RX-3117 in Combination With Abraxane® in Subjects With Me... | NCT03189914

Label	Type	Description	Intervention Names
RX-3117 + Abraxane	Experimental	RX-3117: oral, 500 - 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 75 - 125 mg/m^2, infused once per week for 3 weeks. 1 washout week/ cycle.	Drug: RX-3117

Name	Type	Description	Arm Group Labels	Other Names
RX-3117	Drug	RX-3117 will be administered orally in combination with Abraxane.	RX-3117 + Abraxane

Measure	Description	Time Frame
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0 (Phase 1 and 2)	Number of participants who experienced a treatment-related adverse event	9 months
Number of Participants With Clinical Laboratory Abnormalities (Phase 1 and 2)	Participants with adverse events coded using the MedDRA Dictionary (Version 20.0) to Investigations. Due to the underlying disease, not all abnormal labs are reported.	9 months
Number of Participants With Vital Sign Abnormalities (Phase 1 and 2)	Number of participants with clinically significant vital sign abnormalities (Phase 1 and 2) including heart rate, respiration rate, and blood pressure	9 months
Number of Participants With Electrocardiogram (ECG) Abnormalities (Phase 1 and 2)	Number of participants with clinically significant ECG abnormalities (Phase 1 and 2)	1 month
Number of Dose-limiting Toxicities (DLTs) (Phase 1)		4 weeks
Number of Participants With Progression Free Survival (PFS) and/or Objective Clinical Response (Phase 2)	Participants must have progression Free Survival (PFS) > 4 months or objective clinical response (complete or partial response).	9 months

Measure	Description	Time Frame
Area Under the Plasma Concentration Versus Time Curve (AUC) of RX-3117 (Phase 1 and Phase 2) - Day 1		Cycle 1 Day 1 (pre-infusion, post-infusion, pre-dose RX-3117, and 0.5, 1, 2, 4, 6, and 24 hours after administration)
Time to Maximum Observed Concentration [Tmax] of RX-3117 (Phase 1 and Phase 2) - Day 1

Measure	Description	Time Frame
Exploratory Measurement of Protein Biomarkers Related to RX-3117 or Pancreatic Cancer (Phase 1 and 2)		Baseline, and at 8, 6, 24, and 32 weeks
Tumor Burden Response (Phase 2)	Changes in tumor burden response via tumor marker measurement

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Disease Related

Subject has confirmed histologic or cytologic evidence of metastatic pancreatic cancer and has no prior treatment for metastatic pancreatic cancer.
Subject has measurable disease using Response Evaluation Criteria in Solid Tumors (RECIST) v 1.1.
Subject has a life expectancy of at least 3 months.
Subject has an Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1.

Demographic
Males or females ≥ 18 years of age
Subject must be able to swallow capsules
Subject must have adequate venous access for intravenous (IV) infusion

Laboratory
Subject has hemoglobin ≥ 9.0 g/dL at Screening
Subject has absolute neutrophil count (ANC) ≥ 1.5 x 109/L at Screening
Subject has platelet count ≥ 100 x 109/L at Screening
Subject has serum creatinine ≤ 1.5 times the upper limit of normal (ULN) at Screening. Subjects with serum creatinine levels > 1.5 times the ULN must have a 24-hour urine creatinine clearance ≥ 60 mL/min
Subject has serum bilirubin ≤ 1.5 times the ULN (except in subjects with Gilbert's Syndrome who must have serum bilirubin < 3.0 x ULN)
Subject has aspartate aminotransferase (AST; SGOT) and alanine aminotransferase (ALT; SGPT) ≤ 2.5 times the ULN (OR, AST and ALT ≤ 5 times the ULN in the presence of known liver metastases)
Subject has alkaline phosphatase ≤ 2.5 times the ULN (OR ≤ 5 times the ULN in the presence of known liver or bone metastases)
Subject has normal coagulation parameters (prothrombin time [PT] and/or international normalized ratio [INR], and partial thromboplastin time [PTT] within normal limits [<1.2 x ULN])
Subject has potassium concentration within normal range, or correctable with supplements.
Oxygen saturation by pulse oximetry ≥ 92% at rest.
For women of childbearing potential: Negative serum pregnancy test during screening and negative serum or urine pregnancy test at start of study therapy (Cycle1 Day 1).

