Trial Outcomes & Findings for Temozolomide With or Without Veliparib in Treating Patients With Relapsed or Refractory Small Cell Lung Cancer (NCT NCT01638546)
NCT ID: NCT01638546
Last Updated: 2026-04-29
Results Overview
Compared across the two arms using a Fisher exact test.
Recruitment status
ACTIVE_NOT_RECRUITING
Study phase
PHASE2
Target enrollment
97 participants
Primary outcome timeframe
From randomization to time of progression or death, whichever occurs first, assessed at 4 months
Results posted on
2026-04-29
Participant Flow
Participant milestones
| Measure |
Arm I (Veliparib and Temozolomide)
Patients receive veliparib PO BID on days 1-7 and temozolomide PO on days 1-5.
Laboratory Biomarker Analysis: Correlative studies
Temozolomide: Given PO
Veliparib: Given PO
|
Arm II (Placebo and Temozolomide)
Patients receive placebo PO BID on days 1-7 and temozolomide as in Arm I.
Laboratory Biomarker Analysis: Correlative studies
Placebo: Given PO
Temozolomide: Given PO
|
|---|---|---|
|
Overall Study
STARTED
|
52
|
45
|
|
Overall Study
COMPLETED
|
51
|
42
|
|
Overall Study
NOT COMPLETED
|
1
|
3
|
Reasons for withdrawal
| Measure |
Arm I (Veliparib and Temozolomide)
Patients receive veliparib PO BID on days 1-7 and temozolomide PO on days 1-5.
Laboratory Biomarker Analysis: Correlative studies
Temozolomide: Given PO
Veliparib: Given PO
|
Arm II (Placebo and Temozolomide)
Patients receive placebo PO BID on days 1-7 and temozolomide as in Arm I.
Laboratory Biomarker Analysis: Correlative studies
Placebo: Given PO
Temozolomide: Given PO
|
|---|---|---|
|
Overall Study
Adverse Event
|
1
|
3
|
Baseline Characteristics
Temozolomide With or Without Veliparib in Treating Patients With Relapsed or Refractory Small Cell Lung Cancer
Baseline characteristics by cohort
| Measure |
Arm I (Veliparib and Temozolomide)
n=52 Participants
Patients receive veliparib PO BID on days 1-7 and temozolomide PO on days 1-5.
Laboratory Biomarker Analysis: Correlative studies
Temozolomide: Given PO
Veliparib: Given PO
|
Arm II (Placebo and Temozolomide)
n=45 Participants
Patients receive placebo PO BID on days 1-7 and temozolomide as in Arm I.
Laboratory Biomarker Analysis: Correlative studies
Placebo: Given PO
Temozolomide: Given PO
|
Total
n=97 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=9 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=23 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
31 Participants
n=9 Participants
|
31 Participants
n=24 Participants
|
62 Participants
n=23 Participants
|
|
Age, Categorical
>=65 years
|
21 Participants
n=9 Participants
|
14 Participants
n=24 Participants
|
35 Participants
n=23 Participants
|
|
Sex: Female, Male
Female
|
29 Participants
n=9 Participants
|
23 Participants
n=24 Participants
|
52 Participants
n=23 Participants
|
|
Sex: Female, Male
Male
|
23 Participants
n=9 Participants
|
22 Participants
n=24 Participants
|
45 Participants
n=23 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=9 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=23 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=9 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=23 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=9 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=23 Participants
|
|
Race (NIH/OMB)
Black or African American
|
4 Participants
n=9 Participants
|
1 Participants
n=24 Participants
|
5 Participants
n=23 Participants
|
|
Race (NIH/OMB)
White
|
47 Participants
n=9 Participants
|
43 Participants
n=24 Participants
|
90 Participants
n=23 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=9 Participants
|
0 Participants
n=24 Participants
|
0 Participants
n=23 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=9 Participants
|
1 Participants
n=24 Participants
|
2 Participants
n=23 Participants
|
PRIMARY outcome
Timeframe: From randomization to time of progression or death, whichever occurs first, assessed at 4 monthsCompared across the two arms using a Fisher exact test.
Outcome measures
| Measure |
Arm I (Veliparib and Temozolomide)
n=51 Participants
Patients receive veliparib PO BID on days 1-7 and temozolomide PO on days 1-5.
