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NCT00770809

Paclitaxel and Trastuzumab With or Without Lapatinib in Treating Patients With Stage II or Stage III Breast Cancer That Can Be Removed by Surgery

Completed Phase 3 Results posted Last updated 15 August 2025
What this trial tests

Phase 3 trial testing Laboratory Biomarker Analysis in Male Breast Carcinoma in 305 participants. Completed in 15 February 2024.

Timeline
24 February 2009
Primary endpoint
31 January 2014
15 February 2024

Quick facts

Lead sponsorNational Cancer Institute (NCI)
PhasePhase 3
StatusCompleted
Study typeINTERVENTIONAL
Allocationrandomized
Designparallel
Maskingnone
Primary purposetreatment
Enrollment305
Start date24 February 2009
Primary completion31 January 2014
Estimated completion15 February 2024
Sites318 locations across Puerto Rico, United States

Drugs / interventions tested

Conditions studied

Sponsor

National Cancer Institute (NCI)

Who can join

18 and older, any sex, with Male Breast Carcinoma or Stage IIA Breast Cancer AJCC v6 and v7. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

pCR Rate Primary · At time of surgery

Complete pathological response is defined as the absence of residual invasive carcinoma in the breast at the time of definitive surgical removal. Pathologic complete response in the lymph nodes is defined as no detectable invasive tumor by H\&E. Analysis will use a chi-square test for the difference in proportions of patients on the THL arm versus the TH arm who achieve a pCR. Exact binomial methods will be used to construct 95% confidence intervals around the pCR incidence for each arm.

GroupValue95% CI
Arm I (THL)5647 – 65
Arm II (TH)4635 – 55
Arm III (TL)3222 – 45

Adverse events — posted to ClinicalTrials.gov

Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Arm I (THL)
Serious: 15/115 (13%)
Deaths:
Arm II (TH)
Serious: 12/115 (10%)
Deaths:
Arm III (TL)
Serious: 6/65 (9%)
Deaths:

Serious adverse events (41 terms)

ReactionSystemArm I (THL)Arm II (TH)Arm III (TL)
DiarrheaGastrointestinal disorders
FatigueGeneral disorders
Hemoglobin decreasedBlood and lymphatic system disorders
NauseaGastrointestinal disorders
Peripheral sensory neuropathyNervous system disorders
Neutrophil count decreasedInvestigations
Rash desquamatingSkin and subcutaneous tissue disorders
VomitingGastrointestinal disorders
Alanine aminotransferase increasedInvestigations
Serum potassium decreasedMetabolism and nutrition disorders
MyalgiaMusculoskeletal and connective tissue disorders
Urinary tract infectionInfections and infestations
Aspartate aminotransferase increasedInvestigations
Febrile neutropeniaBlood and lymphatic system disorders
Cardiopulmonary arrestCardiac disorders
PericarditisCardiac disorders
Abdominal painGastrointestinal disorders
ConstipationGastrointestinal disorders
PancreatitisGastrointestinal disorders
Edema limbsGeneral disorders
HypersensitivityImmune system disorders
Gallbladder infectionInfections and infestations
InfectionInfections and infestations
PneumoniaInfections and infestations
SepsisInfections and infestations
Other adverse events (131 terms — click to expand)

ReactionSystemArm I (THL)Arm II (TH)Arm III (TL)
FatigueGeneral disorders
DiarrheaGastrointestinal disorders
Peripheral sensory neuropathyNervous system disorders
Rash desquamatingSkin and subcutaneous tissue disorders
Hemoglobin decreasedBlood and lymphatic system disorders
NauseaGastrointestinal disorders
Neutrophil count decreasedInvestigations
MyalgiaMusculoskeletal and connective tissue disorders
VomitingGastrointestinal disorders
Alanine aminotransferase increasedInvestigations
HypersensitivityImmune system disorders
Aspartate aminotransferase increasedInvestigations
AlopeciaSkin and subcutaneous tissue disorders
Nail disorderSkin and subcutaneous tissue disorders
Serum potassium decreasedMetabolism and nutrition disorders
Hot flashesVascular disorders
Leukocyte count decreasedInvestigations
ConstipationGastrointestinal disorders
Edema limbsGeneral disorders
InsomniaPsychiatric disorders
EpistaxisRespiratory, thoracic and mediastinal disorders
Rash acneiformSkin and subcutaneous tissue disorders
Left ventricular failureCardiac disorders
DyspepsiaGastrointestinal disorders
Blood glucose increasedMetabolism and nutrition disorders
HeadacheNervous system disorders
PruritusSkin and subcutaneous tissue disorders
HypertensionVascular disorders
Mucositis oralGastrointestinal disorders
PainGeneral disorders
Platelet count decreasedInvestigations
AnorexiaMetabolism and nutrition disorders
DizzinessNervous system disorders
DyspneaRespiratory, thoracic and mediastinal disorders
Dry skinSkin and subcutaneous tissue disorders
Skin disorderSkin and subcutaneous tissue disorders
Vision blurredEye disorders
Abdominal painGastrointestinal disorders
Oral hemorrhageGastrointestinal disorders
ChillsGeneral disorders

Most-reported serious reactions: Diarrhea, Fatigue, Hemoglobin decreased, Nausea, Peripheral sensory neuropathy, Neutrophil count decreased, Rash desquamating, Vomiting.

