Trial Outcomes & Findings for Phase 1 Study of Tremelimumab, Durvalumab, High-dose Chemotherapy, + Autologous Stem Cell Transplant (NCT NCT02716805)

Last Updated: 2022-10-12

Results Overview

Toxicity was graded in accordance with the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 4.03. Treatment-emergent adverse events (TEAEs) were reported based on clinical laboratory tests, physical examinations, and vital signs from the time of enrollment through the end of the study period. DLTs were assessed from the first dose of study drug through the Cycle 2 administration of tremelimumab ± durvalumab post ASCT. DLTs were defined per protocol as lack of neutrophil/platelet engraftment by Day 30 post ASCT; Grade 5 toxicity (treatment-related death); Grade 4 non-hematological toxicity; Grade 3 non-hematological toxicity (with exclusions); isolated Grade 3 electrolyte abnormalities; or immune-related AEs resulting in discontinuation of treatment.

Recruitment status

TERMINATED

Study phase

PHASE1

Target enrollment

6 participants

Primary outcome timeframe

up to 14 months

Results posted on

2022-10-12

Participant Flow

Participant milestones

Participant milestones
Measure	Cohort 1 "Late" post-autologous stem cell transplant (ASCT) treatment (tremelimumab alone in Cycles 1-2, durvalumab alone in Cycles 3+)	Cohort 2 "Early" post-ASCT treatment (tremelimumab alone in Cycles 1-2, durvalumab alone in Cycles 3+)	Cohort 3 "Late" post-ASCT treatment (tremelimumab + durvalumab in Cycles 1-2, durvalumab alone in Cycles 3+)	Cohort 4 "Early" post-ASCT treatment (tremelimumab + durvalumab in Cycles 1-2, durvalumab alone in Cycles 3+)
Overall Study STARTED	6	0	0	0
Overall Study COMPLETED	4	0	0	0
Overall Study NOT COMPLETED	2	0	0	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Cohort 1 "Late" post-autologous stem cell transplant (ASCT) treatment (tremelimumab alone in Cycles 1-2, durvalumab alone in Cycles 3+)	Cohort 2 "Early" post-ASCT treatment (tremelimumab alone in Cycles 1-2, durvalumab alone in Cycles 3+)	Cohort 3 "Late" post-ASCT treatment (tremelimumab + durvalumab in Cycles 1-2, durvalumab alone in Cycles 3+)	Cohort 4 "Early" post-ASCT treatment (tremelimumab + durvalumab in Cycles 1-2, durvalumab alone in Cycles 3+)
Overall Study Progressive disease	2	0	0	0

Baseline Characteristics

Phase 1 Study of Tremelimumab, Durvalumab, High-dose Chemotherapy, + Autologous Stem Cell Transplant

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Cohort 1 n=6 Participants "Late" post-ASCT treatment (tremelimumab alone in Cycles 1-2, durvalumab alone in Cycles 3+)
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	5 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Age, Continuous	59.2 years STANDARD_DEVIATION 6.9 • n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	6 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	1 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	2 Participants n=5 Participants
Race (NIH/OMB) White	3 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
Region of Enrollment United States	6 Participants n=5 Participants
Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0	6 Participants n=5 Participants
Eastern Cooperative Oncology Group (ECOG) performance status (PS) ≥ 1	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: up to 14 months

Population: All treated subjects.

Outcome measures

Outcome measures
Measure	Cohort 1 n=6 Participants "Late" post-ASCT treatment (tremelimumab alone in Cycles 1-2, durvalumab alone in Cycles 3+)
Number of Subjects With Treatment-emergent Adverse Events Any TEAE	6 Participants
Number of Subjects With Treatment-emergent Adverse Events Maximum grade 2 TEAE	2 Participants
Number of Subjects With Treatment-emergent Adverse Events Maximum grade 4 TEAE	4 Participants
Number of Subjects With Treatment-emergent Adverse Events Immune-related TEAE	1 Participants
Number of Subjects With Treatment-emergent Adverse Events Tremelimumab-related TEAE	5 Participants
Number of Subjects With Treatment-emergent Adverse Events Durvalumab-related TEAE	1 Participants
Number of Subjects With Treatment-emergent Adverse Events Serious TEAE	1 Participants
Number of Subjects With Treatment-emergent Adverse Events TEAE Leading to Treatment Discontinuation	0 Participants
Number of Subjects With Treatment-emergent Adverse Events TEAE Meeting DLT Criteria	0 Participants

SECONDARY outcome

Timeframe: Up to 14 months

Population: All subjects who received at least 1 dose of study treatment and underwent at least 1 post-baseline disease assessment.