Reproductive
For female subjects of childbearing potential, willingness to abstain from heterosexual intercourse or use a protocol-recommended method of contraception from the screening visit throughout the study treatment period and for 30 days following the last dose of study drug.
Female subjects of non-childbearing potential defined as having amenorrhea for at least 24 consecutive months, a documented hysterectomy, or a documented bilateral oophorectomy)
For fertile male subjects having intercourse with females of childbearing potential, willingness to abstain from heterosexual intercourse or use a protocol-recommended method of contraception from the start of study therapy throughout the study treatment period and for 30 days following the last dose of study drug and to refrain from sperm donation from the start of study treatment throughout the study treatment period and for 30 days following the last dose of study drug.

Ethical
In the judgment of the investigator, participation in the protocol offers an acceptable benefit-to-risk ratio when considering current disease status, medical condition, and the potential benefits and risks of alternative treatments for the subject's cancer.
Before any study-specific procedure, the appropriate written informed consent must be obtained

Exclusion Criteria:

Disease Related

Subject has primary brain tumors or clinical evidence of active brain metastasis
Subject has undergone major surgery within 4 weeks of the start of study treatment. Laparoscopy and central venous catheter placement are not considered major surgery

Medications
Subject has a history of systemic corticosteroid use within 7 days before Day 1 of Cycle 1

General
Subject has an active infection requiring parenteral or oral antibiotics within 2 weeks before planned start of study therapy
Subject has uncontrolled diabetes as assessed by the investigator
Subject has a second malignancy other than curatively resected basal cell carcinoma of the skin, squamous cell carcinoma of the skin, in situ carcinoma of the cervix, or other cancers treated with curative intent and no known active disease within 3 years before planned start of study therapy
Subject has an active infection of hepatitis B, hepatitis C or human immunodeficiency virus
Female subjects who are pregnant, planning a pregnancy or breast feeding during the study
Subject has a high cardiovascular risk, including, but not limited to, subjects with congestive heart failure (New York Heart Association [NYHA] Class III or IV), cardiac arrhythmia, unstable angina, coronary stenting or acute coronary syndromes within 6 months before planned start of study therapy or r myocardial infarction within one year before planned start of study therapy
Criterion removed
Subject has a history of prior allogeneic bone marrow progenitor cell or solid organ transplantation.
Subject has known acute or chronic pancreatitis.
Subject has persistent diarrhea, malabsorption, or known sub-acute bowel obstruction ≥ NCI CTCAE Grade 2, despite medical management.
Subject has any disorder that may interfere with drug absorption, distribution, metabolism, or excretion (including gastrointestinal surgery and bariatric surgery)
All acute toxic effects of any prior antitumor therapy resolved to Grade ≤ 1 before the start of study therapy (with the exception of alopecia [Grade 1 or 2 permitted], or neurotoxicity [Grade 1 or 2 permitted], or anemia [Grade 2 permitted])
Subject has 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
Subject has a history of interstitial lung disease, slowly progressive dyspnea and unproductive cough, sarcoidosis, silicosis, idiopathic pulmonary fibrosis, pulmonary hypersensitivity pneumonitis or multiple allergies. Any lung disease that may interfere with the detection or management of suspected drug-related pulmonary toxicity.
Subject is currently enrolled in any other clinical protocol or investigational trial that involves administration of experimental therapy and/or therapeutic devices, or investigational drug.
Subject has a history of hypersensitivity to RX-3117, gemcitabine, azacytidine cytosine arabinoside, paclitaxel, nab-paclitaxel, or their excipients.
Subject is unwilling or unable to comply with study requirements or planned unavailability for follow-up assessments.

