Trial Outcomes & Findings for Maintenance Rucaparib in BRCA1, BRCA2 or PALB2 Mutated Pancreatic Cancer That Has Not Progressed on Platinum-based Therapy (NCT NCT03140670)

Last Updated: 2023-09-28

Results Overview

Time from initiation of rucaparib until progression or death from any cause. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions. Only if absolute increase is equal to or greater than 5mm.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

46 participants

Primary outcome timeframe

6 months

Results posted on

2023-09-28

Participant Flow

This trial was performed at the Abramson Cancer Center at the University of Pennsylvania. Patients were enrolled from September 2017 through October 2019.

Of 46 enrolled patients, 42 patients were evaluable for efficacy analysis. Thus, the enrollment goal was met, per the Protocol.

Participant milestones

Participant milestones
Measure	Single Arm RUCAPARIB: Rucaparib was used as a maintenance therapy for eligible PDAC patients who received at least 16 weeks of platinum-based chemotherapy without evidence of platinum resistance. Chemotherapy was discontinued and patients received rucaparib 600 mg orally twice a day until progression.
Overall Study STARTED	46
Overall Study COMPLETED	42
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Reasons for withdrawal
Measure	Single Arm RUCAPARIB: Rucaparib was used as a maintenance therapy for eligible PDAC patients who received at least 16 weeks of platinum-based chemotherapy without evidence of platinum resistance. Chemotherapy was discontinued and patients received rucaparib 600 mg orally twice a day until progression.
Overall Study Withdrawal by Subject	2
Overall Study Lost to Follow-up	1
Overall Study Physician Decision	1

Baseline Characteristics

Maintenance Rucaparib in BRCA1, BRCA2 or PALB2 Mutated Pancreatic Cancer That Has Not Progressed on Platinum-based Therapy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Single Arm n=42 Participants RUCAPARIB: Rucaparib is a PARP inhibitor used as an anti-cancer agent. Rucaparib is a first-in-class pharmaceutical drug targeting the DNA repair enzyme poly-ADP ribose polymerase-1.
Age, Continuous	61.5 years n=5 Participants
Sex: Female, Male Female	24 Participants n=5 Participants
Sex: Female, Male Male	18 Participants n=5 Participants
Race/Ethnicity, Customized White Non-Hispanic	40 Participants n=5 Participants
Race/Ethnicity, Customized Black or African American	2 Participants n=5 Participants
Race/Ethnicity, Customized Asian	0 Participants n=5 Participants
Race/Ethnicity, Customized Hispanic or Latino	3 Participants n=5 Participants
Mutations at baseline: gBRCA2, gBRCA1, gPALB2, sBRCA2. Germline BRCA1	7 Participants n=5 Participants
Mutations at baseline: gBRCA2, gBRCA1, gPALB2, sBRCA2. Germline BRCA2	27 Participants n=5 Participants
Mutations at baseline: gBRCA2, gBRCA1, gPALB2, sBRCA2. Germline PALB2	6 Participants n=5 Participants
Mutations at baseline: gBRCA2, gBRCA1, gPALB2, sBRCA2. Somatic BRCA2	2 Participants n=5 Participants
Tumor histology Adenocarcinoma	40 Participants n=5 Participants
Tumor histology Acinar	1 Participants n=5 Participants
Tumor histology Squamous cell carcinoma	1 Participants n=5 Participants
Disease stage at study start Metastatic	40 Participants n=5 Participants
Disease stage at study start Locally advanced	2 Participants n=5 Participants
Platinum treatment duration 4-6 months	26 Participants n=5 Participants
Platinum treatment duration 6-12 months	5 Participants n=5 Participants
Platinum treatment duration More than 12 months	3 Participants n=5 Participants
Platinum treatment duration Less than 4 months	8 Participants n=5 Participants

PRIMARY outcome

Timeframe: 6 months

Outcome measures

Outcome measures
Measure	Single Arm n=42 Participants RUCAPARIB: Rucaparib was used as a maintenance therapy for eligible PDAC patients who received at least 16 weeks of platinum-based chemotherapy without evidence of platinum resistance. Chemotherapy was discontinued and patients received rucaparib 600 mg orally twice a day until progression.
Progression-Free Survival (PFS) at 6 Months (PFS6)	59.5 Percentage of participants Interval 44.6 to 74.4

