Trial Outcomes & Findings for Pembrolizumab With or Without Radiation in Patients With Recurrent or Metastatic Adenoid Cystic Carcinoma (NCT NCT03087019)

NCT ID: NCT03087019

Last Updated: 2022-10-14

Results Overview

Objective response will be assessed in non-irradiated lesions among all eligible and treated patients pursuing RECIST 1.1. Per RECIST guidelines for target lesions, Complete Response (CR): disappearance of all target lesions; Partial Response (PR): \>=30% decrease in the sum of the longest diameter of target lesions; Progressive Disease (PD): \>20% increase in sum of the longest diameter (LD) of measured lesions, referencing the smallest sum LD, or new lesions; Stable Disease (SD): neither PR nor PD. Objective response refers to tumor shrinkage at least qualifying as PR.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 21 participants
• Primary outcome timeframe: 2 years
• Results posted on: 2022-10-14

Participant Flow

Participant milestones

Measure	Pembrolizumab + Radiation * Up to 5 metastatic lesions targeted with radiation * Over 5 fractions starting within 7 calendar days following the first dose of pembrolizumab * Pembrolizumab will be administered on day 1 of each 21day cycle * Pembrolizumab is delivered intravenously Radiation: Standard use of radiation is administered Pembrolizumab: Pembrolizumab is designed to restore the natural ability of the immune system to recognize and target cancer cells.	Pembrolizumab * Pembrolizumab will be administered on day 1 of each 21day cycle * Pembrolizumab is delivered intravenously Pembrolizumab: Pembrolizumab is designed to restore the natural ability of the immune system to recognize and target cancer cells.
Overall Study STARTED	11	10
Overall Study COMPLETED	10	10
Overall Study NOT COMPLETED	1	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pembrolizumab With or Without Radiation in Patients With Recurrent or Metastatic Adenoid Cystic Carcinoma

Baseline characteristics by cohort

Measure	Pembrolizumab + Radiation n=11 Participants * Up to 5 metastatic lesions targeted with radiation * Over 5 fractions starting within 7 calendar days following the first dose of pembrolizumab * Pembrolizumab will be administered on day 1 of each 21day cycle * Pembrolizumab is delivered intravenously Radiation: Standard use of radiation is administered Pembrolizumab: Pembrolizumab is designed to restore the natural ability of the immune system to recognize and target cancer cells.	Pembrolizumab n=10 Participants * Pembrolizumab will be administered on day 1 of each 21day cycle * Pembrolizumab is delivered intravenously Pembrolizumab: Pembrolizumab is designed to restore the natural ability of the immune system to recognize and target cancer cells.	Total n=21 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Age, Categorical Between 18 and 65 years	5 Participants n=93 Participants	7 Participants n=4 Participants	12 Participants n=27 Participants
Age, Categorical >=65 years	6 Participants n=93 Participants	3 Participants n=4 Participants	9 Participants n=27 Participants
Sex: Female, Male Female	7 Participants n=93 Participants	9 Participants n=4 Participants	16 Participants n=27 Participants
Sex: Female, Male Male	4 Participants n=93 Participants	1 Participants n=4 Participants	5 Participants n=27 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Asian	1 Participants n=93 Participants	0 Participants n=4 Participants	1 Participants n=27 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Black or African American	0 Participants n=93 Participants	1 Participants n=4 Participants	1 Participants n=27 Participants
Race (NIH/OMB) White	9 Participants n=93 Participants	9 Participants n=4 Participants	18 Participants n=27 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=93 Participants	0 Participants n=4 Participants	1 Participants n=27 Participants

PRIMARY outcome

Timeframe: 2 years

Population: 21 subjects were enrolled. One subject withdrew consent before starting treatment and was replaced. A total of 20 patients (10 per each arm) received protocol treatment. Among 20 patients treated, 1 patient withdrew consent in order to pursue hospice care after receiving a single dose of pembrolizumab and was not evaluable for response.

Objective response will be assessed in non-irradiated lesions among all eligible and treated patients pursuing RECIST 1.1. Per RECIST guidelines for target lesions, Complete Response (CR): disappearance of all target lesions; Partial Response (PR): >=30% decrease in the sum of the longest diameter of target lesions; Progressive Disease (PD): >20% increase in sum of the longest diameter (LD) of measured lesions, referencing the smallest sum LD, or new lesions; Stable Disease (SD): neither PR nor PD. Objective response refers to tumor shrinkage at least qualifying as PR.