Laboratory Biomarker Analysis: Correlative studies
Temozolomide: Given PO
Veliparib: Given PO
|
Arm II (Placebo and Temozolomide)
n=42 Participants
Patients receive placebo PO BID on days 1-7 and temozolomide as in Arm I.
Laboratory Biomarker Analysis: Correlative studies
Placebo: Given PO
Temozolomide: Given PO
|
|---|---|---|
|
Progression-free Survival, Calculated as the Proportion of Patients Alive and Without Evidence of Disease
|
18 participants
|
11 participants
|
SECONDARY outcome
Timeframe: From date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 4 monthsCorresponding exact two-sided 95% confidence intervals will be calculated and reported in both arms of the study. Comparisons between treatment arms will be performed using Fisher-exact test.
Outcome measures
| Measure |
Arm I (Veliparib and Temozolomide)
n=51 Participants
Patients receive veliparib PO BID on days 1-7 and temozolomide PO on days 1-5.
Laboratory Biomarker Analysis: Correlative studies
Temozolomide: Given PO
Veliparib: Given PO
|
Arm II (Placebo and Temozolomide)
n=42 Participants
Patients receive placebo PO BID on days 1-7 and temozolomide as in Arm I.
Laboratory Biomarker Analysis: Correlative studies
Placebo: Given PO
Temozolomide: Given PO
|
|---|---|---|
|
Overall Response (ORR) by RECIST 1.1 Criteria
|
20 participants
|
6 participants
|
SECONDARY outcome
Timeframe: From randomization to time of deathEstimated in each treatment group using Kaplan-Meier method. Group comparisons will be performed using log-rank test.
Outcome measures
| Measure |
Arm I (Veliparib and Temozolomide)
n=51 Participants
Patients receive veliparib PO BID on days 1-7 and temozolomide PO on days 1-5.
Laboratory Biomarker Analysis: Correlative studies
Temozolomide: Given PO
Veliparib: Given PO
|
Arm II (Placebo and Temozolomide)
n=42 Participants
Patients receive placebo PO BID on days 1-7 and temozolomide as in Arm I.
Laboratory Biomarker Analysis: Correlative studies
Placebo: Given PO
Temozolomide: Given PO
|
|---|---|---|
|
Overall Survival
|
8.2 months
Interval 6.4 to 12.2
|
7 months
Interval 5.3 to 9.5
|
SECONDARY outcome
Timeframe: From the start of treatment until 30 days from coming off treatmentTabulated According to the National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0. Summary level.
Outcome measures
| Measure |
Arm I (Veliparib and Temozolomide)
n=52 Participants
Patients receive veliparib PO BID on days 1-7 and temozolomide PO on days 1-5.
Laboratory Biomarker Analysis: Correlative studies
Temozolomide: Given PO
Veliparib: Given PO
|
Arm II (Placebo and Temozolomide)
n=45 Participants
Patients receive placebo PO BID on days 1-7 and temozolomide as in Arm I.
Laboratory Biomarker Analysis: Correlative studies
Placebo: Given PO
Temozolomide: Given PO
|
|---|---|---|
|
Number of Participants With Adverse Events
|
52 Participants
|
45 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Up to 5 yearsFisher's exact test will be used to correlate response and log-rank test to correlate with progression free survival and overall survival.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline to up to 5 yearsCorrelated with outcome in the two treatment arms.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Up to 5 yearsWilcoxon test will be used to compare the percentage increase of gammaH2AX positive cells between the two treatment groups.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Up to 5 yearsResults will be expressed as binary variables. Associations with objective response, with progression free survival and with overall survival will be tested using Fisher's exact test and log-rank test, respectively.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Up to 5 yearsThe number of CTCs will be correlated with PFS and OS using Cox proportional hazards model. The change in CTCs will be correlated with radiographic response. The number of CTCs at baseline will be correlated with patient characteristics (disease burden, location of metastases, and progression at existing sites or new sites of disease). The number of CTC will be explored as a continuous variable and the presence of a threshold predictive of the outcome will be investigated.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Up to 5 yearsFisher's exact test will be used to correlate response and log-rank test to correlate with progression free survival and overall survival.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Up to 5 yearsResults will be expressed as binary variables. Associations with objective response, with progression free survival and with overall survival will be tested using Fisher's exact test and log-rank test, respectively.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Up to 5 yearsFisher's exact test will be used to correlate response and log-rank test to correlate with progression free survival and overall survival.
