Trial Outcomes & Findings for RX-3117 in Combination With Abraxane® in Subjects With Metastatic Pancreatic Cancer (NCT NCT03189914)

NCT ID: NCT03189914

Last Updated: 2023-12-06

Results Overview

Number of participants who experienced a treatment-related adverse event

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

46 participants

Primary outcome timeframe

9 months

Results posted on

2023-12-06

Participant Flow

Phase 1: The arm was designed as a dose de-escalation. De-escalation was not required during Phase 1, therefore, the starting dose 700mg RX-3117 + 125 mg/m\^2 Abraxane was the only dose tested.

Participant milestones

Participant milestones
Measure	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.	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. (MTD) RX-3117: RX-3117 will be administered orally in combination with Abraxane.
Overall Study STARTED	8	38
Overall Study Completed Cycle 1	6	31
Overall Study COMPLETED	1	4
Overall Study NOT COMPLETED	7	34

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

RX-3117 in Combination With Abraxane® in Subjects With Metastatic Pancreatic Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	RX-3117 + Abraxane Phase 1 n=8 Participants 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.	RX-3117 + Abraxane Phase 2 n=38 Participants 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.	Total n=46 Participants Total of all reporting groups
Age, Continuous	66 years STANDARD_DEVIATION 70.5 • n=5 Participants	66.3 years STANDARD_DEVIATION 66 • n=7 Participants	66.3 years STANDARD_DEVIATION 67 • n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants	19 Participants n=7 Participants	24 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants	19 Participants n=7 Participants	22 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants	5 Participants n=7 Participants	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	7 Participants n=5 Participants	32 Participants n=7 Participants	39 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	4 Participants n=7 Participants	4 Participants n=5 Participants
Race (NIH/OMB) White	8 Participants n=5 Participants	34 Participants n=7 Participants	42 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	8 participants n=5 Participants	38 participants n=7 Participants	46 participants n=5 Participants

PRIMARY outcome

Timeframe: 9 months

Number of participants who experienced a treatment-related adverse event

Outcome measures

Outcome measures
Measure	RX-3117 + Abraxane Phase 1 n=8 Participants 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.	RX-3117 + Abraxane Phase 2 n=38 Participants 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.
Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0 (Phase 1 and 2)	8 Participants	38 Participants

PRIMARY outcome

Timeframe: 9 months

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.

Outcome measures

Outcome measures
Measure	RX-3117 + Abraxane Phase 1 n=8 Participants 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.	RX-3117 + Abraxane Phase 2 n=38 Participants 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.
Number of Participants With Clinical Laboratory Abnormalities (Phase 1 and 2)	4 Participants	18 Participants

PRIMARY outcome

Timeframe: 9 months

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

Outcome measures

Outcome measures
Measure	RX-3117 + Abraxane Phase 1 n=8 Participants 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.	RX-3117 + Abraxane Phase 2 n=38 Participants 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.
Number of Participants With Vital Sign Abnormalities (Phase 1 and 2)	0 Participants	0 Participants

PRIMARY outcome

Timeframe: 1 month

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

Outcome measures

Outcome measures
Measure	RX-3117 + Abraxane Phase 1 n=8 Participants 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.	RX-3117 + Abraxane Phase 2 n=38 Participants 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.
Number of Participants With Electrocardiogram (ECG) Abnormalities (Phase 1 and 2)	0 Participants	0 Participants

PRIMARY outcome

Timeframe: 4 weeks

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

Outcome measures

Outcome measures
Measure	RX-3117 + Abraxane Phase 1 n=6 Participants 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.	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.
Number of Dose-limiting Toxicities (DLTs) (Phase 1)	0 Participants	—

PRIMARY outcome

Timeframe: 9 months

Population: Modified ITT population

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

Outcome measures

Outcome measures
Measure	RX-3117 + Abraxane Phase 1 n=33 Participants 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.	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.
Number of Participants With Progression Free Survival (PFS) and/or Objective Clinical Response (Phase 2)	17 Participants	—

SECONDARY outcome

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

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

Outcome measures

Outcome measures
Measure	RX-3117 + Abraxane Phase 1 n=8 Participants 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.	RX-3117 + Abraxane Phase 2 n=12 Participants 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.
Area Under the Plasma Concentration Versus Time Curve (AUC) of RX-3117 (Phase 1 and Phase 2) - Day 1	10500 ng*hr/mL Standard Deviation 6490	8780 ng*hr/mL Standard Deviation 4460

SECONDARY outcome

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

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

Outcome measures

Outcome measures
Measure	RX-3117 + Abraxane Phase 1 n=8 Participants 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.	RX-3117 + Abraxane Phase 2 n=12 Participants 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.
Time to Maximum Observed Concentration [Tmax] of RX-3117 (Phase 1 and Phase 2) - Day 1	2.3 hr Standard Deviation 0.89	4.51 hr Standard Deviation 6.18

SECONDARY outcome

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

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

Outcome measures

Outcome measures
Measure	RX-3117 + Abraxane Phase 1 n=8 Participants 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.	RX-3117 + Abraxane Phase 2 n=12 Participants 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.
Maximum Observed Concentration [Cmax] of RX-3117 (Phase 1 and Phase 2) - Day 1	1120 ng/mL Standard Deviation 516	947 ng/mL Standard Deviation 422

SECONDARY outcome

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

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

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.

