Trial Outcomes & Findings for Efavirenz as Second-Line Therapy in Treating Patients With Metastatic Pancreatic Cancer (NCT NCT00964171)

Last Updated: 2025-08-28

Results Overview

Non-progression is defined as partial response, complete response or stable disease according to RECIST V1.0. Complete response is defined as the disappearance of all target lesions and partial response is defined as at least a 30% decrease in the sum of the longest diameters (SLD) of target lesions, taking as reference the baseline SLD (RECIST V1.0.).Stable disease is defined as no decrease or increase sufficient to qualify as partial response or progression with reference to SLD since the start of treatment. Radiologic assessment was carried out every 8 weeks.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

19 participants

Primary outcome timeframe

2 months

Results posted on

2025-08-28

Participant Flow

Participant milestones

Participant milestones
Measure	Efavirenz Efavirenz will be provided by the sponsor as follows: capsule of efavirenz 200 mg. A bottle contains 90 capsules. Investigator will dispense efavirenz to the patients according to the dose adjustment. Each patient will receive efavirenz 600 mg/day until morphological progression. Study drug will be taken every day by oral route at bedtime and in fast condition (1-2 hours far from dinner). Efavirenz 600mg
Overall Study STARTED	19
Overall Study COMPLETED	14
Overall Study NOT COMPLETED	5

Reasons for withdrawal

Reasons for withdrawal
Measure	Efavirenz Efavirenz will be provided by the sponsor as follows: capsule of efavirenz 200 mg. A bottle contains 90 capsules. Investigator will dispense efavirenz to the patients according to the dose adjustment. Each patient will receive efavirenz 600 mg/day until morphological progression. Study drug will be taken every day by oral route at bedtime and in fast condition (1-2 hours far from dinner). Efavirenz 600mg
Overall Study Death	5

Baseline Characteristics

Efavirenz as Second-Line Therapy in Treating Patients With Metastatic Pancreatic Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Efavirenz n=19 Participants Efavirenz will be provided by the sponsor as follows: capsule of efavirenz 200 mg. A bottle contains 90 capsules. Investigator will dispense efavirenz to the patients according to the dose adjustment. Each patient will receive efavirenz 600 mg/day until morphological progression. Study drug will be taken every day by oral route at bedtime and in fast condition (1-2 hours far from dinner). Efavirenz 600mg
Age, Continuous	56 years n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	13 Participants n=5 Participants
Region of Enrollment France	19 participants n=5 Participants

PRIMARY outcome

Timeframe: 2 months

Population: 5 patients deceased at 2 months

Outcome measures

Outcome measures
Measure	Efavirenz n=14 Participants Efavirenz will be provided by the sponsor as follows: capsule of efavirenz 200 mg. A bottle contains 90 capsules. Investigator will dispense efavirenz to the patients according to the dose adjustment. Each patient will receive efavirenz 600 mg/day until morphological progression. Study drug will be taken every day by oral route at bedtime and in fast condition (1-2 hours far from dinner). Efavirenz 600mg
Percentage of Patients in Non-progression at 2 Months	14.3 percentage of participants Interval 1.8 to 42.8

SECONDARY outcome

Timeframe: 4 months

Population: 8 patients deceased at 4 months

Outcome measures

Outcome measures
Measure	Efavirenz n=11 Participants Efavirenz will be provided by the sponsor as follows: capsule of efavirenz 200 mg. A bottle contains 90 capsules. Investigator will dispense efavirenz to the patients according to the dose adjustment. Each patient will receive efavirenz 600 mg/day until morphological progression. Study drug will be taken every day by oral route at bedtime and in fast condition (1-2 hours far from dinner). Efavirenz 600mg
Percentage of Patients in Non-progression at 4 Months	9.1 percentage of participants Interval 0.2 to 41.3

SECONDARY outcome

Timeframe: 2 months

Population: 5 patients deceased at 2 months

Non-biological progression was assessed using CA 19-9 every 28 days until end of treatment, and was defined as a 2-month CA19.9 concentration lower than 1.5 times the baseline CA 19-9 concentration.

Outcome measures

Outcome measures
Measure	Efavirenz n=14 Participants Efavirenz will be provided by the sponsor as follows: capsule of efavirenz 200 mg. A bottle contains 90 capsules. Investigator will dispense efavirenz to the patients according to the dose adjustment. Each patient will receive efavirenz 600 mg/day until morphological progression. Study drug will be taken every day by oral route at bedtime and in fast condition (1-2 hours far from dinner). Efavirenz 600mg
Percentage of Patients in Non-biological Progression at 2 Months	31.4 percentage of participants Interval 4.7 to 50.8

SECONDARY outcome

Timeframe: Through Database Cutoff Date of 06-July-2011 ( median follow-up time of 397 days)

OS was was defined as the time from the inclusion to death due to any cause. Participants without documented death were censored at the date of the last follow-up or last patient contact. The OS was calculated using the product-limit (Kaplan-Meier) method for censored data.

