Trial Outcomes & Findings for Study of Arsenic Trioxide in Small Cell Lung Cancer (NCT NCT01470248)

Last Updated: 2021-02-17

Results Overview

Response rate (complete response \[CR\]+ partial response \[PR\]) was evaluated using the revised Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1). * Complete response (CR): Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \< 10 mm. * Partial response (PR): At least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. * Progressive disease (PD): At least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. * Stable disease (SD): Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

20 participants

Primary outcome timeframe

Every 8 weeks

Results posted on

2021-02-17

Participant Flow

All patients were enrolled through the thoracic medical oncology clinic of the Winship Cancer Institute of Emory University, between August 2011 and April 2014.

Participant milestones

Participant milestones
Measure	Arsenic Trioxide Treatment This is a single arm study. All patients will be treated with the investigational agent, Arsenic Trioxide, according to the dose and schedule indicated in the protocol. Arsenic Trioxide: Drug will be given as a loading dose of 0.32mg/kg/day for 4 days in Week 1, followed by 0.25mg/kg/day twice per week for 5 weeks, followed by 2 weeks of rest, at which time response assessment will be performed. Patients will be restaged prior to the beginning of a new cycle, every 2 months on average. Maximum of 6 cycles of therapy will be administered in the absence of tumor progression or excessive side effects
Overall Study STARTED	20
Overall Study COMPLETED	19
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Arsenic Trioxide Treatment This is a single arm study. All patients will be treated with the investigational agent, Arsenic Trioxide, according to the dose and schedule indicated in the protocol. Arsenic Trioxide: Drug will be given as a loading dose of 0.32mg/kg/day for 4 days in Week 1, followed by 0.25mg/kg/day twice per week for 5 weeks, followed by 2 weeks of rest, at which time response assessment will be performed. Patients will be restaged prior to the beginning of a new cycle, every 2 months on average. Maximum of 6 cycles of therapy will be administered in the absence of tumor progression or excessive side effects
Overall Study Withdrawal by Subject	1

Baseline Characteristics

Study of Arsenic Trioxide in Small Cell Lung Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Arsenic Trioxide Treatment n=20 Participants This is a single arm study. All patients will be treated with the investigational agent, Arsenic Trioxide, according to the dose and schedule indicated in the protocol. Arsenic Trioxide: Drug will be given as a loading dose of 0.32mg/kg/day for 4 days in Week 1, followed by 0.25mg/kg/day twice per week for 5 weeks, followed by 2 weeks of rest, at which time response assessment will be performed. Patients will be restaged prior to the beginning of a new cycle, every 2 months on average. Maximum of 6 cycles of therapy will be administered in the absence of tumor progression or excessive side effects
Age, Continuous	63.40 years STANDARD_DEVIATION 10.67 • n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	13 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	4 Participants n=5 Participants
Race (NIH/OMB) White	16 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	20 participants n=5 Participants

PRIMARY outcome

Timeframe: Every 8 weeks

Population: Two patients were not analyzed because only those patients who have measurable disease present at baseline, have received at least one cycle of therapy, and have had their disease re-evaluated were considered evaluable for response.

Outcome measures

Outcome measures
Measure	Arsenic Trioxide Treatment n=17 Participants This is a single arm study. All patients will be treated with the investigational agent, Arsenic Trioxide, according to the dose and schedule indicated in the protocol. Arsenic Trioxide: Drug will be given as a loading dose of 0.32mg/kg/day for 4 days in Week 1, followed by 0.25mg/kg/day twice per week for 5 weeks, followed by 2 weeks of rest, at which time response assessment will be performed. Patients will be restaged prior to the beginning of a new cycle, every 2 months on average. Maximum of 6 cycles of therapy will be administered in the absence of tumor progression or excessive side effects
Response Rate (RR) Complete response	0 Participants
Response Rate (RR) Partial response	0 Participants
Response Rate (RR) Stable disease	2 Participants
Response Rate (RR) Progressive disease	15 Participants

PRIMARY outcome

Timeframe: After completing at least 1 cycle (8 weeks) of treatment

Population: An alternative endpoint of clinical benefit rate was pre-specified in the event that the study failed to meet its overall response rate (ORR) endpoint either at the end of stage I accrual or at final analysis. However, this study met its endpoint. Please see primary outcome measure 1.

Sum of complete response (CR), partial response (PR) and stable disease (SD) in patients eligible for efficacy analysis.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Every 8 weeks

Defined as the duration of time from start of treatment to time of progression or death, whichever occurs first. Progression was evaluated using the revised Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1). Progressive Disease (PD): At least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm.