Name	Affiliation	Role
Ely Benaim, MD	Rexahn Pharmaceuticals, Inc.	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
Rexahn Site		Tucson	Arizona	85724	United States
Rexahn Site

ID	Title	Description
FG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once per week for 3 weeks. 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.
FG001	RX-3117 + Abraxane Phase 2

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	Aug 8, 2018	Oct 12, 2020

ID	Title	Description
BG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once per week for 3 weeks. 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.
BG001	RX-3117 + Abraxane Phase 2	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once per week for 3 weeks. 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.
BG002	Total	Total of all reporting groups

Units	Counts
Participants	BG0008 BG00138 BG00246

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0 (Phase 1 and 2)

Number of participants who experienced a treatment-related adverse event

Posted

Count of Participants

Participants

9 months

ID	Title	Description
OG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.
OG001	RX-3117 + Abraxane Phase 2	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.

Units	Counts
Participants	OG0008 OG00138

Title

Denominators

Categories

Title	Measurements
	OG0008 OG00138

Primary

Number of Participants With Clinical Laboratory Abnormalities (Phase 1 and 2)

Participants with adverse events coded using the MedDRA Dictionary (Version 20.0) to Investigations. Due to the underlying disease, not all abnormal labs are reported.

Posted

Count of Participants

Participants

9 months

ID	Title	Description
OG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.
OG001	RX-3117 + Abraxane Phase 2	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.

Units	Counts
Participants	OG000

Primary

Number of Participants With Vital Sign Abnormalities (Phase 1 and 2)

Number of participants with clinically significant vital sign abnormalities (Phase 1 and 2) including heart rate, respiration rate, and blood pressure

Posted

Count of Participants

Participants

9 months

ID	Title	Description
OG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.
OG001	RX-3117 + Abraxane Phase 2	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.

Units	Counts
Participants	OG000

Primary

Number of Participants With Electrocardiogram (ECG) Abnormalities (Phase 1 and 2)

Number of participants with clinically significant ECG abnormalities (Phase 1 and 2)

Posted

Count of Participants

Participants

1 month

ID	Title	Description
OG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.
OG001	RX-3117 + Abraxane Phase 2	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.

Units	Counts
Participants	OG000

Primary

Number of Dose-limiting Toxicities (DLTs) (Phase 1)

Phase 1 participants receiving at least 10 doses in Cycle 1 and eligible for DLT evaluation.

Posted

Count of Participants

Participants

4 weeks

ID	Title	Description
OG000	RX-3117 + Abraxane	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.

Units	Counts
Participants	OG0006

Primary

Number of Participants With Progression Free Survival (PFS) and/or Objective Clinical Response (Phase 2)

Participants must have progression Free Survival (PFS) > 4 months or objective clinical response (complete or partial response).

Modified ITT population

Posted

Count of Participants

Participants

9 months

ID	Title	Description
OG000	RX-3117 + Abraxane Phase 2	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.

Units	Counts
Participants	OG000

Secondary

Area Under the Plasma Concentration Versus Time Curve (AUC) of RX-3117 (Phase 1 and Phase 2) - Day 1

Pharmacokinetic sampling only performed in Phase 1 and Stage 1 of Phase 2. Twelve patients were dosed as a part of Stage 1.

Posted

Mean

Standard Deviation

ng*hr/mL

Cycle 1 Day 1 (pre-infusion, post-infusion, pre-dose RX-3117, and 0.5, 1, 2, 4, 6, and 24 hours after administration)

ID	Title	Description
OG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once per week for 3 weeks. 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.
OG001	RX-3117 + Abraxane Phase 2	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once per week for 3 weeks. 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.

Units	Counts
Participants

Secondary

Time to Maximum Observed Concentration [Tmax] of RX-3117 (Phase 1 and Phase 2) - Day 1

Pharmacokinetic sampling only performed in Phase 1 and Stage 1 of Phase 2. Twelve patients were dosed as a part of Stage 1.

