Dose-Finding and Safety Study for Oral Single-Agent to Treat Advanced Malignancies

November 14, 2023 updated by: Processa Pharmaceuticals

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

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).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

127

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
        • Rexahn Site
    • Arizona
      • Tucson, Arizona, United States, 85724
        • Rexahn Site
    • California
      • Duarte, California, United States, 91010
        • Rexahn Site
    • Florida
      • Miami, Florida, United States, 33136
        • Rexahn Site
      • Miami Lakes, Florida, United States, 33014
        • Rexahn Site
    • Illinois
      • Skokie, Illinois, United States, 60077
        • Rexahn Site
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Rexahn Site
    • Nevada
      • Las Vegas, Nevada, United States, 89119
        • Rexahn Site
    • New York
      • New York, New York, United States, 10021
        • Rexahn Site
    • Texas
      • San Antonio, Texas, United States, 78229
        • Rexahn Site
    • Utah
      • Salt Lake City, Utah, United States, 84112
        • Rexahn Site
    • Virginia
      • Fairfax, Virginia, United States, 22031
        • Rexahn Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: RX-3117
All subjects will receive RX-3117.
Drug: RX-3117

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Overall Safety Profile Characterized by # of Subjects With Dose-limiting Toxicities (DLTs) in Phase 1
Time Frame: 28 days
Number of subjects participating in Phase 1 that experienced a DLT during the first cycle of treatment (28 days)
28 days
Overall Safety Profile Characterized by Number of Subjects Experiencing Serious Adverse Events in Phase 1
Time Frame: through study completion, up to 224 days (8 cycles of treatment)
Number of subjects participating in Phase 1 that experience any SAEs
through study completion, up to 224 days (8 cycles of treatment)
Overall Safety Profile Characterized by the Number of Subjects That Discontinue Study Treatment - Phase 1
Time Frame: through study completion, up to 224 days (8 cycles of treatment)
Number of subjects participating in Phase 1 of study that discontinued study treatment due to a treatment emergent adverse event.
through study completion, up to 224 days (8 cycles of treatment)
Overall Safety Profile Characterized by Number of Subjects Experiencing a Treatment Emergent Adverse Event- Phase 1
Time Frame: through study completion, up to 224 days (8 cycles of treatment)
Number of subjects that experience any treatment-related adverse event.
through study completion, up to 224 days (8 cycles of treatment)
Progression Free Survival (Phase 2)
Time Frame: 4 months
Progression Free Survival in Phase 2 of the study for pancreatic and bladder cancer subjects.
4 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
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
Best Overall Response Rate (Phase 2)
Time Frame: Baseline and at 4, 8, 12, 16 and 32 weeks
Best Overall Response Rate (includes Complete Response, Partial Response, and Stable Disease)
Baseline and at 4, 8, 12, 16 and 32 weeks

Other Outcome Measures

Outcome Measure
Measure Description
Time Frame
Biomarker Concentrations in Blood (Phase 1 and Phase 2)
Time Frame: Baseline and 4, 8, 12, 16 and 32 weeks
Analysis of biomarker data not conducted. Biomarker samples were not analyzed due to previous sponsor terminating clinical program.
Baseline and 4, 8, 12, 16 and 32 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Processa Pharmaceuticals

Investigators

  • Study Director: Ely Benaim, MD, Rexahn Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

December 1, 2013

Primary Completion (Actual)

July 1, 2019

Study Completion (Actual)

December 1, 2019

Study Registration Dates

First Submitted

December 24, 2013

First Submitted That Met QC Criteria

January 6, 2014

First Posted (Estimated)

January 8, 2014

Study Record Updates

Last Update Posted (Estimated)

December 6, 2023

Last Update Submitted That Met QC Criteria

November 14, 2023

Last Verified

November 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RX-3117-P1-01

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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