Trial Outcomes & Findings for Sorafenib in Myelodysplastic Syndrome (NCT NCT00510289)

Last Updated: 2016-04-19

Results Overview

Hematological response is defined as the number of subjects who achieve either a complete response (CR), Partial response (PR) or Hematologic improvement.(HI). HI is defined as peripheral blood counts with hemoglobin ≥11 g/dL, absolute neutrophil count ≥1x10(9)/L and platelet count ≥100x10(9)/L, and normal bone marrow morphology with no evidence of dysplasia or blasts. CR is defined as the disappearance of all signs and symptoms related to disease, along with HI. PR is defined as fulfilling the criteria for CR in the peripheral blood but blasts decreasing by 50% or more in the bone marrow or to a less advanced WHO classification pretreatment.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

19 participants

Primary outcome timeframe

During treatment - up to a maximum of 5 years

Results posted on

2016-04-19

Participant Flow

Patients 18 years or older with an ECOG performance status of 0-2, and adequate renal and hepatic function with a diagnosis of primary or therapy-related MDS were eligible for this study. Patients were enrolled from 2006 to 2011 at Duke and Duke Oncology Network sites. The study was closed prematurely due to poor response and patient withdrawals.

Three patients were ineligible due to the presence of AML in the BM done at the time of screening.

Participant milestones

Participant milestones
Measure	All Patients All patients who signed consent
Overall Study STARTED	19
Overall Study Started Study Drug	16
Overall Study COMPLETED	7
Overall Study NOT COMPLETED	12

Reasons for withdrawal

Reasons for withdrawal
Measure	All Patients All patients who signed consent
Overall Study Withdrawal by Subject	7
Overall Study Refused bone marrow biopsy	2
Overall Study Lost to Follow-up	1
Overall Study Progressed prior to starting drug	2

Baseline Characteristics

Sorafenib in Myelodysplastic Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Patients n=19 Participants All patients who signed consent
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=5 Participants
Age, Categorical >=65 years	15 Participants n=5 Participants
Sex: Female, Male Female	6 Participants n=5 Participants
Sex: Female, Male Male	13 Participants n=5 Participants
Region of Enrollment United States	19 participants n=5 Participants

PRIMARY outcome

Timeframe: During treatment - up to a maximum of 5 years

Population: There were 16 subjects enrolled in the trial. 9 subjects refused further bone marrow biopsy to assess response to therapy. Therefore, there were 7 evaluable subjects. One subject experienced PR

Outcome measures

Outcome measures
Measure	Evaluable Subjects n=7 Participants Patients who received at least one cycle of study drug and underwent bone marrow assessments
Number of Subjects Achieving Hematological Response Partial Response	1 participants
Number of Subjects Achieving Hematological Response Complete Response	0 participants
Number of Subjects Achieving Hematological Response Hematologic Improvement	0 participants

SECONDARY outcome

Timeframe: While on study drug, a maximum of 5 years

Population: 7 Subjects completed beyond cycle 1. 4 of those subjects had dose reductions during the time they took study drug.

The number of subjects who took study drug for more than 1 cycle and required a dose reduction down to the next dose level.

Outcome measures

Outcome measures
Measure	Evaluable Subjects n=7 Participants Patients who received at least one cycle of study drug and underwent bone marrow assessments
Number of Subjects Requiring Dose Reductions	4 participants

SECONDARY outcome

Timeframe: 5 years

Population: 7 patients completed cycle one and had bone marrow biopsies to confirm response.

Time to progression will be defined as the number of months between on-study and the date of progression or death, whichever comes first, in subjects who took study drug for at least cycle 1.

Outcome measures

Outcome measures
Measure	Evaluable Subjects n=7 Participants Patients who received at least one cycle of study drug and underwent bone marrow assessments
Time to Progression	3.5 months Interval 2.0 to 7.0

SECONDARY outcome

Timeframe: 1 year from the last dose of study drug

Population: 7 subjects completed cycle 1 or beyond.

Overall Survival is defined as the number of months from enrollment onto the study until death from any cause in subjects who took study drug for at least cycle 1.

Outcome measures

Outcome measures
Measure	Evaluable Subjects n=7 Participants Patients who received at least one cycle of study drug and underwent bone marrow assessments
Overall Survival	15 months Interval 5.0 to 40.0

SECONDARY outcome

Timeframe: Measured before and after treatment

Population: This outcome was not analysed due to the early closure of the study.

Microvessel density will be measured before and after treatment, and the distribution of change across time will be summarized with descriptive statistics.

Outcome measures

Outcome data not reported

Adverse Events

All Patients

Serious events: 5 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	All Patients n=9 participants at risk；n=16 participants at risk SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Gastrointestinal disorders Mucositis	6.2% 1/16 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Infections and infestations Myositis	6.2% 1/16 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Infections and infestations Febrile Neutropenia	6.2% 1/16 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Cardiac disorders hypotension	6.2% 1/16 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Blood and lymphatic system disorders Hemoglobin	6.2% 1/16 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Cardiac disorders atrial fibrillation	6.2% 1/16 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Gastrointestinal disorders small bowel obstruction	6.2% 1/16 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Nervous system disorders syncope	6.2% 1/16 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.