Posted

Mean

Standard Deviation

Cycle 1 Days 1 (pre-infusion, post-infusion, pre-dose RX-3117, and 0.5, 1, 2, 4, 6, and 24 hours after administration)

ID	Title	Description
OG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.
OG001	RX-3117 + Abraxane Phase 2	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.

Units	Counts
Participants

Secondary

Maximum Observed Concentration [Cmax] of RX-3117 (Phase 1 and Phase 2) - Day 1

Pharmacokinetic sampling only performed in Phase 1 and Stage 1 of Phase 2. Twelve patients were dosed as a part of Stage 1.

Posted

Mean

Standard Deviation

ng/mL

Cycle 1 Days 1 (pre-infusion, post-infusion, pre-dose RX-3117, and 0.5, 1, 2, 4, 6, and 24 hours after administration)

ID	Title	Description
OG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.
OG001	RX-3117 + Abraxane Phase 2	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.

Units	Counts
Participants

Secondary

Overall Response Rate [ORR] (Phase 1 and Phase 2)

Overall Response Rate will be based on the RECIST v1.1 and is defined as the percentage of subjects meeting criteria of Complete Response (CR) or Partial Response (PR). CR or PR must be confirmed at least 4 weeks after the date of the original CR or PR.

The overall number of participants analyzed is based on the modified intent-to-treat population.

Posted

Count of Participants

Participants

Every 8 weeks until progression or discontinuation, whichever came first, assessed up to 32 weeks

ID	Title	Description
OG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.
OG001	RX-3117 + Abraxane Phase 2	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.

Units	Counts

Secondary

Time to Response [TTR] (Phase 1)

Posted

Median

95% Confidence Interval

weeks

Up to 32 weeks

ID	Title	Description
OG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.

Units	Counts
Participants	OG0006

Title

Secondary

Duration of Response [DOR] (Phase 1 and Phase 2)

Duration of response is defined as the time from documentation of response to disease progression.

Duration of Response was not evaluable

Posted

Median

95% Confidence Interval

weeks

Up to 32 weeks

ID	Title	Description
OG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.
OG001	RX-3117 + Abraxane Phase 2	RX-3117: oral, 700/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.

Units	Counts
Participants	OG000

Secondary

Progression-free Survival [PFS] (Phase 1)

Progression Free Survival defined as the percentage of subjects who are alive in the study and not in progression.

The overall number of participants analyzed is based on the modified intent-to-treat population.

Posted

Median

95% Confidence Interval

weeks

Every 8 weeks until progression or discontinuation, whichever came first, assessed up to 32 weeks

ID	Title	Description
OG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.
OG001	RX-3117 + Abraxane Phase 2	RX-3117: oral, 500/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once per week for 3 weeks. 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.

Units	Counts
Participants

Secondary

Time to Progression (Phase 2)

Time to Progression defined as the time from first treatment administration to first documentation of RECIST-defined objective tumor progression.

The overall number of participants analyzed is based on the modified intent-to-treat population.

Posted

Median

95% Confidence Interval

weeks

Every 8 weeks until progression or discontinuation, whichever came first, assessed up to 32 weeks

ID	Title	Description
OG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.
OG001	RX-3117 + Abraxane Phase 2	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.

Units	Counts
Participants

Secondary

Area Under the Plasma Concentration Versus Time Curve (AUC) of RX-3117 and Abraxane® (Phase 1 and Phase 2) - Day 15

Posted

Mean

Standard Deviation

ng*hr/mL

Day 15 (pre-infusion, post-infusion, pre-dose RX-3117, and 0.5, 1, 2, 4, and 6 hours after administration)

ID	Title	Description
OG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.
OG001	RX-3117 + Abraxane Phase 2	RX-3117: oral, 700/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.

Units	Counts
Participants	OG000

Secondary

Area Under the Plasma Concentration Versus Time Curve (AUC) of Abraxane® (Phase 1 and Phase 2) - Day 1

Posted

Mean

Standard Deviation

ng*hr/mL

Cycle 1 Day 1 (pre-infusion, post-infusion, pre-dose RX-3117, and 0.5, 1, 2, 4, 6, and 24 hours after administration)

ID	Title	Description
OG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once per week for 3 weeks. 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.
OG001	RX-3117 + Abraxane Phase 2	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once per week for 3 weeks. 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.