Data from ClinicalTrials.gov NCT00770809 adverse events section.

Sponsor's own description

This randomized phase III trial studies paclitaxel and trastuzumab with or without lapatinib to see how well they work in treating patients with stage II or stage III breast cancer that can be removed by surgery. Drugs used in chemotherapy, such as paclitaxel, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Monoclonal antibodies, such as trastuzumab, can block tumor growth in different ways. Some block the ability of tumor cells to grow and spread. Others find tumor cells and help kill them or carry tumor-killing substances to them. Lapatinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving paclitaxel with trastuzumab and/or lapatinib before surgery may make the tumor smaller and reduce the amount of normal tissue that needs to be removed. It is not yet known which regimen is more effective in treating patients with breast cancer.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Molecular Heterogeneity and Response to Neoadjuvant Human Epidermal Growth Factor Receptor 2 Targeting in CALGB 40601, a Randomized Phase III Trial of Paclitaxel Plus Trastuzumab With or Without Lapatinib.
    Carey LA, Berry DA, Cirrincione CT, Barry WT, et al · · 2016 · cited 306× · PMID 26527775 · DOI 10.1200/jco.2015.62.1268
  2. Survival, Pathologic Response, and Genomics in CALGB 40601 (Alliance), a Neoadjuvant Phase III Trial of Paclitaxel-Trastuzumab With or Without Lapatinib in HER2-Positive Breast Cancer.
    Fernandez-Martinez A, Krop IE, Hillman DW, Polley MY, et al · · 2020 · cited 103× · PMID 33095682 · DOI 10.1200/jco.20.01276
  3. Interaction of host immunity with HER2-targeted treatment and tumor heterogeneity in HER2-positive breast cancer.
    Griguolo G, Pascual T, Dieci MV, Guarneri V, et al · · 2019 · cited 91× · PMID 30922362 · DOI 10.1186/s40425-019-0548-6
  4. HER2-targeted therapies in cancer: a systematic review.
    Zhu K, Yang X, Tai H, Zhong X, et al · · 2024 · cited 83× · PMID 38308374 · DOI 10.1186/s40364-024-00565-1
  5. Integrated Analysis of RNA and DNA from the Phase III Trial CALGB 40601 Identifies Predictors of Response to Trastuzumab-Based Neoadjuvant Chemotherapy in HER2-Positive Breast Cancer.
    Tanioka M, Fan C, Parker JS, Hoadley KA, et al · · 2018 · cited 71× · PMID 30037817 · DOI 10.1158/1078-0432.ccr-17-3431
  6. Prognostic and Predictive Value of Immune-Related Gene Expression Signatures vs Tumor-Infiltrating Lymphocytes in Early-Stage ERBB2/HER2-Positive Breast Cancer: A Correlative Analysis of the CALGB 40601 and PAMELA Trials.
    Fernandez-Martinez A, Pascual T, Singh B, Nuciforo P, et al · · 2023 · cited 63× · PMID 36602784 · DOI 10.1001/jamaoncol.2022.6288
  7. Drug-resistant HER2-positive breast cancer: Molecular mechanisms and overcoming strategies.
    Wu X, Yang H, Yu X, Qin JJ. · · 2022 · cited 46× · PMID 36210846 · DOI 10.3389/fphar.2022.1012552
  8. Factors Associated with Nodal Pathologic Complete Response Among Breast Cancer Patients Treated with Neoadjuvant Chemotherapy: Results of CALGB 40601 (HER2+) and 40603 (Triple-Negative) (Alliance).
    Weiss A, Campbell J, Ballman KV, Sikov WM, et al · · 2021 · cited 29× · PMID 33821344 · DOI 10.1245/s10434-021-09897-w

Verify or expand the search:

Other trials of Laboratory Biomarker Analysis

Trials testing the same drug.

Other recruiting trials for Male Breast Carcinoma

Currently open trials in the same condition.

Other National Cancer Institute (NCI) trials

Trials by the same sponsor.

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