
* Measurable or evaluable disease using Response Evaluation Criteria in Solid Tumors
* Life expectancy of at least 3 months
* ECOG performance status of 0 or 1
* Provide written informed consent

Exclusion Criteria:

* Primary brain tumors or clinical evidence of active brain metastasis
* Systemic corticosteroid use within 7 days before planned start of study therapy
* Active infection requiring parenteral or oral antibiotics within 2 weeks before planned start of study therapy
* Uncontrolled diabetes as assessed by the investigator
* Prior or current history of hepatitis B, hepatitis C or human immunodeficiency virus
* History of bone marrow of solid organ transplantation
* History of congestive heart failure, arrhythmias, acute coronary syndrome or torsades de pointes
* Any other medical, psychiatric, or social condition, which in the opinion of the investigator, would preclude participation in the study, pose an undue medical hazard, interfere with the conduct of the study, or interfere with interpretation of the study results
* Known hypersensitivity to gemcitabine, azacytidine or cytosine arabinoside
* Pregnant, planning a pregnancy or breast feeding during the study
* Concurrent participation in another therapeutic clinical trial

Primary outcomes:
measure: Overall Safety Profile Characterized by # of Subjects With Dose-limiting Toxicities (DLTs) in Phase 1
timeFrame: 28 days
measure: Overall Safety Profile Characterized by Number of Subjects Experiencing Serious Adverse Events in Phase 1
timeFrame: through study completion, up to 224 days (8 cycles of treatment)
measure: Overall Safety Profile Characterized by the Number of Subjects That Discontinue Study Treatment - Phase 1
timeFrame: through study completion, up to 224 days (8 cycles of treatment)
measure: Overall Safety Profile Characterized by Number of Subjects Experiencing a Treatment Emergent Adverse Event- Phase 1
timeFrame: through study completion, up to 224 days (8 cycles of treatment)
measure: Progression Free Survival (Phase 2)
timeFrame: 4 months
Secondary outcomes:
measure: Area Under the Plasma Concentration Time Curve (AUC) (Phase 1)
timeFrame: Pre-dose and at 0.5, 1, 2, 3, 4, 6, 8, 24 and 48 hours after oral administration in Cycle 1 Days 1 and 15
measure: Best Overall Response Rate (Phase 2)
timeFrame: Baseline and at 4, 8, 12, 16 and 32 weeks
Other outcomes:
measure: Biomarker Concentrations in Blood (Phase 1 and Phase 2)
timeFrame: Baseline and 4, 8, 12, 16 and 32 weeks
Locations:
city: Birmingham
country: United States
facility: Rexahn Site
state: Alabama
city: Tucson
country: United States
facility: Rexahn Site
state: Arizona
city: Duarte
country: United States
facility: Rexahn Site
state: California
city: Miami Lakes
country: United States
facility: Rexahn Site
state: Florida
city: Miami
country: United States
facility: Rexahn Site
state: Florida
city: Skokie
country: United States
facility: Rexahn Site
state: Illinois
city: Saint Louis
country: United States
facility: Rexahn Site
state: Missouri
city: Las Vegas
country: United States
facility: Rexahn Site
state: Nevada
city: New York
country: United States
facility: Rexahn Site
state: New York
city: San Antonio
country: United States
facility: Rexahn Site
state: Texas
city: Salt Lake City
country: United States
facility: Rexahn Site
state: Utah
city: Fairfax
country: United States
facility: Rexahn Site
state: Virginia
Study officials:
affiliation: Rexahn Pharmaceuticals, Inc.
name: Ely Benaim, MD
role: STUDY_DIRECTOR
References: