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neratinib

Phase 3

Breast Cancer | Small molecule | Oncology |Puma Biotechnology Inc|Last Updated: May 11, 2026

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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials7
Total Enrollment3,705
FDA Designations
No designations recorded
Clinical trial landscape

neratinib · 35 trials · 32 indications

Phase 3 2Phase 2 11Phase 1 22
NCT01808573A Study of Neratinib Plus Capecitabine Versus Lapatinib Plus Capecitabine in Patients With HER2+ Metastatic Breast Cancer Who Have Received Two or More Prior HER2 Directed Regimens in the Metastatic SettingHER2+ Metastatic Breast Cancer (MBC)
COMPLETED621 Analytics
NCT00878709Study Evaluating The Effects Of Neratinib After Adjuvant Trastuzumab In Women With Early Stage Breast CancerBreast Cancer
COMPLETED2,840 Analytics
PHASE3COMPLETED
A Study of Neratinib Plus Capecitabine Versus Lapatinib Plus Capecitabine in Patients With HER2+ Metastatic Breast Cancer Who Have Received Two or More Prior HER2 Directed Regimens in the Metastatic Setting
HER2+ Metastatic Breast Cancer (MBC)Unlock trial analytics
PHASE3COMPLETED
Study Evaluating The Effects Of Neratinib After Adjuvant Trastuzumab In Women With Early Stage Breast Cancer
Breast CancerUnlock trial analytics
Study Endpoints
Primary Endpoints
Centrally Assessed Progression Free Survival
From randomization date to recurrence, progression or death, assessed up to 38 months. The result is based on primary analysis data cut.

Progression Free Survival (PFS), Measured in Months, for Randomized Subjects of the Central Assessment. The time interval from the date of randomization until the first date on which recurrence, progression (per Response Evaluation Criteria in Solid Tumors Criteria (RECIST) v1.1), or death due to any cause, is documented. For subjects without recurrence, progression or death, it is censored at the last valid tumor assessment. Progression is defined using Response Evaluation Criteria in Solid Tumors Criteria (RECIST v1.1), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions. Here, the time to event was reported as the restricted mean survival time. The restricted mean survival time was defined as the area under the curve of the survival function up to 24 months.

Overall Survival
From randomization date to death, assessed up to 59 months.The result is based on primary analysis data cut.

Overall survival (OS) is defined as the time from randomization to death due to any cause, censored at the last date known alive on or prior to the data cutoff employed for the analysis, whichever was earlier. Here, the time to event was reported as the restricted mean survival time. The restricted mean survival time was defined as the area under the curve of the survival function up to 48 months.

Invasive Disease-free Survival (iDFS) in Neratinib Arm Compared to Placebo Arm at Year 2
From randomization until time of event up to 2 years

Invasive disease-free survival time is defined as the time from date of randomization until the first disease recurrence of the following events: invasive ipsilateral breast tumor recurrence, invasive contralateral breast cancer, local/regional invasive recurrence, distant recurrence and death from any cause.

Kaplan-Meier Estimates of Invasive Disease-free Survival (iDFS) at Year 2 by Treatment Arms
From randomization until time of event up to 2 years
Percentage of Participants With Grade 3 or Greater Diarrhea
Up to 6 weeks

Percentage of participants with clinically assessed grade 3 or greater diarrhea reported within the first 2 cycles (each cycle is 21 days) of neratinib while using anti-diarrheal strategies. Reports of diarrhea will be graded according to NCI CTCAE version 4.0.

Percentage of Participants Who Did Not Require Loperamide During Multiple Cycles of Treatment
Up to 55 weeks

Percentage of participants who did not require loperamide during cancer treatment for at least 5 cycles will be reported.

Percentage of Participants Who Discontinued Anti-diarrheal Medications for Multiple Cycles of Treatment
Up to 55 weeks

The percentage of participants who discontinued all anti-diarrheal medications during cancer treatment for at least 5 cycles will be reported.

Percentage of Participants With Treatment-related Adverse Events
Up to 55 weeks

Percentage of participants with reported serious adverse events and adverse events of interest of any grade that have been determined to be related to the anti-diarrhea treatment will be reported by worst grade and associated toxicity.

Number of Participants With Neratinib Dose Holds
Up to 55 weeks

The number of participants who experienced a dose hold of neratinib during the course of study therapy will be reported

Number of Participants Who Discontinued Neratinib Early
Up to 55 weeks

The number of participants who discontinued neratinib earlier than expected during the course of study therapy will be reported

Number of Participants With Neratinib Dose-reductions
Up to 55 weeks

The number of participants whose dose was reduced at any time during the course of therapy will be reported

Part I Only: Clinical Benefit Rate (CR+PR+SD≥6months) of Patients Who Received Neratinib Alone
Through completion of treatment (median treatment time of 90 days, full range 54-716 days)

* Imaging for clinical benefit rate occurred at baseline (for an initial comparison scan) and every 2 cycles (28 day length) thereafter. * 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. Disappearance of all non-target lesions and normalization of tumor marker level. * Partial response (PR): At least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. * 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. Persistence of one or more non-target lesion(s) and/or maintenance of tumor marker level above the normal limits. Must have had stable disease for at least 6 months.

Part II ER-cohort Only: Clinical Benefit Rate (CR+PR+SD≥6months) of Neratinib in Patients With Metastatic HER2-, ER- Breast Cancer That Carry HER2 Mutation
Through completion of treatment (median treatment time of 62 days, full range 56-413 days)

* Imaging for clinical benefit rate occurred at baseline (for an initial comparison scan) and every 2 cycles (28 day length) thereafter. * 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. Disappearance of all non-target lesions and normalization of tumor marker level. * Partial response (PR): At least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. * 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. Persistence of one or more non-target lesion(s) and/or maintenance of tumor marker level above the normal limits. Must have had stable disease for at least 6 months.

Part II Fulvestrant-naive ER+ Cohort Only: Clinical Benefit (CR+PR+SD≥6 Months) of Neratinib + Fulvestrant in Patients With Metastatic HER2- ER+ Fulvestrant-naive Breast Cancer That Carry HER2 Mutation
Through completion of treatment (median treatment time of 140.5 days, full range 48-770 days)

* Imaging for clinical benefit rate occurred at baseline (for an initial comparison scan) and every 2 cycles (28 day length) thereafter. * 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. Disappearance of all non-target lesions and normalization of tumor marker level. * Partial response (PR): At least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. * 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. Persistence of one or more non-target lesion(s) and/or maintenance of tumor marker level above the normal limits. Must have had stable disease for at least 6 months.

Part II Fulvestrant-treated ER+ Cohort Only: Clinical Benefit Rate (CR+PR+SD≥6months) of Neratinib + Fulvestrant in Patients With Metastatic HER2- ER+ Fulvestrant-treated Breast Cancer That Carry HER2 Mutation
Through completion of treatment (median treatment time of 168 days, full range 28-671 days)

* Imaging for clinical benefit rate occurred at baseline (for an initial comparison scan) and every 2 cycles (28 day length) thereafter. * 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. Disappearance of all non-target lesions and normalization of tumor marker level. * Partial response (PR): At least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. * 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. Persistence of one or more non-target lesion(s) and/or maintenance of tumor marker level above the normal limits. Must have had stable disease for at least 6 months.

The incidence of neratinib discontinuations due to diarrhoea at the end of 3 cycles (1 cycle= 28 days) of neratinib treatment.
Up to 3 months

The proportion of patients who discontinue the treatment with neratinib due to diarrhoea within this time period.

Pathologic Complete Response (pCR)
24 weeks

pCR is defined as the lack of all signs of invasive cancer in the breast removed during surgery

Changes in Colon Pathology
From baseline to second colonoscopy, which is 88 days after start of neratinib treatment.

The primary endpoint is to describe the changes in colon pathology between the baseline colonoscopy and the second colonoscopy.

Overall Survival in Experimental Arms Compared with Standard Therapy
2 years
Percentage of Patients With Grade 3 or Higher Diarrhea, According to NCI CTCAE v4.0.
From first dose of investigational product through 28 days after last dose, up to 15.5 months.