Response was evaluated by appropriate imaging and myeloma serum/urine tests at the start of Cycle 1 and end of study, with response categorized per IMWG consensus criteria, as follows: stringent complete response (sCR) - CR criteria + normal free light chain (FLC) ratio + no clonal cells in bone marrow; CR - negative immunofixation on serum/urine + no soft tissue plasmacytomas + \<5% plasma cells in bone marrow; very good partial response (VGPR) - serum/urine M-protein detectable by immunofixation but not electrophoresis OR ≥90% reduction in serum M-protein + urine \<100 mg/24h; PR - ≥50% and ≥90% (or \<200 mg/24h) reduction of serum + urine M-protein, respectively; progressive disease - increase of ≥25% serum and/or urine M-component, increase of \>10 mg/dL in involved and uninvolved FLC levels, bone marrow plasma cells ≥10%, new or larger lesions, or corrected serum calcium \>11.5 mg/dL or 2.65 mmol/L \[Rajkumar et al. Blood 2011;117:4691-5; Durie et al. Leukemia 2006;20:1467-73\].

Outcome measures

Outcome measures
Measure	Cohort 1 n=4 Participants "Late" post-ASCT treatment (tremelimumab alone in Cycles 1-2, durvalumab alone in Cycles 3+)
Number of Subjects With Best Response According to International Myeloma Working Group (IMWG) Consensus Criteria sCR	1 Participants
Number of Subjects With Best Response According to International Myeloma Working Group (IMWG) Consensus Criteria VGPR	2 Participants
Number of Subjects With Best Response According to International Myeloma Working Group (IMWG) Consensus Criteria Progressive disease	1 Participants

Adverse Events

Cohort 1

Serious events: 1 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Cohort 1 n=6 participants at risk "Late" post-ASCT treatment (tremelimumab alone in Cycles 1-2, durvalumab alone in Cycles 3+)
Renal and urinary disorders Acute kidney injury	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Metabolism and nutrition disorders Hypercalcaemia	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.

Other adverse events

Other adverse events
Measure	Cohort 1 n=6 participants at risk "Late" post-ASCT treatment (tremelimumab alone in Cycles 1-2, durvalumab alone in Cycles 3+)
Gastrointestinal disorders Nausea	83.3% 5/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Gastrointestinal disorders Diarrhoea	33.3% 2/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Gastrointestinal disorders Vomiting	33.3% 2/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Gastrointestinal disorders Abdominal pain upper	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Gastrointestinal disorders Constipation	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Gastrointestinal disorders Pancreatitis	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Skin and subcutaneous tissue disorders Pruritus	33.3% 2/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Skin and subcutaneous tissue disorders Rash maculo-papular	33.3% 2/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Skin and subcutaneous tissue disorders Blood blister	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Skin and subcutaneous tissue disorders Rash	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
General disorders Fatigue	33.3% 2/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
General disorders Influenza like illness	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
General disorders Oedema	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
General disorders Oedema peripheral	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
General disorders Peripheral swelling	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Investigations Weight decreased	50.0% 3/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Investigations Amylase increased	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Investigations Aspartate aminotransferase increased	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Investigations Blood alkaline phosphatase increased	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Investigations Blood creatinine increased	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Investigations Thyroxine decreased	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Nervous system disorders Dizziness	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Nervous system disorders Dysgeusia	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Nervous system disorders Headache	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Nervous system disorders Jugular vein occlusion	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Nervous system disorders Memory impairment	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Nervous system disorders Neuropathy peripheral	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Respiratory, thoracic and mediastinal disorders Cough	50.0% 3/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Respiratory, thoracic and mediastinal disorders Dyspnoea exertional	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Respiratory, thoracic and mediastinal disorders Throat irritation	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Blood and lymphatic system disorders Leukopenia	50.0% 3/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Blood and lymphatic system disorders Lymphopenia	50.0% 3/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Blood and lymphatic system disorders Neutropenia	50.0% 3/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Blood and lymphatic system disorders Anaemia	33.3% 2/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Blood and lymphatic system disorders Thrombocytopenia	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Cardiac disorders Sinus tachycardia	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Cardiac disorders Tachycardia	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Infections and infestations Diverticulitis	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Infections and infestations Upper respiratory tract infection	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Musculoskeletal and connective tissue disorders Back pain	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Musculoskeletal and connective tissue disorders Pain in jaw	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Renal and urinary disorders Nocturia	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Hepatobiliary disorders Hepatitis	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Psychiatric disorders Restlessness	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.
Vascular disorders Hypertension	16.7% 1/6 • All adverse events (AEs) occurring between the signing of informed consent and the off-study date were documented, regardless of the causal relationship to study drug. AEs occurring after the first dose of study drug were considered treatment emergent (i.e., TEAEs). The AE reporting period for this study was up to 14 months. AE documentation included onset/resolution dates, severity using the NCI CTCAE (version 4.03), seriousness, relationship to study drug, study drug action taken, treatment, and outcome. In summaries, treatment-related AEs included those with a "possible", "probable", or "definite" relationship to the indicated study drug; preferred terms were counted only once per subject at the maximum reported grade.

Additional Information

Jonathan Skipper PhD

Ludwig Institute for Cancer Research

Phone: (212) 450-1539

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60