Trial Outcomes & Findings for Tamoxifen Citrate in Treating Patients With Metastatic or Recurrent Breast Cancer (NCT NCT01124695)

NCT ID: NCT01124695

Last Updated: 2023-06-29

Results Overview

Progression-free survival is defined as the time from registration to progression or death, whichever occurs first. Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1) was used to evaluate progression. Progression is defined as appearance of one or more new lesions or unequivocal progression of existing non-target lesions or 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.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 124 participants
• Primary outcome timeframe: Assessed every 3 months for 2 years, then every 6 months up to 5 years
• Results posted on: 2023-06-29

Participant Flow

The study was activated on September 21, 2010, accrued its first patient on January 7, 2011, and closed on October 22, 2015 for a total of 124 patients enrolled.

Participant milestones

Measure	Tamoxifen Patients receive oral tamoxifen citrate at 20 mg once daily on days 1-28. Treatment repeats every 28 days in the absence of disease progression or unacceptable toxicities.
Overall Study STARTED	124
Overall Study Treated	122
Overall Study Eligible and Treated	113
Overall Study Eligible and Treated and Had CYP2D6 Score	85
Overall Study Eligible and Treated and Had Cycle 3 Edoxifen Evaluation	63
Overall Study Eligible and Treated Patients With 3-month Endoxifen and Were Progression-free and Alive at 3 Months	43
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	123

Reasons for withdrawal

Measure	Tamoxifen Patients receive oral tamoxifen citrate at 20 mg once daily on days 1-28. Treatment repeats every 28 days in the absence of disease progression or unacceptable toxicities.
Overall Study Disease progression	77
Overall Study Adverse Event	11
Overall Study Death	1
Overall Study Withdrawal by Subject	7
Overall Study Physician Decision	3
Overall Study Alternative therapy	9
Overall Study No documentation that patient went off treatment	4
Overall Study Ineligible	9
Overall Study Ineligible and never start treatment	2

Baseline Characteristics

Tamoxifen Citrate in Treating Patients With Metastatic or Recurrent Breast Cancer

Baseline characteristics by cohort

Measure	Tamoxifen n=113 Participants Patients receive oral tamoxifen citrate at 20 mg once daily on days 1-28. Treatment repeats every 28 days in the absence of disease progression or unacceptable toxicities.
Age, Continuous	62 years n=5 Participants
Sex: Female, Male Female	111 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	33 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	77 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	3 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	7 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	6 Participants n=5 Participants
Race (NIH/OMB) White	97 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=5 Participants
CYP2D6 phenotype Poor metabolizer	2 Participants n=5 Participants
CYP2D6 phenotype Intermediate metabolizer	27 Participants n=5 Participants
CYP2D6 phenotype Normal metabolizer	50 Participants n=5 Participants
CYP2D6 phenotype Ultra-rapid metabolizer	6 Participants n=5 Participants
CYP2D6 phenotype Missing	28 Participants n=5 Participants

PRIMARY outcome

Timeframe: Assessed every 3 months for 2 years, then every 6 months up to 5 years

Population: Eligible and treated patients who had a sample from baseline evaluable for CYP2D6 phenotype

Progression-free survival is defined as the time from registration to progression or death, whichever occurs first. Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1) was used to evaluate progression. Progression is defined as appearance of one or more new lesions or unequivocal progression of existing non-target lesions or 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

Measure	CYP2D6 - PM n=2 Participants Patients with CYP2D6 score of 0 are considered as poor metabolizer (PM).	CYP2D6 - (IM+NM+UM) n=83 Participants Patients with CYP2D6 score \>0 are included in the IM+NM+UM group. Intermediate metabolizer (IM): CYP2D6 score \>0 and \<=1 Normal metabolizer (NM): CYP2D6 score \>1 and \<=2 Ultra-rapid metabolizer (UM): CYP2D6 score \>2	CYP2D6 - NM+UM Patients with CYP2D6 score \>1 are included in the NM+UM group. Normal metabolizer (NM): CYP2D6 score \>1 and \<=2 Ultra-rapid metabolizer (UM): CYP2D6 score \>2
Progression-free Survival by CYP2D6 Status in 2 Categories	12.9 months Only 2 patients were in this category. Among these two, one was censored at baseline. As only one patient contributed data, the 95% CI could not be calculated.	6.9 months Interval 4.4 to 8.5	—