Outcome measures

Outcome measures
Measure	RX-3117 + Abraxane Phase 1 n=6 Participants 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.	RX-3117 + Abraxane Phase 2 n=33 Participants 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.
Overall Response Rate [ORR] (Phase 1 and Phase 2)	2 Participants	7 Participants

SECONDARY outcome

Timeframe: Up to 32 weeks

Outcome measures

Outcome measures
Measure	RX-3117 + Abraxane Phase 1 n=6 Participants 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.	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.
Time to Response [TTR] (Phase 1)	23.6 weeks Interval 7.4 to 23.6	—

SECONDARY outcome

Timeframe: Up to 32 weeks

Population: Duration of Response was not evaluable

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

Outcome measures

Outcome measures
Measure	RX-3117 + Abraxane Phase 1 n=6 Participants 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.	RX-3117 + Abraxane Phase 2 n=33 Participants 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.
Duration of Response [DOR] (Phase 1 and Phase 2)	NA weeks Interval 19.1 to The small number of patients is insufficient to calculate the median duration of response	NA weeks Interval 16.1 to The small number of patients is insufficient to calculate the median duration of response

SECONDARY outcome

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

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

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

Outcome measures

Outcome measures
Measure	RX-3117 + Abraxane Phase 1 n=6 Participants 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.	RX-3117 + Abraxane Phase 2 n=33 Participants 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.
Progression-free Survival [PFS] (Phase 1)	13.8 weeks Interval 7.0 to The value is not available because the data set is small and the upper limit of the confidence interval is non-evaluable.	23.3 weeks Interval 15.1 to 31.1

SECONDARY outcome

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

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

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

Outcome measures

Outcome measures
Measure	RX-3117 + Abraxane Phase 1 n=6 Participants 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.	RX-3117 + Abraxane Phase 2 n=33 Participants 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.
Time to Progression (Phase 2)	13.8 weeks Interval 7.0 to The value is not available because the data set is small and the upper limit of the confidence interval is non-evaluable.	23.4 weeks Interval 15.1 to 32.6

SECONDARY outcome

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

Outcome measures

Outcome measures
Measure	RX-3117 + Abraxane Phase 1 n=6 Participants 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.	RX-3117 + Abraxane Phase 2 n=9 Participants 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.
Area Under the Plasma Concentration Versus Time Curve (AUC) of RX-3117 and Abraxane® (Phase 1 and Phase 2) - Day 15	1820 ng*hr/mL Standard Deviation 510	3160 ng*hr/mL Standard Deviation 1490

SECONDARY outcome

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

Outcome measures

Outcome measures
Measure	RX-3117 + Abraxane Phase 1 n=8 Participants 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.	RX-3117 + Abraxane Phase 2 n=12 Participants 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.
Area Under the Plasma Concentration Versus Time Curve (AUC) of Abraxane® (Phase 1 and Phase 2) - Day 1	5560 ng*hr/mL Standard Deviation 2410	5290 ng*hr/mL Standard Deviation 2120

SECONDARY outcome

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

Outcome measures

Outcome measures
Measure	RX-3117 + Abraxane Phase 1 n=5 Participants 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.	RX-3117 + Abraxane Phase 2 n=8 Participants 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.
Area Under the Plasma Concentration Versus Time Curve (AUC) of Abraxane®(Phase 1 and Phase 2) - Day 15	4020 ng*hr/mL Standard Deviation 1710	3670 ng*hr/mL Standard Deviation 2080

SECONDARY outcome

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

Outcome measures

Outcome measures
Measure	RX-3117 + Abraxane Phase 1 n=6 Participants 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.	RX-3117 + Abraxane Phase 2 n=9 Participants 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.
Time to Maximum Observed Concentration [Tmax] of RX-3117 (Phase 1 and Phase 2) - Day 15	1.84 hr Standard Deviation 0.41	3.75 hr Standard Deviation 1.35

