Solid TumorPhase 1/2NCT02030067

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

A Phase 1/2 study testing RX-3117 in people with Solid Tumor and Metastatic Bladder Cancer, run by Processa Pharmaceuticals at 12 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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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). Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Solid Tumor, Metastatic Bladder Cancer
What is being tested?: RX-3117
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 127 participants
Where: 12 sitesUnited States
Study dates: Dec 2013to Dec 2019
Phase: Phase 1/2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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

Who cannot join

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

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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

Trial locations and contacts

12 sites in 1 country.

City, postcode or hospital

12 sites in 1 country

United States12 sites

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

Other trials in United States

About this study

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

Read the 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.

Conditions: Solid Tumor, Metastatic Bladder Cancer
Being tested: RX-3117
Keywords: oncology, tumor, metastatic, bladder
Medical subject headings: Urinary Bladder Neoplasms, Neoplasms, Neoplasm Metastasis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

RX-3117Drug

Study groups in NCT02030067
Study group	Role	What they receive
RX-3117	Experimental	All subjects will receive RX-3117. Receives: Drug: RX-3117

What is being measured

The primary outcome is the main question the study is designed to answer.

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)
Measured: 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
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 4 months

Secondary outcomes (2)

Area Under the Plasma Concentration Time Curve (AUC) (Phase 1)
Measured: 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)
Best Overall Response Rate (includes Complete Response, Partial Response, and Stable Disease)
Measured: Baseline and at 4, 8, 12, 16 and 32 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Dec 2013Dec 2019

First posted8 Jan 2014 (estimated)
Study startDec 2013
Primary completionJul 2019
Study completionDec 2019

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Solid TumorStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1/2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 127 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02030067
Sponsor's protocol ID: RX-3117-P1-01
Status last verified: Nov 2023
First submitted: 24 Dec 2013
Last updated: 6 Dec 2023
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruiting
A Collaborative Intervention for Improving Cancer Pain Management in Cancer Survivors
Hematopoietic and Lymphoid System Neoplasm, Malignant Solid Neoplasm
RecruitingObservational study
NCI Childhood Cancer Data Initiative (CCDI) Led Pediatric, Adolescent, and Young Adult Rare Cancer Registry for Very Rare Solid Tumors
Very Rare Tumors, Very Rare Cancers
Active, not recruitingPhase 1
Research Study Utilizing Expanded Multi-antigen Specific Lymphocytes for the Treatment of Solid Tumors
Solid Tumors
RecruitingObservational study
Natural History and Biospecimen Acquisition for Children and Adults With Rare Solid Tumors
Malignant Solid Tumors, Other Neoplasms Solid Tumors
Enrolling by invitationObservational study
Collection of Tissue Specimens From Patients With Solid Tumors or Blood Disorders and Their HLA-Compatible Family Members
Neoplasms, Hematologic Neoplasms

All Solid Tumor trials

Study at a glance

From the registry record, last updated by the sponsor on 6 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT02030067
Acronym / sponsor ID: RX-3117-P1-01
Conditions: Solid Tumor, Metastatic Bladder Cancer
Keywords: oncology, tumor, metastatic, bladder
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 127 participants (actual)
Lead sponsor: Processa PharmaceuticalsIndustry (pharma, biotech, devices)
Study start: Dec 2013
Primary completion: Jul 2019 (actual)
Study completion: Dec 2019 (actual)
First posted: 8 Jan 2014 (estimated)
Results: Posted 6 Dec 2023

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
RX-3117	Experimental	Drug: RX-3117All subjects will receive RX-3117.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (8 listed)

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

Key exclusion criteria (11 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
RX-3117	Drug	RX-3117

Primary outcome

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)
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
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.
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.
Progression Free Survival (Phase 2)
Time frame: 4 months
Progression Free Survival in Phase 2 of the study for pancreatic and bladder cancer subjects.

2 secondary outcomes

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
Best Overall Response Rate (Phase 2)
Time frame: Baseline and at 4, 8, 12, 16 and 32 weeks

Results and safety

Results posted to the registry on 6 Dec 2023, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
RX-3117 30 mg, 3 Times/Week	1	0
RX-3117 60 mg, 3 Times/Week	1	0
RX-3117 100 mg, 3 Times/Week	3	1
RX-3117 150 mg, 3 Times/Week	3	0
RX-3117 200 mg, 3 Times/Week	3	0
RX-3117 500 mg, 3 Times/Week	3	0
RX-3117 1000 mg, 3 Times/Week	3	0
RX 3117 1500 mg, 3 Times/Week	4	0
RX-3117 2000 mg, 3 Times/Week	6	0
RX-3117 500 mg, 5 Times/Week	3	0
RX-3117 700 mg, 5 Times/Week	11	0
RX-3117 500 mg, 7 Times/Week	3	0
RX-3117 700 mg 5 Times/Week (Phase 2 Portion of Study)	80	0

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

Time frame: 28 days. Count of participants. Unit: Participants

Measure	RX-3117 30 mg, 3 Times/Week n = 1	RX-3117 60 mg, 3 Times/Week n = 1	RX-3117 100 mg, 3 Times/Week n = 3	RX-3117 150 mg, 3 Times/Week n = 3	RX-3117 200 mg, 3 Times/Week n = 3	RX-3117 500 mg, 3 Times/Week n = 3	RX-3117 1000 mg, 3 Times/Week n = 3	RX 3117 1500 mg, 3 Times/Week n = 4	RX-3117 2000 mg, 3 Times/Week n = 6	RX-3117 500 mg, 5 Times/Week n = 3	RX-3117 700 mg, 5 Times/Week n = 11	RX-3117 500 mg, 7 Times/Week n = 3
Value	0	0	0	0	0	0	0	0	1	0	0	2