Other adverse events

Other adverse events
Measure	All Patients n=9 participants at risk；n=16 participants at risk SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Blood and lymphatic system disorders Hemoglobin	33.3% 3/9 • Number of events 3 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Eye disorders Ocular / Visual - Other	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Eye disorders Blurred vision	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Gastrointestinal disorders Constipation	33.3% 3/9 • Number of events 4 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Gastrointestinal disorders Diarrhea	55.6% 5/9 • Number of events 7 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Gastrointestinal disorders Dysphagia (difficulty swallowing)	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Gastrointestinal disorders Esophagitis	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Gastrointestinal disorders Hemorrhage, GI - Anus	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Gastrointestinal disorders Mucositis / stomatitis	33.3% 3/9 • Number of events 3 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Gastrointestinal disorders Nausea	33.3% 3/9 • Number of events 6 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Gastrointestinal disorders Obstruction, GI - Small bowel NOS	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Gastrointestinal disorders Pain - Abdomen	22.2% 2/9 • Number of events 2 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Gastrointestinal disorders Vomiting	22.2% 2/9 • Number of events 3 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
General disorders Fatigue (asthenia, lethargy, malaise)	55.6% 5/9 • Number of events 11 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
General disorders Fever (in the absence of neutropenia)	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
General disorders Pain - Chest / thorax NOS	22.2% 2/9 • Number of events 2 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
General disorders Pain - Other	22.2% 2/9 • Number of events 3 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
General disorders Rigors / chills	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Infections and infestations Infection - Lung (pneumonia)	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Infections and infestations Infection with unknown ANC - Mucosa	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Injury, poisoning and procedural complications Bruising	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Investigations ALT, SGPT (serum glutamic pyruvic transaminase)	33.3% 3/9 • Number of events 4 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Investigations AST, SGOT (serum glutamic oxaloacetic transaminase)	22.2% 2/9 • Number of events 3 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Investigations Bilirubin (hyperbilirubinemia)	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Investigations Creatinine	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Investigations Metabolic / Laboratory - Other	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Investigations Platelets	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Investigations Weight loss	22.2% 2/9 • Number of events 3 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Metabolism and nutrition disorders Albumin, serum-low (hypoalbuminemia)	11.1% 1/9 • Number of events 2 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Metabolism and nutrition disorders Anorexia	33.3% 3/9 • Number of events 5 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Metabolism and nutrition disorders Calcium, serum-low (hypocalcemia)	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Metabolism and nutrition disorders Glucose, serum-low (hypoglycemia)	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Metabolism and nutrition disorders Sodium, serum-low (hyponatremia)	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Musculoskeletal and connective tissue disorders Muscle weakness, generalized or specific area (not due to neuropathy) - Extremity-lower	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Musculoskeletal and connective tissue disorders Muscle weakness, generalized or specific area (not due to neuropathy) - Whole body / generalized	22.2% 2/9 • Number of events 2 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Musculoskeletal and connective tissue disorders Musculoskeletal / Soft Tissue - Other	22.2% 2/9 • Number of events 2 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Musculoskeletal and connective tissue disorders Myositis (inflammation / damage of muscle)	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Musculoskeletal and connective tissue disorders Pain - Extremity-limb	11.1% 1/9 • Number of events 2 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Nervous system disorders Dizziness	33.3% 3/9 • Number of events 4 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Nervous system disorders Neurology - Other	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Nervous system disorders Pain - Head / headache	22.2% 2/9 • Number of events 2 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Psychiatric disorders Insomnia	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Psychiatric disorders Mood Alteration - Anxiety	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Psychiatric disorders Mood Alteration - Depression	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Renal and urinary disorders Renal / Genitourinary - Other	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Respiratory, thoracic and mediastinal disorders Dyspnea (shortness of breath)	44.4% 4/9 • Number of events 5 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Respiratory, thoracic and mediastinal disorders Hemorrhage, pulmonary / upper respiratory - Respiratory tract NOS	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Respiratory, thoracic and mediastinal disorders Hypoxia	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Respiratory, thoracic and mediastinal disorders Pain - Throat / pharynx / larynx	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Skin and subcutaneous tissue disorders Dry Skin	33.3% 3/9 • Number of events 3 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Skin and subcutaneous tissue disorders Hair Loss / Alopecia (scalp or body)	22.2% 2/9 • Number of events 2 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Skin and subcutaneous tissue disorders Pruritus / itching	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Skin and subcutaneous tissue disorders Rash / desquamation	33.3% 3/9 • Number of events 4 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Skin and subcutaneous tissue disorders Sweating (diaphoresis)	11.1% 1/9 • Number of events 1 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Vascular disorders Hypertension	22.2% 2/9 • Number of events 2 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.
Vascular disorders Hypotension	22.2% 2/9 • Number of events 2 • 30 days after the last dose of study drug Serious Adverse Events/Other Adverse events are recorded whether or not they are thought to be related to the study drug. SAEs are listed for all patients who took at least one dose of study drug. Due to early study closure and patient withdrawals, Other AEs are listed for 9 patients only.

Additional Information

David Rizzieri, MD

Duke University Medical Center

Phone: 919-668-1014

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place