Trial Outcomes & Findings for Gamma Secretase Inhibitor RO4929097 in Previously Treated Metastatic Pancreas Cancer (NCT NCT01232829)

Last Updated: 2014-11-26

Results Overview

The primary endpoint of the study was 6-month survival. The proportion of successes was estimated by the number of successes divided by the total number of evaluable patients.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

18 participants

Primary outcome timeframe

6 months

Results posted on

2014-11-26

Participant Flow

This was a phase II, single-arm study. From December 2010 to May 2012, a total of eighteen patients were enrolled at the University of Colorado Cancer Center and Johns Hopkins Hospital.

Subjects were eligible if they were at least 18 years old, had a Karnofsky Performance Status of ≥70, at least one previous chemotherapy for metastatic disease, histologically or cytologically metastatic adenocarcinoma of the pancreas, and measurable disease. Patients with islet cell neoplasms and locally advanced disease were excluded.

Participant milestones

Participant milestones
Measure	Treatment (RO4929097) RO4929097 was administered at a dose of 20 mg daily on days 1-3, 8-10 and 15-17 of 21-day cycles.
Overall Study STARTED	18
Overall Study COMPLETED	18
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Gamma Secretase Inhibitor RO4929097 in Previously Treated Metastatic Pancreas Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (RO4929097) n=18 Participants RO4929097 was administered at a dose of 20 mg daily on days 1-3, 8-10 and 15-17 of 21-day cycles.
Age, Continuous	65.0 Years STANDARD_DEVIATION 11.9 • n=93 Participants
Sex: Female, Male Female	10 Participants n=93 Participants
Sex: Female, Male Male	8 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants
Race (NIH/OMB) Black or African American	1 Participants n=93 Participants
Race (NIH/OMB) White	17 Participants n=93 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=93 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	16 Participants n=93 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=93 Participants
Region of Enrollment United States	18 participants n=93 Participants

PRIMARY outcome

Timeframe: 6 months

Population: All patients meeting the eligibility criteria and who received treatment were considered evaluable for the primary endpoint. All patients treated per protocol (n=18) were evaluable for the 6-month survival endpoint.

The primary endpoint of the study was 6-month survival. The proportion of successes was estimated by the number of successes divided by the total number of evaluable patients.

Outcome measures

Outcome measures
Measure	Treatment (RO4929097) n=18 Participants RO4929097 was administered at a dose of 20 mg daily on days 1-3, 8-10 and 15-17 of 21-day cycles.
Survival Rate	5 participants

SECONDARY outcome

Timeframe: From registration to death due to any cause, assessed up to 2 years

Population: All patients meeting the eligibility criteria and who received treatment per protocol (n=18) were evaluable for the overall survival endpoint.

Survival was estimated using the Kaplan-Meier (1958) method.

Outcome measures

Outcome measures
Measure	Treatment (RO4929097) n=18 Participants RO4929097 was administered at a dose of 20 mg daily on days 1-3, 8-10 and 15-17 of 21-day cycles.
Survival	4.1 months Interval 2.7 to 5.8

SECONDARY outcome

Timeframe: From registration to documentation of disease progression, assessed up to 2 years

Population: All patients meeting the eligibility criteria and who received treatment per protocol (n=18) were evaluable for the progression-free survival.

Eighteen patients were evaluable for the time to disease progression endpoint.

Outcome measures

Outcome measures
Measure	Treatment (RO4929097) n=18 Participants RO4929097 was administered at a dose of 20 mg daily on days 1-3, 8-10 and 15-17 of 21-day cycles.
Time to Disease Progression	1.5 months Interval 1.3 to 1.6

Adverse Events

Treatment (RO4929097)

Serious events: 1 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Treatment (RO4929097) n=18 participants at risk RO4929097 was administered at a dose of 20 mg daily on days 1-3, 8-10 and 15-17 of 21-day cycles.
Psychiatric disorders Confusion	5.6% 1/18 • Adverse events were assessed within 21 days of receiving the study drug. Within the 21 days of receiving the study drug, routine blood tests and physical examinations were done.

Other adverse events

Other adverse events
Measure	Treatment (RO4929097) n=18 participants at risk RO4929097 was administered at a dose of 20 mg daily on days 1-3, 8-10 and 15-17 of 21-day cycles.
Metabolism and nutrition disorders Hyperglycemia	11.1% 2/18 • Adverse events were assessed within 21 days of receiving the study drug. Within the 21 days of receiving the study drug, routine blood tests and physical examinations were done.
General disorders Fatigue	11.1% 2/18 • Adverse events were assessed within 21 days of receiving the study drug. Within the 21 days of receiving the study drug, routine blood tests and physical examinations were done.
Nervous system disorders Dysgeusia	5.6% 1/18 • Adverse events were assessed within 21 days of receiving the study drug. Within the 21 days of receiving the study drug, routine blood tests and physical examinations were done.
Investigations Lymphocyte Count Decreased	11.1% 2/18 • Adverse events were assessed within 21 days of receiving the study drug. Within the 21 days of receiving the study drug, routine blood tests and physical examinations were done.
Metabolism and nutrition disorders Hypophosphatemia	5.6% 1/18 • Adverse events were assessed within 21 days of receiving the study drug. Within the 21 days of receiving the study drug, routine blood tests and physical examinations were done.

Additional Information

Dr. Wells Messersmith

University of Colorado Cancer Center

Phone: 303-724-3808

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60