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

Solid Tumor Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02030067
• Other study ID #: RX-3117-P1-01
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: December 2013
• Est. completion date: December 2019

Study information

• Verified date: November 2023
• Source: Processa Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 127
• Est. completion date: December 2019
• Est. primary completion date: July 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Metastatic Bladder Cancer
• Solid Tumor
• Urinary Bladder Neoplasms

Intervention

Drug:
• RX-3117

Locations

Country	Name	City	State
United States	Rexahn Site	Birmingham	Alabama
United States	Rexahn Site	Duarte	California
United States	Rexahn Site	Fairfax	Virginia
United States	Rexahn Site	Las Vegas	Nevada
United States	Rexahn Site	Miami	Florida
United States	Rexahn Site	Miami Lakes	Florida
United States	Rexahn Site	New York	New York
United States	Rexahn Site	Saint Louis	Missouri
United States	Rexahn Site	Salt Lake City	Utah
United States	Rexahn Site	San Antonio	Texas
United States	Rexahn Site	Skokie	Illinois
United States	Rexahn Site	Tucson	Arizona

Sponsors (1)

Lead Sponsor	Collaborator
Processa Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Biomarker Concentrations in Blood (Phase 1 and Phase 2)	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
Primary	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
Primary	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)
Primary	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)
Primary	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)
Primary	Progression Free Survival (Phase 2)	Progression Free Survival in Phase 2 of the study for pancreatic and bladder cancer subjects.	4 months
Secondary	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
Secondary	Best Overall Response Rate (Phase 2)	Best Overall Response Rate (includes Complete Response, Partial Response, and Stable Disease)	Baseline and at 4, 8, 12, 16 and 32 weeks