Units	Counts
Participants	OG000

Secondary

Area Under the Plasma Concentration Versus Time Curve (AUC) of Abraxane®(Phase 1 and Phase 2) - Day 15

Posted

Mean

Standard Deviation

ng*hr/mL

Day 15 (pre-infusion, post-infusion, pre-dose RX-3117, and 0.5, 1, 2, 4, and 6 hours after administration)

ID	Title	Description
OG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once per week for 3 weeks. 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.
OG001	RX-3117 + Abraxane Phase 2	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once per week for 3 weeks. 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.

Units	Counts
Participants	OG000

Secondary

Time to Maximum Observed Concentration [Tmax] of RX-3117 (Phase 1 and Phase 2) - Day 15

Posted

Mean

Standard Deviation

Day 15 (pre-infusion, post-infusion, pre-dose RX-3117, and 0.5, 1, 2, 4, and 6 hours after administration)

ID	Title	Description
OG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.
OG001	RX-3117 + Abraxane Phase 2	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.

Units	Counts
Participants	OG000

Secondary

Time to Maximum Observed Concentration [Tmax] of Abraxane® (Phase 1 and Phase 2) - Day 1

Posted

Mean

Standard Deviation

Cycle 1 Days 1 (pre-infusion, post-infusion, pre-dose RX-3117, and 0.5, 1, 2, 4, 6, and 24 hours after administration)

ID	Title	Description
OG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.
OG001	RX-3117 + Abraxane Phase 2	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.

Units	Counts
Participants	OG000

Secondary

Time to Maximum Observed Concentration [Tmax] of Abraxane® (Phase 1 and Phase 2) - Day 15

Posted

Mean

Standard Deviation

Day 15 (pre-infusion, post-infusion, pre-dose RX-3117, and 0.5, 1, 2, 4, and 6 hours after administration)

ID	Title	Description
OG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.
OG001	RX-3117 + Abraxane Phase 2	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.

Units	Counts
Participants	OG000

Other Pre-specified

Exploratory Measurement of Protein Biomarkers Related to RX-3117 or Pancreatic Cancer (Phase 1 and 2)

Not Posted

Baseline, and at 8, 6, 24, and 32 weeks

Participants

Other Pre-specified

Tumor Burden Response (Phase 2)

Changes in tumor burden response via tumor marker measurement

Not Posted

Baseline, and at 8, 6, 24, and 32 weeks

Participants

Other Pre-specified

Quality of Life (QOL) (Phase 1 and 2)

Weekly patient reported quality of life measures using validated QOL questionnaires

Not Posted

9 months

Participants

Secondary

Maximum Observed Concentration [Cmax] of RX-3117 (Phase 1 and Phase 2) - Day 15

Posted

Mean

Standard Deviation

ng/mL

Day 15 (pre-infusion, post-infusion, pre-dose RX-3117, and 0.5, 1, 2, 4, and 6 hours after administration)

ID	Title	Description
OG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.
OG001	RX-3117 + Abraxane Phase 2	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.

Units	Counts
Participants	OG000

Secondary

Maximum Observed Concentration [Cmax] of Abraxane (Phase 1 and Phase 2) - Day 1

Posted

Mean

Standard Deviation

ng/mL

Cycle 1 Days 1 (pre-infusion, post-infusion, pre-dose RX-3117, and 0.5, 1, 2, 4, 6, and 24 hours after administration)

ID	Title	Description
OG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.
OG001	RX-3117 + Abraxane Phase 2	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.

Units	Counts
Participants	OG000

Secondary

Maximum Observed Concentration [Cmax] of Abraxane (Phase 1 and Phase 2) - Day 15

Posted

Mean

Standard Deviation

ng/mL

Day 15 (pre-infusion, post-infusion, pre-dose RX-3117, and 0.5, 1, 2, 4, and 6 hours after administration)

ID	Title	Description
OG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.
OG001	RX-3117 + Abraxane Phase 2	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.