Outcome measures

Outcome measures
Measure	Efavirenz n=19 Participants Efavirenz will be provided by the sponsor as follows: capsule of efavirenz 200 mg. A bottle contains 90 capsules. Investigator will dispense efavirenz to the patients according to the dose adjustment. Each patient will receive efavirenz 600 mg/day until morphological progression. Study drug will be taken every day by oral route at bedtime and in fast condition (1-2 hours far from dinner). Efavirenz 600mg
Overall Survival	216 days Interval 59.0 to 390.0

SECONDARY outcome

Timeframe: Through Database Cutoff Date of 06-July-2011 ( median follow-up time of 397 days)

Event-free survival (EFS) was defined as the delay between trial inclusion and the first of the following events: progression (biological or morphological), death, treatment interruption due to toxicity, initiation of another treatment. Morphological 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. Biological progression is defined as a confirmed increase in CA 19-9 concentration to more than 1.5 times the baseline value, sustained over two consecutive monthly assessments. Patients without any of the aforementioned events at the time of the statistical analysis will be censored at the time they were last known to be event-free. The EFS was calculated using the product-limit (Kaplan-Meier) method for censored data.

Outcome measures

Outcome measures
Measure	Efavirenz n=19 Participants Efavirenz will be provided by the sponsor as follows: capsule of efavirenz 200 mg. A bottle contains 90 capsules. Investigator will dispense efavirenz to the patients according to the dose adjustment. Each patient will receive efavirenz 600 mg/day until morphological progression. Study drug will be taken every day by oral route at bedtime and in fast condition (1-2 hours far from dinner). Efavirenz 600mg
Event-free Survival	52 days Interval 22.0 to 56.0

SECONDARY outcome

Timeframe: Through Database Cutoff Date of 06-July-2011 ( median follow-up time of 397 days)

Progression was defined as morphological or biological progression. Progression-free survival was defined as the delay between trial inclusion and progression (morphological or biological) or death for any reason. Morphological 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. Biological progression is defined as a confirmed increase in CA 19-9 concentration to more than 1.5 times the baseline value, sustained over two consecutive monthly assessments. Patients who have not progressed nor died at the time of the statistical analysis will be censored at the time they were last known to be progression-free. The EFS was calculated using the product-limit (Kaplan-Meier) method for censored data.

Outcome measures

Outcome measures
Measure	Efavirenz n=19 Participants Efavirenz will be provided by the sponsor as follows: capsule of efavirenz 200 mg. A bottle contains 90 capsules. Investigator will dispense efavirenz to the patients according to the dose adjustment. Each patient will receive efavirenz 600 mg/day until morphological progression. Study drug will be taken every day by oral route at bedtime and in fast condition (1-2 hours far from dinner). Efavirenz 600mg
Progression-free Survival	54 days Interval 23.0 to 56.0

SECONDARY outcome

Timeframe: 2 months

EORTC-QLQ-C30 is a cancer-specific instrument with 30 questions to evaluate quality of life. The first 28 use a 4-point scale (1=not at all, 2=a little, 3=quite a bit, 4=very much) assessing 5 functional scales (physical, role, emotional, cognitive, social), 3 symptom scales (fatigue, nausea/vomiting, pain), six single symptoms (dyspnea, insomnia, appetite loss, constipation, diarrhea, financial difficulties), and two global health/QoL questions. Items 29 and 30 use a 7-point scale (1=very poor to 7=excellent).Scores are linearly transformed from 0 to 100 per EORTC guidelines. Higher scores mean better functioning or QoL for functional/global scales, but worse symptoms for symptom scales and items. Score ranges (0-100): higher Functional scores = better QoL; higher Symptom scores= worse QoL; Higher Global health score= better QoL.