Outcome measures

Outcome measures
Measure	Arsenic Trioxide Treatment n=17 Participants This is a single arm study. All patients will be treated with the investigational agent, Arsenic Trioxide, according to the dose and schedule indicated in the protocol. Arsenic Trioxide: Drug will be given as a loading dose of 0.32mg/kg/day for 4 days in Week 1, followed by 0.25mg/kg/day twice per week for 5 weeks, followed by 2 weeks of rest, at which time response assessment will be performed. Patients will be restaged prior to the beginning of a new cycle, every 2 months on average. Maximum of 6 cycles of therapy will be administered in the absence of tumor progression or excessive side effects
Progression-free Survival	6.26 weeks Standard Deviation 3.9

SECONDARY outcome

Timeframe: From enrolment till death on average up to 2 years

Duration of time from enrollment on study until death

Outcome measures

Outcome measures
Measure	Arsenic Trioxide Treatment n=17 Participants This is a single arm study. All patients will be treated with the investigational agent, Arsenic Trioxide, according to the dose and schedule indicated in the protocol. Arsenic Trioxide: Drug will be given as a loading dose of 0.32mg/kg/day for 4 days in Week 1, followed by 0.25mg/kg/day twice per week for 5 weeks, followed by 2 weeks of rest, at which time response assessment will be performed. Patients will be restaged prior to the beginning of a new cycle, every 2 months on average. Maximum of 6 cycles of therapy will be administered in the absence of tumor progression or excessive side effects
Overall Survival	4.5 months Interval 2.0 to 7.0

Adverse Events

Arsenic Trioxide Treatment

Serious events: 1 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Arsenic Trioxide Treatment n=19 participants at risk This is a single arm study. All patients will be treated with the investigational agent, Arsenic Trioxide, according to the dose and schedule indicated in the protocol. Arsenic Trioxide: Drug will be given as a loading dose of 0.32mg/kg/day for 4 days in Week 1, followed by 0.25mg/kg/day twice per week for 5 weeks, followed by 2 weeks of rest, at which time response assessment will be performed. Patients will be restaged prior to the beginning of a new cycle, every 2 months on average. Maximum of 6 cycles of therapy will be administered in the absence of tumor progression or excessive side effects
Respiratory, thoracic and mediastinal disorders Pleural effusion	5.3% 1/19 • Patients were followed until study closure or until death, whichever occurred first.

Other adverse events

Other adverse events
Measure	Arsenic Trioxide Treatment n=19 participants at risk This is a single arm study. All patients will be treated with the investigational agent, Arsenic Trioxide, according to the dose and schedule indicated in the protocol. Arsenic Trioxide: Drug will be given as a loading dose of 0.32mg/kg/day for 4 days in Week 1, followed by 0.25mg/kg/day twice per week for 5 weeks, followed by 2 weeks of rest, at which time response assessment will be performed. Patients will be restaged prior to the beginning of a new cycle, every 2 months on average. Maximum of 6 cycles of therapy will be administered in the absence of tumor progression or excessive side effects
Respiratory, thoracic and mediastinal disorders Dyspnea	5.3% 1/19 • Patients were followed until study closure or until death, whichever occurred first.
Blood and lymphatic system disorders Anemia	5.3% 1/19 • Patients were followed until study closure or until death, whichever occurred first.
Musculoskeletal and connective tissue disorders Back pain	5.3% 1/19 • Patients were followed until study closure or until death, whichever occurred first.
Renal and urinary disorders Elevated creatinine	5.3% 1/19 • Patients were followed until study closure or until death, whichever occurred first.
Skin and subcutaneous tissue disorders Facial edema around eyes	5.3% 1/19 • Patients were followed until study closure or until death, whichever occurred first.
General disorders Generalized weakness	5.3% 1/19 • Patients were followed until study closure or until death, whichever occurred first.
Blood and lymphatic system disorders Hyperbilirubinemia	5.3% 1/19 • Patients were followed until study closure or until death, whichever occurred first.
Metabolism and nutrition disorders Hyperglycemia	5.3% 1/19 • Patients were followed until study closure or until death, whichever occurred first.
Blood and lymphatic system disorders Hypoalbuminemia	15.8% 3/19 • Patients were followed until study closure or until death, whichever occurred first.
Blood and lymphatic system disorders Hypocalcemia	5.3% 1/19 • Patients were followed until study closure or until death, whichever occurred first.
Blood and lymphatic system disorders Hyponatremia	5.3% 1/19 • Patients were followed until study closure or until death, whichever occurred first.
Blood and lymphatic system disorders Hypophosphatemia	5.3% 1/19 • Patients were followed until study closure or until death, whichever occurred first.
Blood and lymphatic system disorders Increased alkaline phosphatase	5.3% 1/19 • Patients were followed until study closure or until death, whichever occurred first.
Blood and lymphatic system disorders Increased lipase	5.3% 1/19 • Patients were followed until study closure or until death, whichever occurred first.
Blood and lymphatic system disorders Leukopenia	10.5% 2/19 • Patients were followed until study closure or until death, whichever occurred first.
Blood and lymphatic system disorders Neutrophil count decreased	5.3% 1/19 • Patients were followed until study closure or until death, whichever occurred first.

Additional Information

Taofeek K. Owonikoko, MD, PhD, MSCR

Emory University

Phone: 404-778-1900

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place