Outcome measures

Measure	Pembrolizumab + Radiation n=10 Participants * Up to 5 metastatic lesions targeted with radiation * Over 5 fractions starting within 7 calendar days following the first dose of pembrolizumab * Pembrolizumab will be administered on day 1 of each 21day cycle * Pembrolizumab is delivered intravenously Radiation: Standard use of radiation is administered Pembrolizumab: Pembrolizumab is designed to restore the natural ability of the immune system to recognize and target cancer cells.	Pembrolizumab n=9 Participants * Pembrolizumab will be administered on day 1 of each 21day cycle * Pembrolizumab is delivered intravenously Pembrolizumab: Pembrolizumab is designed to restore the natural ability of the immune system to recognize and target cancer cells.
Patients Demonstrating Objective Response In Non-Irradiated Lesions (Tumor Size on Scans)	0 Participants	0 Participants

SECONDARY outcome

Timeframe: 2 years

Population: 21 subjects were enrolled. One subject withdrew consent before starting treatment and was replaced. A total of 20 patients (10 per each arm) received protocol treatment.

Progression-free survival is defined as the time from randomization to disease progression or death, whichever occurs first. Patients who are alive without disease progression will be censored at the date of last disease assessment. Progression-free survival will be evaluated using both RECIST 1.1 and immune-related response criteria (irRC), and both sets of results will be reported in each arm. Per RECIST criteria, progression is defined as a >=20% increase in the sum of the longest diameter of the measured lesions, referencing the smallest longest diameter sum, or the appearance of at least one new lesion.

Outcome measures

Measure	Pembrolizumab + Radiation n=10 Participants * Up to 5 metastatic lesions targeted with radiation * Over 5 fractions starting within 7 calendar days following the first dose of pembrolizumab * Pembrolizumab will be administered on day 1 of each 21day cycle * Pembrolizumab is delivered intravenously Radiation: Standard use of radiation is administered Pembrolizumab: Pembrolizumab is designed to restore the natural ability of the immune system to recognize and target cancer cells.	Pembrolizumab n=10 Participants * Pembrolizumab will be administered on day 1 of each 21day cycle * Pembrolizumab is delivered intravenously Pembrolizumab: Pembrolizumab is designed to restore the natural ability of the immune system to recognize and target cancer cells.
Progression Free Survival (Time From Randomization to Disease Progression or Death)	4.5 months Interval 2.4 to 20.6	6.6 months Interval 2.4 to 13.1

SECONDARY outcome

Timeframe: 2 years

Population: 21 subjects were enrolled. One subject withdrew consent before starting treatment and was replaced. A total of 20 patients (10 per each arm) received protocol treatment.

Overall survival is defined as the time from randomization to death or date last known alive. Overall survival will be evaluated using both RECIST 1.1 and immune-related response criteria (irRC), and both sets of results will be reported in each arm. will be evaluated using both RECIST 1.1 and immune-related response criteria (irRC), and both sets of results will be reported in each arm.

Outcome measures

Measure	Pembrolizumab + Radiation n=10 Participants * Up to 5 metastatic lesions targeted with radiation * Over 5 fractions starting within 7 calendar days following the first dose of pembrolizumab * Pembrolizumab will be administered on day 1 of each 21day cycle * Pembrolizumab is delivered intravenously Radiation: Standard use of radiation is administered Pembrolizumab: Pembrolizumab is designed to restore the natural ability of the immune system to recognize and target cancer cells.	Pembrolizumab n=10 Participants * Pembrolizumab will be administered on day 1 of each 21day cycle * Pembrolizumab is delivered intravenously Pembrolizumab: Pembrolizumab is designed to restore the natural ability of the immune system to recognize and target cancer cells.
Overall Survival (Time From Randomization to Death)	NA months Interval 10.1 to upper limit not reached	27.2 months Interval 22.9 to upper limit not reached

SECONDARY outcome

Timeframe: 2 years

Population: 21 subjects were enrolled. One subject withdrew consent before starting treatment and was replaced. A total of 20 patients (10 per each arm) received protocol treatment.