SECONDARY outcome

Timeframe: Assessed every 3 months for 2 years, then every 6 months up to 5 years

Population: Eligible and treated patients who had a sample from baseline evaluable for CYP2D6 phenotype

Progression-free survival is defined as the time from registration to progression or death, whichever occurs first. Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1) was used to evaluate progression. Progression is defined as appearance of one or more new lesions or unequivocal progression of existing non-target lesions or 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

Measure	CYP2D6 - PM n=2 Participants Patients with CYP2D6 score of 0 are considered as poor metabolizer (PM).	CYP2D6 - (IM+NM+UM) n=27 Participants Patients with CYP2D6 score \>0 are included in the IM+NM+UM group. Intermediate metabolizer (IM): CYP2D6 score \>0 and \<=1 Normal metabolizer (NM): CYP2D6 score \>1 and \<=2 Ultra-rapid metabolizer (UM): CYP2D6 score \>2	CYP2D6 - NM+UM n=56 Participants Patients with CYP2D6 score \>1 are included in the NM+UM group. Normal metabolizer (NM): CYP2D6 score \>1 and \<=2 Ultra-rapid metabolizer (UM): CYP2D6 score \>2
Progression-free Survival by CYP2D6 Status in 3 Categories	12.9 months Only 2 patients were in this category. Among these two, one was censored at baseline. As only one patient contributed data, the 95% CI could not be calculated.	4.8 months Interval 2.7 to 13.8	7.4 months Interval 4.9 to 10.3

SECONDARY outcome

Timeframe: Assessed every 3 months for 6 months

Population: Eligible and treated patients who had a sample from baseline evaluable for CYP2D6 phenotype

Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1) was used to evaluate progression. Progression is defined as appearance of one or more new lesions or unequivocal progression of existing non-target lesions or 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

Measure	CYP2D6 - PM n=2 Participants Patients with CYP2D6 score of 0 are considered as poor metabolizer (PM).	CYP2D6 - (IM+NM+UM) n=83 Participants Patients with CYP2D6 score \>0 are included in the IM+NM+UM group. Intermediate metabolizer (IM): CYP2D6 score \>0 and \<=1 Normal metabolizer (NM): CYP2D6 score \>1 and \<=2 Ultra-rapid metabolizer (UM): CYP2D6 score \>2	CYP2D6 - NM+UM Patients with CYP2D6 score \>1 are included in the NM+UM group. Normal metabolizer (NM): CYP2D6 score \>1 and \<=2 Ultra-rapid metabolizer (UM): CYP2D6 score \>2
Proportion of Patients Progression-free at 6 Months	1.0 proportion of participants Interval 0.16 to 1.0	0.50 proportion of participants Interval 0.39 to 0.62	—

SECONDARY outcome

Timeframe: Assessed every 3 months for 2 years, then every 6 months up to 5 years

Population: Eligible and treated patients who had a sample from baseline evaluable for CYP2D6 phenotype

Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1) was used to evaluate response. Either complete response (CR) or partial response (PR) is considered as response. CR is defined as disappearance of all lesions. PR is defined as at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters.

Outcome measures

Measure	CYP2D6 - PM n=2 Participants Patients with CYP2D6 score of 0 are considered as poor metabolizer (PM).	CYP2D6 - (IM+NM+UM) n=83 Participants Patients with CYP2D6 score \>0 are included in the IM+NM+UM group. Intermediate metabolizer (IM): CYP2D6 score \>0 and \<=1 Normal metabolizer (NM): CYP2D6 score \>1 and \<=2 Ultra-rapid metabolizer (UM): CYP2D6 score \>2	CYP2D6 - NM+UM Patients with CYP2D6 score \>1 are included in the NM+UM group. Normal metabolizer (NM): CYP2D6 score \>1 and \<=2 Ultra-rapid metabolizer (UM): CYP2D6 score \>2
Proportion of Patients With Response	0 proportion of participants Interval 0.0 to 0.84	0.13 proportion of participants Interval 0.07 to 0.22	—

SECONDARY outcome

Timeframe: Endoxifen was assessed at cycle 3; response was assessed every 3 months for 2 years, then every 6 months up to 5 years

Population: Eligible and treated patients who had a sample at cycle 3 for endoxifen concentration evaluation

Endoxifen (ng/ml) was assessed at cycle 3.

Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1) was used to evaluate response. Either complete response (CR) or partial response (PR) is considered as response. CR is defined as disappearance of all lesions. PR is defined as at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters.

Outcome measures

Measure	CYP2D6 - PM n=6 Participants Patients with CYP2D6 score of 0 are considered as poor metabolizer (PM).	CYP2D6 - (IM+NM+UM) n=57 Participants Patients with CYP2D6 score \>0 are included in the IM+NM+UM group. Intermediate metabolizer (IM): CYP2D6 score \>0 and \<=1 Normal metabolizer (NM): CYP2D6 score \>1 and \<=2 Ultra-rapid metabolizer (UM): CYP2D6 score \>2	CYP2D6 - NM+UM Patients with CYP2D6 score \>1 are included in the NM+UM group. Normal metabolizer (NM): CYP2D6 score \>1 and \<=2 Ultra-rapid metabolizer (UM): CYP2D6 score \>2
Endoxifen Concentration by Response	17.5 ng/ml Interval 7.5 to 45.2	17.2 ng/ml Interval 2.0 to 67.9	—

SECONDARY outcome

Timeframe: Assessed every 3 months for 2 years, then every 6 months up to 5 years

Population: Eligible and treated patients who had endoxifen level assessed at 3 months and were progression-free and alive at 3 months from registration

This is a landmark progression-free survival analysis at 3 months post registration. Only patients who were progression-free and alive at 3 months were included.

Progression-free survival in this analysis is defined as the time from 3 months post registration to progression or death, whichever occurs first. Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1) was used to evaluate progression. Progression is defined as appearance of one or more new lesions or unequivocal progression of existing non-target lesions or 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

Measure	CYP2D6 - PM n=21 Participants Patients with CYP2D6 score of 0 are considered as poor metabolizer (PM).	CYP2D6 - (IM+NM+UM) n=22 Participants Patients with CYP2D6 score \>0 are included in the IM+NM+UM group. Intermediate metabolizer (IM): CYP2D6 score \>0 and \<=1 Normal metabolizer (NM): CYP2D6 score \>1 and \<=2 Ultra-rapid metabolizer (UM): CYP2D6 score \>2	CYP2D6 - NM+UM Patients with CYP2D6 score \>1 are included in the NM+UM group. Normal metabolizer (NM): CYP2D6 score \>1 and \<=2 Ultra-rapid metabolizer (UM): CYP2D6 score \>2
Progression-free Survival From 3 Months Post Registration	13.8 months Interval 6.5 to 16.6	11.1 months Interval 5.3 to 19.3	—