Outcome measures

Outcome measures
Measure	Efavirenz n=19 Participants Efavirenz will be provided by the sponsor as follows: capsule of efavirenz 200 mg. A bottle contains 90 capsules. Investigator will dispense efavirenz to the patients according to the dose adjustment. Each patient will receive efavirenz 600 mg/day until morphological progression. Study drug will be taken every day by oral route at bedtime and in fast condition (1-2 hours far from dinner). Efavirenz 600mg
Quality of Life Score Physical functionning score	73.3 score on a scale Interval 53.3 to 100.0
Quality of Life Score Role functionning score	75 score on a scale Interval 50.0 to 100.0
Quality of Life Score Cognitive Functioning score	75 score on a scale Interval 66.7 to 100.0
Quality of Life Score Social Functioning score	75 score on a scale Interval 50.0 to 100.0
Quality of Life Score Global health status score	58.3 score on a scale Interval 50.0 to 83.3
Quality of Life Score Fatigue score	44.4 score on a scale Interval 22.2 to 100.0
Quality of Life Score Nausea / Vomiting score	0 score on a scale Interval 0.0 to 16.7
Quality of Life Score Dyspnoea score	33.3 score on a scale Interval 0.0 to 33.3
Quality of Life Score Insomnia score	33.3 score on a scale Interval 0.0 to 66.7
Quality of Life Score Appetite loss score	16.7 score on a scale Interval 0.0 to 66.7
Quality of Life Score Constipation score	0 score on a scale Interval 0.0 to 66.7
Quality of Life Score Diarhoea score	16.7 score on a scale Interval 0.0 to 66.7
Quality of Life Score Financial Problems score	0 score on a scale Interval 0.0 to 33.3
Quality of Life Score Emotional Functioning score	83.3 score on a scale Interval 66.7 to 83.3
Quality of Life Score Pain score	41.7 score on a scale Interval 0.0 to 66.7

Adverse Events

Efavirenz

Serious events: 12 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Efavirenz n=19 participants at risk Efavirenz will be provided by the sponsor as follows: capsule of efavirenz 200 mg. A bottle contains 90 capsules. Investigator will dispense efavirenz to the patients according to the dose adjustment. Each patient will receive efavirenz 600 mg/day until morphological progression. Study drug will be taken every day by oral route at bedtime and in fast condition (1-2 hours far from dinner). Efavirenz 600mg
Respiratory, thoracic and mediastinal disorders Respiratory distress	5.3% 1/19 • Number of events 1 Only serious adverse events were monitored. Adverse events (non-serious) were not assessed/monitored during the study.
Gastrointestinal disorders Vomiting	5.3% 1/19 • Number of events 1 Only serious adverse events were monitored. Adverse events (non-serious) were not assessed/monitored during the study.
Gastrointestinal disorders Abdominal pain	5.3% 1/19 • Number of events 1 Only serious adverse events were monitored. Adverse events (non-serious) were not assessed/monitored during the study.
Infections and infestations Kidney infection	5.3% 1/19 • Number of events 1 Only serious adverse events were monitored. Adverse events (non-serious) were not assessed/monitored during the study.
General disorders Disease progression	10.5% 2/19 • Number of events 2 Only serious adverse events were monitored. Adverse events (non-serious) were not assessed/monitored during the study.
Cardiac disorders Pericardial constriction	5.3% 1/19 • Number of events 1 Only serious adverse events were monitored. Adverse events (non-serious) were not assessed/monitored during the study.
Psychiatric disorders Suicidal ideation	5.3% 1/19 • Number of events 1 Only serious adverse events were monitored. Adverse events (non-serious) were not assessed/monitored during the study.
Psychiatric disorders Euphoria	5.3% 1/19 • Number of events 1 Only serious adverse events were monitored. Adverse events (non-serious) were not assessed/monitored during the study.
Respiratory, thoracic and mediastinal disorders Lung infection	5.3% 1/19 • Number of events 1 Only serious adverse events were monitored. Adverse events (non-serious) were not assessed/monitored during the study.
Hepatobiliary disorders Biliary stent blockage	5.3% 1/19 • Number of events 1 Only serious adverse events were monitored. Adverse events (non-serious) were not assessed/monitored during the study.
Gastrointestinal disorders Colonic obstruction	5.3% 1/19 • Number of events 1 Only serious adverse events were monitored. Adverse events (non-serious) were not assessed/monitored during the study.
Vascular disorders Thromboembolic event	5.3% 1/19 • Number of events 1 Only serious adverse events were monitored. Adverse events (non-serious) were not assessed/monitored during the study.
Gastrointestinal disorders Esophageal varices hemorrhage	5.3% 1/19 • Number of events 1 Only serious adverse events were monitored. Adverse events (non-serious) were not assessed/monitored during the study.

Other adverse events

Adverse event data not reported

Additional Information

Dr Marianne Fonck

Institut Bergonié

Phone: 05.56.33.32.42

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place