Trial Outcomes & Findings for Pembrolizumab in Treating Patients With Recurrent Glioblastoma (NCT NCT02337686)

Last Updated: 2025-08-15

Results Overview

Progression-free survival, defined as the time from study enrollment until the time of first occurrence of disease progression, relapse, or death due to disease. Patients who are alive without progression or relapse will be censored at the time of last contact. Response Assessment in Neuro-Oncology (RANO) Criteria for Evaluating Response.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE2

Target enrollment

18 participants

Primary outcome timeframe

At 6 months

Results posted on

2025-08-15

Participant Flow

All participants recruited at MD Anderson Cancer Center.

A total of 18 participants were consented; 3 screen failures.

Participant milestones

Participant milestones
Measure	Treatment (Pembrolizumab, Surgery) 200 mg, intravenously (IV) once every 3-weeks prior to surgery for two doses and then restarting 200 mg Q 2-wk following surgical resection
Overall Study STARTED	15
Overall Study COMPLETED	14
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Treatment (Pembrolizumab, Surgery) 200 mg, intravenously (IV) once every 3-weeks prior to surgery for two doses and then restarting 200 mg Q 2-wk following surgical resection
Overall Study Adverse Event	1

Baseline Characteristics

Pembrolizumab in Treating Patients With Recurrent Glioblastoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Pembrolizumab, Surgery) n=15 Participants 200 mg, intravenously (IV) once every 3-weeks prior to surgery for two doses and then restarting 200 mg Q 2-wk following surgical resection
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	13 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants
Sex: Female, Male Female	9 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	14 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Region of Enrollment United States	15 participants n=5 Participants

PRIMARY outcome

Timeframe: At 6 months

Outcome measures

Outcome measures
Measure	Treatment (Pembrolizumab, Surgery) n=15 Participants 200 mg, intravenously (IV) once every 3-weeks prior to surgery for two doses and then restarting 200 mg Q 2-wk following surgical resection
Number of Participants With Progression Free Survival at 6 Months	15 Participants

SECONDARY outcome

Timeframe: The time from study enrollment until the time of first occurrence of disease progression, relapse, or death due to disease, assessed up to 2 years

Defined as the length of time from the date of diagnosis or the start of treatment for a disease until the disease starts to get worse or spread to other parts of the body. Time to Progression measured by the method of Kaplan and Meier.

Outcome measures

Outcome measures
Measure	Treatment (Pembrolizumab, Surgery) n=15 Participants 200 mg, intravenously (IV) once every 3-weeks prior to surgery for two doses and then restarting 200 mg Q 2-wk following surgical resection
Number of Participants With Progression	10 Participants

SECONDARY outcome

Timeframe: Up to 2 years

Overall survival time is defined as the time from registration to death due to any cause. The distribution of survival time will be estimated using the method of Kaplan-Meier.

Outcome measures

Outcome measures
Measure	Treatment (Pembrolizumab, Surgery) n=15 Participants 200 mg, intravenously (IV) once every 3-weeks prior to surgery for two doses and then restarting 200 mg Q 2-wk following surgical resection
Overall Survival	20 months Interval 8.64 to 28.45

SECONDARY outcome

Timeframe: Up to 2 years

Logistic regression will be utilized to assess the effect of patient prognostic factors on the response rate. Per the Response Assessment in Neuro-Oncology (RANO) Criteria for Evaluating Response for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=50% decrease in the sum of the products of perpendicular diameter of measurable lesions; Overall Response (OR) = CR + PR.

Outcome measures

Outcome measures
Measure	Treatment (Pembrolizumab, Surgery) n=14 Participants 200 mg, intravenously (IV) once every 3-weeks prior to surgery for two doses and then restarting 200 mg Q 2-wk following surgical resection
Number of Participants With Response Rate	3 Participants

SECONDARY outcome

Timeframe: Up to 30 days after completion of study treatment

Adverse events as defined by the Common Terminology Criteria for Adverse Events (CTCAE) version 4 for grades 1, 2, and 3. There were no grade 4 AEs.

Outcome measures

Outcome measures
Measure	Treatment (Pembrolizumab, Surgery) n=15 Participants 200 mg, intravenously (IV) once every 3-weeks prior to surgery for two doses and then restarting 200 mg Q 2-wk following surgical resection
Number of Adverse Events Adverse Event Grade 1	31 number of adverse events
Number of Adverse Events Adverse Event Grade 2	18 number of adverse events
Number of Adverse Events Adverse Event Grade 3	6 number of adverse events

Adverse Events

Treatment (Pembrolizumab, Surgery)

Serious events: 5 serious events

Other events: 12 other events

Deaths: 14 deaths

Serious adverse events

Serious adverse events
Measure	Treatment (Pembrolizumab, Surgery) n=15 participants at risk 200 mg, intravenously (IV) once every 3-weeks prior to surgery for two doses and then restarting 200 mg Q 2-wk following surgical resection
Nervous system disorders Hydrocephalus	33.3% 5/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Musculoskeletal and connective tissue disorders Muscle weakness left-sided	20.0% 3/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Nervous system disorders Edema cerebral	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Nervous system disorders Wound dehiscence	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign malignant and unspecified (incl and polyps (other)	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Infections and infestations Enterocolitis infections	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.

Other adverse events

Other adverse events
Measure	Treatment (Pembrolizumab, Surgery) n=15 participants at risk 200 mg, intravenously (IV) once every 3-weeks prior to surgery for two doses and then restarting 200 mg Q 2-wk following surgical resection
General disorders Fatigue	13.3% 2/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Musculoskeletal and connective tissue disorders Gait disturbance	26.7% 4/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Nervous system disorders Dysphasia	33.3% 5/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Nervous system disorders Headache	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Nervous system disorders Seizure	13.3% 2/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Gastrointestinal disorders Diarrhea	20.0% 3/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Nervous system disorders Dizziness	13.3% 2/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Gastrointestinal disorders Nausea	33.3% 5/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Renal and urinary disorders Urinary tract Infection	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Infections and infestations Edema face	13.3% 2/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Musculoskeletal and connective tissue disorders Pain Extremity	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Infections and infestations Lung Infection	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Ear and labyrinth disorders Otitis Media	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Musculoskeletal and connective tissue disorders Tremor	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Vascular disorders Hypotension	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
General disorders Chill	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Psychiatric disorders Insomia	13.3% 2/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Gastrointestinal disorders Anorexia	13.3% 2/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Skin and subcutaneous tissue disorders Pruritius	13.3% 2/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Musculoskeletal and connective tissue disorders General muscle weakness	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Nervous system disorders Paresthesia	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Musculoskeletal and connective tissue disorders Ataxia	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Nervous system disorders Cognitive disorder	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Musculoskeletal and connective tissue disorders Arthralgia	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Psychiatric disorders Confusion	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Skin and subcutaneous tissue disorders Dry Skin	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
General disorders Fever	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Vascular disorders Flushing	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Gastrointestinal disorders Gastrointestinal Disorder	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Endocrine disorders Hyperthyroidism	13.3% 2/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Endocrine disorders Hypothyroidism	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Nervous system disorders Memory Impairment	13.3% 2/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Renal and urinary disorders Urinary Frequency	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.
Renal and urinary disorders Urinary Incontinence	6.7% 1/15 • adverse events will be recorded from the baseline through 30 days following the end of treatment and at each examination. Serious adverse events will be collected for 90 days after the end of treatment. All-Cause Mortality will be assessed for up to 2 years.

Additional Information

Dr. Vinaykumar Puduvalli, MD-Chair, Neuro-Oncology

UT MD Anderson Cancer Center

Phone: (713) 745-2343

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place