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Up to 5 yearsFisher's exact test will be used to correlate response and log-rank test to correlate with progression free survival and overall survival.
Outcome measures
Outcome data not reported
Adverse Events
Arm I (Veliparib and Temozolomide)
Serious events: 22 serious events
Other events: 6 other events
Deaths: 41 deaths
Arm II (Placebo and Temozolomide)
Serious events: 18 serious events
Other events: 35 other events
Deaths: 39 deaths
Serious adverse events
| Measure |
Arm I (Veliparib and Temozolomide)
n=52 participants at risk
Patients receive veliparib PO BID on days 1-7 and temozolomide PO on days 1-5.
Laboratory Biomarker Analysis: Correlative studies
Temozolomide: Given PO
Veliparib: Given PO
|
Arm II (Placebo and Temozolomide)
n=45 participants at risk
Patients receive placebo PO BID on days 1-7 and temozolomide as in Arm I.
Laboratory Biomarker Analysis: Correlative studies
Placebo: Given PO
Temozolomide: Given PO
|
|---|---|---|
|
Renal and urinary disorders
Acute Kidney Injury
|
0.00%
0/52
|
2.2%
1/45 • Number of events 1
|
|
Gastrointestinal disorders
Anorexia
|
0.00%
0/52
|
2.2%
1/45 • Number of events 1
|
|
Gastrointestinal disorders
Dehydration
|
0.00%
0/52
|
2.2%
1/45 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
3.8%
2/52 • Number of events 2
|
4.4%
2/45 • Number of events 2
|
|
General disorders
Fatigue
|
0.00%
0/52
|
2.2%
1/45 • Number of events 1
|
|
Gastrointestinal disorders
Gastric perforation
|
0.00%
0/52
|
2.2%
1/45 • Number of events 1
|
|
Cardiac disorders
Heart failure
|
0.00%
0/52
|
2.2%
1/45 • Number of events 1
|
|
Metabolism and nutrition disorders
Hyponatremia
|
1.9%
1/52 • Number of events 1
|
4.4%
2/45 • Number of events 2
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
0.00%
0/52
|
2.2%
1/45 • Number of events 2
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/52
|
2.2%
1/45 • Number of events 1
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms ben/mal/unk (inc cyst/polyp) Other, spec
|
3.8%
2/52 • Number of events 2
|
6.7%
3/45 • Number of events 3
|
|
Cardiac disorders
Pericardial effusion
|
0.00%
0/52
|
2.2%
1/45 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
0.00%
0/52
|
4.4%
2/45 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
0.00%
0/52
|
2.2%
1/45 • Number of events 1
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/52
|
4.4%
2/45 • Number of events 2
|
|
Metabolism and nutrition disorders
Anemia
|
3.8%
2/52 • Number of events 2
|
0.00%
0/45
|
|
Gastrointestinal disorders
Appendicitis perforated
|
1.9%
1/52 • Number of events 1
|
0.00%
0/45
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
1.9%
1/52 • Number of events 1
|
0.00%
0/45
|
|
Respiratory, thoracic and mediastinal disorders
Bronchopulmonary hemorrhage
|
1.9%
1/52 • Number of events 1
|
0.00%
0/45
|
|
Gastrointestinal disorders
Diarrhea
|
1.9%
1/52 • Number of events 1
|
0.00%
0/45
|
|
Ear and labyrinth disorders
Fall
|
1.9%
1/52 • Number of events 2
|
0.00%
0/45
|
|
Investigations
Febrile Neutropenia
|
5.8%
3/52 • Number of events 3
|
0.00%
0/45
|
|
Ear and labyrinth disorders
Gait disturbance
|
1.9%
1/52 • Number of events 1
|
0.00%
0/45
|
|
Gastrointestinal disorders
Hepatic failure
|
1.9%
1/52 • Number of events 1
|
0.00%
0/45
|
|
Musculoskeletal and connective tissue disorders