Units	Counts
Participants	OG000

Other Adverse Events

Term	Organ System	Assessment Type	Statistical Information
Anaemia	Blood and lymphatic system disorders	Systematic Assessment	EG0002 affected8 at risk EG00119 affected38 at risk
Neutropenia	Blood and lymphatic system disorders	Systematic Assessment	EG0001 affected8 at risk EG0019 affected38 at risk
Palpitations	Cardiac disorders	Systematic Assessment	EG0000 affected8 at risk EG0012 affected38 at risk
Tachycardia	Cardiac disorders	Systematic Assessment	EG0000 affected8 at risk EG0012 affected38 at risk
Vision blurred	Eye disorders	Systematic Assessment	EG0001 affected8 at risk EG0014 affected38 at risk
Diarrhoea	Gastrointestinal disorders	Systematic Assessment	EG0008 affected8 at risk EG00125 affected38 at risk
Nausea	Gastrointestinal disorders	Systematic Assessment	EG0006 affected8 at risk EG00123 affected38 at risk
Abdominal pain	Gastrointestinal disorders	Systematic Assessment	EG0001 affected8 at risk EG00120 affected38 at risk
Vomiting	Gastrointestinal disorders	Systematic Assessment	EG0004 affected8 at risk EG00111 affected38 at risk
Constipation	Gastrointestinal disorders	Systematic Assessment	EG0002 affected8 at risk EG00112 affected38 at risk
Abdominal distension	Gastrointestinal disorders	Systematic Assessment	EG0000 affected8 at risk EG0015 affected38 at risk
Dry mouth	Gastrointestinal disorders	Systematic Assessment	EG0001 affected8 at risk EG0017 affected38 at risk
Dyspepsia	Gastrointestinal disorders	Systematic Assessment	EG0001 affected8 at risk EG0015 affected38 at risk
Flatulence	Gastrointestinal disorders	Systematic Assessment	EG0000 affected8 at risk EG0016 affected38 at risk
Stomatitis	Gastrointestinal disorders	Systematic Assessment	EG0001 affected8 at risk EG0015 affected38 at risk
Abdominal pain upper	Gastrointestinal disorders	Systematic Assessment	EG0002 affected8 at risk EG0013 affected38 at risk
Abdominal discomfort	Gastrointestinal disorders	Systematic Assessment	EG0002 affected8 at risk EG0011 affected38 at risk
Rectal haemorrhage	Gastrointestinal disorders	Systematic Assessment	EG0001 affected8 at risk EG0012 affected38 at risk
Fatigue	Gastrointestinal disorders	Systematic Assessment	EG0006 affected8 at risk EG00123 affected38 at risk
Pyrexia	Gastrointestinal disorders	Systematic Assessment	EG0002 affected8 at risk EG00113 affected38 at risk
Oedema peripheral	Gastrointestinal disorders	Systematic Assessment	EG0001 affected8 at risk EG00113 affected38 at risk
Chills	Gastrointestinal disorders	Systematic Assessment	EG0001 affected8 at risk EG0017 affected38 at risk
Asthenia	Gastrointestinal disorders	Systematic Assessment	EG0002 affected8 at risk EG0015 affected38 at risk
Non-cardiac chest pain	Gastrointestinal disorders	Systematic Assessment	EG0000 affected8 at risk EG0015 affected38 at risk
Influenza like illness	Gastrointestinal disorders	Systematic Assessment	EG0000 affected8 at risk EG0013 affected38 at risk
Urinary tract infection	Infections and infestations	Systematic Assessment	EG0002 affected8 at risk EG0015 affected38 at risk
Oral candidiasis	Infections and infestations	Systematic Assessment	EG0000 affected8 at risk EG0013 affected38 at risk
Pneumonia	Infections and infestations	Systematic Assessment	EG0000 affected8 at risk EG0013 affected38 at risk
Fall	Injury, poisoning and procedural complications	Systematic Assessment	EG0000 affected8 at risk EG0013 affected38 at risk
Weight decreased	Investigations	Systematic Assessment	EG0000 affected8 at risk EG0017 affected38 at risk
Blood bilirubin increased	Investigations	Systematic Assessment	EG0002 affected8 at risk EG0014 affected38 at risk
Blood alkaline phosphatase increased	Investigations	Systematic Assessment	EG0001 affected8 at risk EG0014 affected38 at risk
