Trial Outcomes & Findings for A Phase II Study of Pembrolizumab as Post-Remission Treatment of Patients ≥ 60 With AML (NCT NCT02708641)

NCT ID: NCT02708641

Last Updated: 2021-08-10

Results Overview

Time to recurrence of AML, including only deaths related to recurrence. Relapse of AML is defined as patients reaching remission (bone marrow contains \<5% blast cells, blood cell counts return to within normal limits, no signs disease) followed by a return of leukemia cells in the marrow and a decrease in normal blood cells.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 12 participants
• Primary outcome timeframe: Up to 24 months
• Results posted on: 2021-08-10

Participant Flow

Participant milestones

Measure	Pembro 200 mg - AML Patients Patients with AML (not transplantation eligible) treated with pembrolizumab 200 mg IV administered once every three weeks, post-remission
Overall Study STARTED	12
Overall Study COMPLETED	12
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Phase II Study of Pembrolizumab as Post-Remission Treatment of Patients ≥ 60 With AML

Baseline characteristics by cohort

Measure	Pembro 200 mg - AML Patients n=12 Participants Patients with AML (not transplantation eligible) treated with pembrolizumab 200 mg IV administered once every three weeks, post-remission
Age, Continuous	70.4 years n=5 Participants
Sex: Female, Male Female	6 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	1 Participants n=5 Participants
Race (NIH/OMB) White	11 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
ECOG Performance Status ECOG = 0	4 Participants n=5 Participants
ECOG Performance Status ECOG = 1	8 Participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 24 months

Population: Patients who received at least one cycle of study treatment and were evaluable for response.

Time to recurrence of AML, including only deaths related to recurrence. Relapse of AML is defined as patients reaching remission (bone marrow contains <5% blast cells, blood cell counts return to within normal limits, no signs disease) followed by a return of leukemia cells in the marrow and a decrease in normal blood cells.

Outcome measures

Measure	Pembro 200 mg - AML Patients n=12 Participants Patients with AML (not transplantation eligible) treated with pembrolizumab 200 mg IV administered once every three weeks, post-remission.
Time to Relapse (TTR)	12.14 months Interval 9.0 to Upper bound of CI not reached (due to low accrual number)

PRIMARY outcome

Timeframe: Up to 24 months

Population: Patients who received at least one cycle of study treatment.

Worst Grade of AE experienced, regardless of relatedness to study therapy, per CTCAE v5.0.

Outcome measures

Measure	Pembro 200 mg - AML Patients n=12 Participants Patients with AML (not transplantation eligible) treated with pembrolizumab 200 mg IV administered once every three weeks, post-remission.
Worst Grade of Adverse Events Experienced (Unrelated to Relatedness to Study Therapy) Grade 3	6 Participants
Worst Grade of Adverse Events Experienced (Unrelated to Relatedness to Study Therapy) Grade 2	1 Participants
Worst Grade of Adverse Events Experienced (Unrelated to Relatedness to Study Therapy) Grade 4	5 Participants

PRIMARY outcome

Timeframe: Up to 24 months

Population: Patients who received at least one cycle of study treatment.

Worst Grade of AE experienced, at least probably related to treatment, per CTCAE v5.0.

Outcome measures

Measure	Pembro 200 mg - AML Patients n=12 Participants Patients with AML (not transplantation eligible) treated with pembrolizumab 200 mg IV administered once every three weeks, post-remission.
Worst Grade of Adverse Events Experienced (at Least Probably Related to Treatment) Grade 2	3 Participants
Worst Grade of Adverse Events Experienced (at Least Probably Related to Treatment) Grade 3	5 Participants
Worst Grade of Adverse Events Experienced (at Least Probably Related to Treatment) Grade 4	4 Participants

PRIMARY outcome

Timeframe: Up to 24 months

Population: Patients who received at least one cycle of study treatment.

Worst Grade of AE experienced, at least probably related to treatment, per CTCAE v5.0.

Outcome measures

Measure	Pembro 200 mg - AML Patients n=12 Participants Patients with AML (not transplantation eligible) treated with pembrolizumab 200 mg IV administered once every three weeks, post-remission.
Worst Grade of Adverse Events Experienced (at Least Probably Related to Treatment) Grade 1	3 Participants
Worst Grade of Adverse Events Experienced (at Least Probably Related to Treatment) Grade 2	1 Participants
Worst Grade of Adverse Events Experienced (at Least Probably Related to Treatment) Grade 3	1 Participants
Worst Grade of Adverse Events Experienced (at Least Probably Related to Treatment) Grade 4	1 Participants

SECONDARY outcome

Timeframe: Up to 48 months

Population: Patients eligible for study participation.