The primary objective of this study is to characterize the percentage of patients with Grade 3 or higher diarrhea in patients with early-stage HER2 overexpressed/amplified (HER2+) breast cancer treated with neratinib when administered with intensive loperamide prophylaxis, after prior treatment with trastuzumab. Grade 3: Increase of \>=7 stools per day over baseline; incontinence; hospitalization indicated; severe increase in ostomy output compared to baseline; limiting self care ADL. Grade 4: Life-threatening consequences; urgent intervention indicated. Grade 5: Death.

Objective Response Rate (ORR)
From randomization to last tumor assessment, assessed up to 116.5 weeks. For the Neratinib arm, only tumor assessments prior to crossover were included.

ORR is defined as proportion of subjects who achieved confirmed complete response (CR) or partial response (PR) per Response Evaluation Criteria In Solid Tumors Criteria (RECIST) v1.1: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR. A complete or partial response must be confirmed no less than 4-weeks after the criteria for response are initially met.

Progression-Free Survival
From randomization to disease progression or death, assessed up to 5.3 years

Defined as the interval from the date of randomization until the first date on which recurrence or progression, or death due to any cause, is documented, censored at the last assessable evaluation or at the initiation of new anticancer therapy.

Progression Free Survival
From randomization date to progression or death, assessed up to 69 months

Progression Free Survival, Measured in Months, for Subjects Randomized. Investigator assessment. The time interval from the date of randomization until the earliest date of progression per Response Evaluation Criteria In Solid Tumors Criteria (RECIST) or death due to any cause. For subjects without death or progression, censorship was at the last valid tumor assessment.

16-week Progression Free Survival
From first dose to 16 weeks

16 week progression-free survival (PFS) rate of neratinib in women with human epidermal growth factor receptor 2 (HER2) positive breast cancer, either with prior trastuzumab or no prior trastuzumab therapy, evaluated by independent assessment of tumor scans collected at baseline and then every 8 weeks.

Phase 1: To determine the Recommended phase 2 dose (RP2D) of niraparib and neratinib in patients with advanced solid tumors
4 Months

The RP2D will be identified during the phase 1 dose escalation portion of the study using a modified 3+3 design and evaluated in a phase 1b dose expansion cohort of up to 12 patients with platinum-resistant ovarian cancer.

Phase 1b: To evaluate clinical benefit (≥4-month progression-free survival [PFS]) of niraparib and neratinib in patients with platinum-resistant ovarian cancer.
4 months

Phase 1b: To evaluate clinical benefit (≥4-month progression-free survival \[PFS\]) of niraparib and neratinib given at the RP2D to in patients with platinum-resistant ovarian cancer. To evaluate the clinical benefit, (defined as ≥4-month progression free survival (PFS), using Response Evaluation Criteria in Solid Tumors (RECIST 1.1) criteria in patients with platinum-resistant ovarian cancer.

Determination of Recommended Phase 2 Dose (RP2D)
28 Days

RP2D for the combination of neratinib and sodium valproate that is less than or the same as the maximum tolerated dose (MTD).

AUC(0-t) of neratinib with and without lansoprazole
0, 0.5, 1, 2, 2.75, 3.5, 4.25, 5, 5.75, 6.5, 7.25, 8, 10, 12, 24, 32, 48, 60, and 72 hours post-dose

The area under the plasma concentration-time curve, from time 0 to the last measurable nonzero concentration, as calculated by the linear trapezoidal method.

AUC(0-inf) of neratinib with and without lansoprazole
0, 0.5, 1, 2, 2.75, 3.5, 4.25, 5, 5.75, 6.5, 7.25, 8, 10, 12, 24, 32, 48, 60, and 72 hours post-dose

The area under the plasma concentration-time curve from time 0 extrapolated to infinity. AUC(0-inf) is calculated as the sum of AUC(0-t) plus the ratio of the last measurable plasma concentration to the elimination rate constant.

Cmax of neratinib with and without lansoprazole
0, 0.5, 1, 2, 2.75, 3.5, 4.25, 5, 5.75, 6.5, 7.25, 8, 10, 12, 24, 32, 48, 60, and 72 hours post-dose

Maximum observed concentration.