Neutrophil count decreased	Investigations	Systematic Assessment	EG0001 affected8 at risk EG0014 affected38 at risk
White blood cell count decreased	Investigations	Systematic Assessment	EG0002 affected8 at risk EG0013 affected38 at risk
Decreased appetite	Metabolism and nutrition disorders	Systematic Assessment	EG0004 affected8 at risk EG00115 affected38 at risk
Dehydration	Metabolism and nutrition disorders	Systematic Assessment	EG0002 affected8 at risk EG00110 affected38 at risk
Hypokalaemia	Metabolism and nutrition disorders	Systematic Assessment	EG0002 affected8 at risk EG0018 affected38 at risk
Hypomagnesaemia	Metabolism and nutrition disorders	Systematic Assessment	EG0002 affected8 at risk EG0015 affected38 at risk
Hyperglycaemia	Metabolism and nutrition disorders	Systematic Assessment	EG0000 affected8 at risk EG0013 affected38 at risk
Back pain	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0000 affected8 at risk EG0017 affected38 at risk
Muscular weakness	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0000 affected8 at risk EG0014 affected38 at risk
Pain in extremity	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0001 affected8 at risk EG0013 affected38 at risk
Arthralgia	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0000 affected8 at risk EG0013 affected38 at risk
Myalgia	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0000 affected8 at risk EG0013 affected38 at risk
Dizziness	Nervous system disorders	Systematic Assessment	EG0002 affected8 at risk EG00117 affected38 at risk
Neuropathy peripheral	Nervous system disorders	Systematic Assessment	EG0001 affected8 at risk EG00110 affected38 at risk
Peripheral sensory neuropathy	Nervous system disorders	Systematic Assessment	EG0001 affected8 at risk EG0018 affected38 at risk
Dysgeusia	Nervous system disorders	Systematic Assessment	EG0002 affected8 at risk EG0016 affected38 at risk
Headache	Nervous system disorders	Systematic Assessment	EG0002 affected8 at risk EG0015 affected38 at risk
Depression	Psychiatric disorders	Systematic Assessment	EG0000 affected8 at risk EG0017 affected38 at risk
Insomnia	Psychiatric disorders	Systematic Assessment	EG0000 affected8 at risk EG0015 affected38 at risk
Anxiety	Psychiatric disorders	Systematic Assessment	EG0000 affected8 at risk EG0013 affected38 at risk
Dyspnoea	Respiratory, thoracic and mediastinal disorders	Systematic Assessment	EG0000 affected8 at risk EG00110 affected38 at risk
Epistaxis	Respiratory, thoracic and mediastinal disorders	Systematic Assessment	EG0002 affected8 at risk EG0014 affected38 at risk
Cough	Respiratory, thoracic and mediastinal disorders	Systematic Assessment	EG0000 affected8 at risk EG0014 affected38 at risk
Nasal congestion	Respiratory, thoracic and mediastinal disorders	Systematic Assessment	EG0000 affected8 at risk EG0014 affected38 at risk
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	Systematic Assessment	EG0000 affected8 at risk EG0014 affected38 at risk
Alopecia	Skin and subcutaneous tissue disorders	Systematic Assessment	EG0005 affected8 at risk EG0019 affected38 at risk
Rash	Skin and subcutaneous tissue disorders	Systematic Assessment	EG0002 affected8 at risk EG0017 affected38 at risk
Pruritus	Skin and subcutaneous tissue disorders	Systematic Assessment	EG0003 affected8 at risk EG0013 affected38 at risk
Dry skin	Skin and subcutaneous tissue disorders	Systematic Assessment	EG0000 affected8 at risk EG0014 affected38 at risk
Rash maculo-papular	Skin and subcutaneous tissue disorders	Systematic Assessment	EG0000 affected8 at risk EG0014 affected38 at risk
Nail discolouration	Skin and subcutaneous tissue disorders	Systematic Assessment	EG0000 affected8 at risk EG0013 affected38 at risk
Hypotension	Vascular disorders	Systematic Assessment	EG0000 affected8 at risk EG0019 affected38 at risk

