Trial Outcomes & Findings for Re-challenge Pembrolizumab Study as a Second or Further Line in Patients With Advanced NSCLC (NCT NCT03526887)

NCT ID: NCT03526887

Last Updated: 2025-03-24

Results Overview

Overall survival: Defined as the length of time from either the date of diagnosis or the start of the treatment that patients diagnosed with the disease are still alive.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 73 participants
• Primary outcome timeframe: From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 48 months.
• Results posted on: 2025-03-24

Participant Flow

Participant milestones

Measure	Cohort 1 Patients who experienced progression disease while on treatment progression disease \< 12 weeks after stopping treatment. After that the patients took chemotherapy ≥ 4 cycles and progressed again. After the last progression the patient is included in the study to be retreated with Pembrolizumab 200mg Pembrolizumab: 200 mg, Q3W	Cohort 2 Stop treatment and progression \> 12 weeks after stopping treatment. After the last progression the patient is included in the study to be retreated with Pembrolizumab 200mg Pembrolizumab: 200 mg, Q3W
Overall Study STARTED	55	18
Overall Study COMPLETED	55	18
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Re-challenge Pembrolizumab Study as a Second or Further Line in Patients With Advanced NSCLC

Baseline characteristics by cohort

Measure	Cohort 1 n=55 Participants Patients who experienced progression disease while on treatment progression disease \< 12 weeks after stopping treatment. After that the patients took chemotherapy ≥ 4 cycles and progressed again. After the last progression the patient is included in the study to be retreated with Pembrolizumab 200mg Pembrolizumab: 200 mg, Q3W	Cohort 2 n=18 Participants Stop treatment and progression \> 12 weeks after stopping treatment. After the last progression the patient is included in the study to be retreated with Pembrolizumab 200mg Pembrolizumab: 200 mg, Q3W	Total n=73 Participants Total of all reporting groups
Age, Continuous	63.5 years n=5 Participants	69.8 years n=7 Participants	65 years n=5 Participants
Sex: Female, Male Female	39 Participants n=5 Participants	15 Participants n=7 Participants	54 Participants n=5 Participants
Sex: Female, Male Male	16 Participants n=5 Participants	3 Participants n=7 Participants	19 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	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	53 Participants n=5 Participants	18 Participants n=7 Participants	71 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	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Region of Enrollment Spain	55 participants n=5 Participants	18 participants n=7 Participants	73 participants n=5 Participants
Performance Status ECOG 0	15 Participants n=5 Participants	8 Participants n=7 Participants	23 Participants n=5 Participants
Performance Status ECOG 1	39 Participants n=5 Participants	10 Participants n=7 Participants	49 Participants n=5 Participants
Performance Status ECOG 2	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Performance Status ECOG 3	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Performance Status ECOG4	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Performance Status Not recorded	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Cigarette Smoking History Never smoker	7 Participants n=5 Participants	1 Participants n=7 Participants	8 Participants n=5 Participants
Cigarette Smoking History Former smoker	39 Participants n=5 Participants	15 Participants n=7 Participants	54 Participants n=5 Participants
Cigarette Smoking History Smoker	8 Participants n=5 Participants	2 Participants n=7 Participants	10 Participants n=5 Participants
Cigarette Smoking History Not recorded	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Body Mass Index	26 kg/m^2 n=5 Participants	28 kg/m^2 n=7 Participants	26.5 kg/m^2 n=5 Participants
Histology Adenocarcinoma	32 Participants n=5 Participants	8 Participants n=7 Participants	40 Participants n=5 Participants
Histology Squamous	19 Participants n=5 Participants	8 Participants n=7 Participants	27 Participants n=5 Participants
Histology Adeno-squamous	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Histology Large Cell Carcinoma	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Histology NOS/Undifferentiated	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Histology Other	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Clinical Stage Stage I	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Clinical Stage Stage II	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Clinical Stage Stage III	3 Participants n=5 Participants	1 Participants n=7 Participants	4 Participants n=5 Participants
Clinical Stage Stage IV	52 Participants n=5 Participants	17 Participants n=7 Participants	69 Participants n=5 Participants

PRIMARY outcome

Timeframe: From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 48 months.

Overall survival: Defined as the length of time from either the date of diagnosis or the start of the treatment that patients diagnosed with the disease are still alive.

Outcome measures

Measure	Cohort 1 n=55 Participants Patients who experienced progression disease while on treatment progression disease \< 12 weeks after stopping treatment. After that the patients took chemotherapy ≥ 4 cycles and progressed again. After the last progression the patient is included in the study to be retreated with Pembrolizumab 200mg Pembrolizumab: 200 mg, Q3W	Cohort 2 n=18 Participants Stop treatment and progression \> 12 weeks after stopping treatment. After the last progression the patient is included in the study to be retreated with Pembrolizumab 200mg Pembrolizumab: 200 mg, Q3W
Efficacy of Pembrolizumab Re-challenge Measured by Overall Survival	9.4 months Interval 1.0 to 18.0	19.1 months Interval 3.0 to 48.0

SECONDARY outcome

Timeframe: From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 36 months.