The length of time from date of start of treatment that patients are still alive.

Outcome measures

Measure	Pembro 200 mg - AML Patients n=12 Participants Patients with AML (not transplantation eligible) treated with pembrolizumab 200 mg IV administered once every three weeks, post-remission.
Overall Survival (OS)	42.18 months Interval 14.65 to Upper bound of CI not reached (due to low accrual number)

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 24 months

Determination of activated T cells level (percentages) in peripheral blood. Increased levels of activated T cells may indicate decreasing disease progression.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 24 months

Determination of activated NK cells level (percentages) in peripheral blood. Increased levels of activated T cells may indicate decreasing disease progression.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 24 months

Determination of regulatory T cell (Treg) levels (percentages) in peripheral blood. Treg cells are involved in cancer progression by inhibiting anti-cancer immunity. Increased levels of Treg cells may indicate progressing disease.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 24 months

Determination of cytokine expression levels (percentages) in peripheral blood. Cytokine expression is associated with cancer progression, immuno-suppression, and decreased anti-cancer response.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Up to 24 months

Determination of Granzyme B/perforin expression levels (percentages) in peripheral blood.

Granzyme B/perforin expression is associated with the suppression of cancer progression.

Outcome measures

Outcome data not reported

Adverse Events

AML Patients

Serious events: 11 serious events

Other events: 12 other events

Deaths: 6 deaths

Serious adverse events

Measure	AML Patients n=12 participants at risk pembrolizumab 200 mg given IV once every three weeks
Blood and lymphatic system disorders Anemia	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Blood and lymphatic system disorders Blood and lymphatic system	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Blood and lymphatic system disorders Febrile neutropenia	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Cardiac disorders Cardiac disorders - Other, specify	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Gastrointestinal disorders Colitis	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Gastrointestinal disorders Diarrhea	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
General disorders General disorders and admin. site	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Infections and infestations Appendicitis	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Infections and infestations Infections and infestations	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Infections and infestations Lung infection	25.0% 3/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Infections and infestations Rhinitis infective	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Infections and infestations Skin infection	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Infections and infestations Urinary tract infection	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Investigations Aspartate aminotransferase increased	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Investigations Lymphocyte count decreased	33.3% 4/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Investigations Neutrophil count decreased	50.0% 6/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Investigations Platelet count decreased	50.0% 6/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Investigations White blood cell decreased	58.3% 7/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Metabolism and nutrition disorders Hypocalcemia	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Metabolism and nutrition disorders Hyponatremia	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Metabolism and nutrition disorders Hypophosphatemia	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Renal and urinary disorders Hematuria	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Vascular disorders Hypertension	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Vascular disorders Hypotension	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0