ID	Term
D004067	Digestive System Neoplasms
D009371	Neoplasms by Site
D009369	Neoplasms
D004701	Endocrine Gland Neoplasms
D004066	Digestive System Diseases
D010182	Pancreatic Diseases
D004700	Endocrine System Diseases

ID	Term
C000589325	fluorocyclopentenylcytosine
D000068196	Albumin-Bound Paclitaxel

ID	Term
D017239	Paclitaxel
D043823	Taxoids
D043822	Cyclodecanes
D003516	Cycloparaffins
D006840	Hydrocarbons, Alicyclic
D006844	Hydrocarbons, Cyclic
D006838	Hydrocarbons
D009930	Organic Chemicals
D004224	Diterpenes
D013729	Terpenes
D000418	Albumins
D011506	Proteins
D000602	Amino Acids, Peptides, and Proteins

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	RX-3117 + Abraxane Phase 1	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.	0	8	3	8	8	8
EG001	RX-3117 + Abraxane Phase 2	RX-3117: oral, 700 mg/ day for 5 days on/ 2 days off for 3 weeks. 1 washout week/ cycle. Abraxane: 125 mg/m^2, infused once a week for 3 weeks (Days 1, 8, and 15). 1 washout week/ cycle. RX-3117: RX-3117 will be administered orally in combination with Abraxane.	2	38	18	38	38	38

Term	Organ System	Assessment Type	Statistical Information
Febrile neutropenia	Blood and lymphatic system disorders	Systematic Assessment	EG0001 affected8 at risk EG0011 affected38 at risk
Anaemia	Blood and lymphatic system disorders	Systematic Assessment	EG0001 affected8 at risk EG0010 affected38 at risk
Neutropenia	Blood and lymphatic system disorders	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Myocardial infarction	Cardiac disorders	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Small intestinal obstruction	Gastrointestinal disorders	Systematic Assessment	EG0000 affected8 at risk EG0012 affected38 at risk
Abdominal pain upper	Gastrointestinal disorders	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Stomatitis	Gastrointestinal disorders	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Sudden death	General disorders	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Cholecystitis acute	Hepatobiliary disorders	Systematic Assessment	EG0000 affected8 at risk EG0012 affected38 at risk
Bile duct obstruction	Hepatobiliary disorders	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Hyperbilirubinaemia	Hepatobiliary disorders	Systematic Assessment	EG0001 affected8 at risk EG0010 affected38 at risk
Jaundice	Hepatobiliary disorders	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Catheter site infection	Infections and infestations	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Cholecystitis infective	Infections and infestations	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Clostridium difficile colitis	Infections and infestations	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Emphysematous choleccystitis	Infections and infestations	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Liver abscess	Infections and infestations	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Penumonia	Infections and infestations	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Sepsis	Infections and infestations	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Humerus fracture	Injury, poisoning and procedural complications	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Blood alkaline phosphatase increased	Investigations	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Blood bilirubin increased	Investigations	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Transaminases increased	Investigations	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Dehydration	Metabolism and nutrition disorders	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Back pain	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Musucular weakness	Musculoskeletal and connective tissue disorders	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Pancreatic carcinoma	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Ischaemic stroke	Nervous system disorders	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Deep vein thrombosis	Vascular disorders	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk
Embolism	Vascular disorders	Systematic Assessment	EG0001 affected8 at risk EG0010 affected38 at risk
Hypotension	Vascular disorders	Systematic Assessment	EG0000 affected8 at risk EG0011 affected38 at risk