Complete disappearance of all measurable non-irradiated lesions. All lymph nodes must be non-pathological in size (<10 mm short axis).

Outcome measures

Measure	Pembrolizumab + Radiation n=10 Participants * Up to 5 metastatic lesions targeted with radiation * Over 5 fractions starting within 7 calendar days following the first dose of pembrolizumab * Pembrolizumab will be administered on day 1 of each 21day cycle * Pembrolizumab is delivered intravenously Radiation: Standard use of radiation is administered Pembrolizumab: Pembrolizumab is designed to restore the natural ability of the immune system to recognize and target cancer cells.	Pembrolizumab n=10 Participants * Pembrolizumab will be administered on day 1 of each 21day cycle * Pembrolizumab is delivered intravenously Pembrolizumab: Pembrolizumab is designed to restore the natural ability of the immune system to recognize and target cancer cells.
Number of Participants With Complete Response (Absence of Non-irradiated Lesions on Scans)	0 Participants	0 Participants

SECONDARY outcome

Timeframe: 2 years

Population: 21 subjects were enrolled. One subject withdrew consent before starting treatment and was replaced. A total of 20 patients (10 per each arm) received protocol treatment.

At least a 30% decrease in the longest diameter valuated using RECIST 1.1.

Outcome measures

Measure	Pembrolizumab + Radiation n=10 Participants * Up to 5 metastatic lesions targeted with radiation * Over 5 fractions starting within 7 calendar days following the first dose of pembrolizumab * Pembrolizumab will be administered on day 1 of each 21day cycle * Pembrolizumab is delivered intravenously Radiation: Standard use of radiation is administered Pembrolizumab: Pembrolizumab is designed to restore the natural ability of the immune system to recognize and target cancer cells.	Pembrolizumab n=10 Participants * Pembrolizumab will be administered on day 1 of each 21day cycle * Pembrolizumab is delivered intravenously Pembrolizumab: Pembrolizumab is designed to restore the natural ability of the immune system to recognize and target cancer cells.
Number of Participants With Partial Response (Tumor Size on Scans)	0 Participants	0 Participants

SECONDARY outcome

Timeframe: 2 years

Population: 21 subjects were enrolled. One subject withdrew consent before starting treatment and was replaced. A total of 20 patients (10 per each arm) received protocol treatment.

All patients who receive treatment, regardless of eligibility, will be evaluable for toxicity. Toxicity will be graded according to NCI CTCAE, Version 4.0. The proportions of patients with various toxicities will be reported by treatment arm.

Outcome measures

Measure	Pembrolizumab + Radiation n=10 Participants * Up to 5 metastatic lesions targeted with radiation * Over 5 fractions starting within 7 calendar days following the first dose of pembrolizumab * Pembrolizumab will be administered on day 1 of each 21day cycle * Pembrolizumab is delivered intravenously Radiation: Standard use of radiation is administered Pembrolizumab: Pembrolizumab is designed to restore the natural ability of the immune system to recognize and target cancer cells.	Pembrolizumab n=10 Participants * Pembrolizumab will be administered on day 1 of each 21day cycle * Pembrolizumab is delivered intravenously Pembrolizumab: Pembrolizumab is designed to restore the natural ability of the immune system to recognize and target cancer cells.
Number of Treatment-Emergent Adverse Events	51 treatment-emergent adverse events	24 treatment-emergent adverse events