Dose Limiting Toxicity (DLT) - Percentage of Participants With DLT Events
From first dose date to day 21.

DLT was defined as 1. Grade 3 or 4 non-hematologic toxicity (exceptions listed below as a.-c.), a. Grade 3 asthenia was NOT considered to be a DLT UNLESS it lasted \>3 days. b. Grade 3 nausea or vomiting was NOT considered to be a DLT UNLESS the subject was already receiving optimal medical therapy. c. Grade 3 or 4 infection was NOT considered to be a DLT UNLESS it is associated with grade 3 or 4 neutropenia. 2. Grade 3 diarrhea that lasted \>2 days while the subject was on optimal medical therapy or that was associated with fever (greater than or equal 38.0 ºC) or grade 3 dehydration. 3. Grade 4 neutropenia lasting ≥3 days or grade 4 febrile neutropenia. 4. Grade 4 thrombocytopenia lasting ≥3 days or complicated with bleeding or requiring platelet transfusion. 5. Delayed recovery (to National Cancer Institute \[NCI\] grade 1 or less, or baseline) from any of the toxicities listed above (items 1-4), that was related to study drug, and that delayed the next dose by more than 3 weeks.

Tolerated Dose
From first dose date to day 21.

Six subjects will be initially enrolled (neratinib 240 mg/day; capecitabine 1500 mg/m²/day on days 1 through 14). AEs and DLTs will be assessed from the first dose of investigational product through day 21. Based on the DLT rate in these first 6 subjects, dose tolerability will be confirmed as follows: If ≤1 of the first 6 evaluable subjects experience a DLT by day 21, then this dose is considered tolerable, and enrollment will stop. If ≥3 of the first 6 evaluable subjects experience a DLT by day 21, this dose is considered intolerable. If 2 of the first 6 evaluable subjects experience a DLT by day 21, then an additional 4 subjects will be enrolled at the same dose level. If a total of 10 subjects are enrolled, then the tolerability will be confirmed as follows: If ≤3 of the total 10 subjects experience a DLT by day 21, then this dose will be considered tolerable. If ≥4 of the total 10 subjects experience a DLT by day 21, then the dose will be considered intolerable.

Objective Response Rate (ORR) (Phase II)
From enrollment date to first documented response, or last tumor assessment, assessed up to two years

ORR is defined as proportion of subjects who achieved confirmed complete response (CR) or partial response (PR) per Response Evaluation Criteria In Solid Tumors Criteria (RECIST) v1.1: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR. A complete or partial response must be confirmed no less than 4-weeks after the criteria for response are initially met.

Dose Limiting Toxicity (DLT)
From first dose date to 21st day

Number of participants experiencing DLT of neratinib in combination with vinorelbine in Japanese patients.

Pharmacokinetics (plasma blood concentrations)
4 weeks
Pharmacokinetic parameters: concentration of digoxin in blood and urine
5 days
Probability of Dose-Limiting Toxicity (DLT)
From first dose date to day 28

A DLT was defined as any dose-limiting Adverse Event related to neratinib + Temsirolimus as Grade 3 or higher nonhematologic toxicity (except neutropenia) or Grade 3 or higher diarrhea lasting \>2 days with optimal antidiarrheal therapy etc.

Maximum Tolerated Dose (MTD) of Neratinib in Combination With Temsirolimus
From first dose date to day 28

Identification of the daily neratinib high-dose MTD in combination with weekly temsirolimus.