Other adverse events

Measure	AML Patients n=12 participants at risk pembrolizumab 200 mg given IV once every three weeks
Cardiac disorders Acute coronary syndrome	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Cardiac disorders Sinus bradycardia	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Ear and labyrinth disorders Ear pain	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Ear and labyrinth disorders Hearing impaired	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Endocrine disorders Endocrine disorders - Other, specify	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Endocrine disorders Hyperthyroidism	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Eye disorders Blurred vision	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Eye disorders Conjunctivitis	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Eye disorders Floaters	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Eye disorders Glaucoma	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Gastrointestinal disorders Dry mouth	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Gastrointestinal disorders Gastroesophageal reflux disease	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Gastrointestinal disorders Hemorrhoids	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Gastrointestinal disorders Mucositis oral	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Gastrointestinal disorders Vomiting	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
General disorders Chills	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Infections and infestations Lung infection	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Infections and infestations Rash pustular	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Infections and infestations Scrotal infection	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Injury, poisoning and procedural complications Bruising	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Investigations Alanine aminotransferase increased	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Investigations Alkaline phosphatase increased	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Investigations Cholesterol high	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Investigations Weight loss	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Investigations White blood cell decreased	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Metabolism and nutrition disorders Hypernatremia	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Metabolism and nutrition disorders Hyperuricemia	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Metabolism and nutrition disorders Hypoglycemia	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Metabolism and nutrition disorders Metabolism and nutrition	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Musculoskeletal and connective tissue disorders Chest wall pain	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Musculoskeletal and connective tissue disorders Generalized muscle weakness	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Psychiatric disorders Anxiety	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Psychiatric disorders Confusion	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Renal and urinary disorders Cystitis noninfective	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Renal and urinary disorders Hematuria	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Renal and urinary disorders Hemoglobinuria	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Renal and urinary disorders Urinary incontinence	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Reproductive system and breast disorders Scrotal pain	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Reproductive system and breast disorders Vaginal discharge	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Respiratory, thoracic and mediastinal disorders Pleural effusion	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Respiratory, thoracic and mediastinal disorders Sinus disorder	8.3% 1/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Endocrine disorders Hypothyroidism	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Gastrointestinal disorders Nausea	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Infections and infestations Infections and infestations	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Infections and infestations Vaginal infection	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Injury, poisoning and procedural complications Fall	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Investigations Act. partial thrombop. time prolonged	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Investigations INR increased	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Investigations Investigations - Other, specify	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Metabolism and nutrition disorders Hypomagnesemia	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Musculoskeletal and connective tissue disorders Arthralgia	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Musculoskeletal and connective tissue disorders Myalgia	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Musculoskeletal and connective tissue disorders Pain in extremity	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Nervous system disorders Dizziness	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Nervous system disorders Headache	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Psychiatric disorders Insomnia	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Respiratory, thoracic and mediastinal disorders Dyspnea	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Respiratory, thoracic and mediastinal disorders Postnasal drip	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Skin and subcutaneous tissue disorders Dry skin	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Skin and subcutaneous tissue disorders Pruritus	16.7% 2/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Cardiac disorders Cardiac disorders - Other, specify	25.0% 3/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Gastrointestinal disorders Abdominal pain	25.0% 3/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Gastrointestinal disorders Diarrhea	25.0% 3/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
General disorders General disorders and admin. site	25.0% 3/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Investigations Creatinine increased	25.0% 3/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Investigations Neutrophil count decreased	25.0% 3/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Metabolism and nutrition disorders Hyperglycemia	25.0% 3/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Metabolism and nutrition disorders Hyperkalemia	25.0% 3/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Metabolism and nutrition disorders Hypoalbuminemia	25.0% 3/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Metabolism and nutrition disorders Hypokalemia	25.0% 3/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Musculoskeletal and connective tissue disorders Musculoskeletal and connective tissue	25.0% 3/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Respiratory, thoracic and mediastinal disorders Nasal congestion	25.0% 3/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Respiratory, thoracic and mediastinal disorders Sore throat	25.0% 3/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Skin and subcutaneous tissue disorders Skin and subcut. tissue	25.0% 3/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Vascular disorders Hypertension	25.0% 3/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Vascular disorders Hypotension	25.0% 3/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
General disorders Edema limbs	33.3% 4/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
General disorders Pain	33.3% 4/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Investigations Lymphocyte count decreased	33.3% 4/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Investigations Platelet count decreased	33.3% 4/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Metabolism and nutrition disorders Anorexia	33.3% 4/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Metabolism and nutrition disorders Hypercalcemia	33.3% 4/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Metabolism and nutrition disorders Hypophosphatemia	33.3% 4/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Cardiac disorders Sinus tachycardia	41.7% 5/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Metabolism and nutrition disorders Hyponatremia	41.7% 5/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
General disorders Fever	50.0% 6/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Metabolism and nutrition disorders Hypocalcemia	50.0% 6/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Blood and lymphatic system disorders Anemia	58.3% 7/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
General disorders Fatigue	58.3% 7/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0
Respiratory, thoracic and mediastinal disorders Cough	66.7% 8/12 • Adverse Events monitored up to 24 months, All-Cause Mortality monitored up to 48 months Adverse Events: Grade 1 and Grade 2 events per CTCAE v4.0 Serious Adverse Events: Grade 3 and Grade 4 events per CTCAE v4.0

Additional Information

Barbara Stadterman, MPH, MCCR, Regulatory Supervisor, CRS

UPMC Hillman Cancer Center

Phone: 412-647-5554

Email: stadtermanbm@upmc.edu

Results disclosure agreements

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