Trial Outcomes & Findings for R-(-)-Gossypol Acetic Acid in Treating Patients With Recurrent Extensive-Stage Small Cell Lung Cancer (NCT NCT00773955)
NCT ID: NCT00773955
Last Updated: 2014-05-12
Results Overview
The number of successes will be estimated by counting the number of participants with confirmed responses. A confirmed tumor response is defined to be either a CR or PR noted as the objective status on 2 consecutive evaluations at least 4 weeks apart. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions: A Complete Response (CR) requires the disappearance of all target lesions A Partial Response (PR) requires a \>=30% decrease in the sum of the longest diameter of target lesions from baseline measurements.
COMPLETED
PHASE2
15 participants
During the first 6 courses of treatment
2014-05-12
Participant Flow
From November 2008 to January 2010, 15 patients were accrued to the study from five sites within the Mayo Clinic Phase 2 Consortium and the California Consortium.
One patient discontinued therapy after one cycle of treatment due to persistent grade 1 thrombocytopenia. Therefore, fourteen patients were evaluable for the primary end point at the interim analysis. All 15 patients were included in secondary endpoint analysis.
Participant milestones
| Measure |
Treatment (R-(-)-Gossypol)
Patients receive oral R-(-)-gossypol once daily on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Overall Study
STARTED
|
15
|
|
Overall Study
COMPLETED
|
15
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
R-(-)-Gossypol Acetic Acid in Treating Patients With Recurrent Extensive-Stage Small Cell Lung Cancer
Baseline characteristics by cohort
| Measure |
Treatment (R-(-)-Gossypol)
n=15 Participants
Patients receive oral R-(-)-gossypol once daily on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Age, Continuous
|
67.0 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
9 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
14 participants
n=5 Participants
|
|
Region of Enrollment
Australia
|
1 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: During the first 6 courses of treatmentPopulation: One patient discontinued therapy after one cycle of treatment due to persistent grade 1 thrombocytopenia. Therefore, fourteen patients were evaluable for the primary end point at the interim analysis.
The number of successes will be estimated by counting the number of participants with confirmed responses. A confirmed tumor response is defined to be either a CR or PR noted as the objective status on 2 consecutive evaluations at least 4 weeks apart. Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions: A Complete Response (CR) requires the disappearance of all target lesions A Partial Response (PR) requires a \>=30% decrease in the sum of the longest diameter of target lesions from baseline measurements.
Outcome measures
| Measure |
Treatment (R-(-)-Gossypol)
n=14 Participants
Patients receive oral R-(-)-gossypol once daily on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Number of Participants With Confirmed Tumor Response Defined to be Either a Complete Response (CR) or Partial Response (PR)
Confirmed Partial Response (PR)
|
0 participants
|
|
Number of Participants With Confirmed Tumor Response Defined to be Either a Complete Response (CR) or Partial Response (PR)
Confirmed Complete Response (CR)
|
0 participants
|
SECONDARY outcome
Timeframe: From registration to death due to any cause, assessed up to 5 yearsPopulation: All 15 patients were analyzed for this endpoint.
Estimated using the method of Kaplan-Meier.
Outcome measures
| Measure |
Treatment (R-(-)-Gossypol)
n=15 Participants
Patients receive oral R-(-)-gossypol once daily on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Survival Time
|
8.5 months
Interval 8.1 to 10.2
|
SECONDARY outcome
Timeframe: From registration to the earliest date documentation of disease progression, assessed up to 5 yearsPopulation: All 15 patients were analyzed for this endpoint.
Time to disease progression is defined as the time from registration to the earliest date documentation of disease progression. Estimated using the method of Kaplan-Meier. Per the RECIST criteria, progression is defined as at least a 20% increase in the sum of Longest Dimension (LD) of target lesions taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions.
Outcome measures
| Measure |
Treatment (R-(-)-Gossypol)
n=15 Participants
Patients receive oral R-(-)-gossypol once daily on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Time to Disease Progression
|
1.7 months
Interval 1.5 to 2.0
|
SECONDARY outcome
Timeframe: From the date at which the patient's earliest best objective status is first noted to be either a CR or PR to the earliest date progression is documented, assessed up to 5 yearsPopulation: There were no confirmed responses qualifying for this endpoint.