PFS: Defined as the length of time from the date of randomization to the date of the first documented progression of disease. "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"

Outcome measures

Measure	Cohort 1 n=55 Participants Patients who experienced progression disease while on treatment progression disease \< 12 weeks after stopping treatment. After that the patients took chemotherapy ≥ 4 cycles and progressed again. After the last progression the patient is included in the study to be retreated with Pembrolizumab 200mg Pembrolizumab: 200 mg, Q3W	Cohort 2 n=18 Participants Stop treatment and progression \> 12 weeks after stopping treatment. After the last progression the patient is included in the study to be retreated with Pembrolizumab 200mg Pembrolizumab: 200 mg, Q3W
Efficacy of Pembrolizumab Re-challenge Measured by Progression Free Survival (PFS) Per RECIST v1.1.	1.6 months Interval 0.0 to 11.0	4.1 months Interval 0.2 to 18.0

SECONDARY outcome

Timeframe: From the date of randomization until end of follow up, up to 36 months

To evaluate the best global response of the treatment as measured by investigator-assessed overall response rate (ORR) according to RECIST v1.1. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), >=30% decrease in the sum of the longest diameter of target lesions; Progression is defined 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.

Outcome measures

Measure	Cohort 1 n=55 Participants Patients who experienced progression disease while on treatment progression disease \< 12 weeks after stopping treatment. After that the patients took chemotherapy ≥ 4 cycles and progressed again. After the last progression the patient is included in the study to be retreated with Pembrolizumab 200mg Pembrolizumab: 200 mg, Q3W	Cohort 2 n=18 Participants Stop treatment and progression \> 12 weeks after stopping treatment. After the last progression the patient is included in the study to be retreated with Pembrolizumab 200mg Pembrolizumab: 200 mg, Q3W
Best Global Response Complete response	0 Participants	0 Participants
Best Global Response Partial response	1 Participants	3 Participants
Best Global Response Stable disease	24 Participants	11 Participants
Best Global Response Progression disease	25 Participants	3 Participants
Best Global Response Not evaluable	5 Participants	1 Participants

Adverse Events

Cohort 1

Serious events: 2 serious events

Other events: 29 other events

Deaths: 44 deaths

Cohort 2

Serious events: 1 serious events

Other events: 11 other events

Deaths: 5 deaths

Serious adverse events

Measure	Cohort 1 n=55 participants at risk Patients who experienced progression disease while on treatment progression disease \< 12 weeks after stopping treatment. After that the patients took chemotherapy ≥ 4 cycles and progressed again. After the last progression the patient is included in the study to be retreated with Pembrolizumab 200mg Pembrolizumab: 200 mg, Q3W	Cohort 2 n=18 participants at risk Stop treatment and progression \> 12 weeks after stopping treatment. After the last progression the patient is included in the study to be retreated with Pembrolizumab 200mg Pembrolizumab: 200 mg, Q3W
Gastrointestinal disorders Gastrointestinal disorder	0.00% 0/55 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.	5.6% 1/18 • Number of events 1 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.
Blood and lymphatic system disorders Alkaline phosphatase increased	1.8% 1/55 • Number of events 1 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.	0.00% 0/18 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.
Blood and lymphatic system disorders Blood bilirubin increased	1.8% 1/55 • Number of events 1 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.	0.00% 0/18 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.

Other adverse events

Measure	Cohort 1 n=55 participants at risk Patients who experienced progression disease while on treatment progression disease \< 12 weeks after stopping treatment. After that the patients took chemotherapy ≥ 4 cycles and progressed again. After the last progression the patient is included in the study to be retreated with Pembrolizumab 200mg Pembrolizumab: 200 mg, Q3W	Cohort 2 n=18 participants at risk Stop treatment and progression \> 12 weeks after stopping treatment. After the last progression the patient is included in the study to be retreated with Pembrolizumab 200mg Pembrolizumab: 200 mg, Q3W
Gastrointestinal disorders Diarrhea	14.5% 8/55 • Number of events 8 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.	5.6% 1/18 • Number of events 1 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.
Gastrointestinal disorders Nausea	7.3% 4/55 • Number of events 4 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.	5.6% 1/18 • Number of events 1 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.
Gastrointestinal disorders Stomach pain	5.5% 3/55 • Number of events 3 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.	0.00% 0/18 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.
General disorders Fatigue	12.7% 7/55 • Number of events 7 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.	22.2% 4/18 • Number of events 4 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.
Blood and lymphatic system disorders Alanine aminotransferase increased	5.5% 3/55 • Number of events 3 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.	5.6% 1/18 • Number of events 1 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.
Investigations Aspartate aminotransferase increased	5.5% 3/55 • Number of events 3 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.	11.1% 2/18 • Number of events 2 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.
Investigations Blood bilirubin increased	5.5% 3/55 • Number of events 3 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.	5.6% 1/18 • Number of events 1 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.
Metabolism and nutrition disorders Anorexia	5.5% 3/55 • Number of events 3 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.	0.00% 0/18 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.
Musculoskeletal and connective tissue disorders Arthralgia	5.5% 3/55 • Number of events 3 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.	5.6% 1/18 • Number of events 1 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.
Skin and subcutaneous tissue disorders Pruritus	9.1% 5/55 • Number of events 5 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.	16.7% 3/18 • Number of events 3 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.
Skin and subcutaneous tissue disorders Rash	5.5% 3/55 • Number of events 3 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.	5.6% 1/18 • Number of events 1 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.
Metabolism and nutrition disorders Hypomagnesemia	5.5% 3/55 • Number of events 3 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.	0.00% 0/18 • Adverse Events were assessed an average of 30 months. Deaths were assessed up to 48 months. Any adverse event or breakdown occurring during the course of the study. The investigator will have to collect all adverse events once they have signed informed consent, during treatment and 30 days after the last dose study treatment administration.

Additional Information

Eva Pereira

Fundación GECP

Phone: +34934302006

Email: gecp@gecp.org

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place