SECONDARY outcome

Timeframe: 24 months

Time from initiation of rucaparib until death or last follow-up

Outcome measures

Outcome measures
Measure	Single Arm n=42 Participants RUCAPARIB: Rucaparib was used as a maintenance therapy for eligible PDAC patients who received at least 16 weeks of platinum-based chemotherapy without evidence of platinum resistance. Chemotherapy was discontinued and patients received rucaparib 600 mg orally twice a day until progression.
Overall Survival	23.5 Months Interval 20.0 to 27.0

SECONDARY outcome

Timeframe: 24 months

Population: Evaluable patients with measurable disease

Confirmed Complete Response or Partial Response according to RECIST v1.1. Complete Response (CR) is defined as tumor burden reduced to 0.0 mm or lymph node lesions are smaller than 10mm. Partial Response (PR), tumor burden decreased by greater than 30% but not CR. Overall Response Rate (ORR) is defined as confirmed CR or PR.

Outcome measures

Outcome measures
Measure	Single Arm n=36 Participants RUCAPARIB: Rucaparib was used as a maintenance therapy for eligible PDAC patients who received at least 16 weeks of platinum-based chemotherapy without evidence of platinum resistance. Chemotherapy was discontinued and patients received rucaparib 600 mg orally twice a day until progression.
Overall Response Rate (ORR)	41.7 Percentage of participants Interval 25.5 to 59.2

SECONDARY outcome

Timeframe: 24 months

Population: Evaluable patients with measurable disease

Confirmed complete response, partial response, or stable disease lasting for at least 16 weeks

Outcome measures

Outcome measures
Measure	Single Arm n=36 Participants RUCAPARIB: Rucaparib was used as a maintenance therapy for eligible PDAC patients who received at least 16 weeks of platinum-based chemotherapy without evidence of platinum resistance. Chemotherapy was discontinued and patients received rucaparib 600 mg orally twice a day until progression.
Disease Control Rate (DCR)	66.7 Percentage of evaluable patients Interval 49.0 to 81.4

SECONDARY outcome

Timeframe: 24 months

Population: Evaluable patients with measurable disease

Time from initial response to progression or death from any cause

Outcome measures

Outcome measures
Measure	Single Arm n=36 Participants RUCAPARIB: Rucaparib was used as a maintenance therapy for eligible PDAC patients who received at least 16 weeks of platinum-based chemotherapy without evidence of platinum resistance. Chemotherapy was discontinued and patients received rucaparib 600 mg orally twice a day until progression.
Duration of Response (DOR)	17.3 Months Interval 8.8 to 25.8

SECONDARY outcome

Timeframe: 24 months

Toxicity of rucaparib as maintenance therapy was assessed by examining Adverse Events (AEs) that were at least possibly related to the drug treatment. AEs were classified and graded according to the NCI Common Terminology Criteria of Adverse Events, version 4.1.

Outcome measures

Outcome measures
Measure	Single Arm n=46 Participants RUCAPARIB: Rucaparib was used as a maintenance therapy for eligible PDAC patients who received at least 16 weeks of platinum-based chemotherapy without evidence of platinum resistance. Chemotherapy was discontinued and patients received rucaparib 600 mg orally twice a day until progression.
Toxicity at Least Possibly Related to Rucaparib Thrombocytopenia	39 Percentage of participants
Toxicity at Least Possibly Related to Rucaparib Anemia	74 Percentage of participants
Toxicity at Least Possibly Related to Rucaparib Nausea	48 Percentage of participants
Toxicity at Least Possibly Related to Rucaparib Increased ALT	47 Percentage of participants
Toxicity at Least Possibly Related to Rucaparib Fatigue	45 Percentage of participants
Toxicity at Least Possibly Related to Rucaparib Dysgeusia	37 Percentage of participants

Adverse Events

Single Arm

Serious events: 1 serious events

Other events: 0 other events

Deaths: 33 deaths

Serious adverse events

Serious adverse events
Measure	Single Arm n=42 participants at risk RUCAPARIB: Rucaparib was used as a maintenance therapy for eligible PDAC patients who received at least 16 weeks of platinum-based chemotherapy without evidence of platinum resistance. Chemotherapy was discontinued and patients received rucaparib 600 mg orally twice a day until progression.
Hepatobiliary disorders Hepatic dysfunction	2.4% 1/42 • 24 months Adverse events (AEs) were classified and graded according to the NCI Common Terminology Criteria of Adverse Events, version 4.1.

Other adverse events

Adverse event data not reported

Additional Information

Pancreas Research Program Manager

Abramson Cancer Center

Phone: 2672575567

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place