Adverse Events

Tamoxifen

Serious events: 30 serious events

Other events: 18 other events

Deaths: 69 deaths

Serious adverse events

Measure	Tamoxifen n=122 participants at risk Patients receive oral tamoxifen citrate at 20 mg once daily on days 1-28. Treatment repeats every 28 days in the absence of disease progression or unacceptable toxicities.
Blood and lymphatic system disorders Anemia	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
General disorders Fatigue	1.6% 2/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Investigations Aspartate aminotransferase increased	1.6% 2/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Investigations Lymphocyte count decreased	3.3% 4/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Investigations Platelet count decreased	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Metabolism and nutrition disorders Hypercalcemia	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Metabolism and nutrition disorders Hypertriglyceridemia	2.5% 3/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Musculoskeletal and connective tissue disorders Arthralgia	2.5% 3/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Musculoskeletal and connective tissue disorders Back pain	3.3% 4/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Musculoskeletal and connective tissue disorders Bone pain	3.3% 4/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Nervous system disorders Peripheral motor neuropathy	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Nervous system disorders Peripheral sensory neuropathy	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Vascular disorders Thromboembolic event	1.6% 2/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Ear and labyrinth disorders Hearing impaired	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Cardiac disorders Atrial fibrillation	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Cardiac disorders Heart failure	1.6% 2/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Cardiac disorders Pericardial tamponade	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
General disorders Death NOS	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
General disorders Edema limbs	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Skin and subcutaneous tissue disorders Skin ulceration	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Skin and subcutaneous tissue disorders Urticaria	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Gastrointestinal disorders Constipation	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Gastrointestinal disorders Diarrhea	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Gastrointestinal disorders Obstruction gastric	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Gastrointestinal disorders Pancreatitis	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Gastrointestinal disorders Vomiting	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Hepatobiliary disorders Cholecystitis	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Infections and infestations Joint infection	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Infections and infestations Sepsis	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Infections and infestations Skin infection	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Investigations Alanine aminotransferase increased	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Investigations Alkaline phosphatase increased	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Investigations Creatinine increased	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Investigations Neutrophil count decreased	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Investigations Weight gain	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Investigations White blood cell decreased	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Metabolism and nutrition disorders Acidosis	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Metabolism and nutrition disorders Glucose intolerance	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Metabolism and nutrition disorders Hyperglycemia	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Metabolism and nutrition disorders Hyperkalemia	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Metabolism and nutrition disorders Hypocalcemia	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Metabolism and nutrition disorders Hypoglycemia	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Metabolism and nutrition disorders Obesity	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Musculoskeletal and connective tissue disorders Muscle weakness lower limb	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Musculoskeletal and connective tissue disorders Pain in extremity	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Nervous system disorders Dizziness	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Nervous system disorders Headache	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms - Other	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Psychiatric disorders Confusion	1.6% 2/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Respiratory, thoracic and mediastinal disorders Dyspnea	2.5% 3/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Respiratory, thoracic and mediastinal disorders Respiratory failure	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Renal and urinary disorders Acute kidney injury	1.6% 2/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Renal and urinary disorders Chronic kidney disease	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Surgical and medical procedures Surgical and medical procedures - Other	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Vascular disorders Hypertension	2.5% 3/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Vascular disorders Hypotension	0.82% 1/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.

Other adverse events

Measure	Tamoxifen n=122 participants at risk Patients receive oral tamoxifen citrate at 20 mg once daily on days 1-28. Treatment repeats every 28 days in the absence of disease progression or unacceptable toxicities.
General disorders Fatigue	14.8% 18/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Vascular disorders Hot flashes	9.0% 11/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Blood and lymphatic system disorders Anemia	8.2% 10/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
General disorders Edema limbs	6.6% 8/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Gastrointestinal disorders Constipation	7.4% 9/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Gastrointestinal disorders Nausea	11.5% 14/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Gastrointestinal disorders Vomiting	7.4% 9/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Investigations Aspartate aminotransferase increased	6.6% 8/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Metabolism and nutrition disorders Anorexia	6.6% 8/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Musculoskeletal and connective tissue disorders Arthralgia	5.7% 7/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Musculoskeletal and connective tissue disorders Back pain	13.9% 17/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Musculoskeletal and connective tissue disorders Bone pain	6.6% 8/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Psychiatric disorders Anxiety	5.7% 7/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Psychiatric disorders Insomnia	5.7% 7/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Respiratory, thoracic and mediastinal disorders Dyspnea	6.6% 8/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.
Vascular disorders Hypertension	7.4% 9/122 • Assessed every 4 weeks while on treatment and for 30 days after the end of treatment up to 5 years All patients enrolled are included in all-cause mortality analysis. Patients who received treatment with adverse event data available are included in AE analysis. This is a single-arm study, so adverse events are reported for the Tamoxifen arm only, which is reported per arm.

Additional Information

Study Statistician

ECOG-ACRIN Statistical Office

Phone: 617-632-3012

Email: eatrials@jimmy.harvard.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place

Restriction type: LTE60