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

NCT02030067 | Completed Phase 1 Results DMC

• 1 other identifier: RX-3117-P1-01
• Study Type: interventional
• Target: 127
• Locations: 1 country
• Sites: 12

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).

Conditions

Solid Tumor; Metastatic Bladder Cancer

Keywords

oncology; tumor; metastatic; bladder

Outcome Measures

Primary Outcomes (5)

• Overall Safety Profile Characterized by # of Subjects With Dose-limiting Toxicities (DLTs) in Phase 1

  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

  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

  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

  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)

  Progression Free Survival in Phase 2 of the study for pancreatic and bladder cancer subjects.

  4 months

Secondary Outcomes (2)

• Area Under the Plasma Concentration Time Curve (AUC) (Phase 1)

  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)

  Baseline and at 4, 8, 12, 16 and 32 weeks

Other Outcomes (1)

• Biomarker Concentrations in Blood (Phase 1 and Phase 2)

  Baseline and 4, 8, 12, 16 and 32 weeks

Study Arms (1)

RX-3117

EXPERIMENTAL

All subjects will receive RX-3117.

Drug: RX-3117

Interventions

RX-3117 DRUG

RX-3117

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Processa Pharmaceuticals: lead

Study Sites (12)

Rexahn Site

Birmingham, Alabama, 35294, United States

Location

Rexahn Site

Tucson, Arizona, 85724, United States

Location

Rexahn Site

Duarte, California, 91010, United States

Location

Rexahn Site

Miami, Florida, 33136, United States

Location

Rexahn Site

Miami Lakes, Florida, 33014, United States

Location

Rexahn Site

Skokie, Illinois, 60077, United States

Location

Rexahn Site

St Louis, Missouri, 63110, United States

Location

Rexahn Site

Las Vegas, Nevada, 89119, United States

Location

Rexahn Site

New York, New York, 10021, United States

Location

Rexahn Site

San Antonio, Texas, 78229, United States

Location

Rexahn Site

Salt Lake City, Utah, 84112, United States

Location

Rexahn Site

Fairfax, Virginia, 22031, United States

Location

MeSH Terms

Conditions

Urinary Bladder Neoplasms; Neoplasms; Neoplasm Metastasis

Interventions

fluorocyclopentenylcytosine

Condition Hierarchy (Ancestors)

Urologic Neoplasms; Urogenital Neoplasms; Neoplasms by Site; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Urinary Bladder Diseases; Urologic Diseases; Male Urogenital Diseases; Neoplastic Processes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Director Drug Development and Regulatory Affairs
• Organization: Processa Pharmaceuticals

pfranks@processapharmaceuticals.com

443-631-6614

Study Officials

• Ely Benaim, MD

  Rexahn Pharmaceuticals, Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 24, 2013
• First Posted: January 8, 2014
• Study Start: December 1, 2013
• Primary Completion: July 1, 2019
• Study Completion: December 1, 2019
• Last Updated: December 6, 2023
• Results First Posted: December 6, 2023

Record last verified: 2023-11

Locations

US (12)