Primary outcome: 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). Count of participants. Unit: Participants

Measure	RX-3117 30 mg, 3 Times/Week n = 1	RX-3117 60 mg, 3 Times/Week n = 1	RX-3117 100 mg, 3 Times/Week n = 3	RX-3117 150 mg, 3 Times/Week n = 3	RX-3117 200 mg, 3 Times/Week n = 3	RX-3117 500 mg, 3 Times/Week n = 3	RX-3117 1000 mg, 3 Times/Week n = 3	RX 3117 1500 mg, 3 Times/Week n = 4	RX-3117 2000 mg, 3 Times/Week n = 6	RX-3117 500 mg, 5 Times/Week n = 3	RX-3117 700 mg, 5 Times/Week n = 11	RX-3117 500 mg, 7 Times/Week n = 3
Value	0	1	0	0	0	0	1	0	2	2	5	1

Population analysed: Population includes those subjects participating in the dose escalation phase of the study (Phase 1)

Primary outcome: 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). Count of participants. Unit: Participants

Measure	RX-3117 30 mg, 3 Times/Week n = 1	RX-3117 60 mg, 3 Times/Week n = 1	RX-3117 100 mg, 3 Times/Week n = 3	RX-3117 150 mg, 3 Times/Week n = 3	RX-3117 200 mg, 3 Times/Week n = 3	RX-3117 500 mg, 3 Times/Week n = 3	RX-3117 1000 mg, 3 Times/Week n = 3	RX 3117 1500 mg, 3 Times/Week n = 4	RX-3117 2000 mg, 3 Times/Week n = 6	RX-3117 500 mg, 5 Times/Week n = 3	RX-3117 700 mg, 5 Times/Week n = 11	RX-3117 500 mg, 7 Times/Week n = 3
Value	0	0	0	0	0	0	0	0	0	1	0	1

Population analysed: Population includes subjects in the dose escalation phase of the study (Phase 1)

Primary outcome: 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). Count of participants. Unit: Participants

Measure	RX-3117 30 mg, 3 Times/Week n = 1	RX-3117 60 mg, 3 Times/Week n = 1	RX-3117 100 mg, 3 Times/Week n = 3	RX-3117 150 mg, 3 Times/Week n = 3	RX-3117 200 mg, 3 Times/Week n = 3	RX-3117 500 mg, 3 Times/Week n = 3	RX-3117 1000 mg, 3 Times/Week n = 3	RX 3117 1500 mg, 3 Times/Week n = 4	RX-3117 2000 mg, 3 Times/Week n = 6	RX-3117 500 mg, 5 Times/Week n = 3	RX-3117 700 mg, 5 Times/Week n = 11	RX-3117 500 mg, 7 Times/Week n = 3
Value	0	1	2	3	3	0	2	3	6	3	9	3

Population analysed: Population includes subjects participating in the dose escalation phase of the study (Phase 1)

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: up to 224 days (8 cycles of treatment). Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Phase 1 (Dose Escalation) 30 mg 3x/Week	0 of 1 (0%)	1 of 1 (100%)	0 of 1
Phase 1 (Dose Escalation) 60 mg 3x/Week	1 of 1 (100%)	1 of 1 (100%)	0 of 1
Phase 1 (Dose Escalation) 100 mg 3x/Week	0 of 3 (0%)	3 of 3 (100%)	0 of 3
Phase 1 (Dose Escalation) 150 mg 3x/Week	0 of 3 (0%)	3 of 3 (100%)	0 of 3
Phase 1 (Dose Escalation) 200 mg 3x/Week	0 of 3 (0%)	3 of 3 (100%)	0 of 3
Phase 1 (Dose Escalation) 500 mg 3x/Week	0 of 3 (0%)	0 of 3 (0%)	0 of 3
Phase 1 (Dose Escalation) 1000 mg 3x/Week	1 of 3 (33.3%)	3 of 3 (100%)	0 of 3
Phase 1 (Dose Escalation) 1500 mg 3x/Week	0 of 4 (0%)	4 of 4 (100%)	0 of 4
Phase 1 (Dose Escalation) 2000 mg 3x/Week	2 of 6 (33.3%)	6 of 6 (100%)	0 of 6
Phase 1 (Dose Escalation) 500 mg 5x/Week	2 of 3 (66.7%)	3 of 3 (100%)	0 of 3
Phase 1 (Dose Escalation) 700 mg 5x/Week	5 of 11 (45.5%)	11 of 11 (100%)	1 of 11
Phase 1 (Dose Escalation) 500 mg 7x/Week	1 of 3 (33.3%)	3 of 3 (100%)	0 of 3
Phase 2 700 mg	29 of 80 (36.3%)	78 of 80 (97.5%)	8 of 80

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 6 Dec 2023.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Solid Tumor

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Ely Benaim, MD study director

Rexahn Pharmaceuticals, Inc.

12 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing RX-3117 in people with Solid Tumor and Metastatic Bladder Cancer, run by Processa Pharmaceuticals at 12 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 6 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 12 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 6 Dec 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall Safety Profile Characterized by # of Subjects With Dose-limiting Toxicities (DLTs) in Phase 1 (time frame: 28 days). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02030067.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Dec 2023.