Hip fracture
|
1.9%
1/52 • Number of events 1
|
0.00%
0/45
|
|
Respiratory, thoracic and mediastinal disorders
Lung infection
|
3.8%
2/52 • Number of events 5
|
0.00%
0/45
|
|
Nervous system disorders
Nervous system disorders - Other, specify
|
5.8%
3/52 • Number of events 3
|
0.00%
0/45
|
|
Investigations
Neutrophil count decreased
|
1.9%
1/52 • Number of events 1
|
0.00%
0/45
|
|
Investigations
Platelet count decreased
|
9.6%
5/52 • Number of events 5
|
0.00%
0/45
|
|
General disorders
Sepsis
|
3.8%
2/52 • Number of events 2
|
0.00%
0/45
|
|
Renal and urinary disorders
Urinary retention
|
1.9%
1/52 • Number of events 1
|
0.00%
0/45
|
|
Cardiac disorders
Ventricular tachycardia
|
1.9%
1/52 • Number of events 1
|
0.00%
0/45
|
|
Investigations
White blood cell decreased
|
3.8%
2/52 • Number of events 2
|
0.00%
0/45
|
|
Gastrointestinal disorders
Abdominal pain
|
3.8%
2/52 • Number of events 2
|
2.2%
1/45 • Number of events 1
|
Other adverse events
| Measure |
Arm I (Veliparib and Temozolomide)
n=52 participants at risk
Patients receive veliparib PO BID on days 1-7 and temozolomide PO on days 1-5.
Laboratory Biomarker Analysis: Correlative studies
Temozolomide: Given PO
Veliparib: Given PO
|
Arm II (Placebo and Temozolomide)
n=45 participants at risk
Patients receive placebo PO BID on days 1-7 and temozolomide as in Arm I.
Laboratory Biomarker Analysis: Correlative studies
Placebo: Given PO
Temozolomide: Given PO
|
|---|---|---|
|
Metabolism and nutrition disorders
Hyponatremia
|
0.00%
0/52
|
6.7%
3/45
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
0.00%
0/52
|
8.9%
4/45
|
|
Metabolism and nutrition disorders
Lipase increased
|
0.00%
0/52
|
8.9%
4/45
|
|
Metabolism and nutrition disorders
Serum amylase increased
|
0.00%
0/52
|
6.7%
3/45
|
|
General disorders
Febrile neutropenia
|
5.8%
3/52
|
0.00%
0/45
|
|
Nervous system disorders
Nervous system disorders - Other, specify
|
5.8%
3/52
|
0.00%
0/45
|
|
Blood and lymphatic system disorders
Platelet count decreased
|
9.6%
5/52
|
31.1%
14/45
|
|
Blood and lymphatic system disorders
Alanine aminotransferase increased
|
0.00%
0/52
|
17.8%
8/45
|
|
Metabolism and nutrition disorders
Alkaline phosphatase increased
|
0.00%
0/52
|
13.3%
6/45
|
|
Blood and lymphatic system disorders
Anemia
|
0.00%
0/52
|
40.0%
18/45
|
|
General disorders
Anorexia
|
0.00%
0/52
|
6.7%
3/45
|
|
Blood and lymphatic system disorders
Aspartate aminotransferase increased
|
0.00%
0/52
|
6.7%
3/45
|
|
General disorders
Fatigue
|
0.00%
0/52
|
28.9%
13/45
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
0.00%
0/52
|
31.1%
14/45
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
0.00%
0/52
|
11.1%
5/45
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
0.00%
0/52
|
15.6%
7/45
|
|
Blood and lymphatic system disorders
Lymphocyte count decreased
|
0.00%
0/52
|
51.1%
23/45
|
|
General disorders
Nausea
|
0.00%
0/52
|
6.7%
3/45
|
|
Blood and lymphatic system disorders
Neutrophil count decreased
|
0.00%
0/52
|
13.3%
6/45
|
|
Blood and lymphatic system disorders
White blood cell decreased
|
0.00%
0/52
|
20.0%
9/45
|
|
Metabolism and nutrition disorders
Creatinine increased
|
0.00%
0/52
|
6.7%
3/45
|
Additional Information
Dr. Charles Rudin
Memorial Sloan Kettering Cancer Center
Phone: 646-888-4527
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60