Adverse Events

Pembrolizumab + Radiation

Serious events: 3 serious events

Other events: 10 other events

Deaths: 1 deaths

Pembrolizumab

Serious events: 4 serious events

Other events: 10 other events

Deaths: 1 deaths

Serious adverse events

Measure	Pembrolizumab + Radiation n=10 participants at risk * Up to 5 metastatic lesions targeted with radiation * Over 5 fractions starting within 7 calendar days following the first dose of pembrolizumab * Pembrolizumab will be administered on day 1 of each 21day cycle * Pembrolizumab is delivered intravenously Radiation: Standard use of radiation is administered Pembrolizumab: Pembrolizumab is designed to restore the natural ability of the immune system to recognize and target cancer cells.	Pembrolizumab n=10 participants at risk * Pembrolizumab will be administered on day 1 of each 21day cycle * Pembrolizumab is delivered intravenously Pembrolizumab: Pembrolizumab is designed to restore the natural ability of the immune system to recognize and target cancer cells.
Endocrine disorders Hyperthydroidism	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Respiratory, thoracic and mediastinal disorders Pneumonia	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Gastrointestinal disorders Dysphagia	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Investigations Elevated ALT	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Investigations Elevated AST	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Investigations Elevated Alkaline Phosphatase	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Investigations Elevated Total Bilirubin	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Respiratory, thoracic and mediastinal disorders Aspiration Pneumonia	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Gastrointestinal disorders Diarrhea	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Musculoskeletal and connective tissue disorders Generalized Muscle Weakness	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
General disorders Chest Tightness	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Nervous system disorders Paresthesia (tongue)	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Psychiatric disorders Confusion	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Nervous system disorders Movement Disorder (tongue and lips)	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Infections and infestations Wound Infection	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	20.0% 2/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).

Other adverse events

Measure	Pembrolizumab + Radiation n=10 participants at risk * Up to 5 metastatic lesions targeted with radiation * Over 5 fractions starting within 7 calendar days following the first dose of pembrolizumab * Pembrolizumab will be administered on day 1 of each 21day cycle * Pembrolizumab is delivered intravenously Radiation: Standard use of radiation is administered Pembrolizumab: Pembrolizumab is designed to restore the natural ability of the immune system to recognize and target cancer cells.	Pembrolizumab n=10 participants at risk * Pembrolizumab will be administered on day 1 of each 21day cycle * Pembrolizumab is delivered intravenously Pembrolizumab: Pembrolizumab is designed to restore the natural ability of the immune system to recognize and target cancer cells.
Investigations Alanine Aminotransferase Increased	20.0% 2/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Investigations Alkaline Phosphatase Increased	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Skin and subcutaneous tissue disorders Alopecia	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Metabolism and nutrition disorders Anorexia	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Musculoskeletal and connective tissue disorders Arthralgia	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Investigations Aspartate Aminotransferase Increased	20.0% 2/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Musculoskeletal and connective tissue disorders Back Pain	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
General disorders Chills	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Investigations Creatinine Increased	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Gastrointestinal disorders Diarrhea	40.0% 4/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Gastrointestinal disorders Dry Mouth	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Nervous system disorders Dysgeusia	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Respiratory, thoracic and mediastinal disorders Dyspnea	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Endocrine disorders Endocrine Disorders - Other	20.0% 2/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
General disorders Fatigue	40.0% 4/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	40.0% 4/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Gastrointestinal disorders Gastroesophageal Reflux Disease	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Gastrointestinal disorders Gastrointestinal Disorders - Other	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
General disorders General Disorders and Administration Site Conditions - Other	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Hepatobiliary disorders Hepatobiliary Disorders - Other	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Metabolism and nutrition disorders Hypercalcemia	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Vascular disorders Hypertension	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Endocrine disorders Hyperthyroidism	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Metabolism and nutrition disorders Hyponatremia	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Metabolism and nutrition disorders Hypophosphatemia	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Endocrine disorders Hypothyroidism	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	20.0% 2/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Musculoskeletal and connective tissue disorders Musculoskeletal and Connective Tissue Disorder - Other	20.0% 2/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	20.0% 2/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Musculoskeletal and connective tissue disorders Myalgia	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Gastrointestinal disorders Nausea	20.0% 2/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Musculoskeletal and connective tissue disorders Neck Pain	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Nervous system disorders Nervous System Disorders - Other	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
General disorders Pain	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Respiratory, thoracic and mediastinal disorders Pneumonitis	30.0% 3/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Skin and subcutaneous tissue disorders Pruritis	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Skin and subcutaneous tissue disorders Skin Hyperpigmentation	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	0.00% 0/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Skin and subcutaneous tissue disorders Skin/Subcutaneous Tissue Disorders - Other	40.0% 4/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).
Gastrointestinal disorders Vomiting	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).	10.0% 1/10 • 2 years Adverse event evaluation was performed at every protocol visit using the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).

Additional Information

Jonathan Schoenfeld, MD, MPH

Dana-Farber Cancer Institute

Phone: 617-632-3591

Email: jonathan_schoenfeld@dfci.harvard.edu

Results disclosure agreements

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