Dose-Finding and Safety Study for Oral Single-Agent to Treat Advanced Malignancies
Primary Purpose
Solid Tumor, Metastatic Bladder Cancer
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
RX-3117
Sponsored by
About this trial
This is an interventional treatment trial for Solid Tumor focused on measuring oncology, tumor, metastatic, bladder
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
Sites / Locations
- Rexahn Site
- Rexahn Site
- Rexahn Site
- Rexahn Site
- Rexahn Site
- Rexahn Site
- Rexahn Site
- Rexahn Site
- Rexahn Site
- Rexahn Site
- Rexahn Site
- Rexahn Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
RX-3117
Arm Description
All subjects will receive RX-3117.
Outcomes
Primary Outcome Measures
Incidence of dose limiting toxicities (Phase 1)
Progression free survival and/or objective clinical response rate (Phase 2)
Secondary Outcome Measures
Area under the plasma concentration time curve (AUC) (Phase 1)
Overall response rate, time to progression and duration of response (Phase 2)
Full Information
NCT ID
NCT02030067
First Posted
December 24, 2013
Last Updated
January 3, 2020
Sponsor
Rexahn Pharmaceuticals, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT02030067
Brief Title
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
2. Study Status
Record Verification Date
January 2020
Overall Recruitment Status
Completed
Study Start Date
December 2013 (undefined)
Primary Completion Date
July 2019 (Actual)
Study Completion Date
December 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Rexahn Pharmaceuticals, Inc.
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Solid Tumor, Metastatic Bladder Cancer
Keywords
oncology, tumor, metastatic, bladder
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
124 (Actual)
8. Arms, Groups, and Interventions
Arm Title
RX-3117
Arm Type
Experimental
Arm Description
All subjects will receive RX-3117.
Intervention Type
Drug
Intervention Name(s)
RX-3117
Intervention Description
escalating doses (Phase 1)
Primary Outcome Measure Information:
Title
Incidence of dose limiting toxicities (Phase 1)
Time Frame
4 weeks
Title
Progression free survival and/or objective clinical response rate (Phase 2)
Time Frame
4 months
Secondary Outcome Measure Information:
Title
Area under the plasma concentration time curve (AUC) (Phase 1)
Time Frame
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
Title
Overall response rate, time to progression and duration of response (Phase 2)
Time Frame
Baseline and at 4, 8, 12, 16 and 32 weeks
Other Pre-specified Outcome Measures:
Title
Biomarker concentrations in blood (Phase 1 and Phase 2)
Time Frame
Baseline and 4, 8, 12, 16 and 32 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ely Benaim, MD
Organizational Affiliation
Rexahn Pharmaceuticals, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Rexahn Site
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35294
Country
United States
Facility Name
Rexahn Site
City
Tucson
State/Province
Arizona
ZIP/Postal Code
85724
Country
United States
Facility Name
Rexahn Site
City
Duarte
State/Province
California
ZIP/Postal Code
91010
Country
United States
Facility Name
Rexahn Site
City
Miami Lakes
State/Province
Florida
ZIP/Postal Code
33014
Country
United States
Facility Name
Rexahn Site
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Rexahn Site
City
Skokie
State/Province
Illinois
ZIP/Postal Code
60077
Country
United States
Facility Name
Rexahn Site
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Rexahn Site
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89119
Country
United States
Facility Name
Rexahn Site
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Facility Name
Rexahn Site
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78229
Country
United States
Facility Name
Rexahn Site
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84112
Country
United States
Facility Name
Rexahn Site
City
Fairfax
State/Province
Virginia
ZIP/Postal Code
22031
Country
United States
12. IPD Sharing Statement
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Dose-Finding and Safety Study for Oral Single-Agent to Treat Advanced Malignancies
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