Outcome measures
Outcome data not reported
Adverse Events
Treatment (R-(-)-Gossypol)
Serious adverse events
| Measure |
Treatment (R-(-)-Gossypol)
n=15 participants at risk
Patients receive oral R-(-)-gossypol once daily on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Cardiac disorders
Supraventricular tachycardia
|
6.7%
1/15 • Number of events 1
|
|
Gastrointestinal disorders
Ear, nose and throat examination abnormal
|
6.7%
1/15 • Number of events 1
|
|
Gastrointestinal disorders
Small intestinal obstruction
|
6.7%
1/15 • Number of events 1
|
|
Gastrointestinal disorders
Vomiting
|
6.7%
1/15 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
6.7%
1/15 • Number of events 1
|
Other adverse events
| Measure |
Treatment (R-(-)-Gossypol)
n=15 participants at risk
Patients receive oral R-(-)-gossypol once daily on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Blood and lymphatic system disorders
Hemoglobin decreased
|
66.7%
10/15 • Number of events 20
|
|
Cardiac disorders
Sinus tachycardia
|
13.3%
2/15 • Number of events 7
|
|
Gastrointestinal disorders
Abdominal distension
|
6.7%
1/15 • Number of events 1
|
|
Gastrointestinal disorders
Abdominal pain
|
26.7%
4/15 • Number of events 4
|
|
Gastrointestinal disorders
Constipation
|
53.3%
8/15 • Number of events 13
|
|
Gastrointestinal disorders
Diarrhea
|
33.3%
5/15 • Number of events 7
|
|
Gastrointestinal disorders
Dry mouth
|
6.7%
1/15 • Number of events 1
|
|
Gastrointestinal disorders
Flatulence
|
6.7%
1/15 • Number of events 1
|
|
Gastrointestinal disorders
Gastritis
|
6.7%
1/15 • Number of events 1
|
|
Gastrointestinal disorders
Nausea
|
33.3%
5/15 • Number of events 8
|
|
Gastrointestinal disorders
Rectal pain
|
6.7%
1/15 • Number of events 1
|
|
Gastrointestinal disorders
Stomach pain
|
13.3%
2/15 • Number of events 2
|
|
Gastrointestinal disorders
Vomiting
|
26.7%
4/15 • Number of events 6
|
|
General disorders
Chest pain
|
13.3%
2/15 • Number of events 2
|
|
General disorders
Chills
|
6.7%
1/15 • Number of events 1
|
|
General disorders
Edema limbs
|
13.3%
2/15 • Number of events 2
|
|
General disorders
Fatigue
|
60.0%
9/15 • Number of events 17
|
|
General disorders
Localized edema
|
6.7%
1/15 • Number of events 1
|
|
General disorders
Pain
|
20.0%
3/15 • Number of events 5
|
|
Hepatobiliary disorders
Hepatic failure
|
6.7%
1/15 • Number of events 1
|
|
Investigations
Alanine aminotransferase increased
|
33.3%
5/15 • Number of events 5
|
|
Investigations
Alkaline phosphatase increased
|
20.0%
3/15 • Number of events 3
|
|
Investigations
Aspartate aminotransferase increased
|
33.3%
5/15 • Number of events 7
|
|
Investigations
Bilirubin increased
|
6.7%
1/15 • Number of events 1
|
|
Investigations
Creatinine increased
|
13.3%
2/15 • Number of events 2
|
|
Investigations
Laboratory test abnormal
|
6.7%
1/15 • Number of events 1
|
|
Investigations
Leukocyte count decreased
|
33.3%
5/15 • Number of events 7
|
|
Investigations
Lymphocyte count decreased
|
13.3%
2/15 • Number of events 2
|
|
Investigations
Platelet count decreased
|
13.3%
2/15 • Number of events 2
|
|
Investigations
Weight gain
|
6.7%
1/15 • Number of events 1
|
|
Investigations
Weight loss
|
26.7%
4/15 • Number of events 4
|
|
Metabolism and nutrition disorders
Anorexia
|
60.0%
9/15 • Number of events 12
|
|
Metabolism and nutrition disorders
Blood bicarbonate decreased
|
6.7%
1/15 • Number of events 1
|
|
Metabolism and nutrition disorders
Blood glucose increased
|
33.3%
5/15 • Number of events 7
|
|
Metabolism and nutrition disorders
Dehydration
|
6.7%
1/15 • Number of events 1
|
|
Metabolism and nutrition disorders
Serum albumin decreased
|
13.3%
2/15 • Number of events 2
|
|
Metabolism and nutrition disorders
Serum calcium decreased
|
13.3%
2/15 • Number of events 2
|
|
Metabolism and nutrition disorders
Serum magnesium decreased
|
6.7%
1/15 • Number of events 1
|
|
Metabolism and nutrition disorders
Serum phosphate decreased
|
6.7%
1/15 • Number of events 1
|
|
Metabolism and nutrition disorders
Serum potassium decreased
|
26.7%
4/15 • Number of events 7
|
|
Metabolism and nutrition disorders
Serum potassium increased
|
6.7%
1/15 • Number of events 1
|
|
Metabolism and nutrition disorders
Serum sodium decreased
|
46.7%
7/15 • Number of events 8
|
|
Metabolism and nutrition disorders
Serum sodium increased
|
6.7%
1/15 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
20.0%
3/15 • Number of events 4
|
|
Musculoskeletal and connective tissue disorders
Chest wall pain
|
6.7%
1/15 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Joint pain
|
6.7%
1/15 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
6.7%
1/15 • Number of events 2
|
|
Nervous system disorders
Dizziness
|
6.7%
1/15 • Number of events 1
|
|
Nervous system disorders
Headache
|
6.7%
1/15 • Number of events 1
|
|
Nervous system disorders
Taste alteration
|
6.7%
1/15 • Number of events 1
|
|
Nervous system disorders
Tremor
|
6.7%
1/15 • Number of events 1
|
|
Psychiatric disorders
Anxiety
|
6.7%
1/15 • Number of events 1
|
|
Psychiatric disorders
Insomnia
|
6.7%
1/15 • Number of events 1
|
|
Reproductive system and breast disorders
Vaginal mucositis
|
6.7%
1/15 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm
|
6.7%
1/15 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
20.0%
3/15 • Number of events 3
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
6.7%
1/15 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
6.7%
1/15 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory disorder
|
6.7%
1/15 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Voice alteration
|
6.7%
1/15 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Decubitus ulcer
|
6.7%
1/15 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
6.7%
1/15 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Rash desquamating
|
13.3%
2/15 • Number of events 2
|
|
Skin and subcutaneous tissue disorders
Skin disorder
|
13.3%
2/15 • Number of events 2
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60