SECONDARY outcome

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

Outcome measures

Outcome measures
Measure	RX-3117 + Abraxane Phase 1 n=8 Participants 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.	RX-3117 + Abraxane Phase 2 n=12 Participants 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.
Time to Maximum Observed Concentration [Tmax] of Abraxane® (Phase 1 and Phase 2) - Day 1	0.71 hr Standard Deviation 0.17	0.62 hr Standard Deviation 0.10

SECONDARY outcome

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

Outcome measures

Outcome measures
Measure	RX-3117 + Abraxane Phase 1 n=5 Participants 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.	RX-3117 + Abraxane Phase 2 n=8 Participants 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.
Time to Maximum Observed Concentration [Tmax] of Abraxane® (Phase 1 and Phase 2) - Day 15	0.55 hr Standard Deviation 0.06	0.69 hr Standard Deviation 0.18

SECONDARY outcome

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

Outcome measures

Outcome measures
Measure	RX-3117 + Abraxane Phase 1 n=6 Participants 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.	RX-3117 + Abraxane Phase 2 n=9 Participants 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.
Maximum Observed Concentration [Cmax] of RX-3117 (Phase 1 and Phase 2) - Day 15	528 ng/mL Standard Deviation 166	838 ng/mL Standard Deviation 329

SECONDARY outcome

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

Outcome measures

Outcome measures
Measure	RX-3117 + Abraxane Phase 1 n=8 Participants 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.	RX-3117 + Abraxane Phase 2 n=12 Participants 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.
Maximum Observed Concentration [Cmax] of Abraxane (Phase 1 and Phase 2) - Day 1	4160 ng/mL Standard Deviation 2770	3940 ng/mL Standard Deviation 2320

SECONDARY outcome

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

Outcome measures

Outcome measures
Measure	RX-3117 + Abraxane Phase 1 n=5 Participants 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.	RX-3117 + Abraxane Phase 2 n=8 Participants 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.
Maximum Observed Concentration [Cmax] of Abraxane (Phase 1 and Phase 2) - Day 15	4780 ng/mL Standard Deviation 2380	3320 ng/mL Standard Deviation 2330

OTHER_PRE_SPECIFIED outcome

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

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

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

Changes in tumor burden response via tumor marker measurement

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: 9 months

Weekly patient reported quality of life measures using validated QOL questionnaires

Outcome measures

Outcome data not reported

Adverse Events

RX-3117 + Abraxane Phase 1

Serious events: 3 serious events

Other events: 8 other events

Deaths: 0 deaths

RX-3117 + Abraxane Phase 2

Serious events: 18 serious events

Other events: 38 other events

Deaths: 2 deaths

Serious adverse events

Other adverse events

Additional Information

Doug Swirsky/ Chief Executive Officer

Rexahn

Phone: 12402685300

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	RX-3117 + Abraxane Phase 1 n=8 participants at risk 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.	RX-3117 + Abraxane Phase 2 n=38 participants at risk 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.
Blood and lymphatic system disorders Febrile neutropenia	12.5% 1/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Blood and lymphatic system disorders Anaemia	12.5% 1/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	0.00% 0/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Blood and lymphatic system disorders Neutropenia	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Cardiac disorders Myocardial infarction	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Gastrointestinal disorders Small intestinal obstruction	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	5.3% 2/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Gastrointestinal disorders Abdominal pain upper	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Gastrointestinal disorders Stomatitis	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
General disorders Sudden death	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Hepatobiliary disorders Cholecystitis acute	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	5.3% 2/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Hepatobiliary disorders Bile duct obstruction	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Hepatobiliary disorders Hyperbilirubinaemia	12.5% 1/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	0.00% 0/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Hepatobiliary disorders Jaundice	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Infections and infestations Catheter site infection	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Infections and infestations Cholecystitis infective	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Infections and infestations Clostridium difficile colitis	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Infections and infestations Emphysematous choleccystitis	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Infections and infestations Liver abscess	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Infections and infestations Penumonia	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Infections and infestations Sepsis	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Injury, poisoning and procedural complications Humerus fracture	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Investigations Blood alkaline phosphatase increased	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Investigations Blood bilirubin increased	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Investigations Transaminases increased	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Metabolism and nutrition disorders Dehydration	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Musculoskeletal and connective tissue disorders Back pain	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Musculoskeletal and connective tissue disorders Musucular weakness	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Pancreatic carcinoma	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Nervous system disorders Ischaemic stroke	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Vascular disorders Deep vein thrombosis	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Vascular disorders Embolism	12.5% 1/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	0.00% 0/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.
Vascular disorders Hypotension	0.00% 0/8 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.	2.6% 1/38 • Adverse event data were collected from Cycle 1 Day until Safety Follow Up (30 days after last dose). an average of 9 months.