Maximum Tolerated Dose (MTD) of Temsirolimus in Combination With Neratinib
From first dose date to day 28

Identification of the weekly temsirolimus high-dose MTD in combination with daily neratinib

Adverse Events Causing Dose Limiting Toxicities
From first dose date to day 21

A DLT was defined as any dose-limiting adverse event (AE) related to neratinib + TEMSR as follows: \[1\] Grade 3 or 4 nonhematologic toxicity (Grade 3 or 4 nausea, vomiting, hyperglycemia, hypophosphatemia, hypertriglyceridemia, or hypercholesterolemia was not considered a DLT unless the subject was already receiving optimal medical therapy). \[2\] Grade 3 or 4 diarrhea lasting \>2 days while subject was on optimal vigorous antidiarrheal therapy. \[3\] Grade 4 neutropenia lasting \>3 days or Grade 3 or 4 neutropenia of any duration with sepsis or a fever \>38.5C. \[4\] Platelet value less than or equal to 25,000/mm3 or bleeding requiring a platelet transfusion. \[5\] Delayed recovery from toxicity, which delayed rescheduled re-treatment for \>3 weeks. \[6\] Inability to maintain the original dose during the first 28 days of treatment (at least 21 doses of neratinib and 2 doses of TEMSR at the original specified dose) due to treatment-related toxicity.

Plasma concentrations of neratinib in healthy volunteers compared to hepatically impaired patients through analysis of blod samples collected after neratinib administration in these 2 populations.
4 weeks
Blood will be collected to determine the pharmacokinetics of the various formulations of HKI-272
6 weeks
Number of Participants With Dose Limiting Toxicities
From first dose date to day 21

Number of participants reporting Adverse Events Causing Dose Limiting Toxicities (DLT).

Maximum Tolerated Dose (MTD) of Neratinib
From first dose date to day 21.

MTD reflects the highest dose of neratinib plus capeciteabine that did not cause a selected Grade 3 toxicity in \>= 2 participants, which is any of 1) Grade 3 or 4 non-hematologic toxicity (Grade 3 asthenia was not considered a DLT unless lasting \>3 days, 2) Grade 3 diarrhea lasting \>2 days on optimal medical therapy or associated with fever or dehydration. 3) Grade 4 neutropenia lasting ≥ 3 days or Grade 4 febrile neutropenia, 4) Grade 4 thrombocytopenia lasting ≥3 days or associated with bleeding or requiring platelet transfusion, 5) Delayed recovery \[to ≥ National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) Grade 1 or baseline\] from one of the above listed toxicities that were related to neratinib and/or capecitabine that delayed the initiation of the next dose by more than 3 weeks.

Maximum Tolerated Dose (MTD) of Capecitabine
From first dose date to day 21.

MTD reflects the highest dose of capecitabine in combination with neratinib that did not cause a selected Grade 3 toxicity in \>= 2 participants, which is any of 1) Grade 3 or 4 non-hematologic toxicity (Grade 3 asthenia was not considered a DLT unless lasting \>3 days, 2) Grade 3 diarrhea lasting \>2 days on optimal medical therapy or associated with fever or dehydration. 3) Grade 4 neutropenia lasting ≥ 3 days or Grade 4 febrile neutropenia, 4) Grade 4 thrombocytopenia lasting ≥3 days or associated with bleeding or requiring platelet transfusion, 5) Delayed recovery \[to ≥ National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) Grade 1 or baseline\] from one of the above listed toxicities that were related to neratinib and/or capecitabine that delayed the initiation of the next dose by more than 3 weeks.

Maximum Tolerated Dose
From first dose day through day 28.

The maximum tolerated dose of neratinib, as determined by the incidence of DLTs, in combination with paclitaxel 80 mg/m\^2, in subjects with advanced solid tumors.

Occurence of diarrhea
14 days
QTc interval
3 days
Overall Response Rate
From first dose date to progression or last tumor assessment, up to four years and six months.

Overall Response Rate (ORR), subjects with CR or PR by independent review in subjects with ErbB-2-positive breast cancer treated at the MTD of neratinib in combination with vinorelbine per Response Evaluation Criteria In Solid Tumors Criteria (RECIST) v1.0: Complete Response (CR), disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; and Non-PD for non-target lesions, and no new lesions.

Mass balance, metabolic distribution, PK
10 days
The data from this study along with in vitro data will be used to explore in vitro/in vivo correlation for HKI-272 to support formulation development.
Maximum Tolerated Dose (MTD)
First dose date through 21 days

MTD is defined as the prior dose level of the dose level which has \>=2 of 3 to 6 subjects that experience a neratinib-related DLT during 21 days from first dose date. A DLT is defined as any HKI-272-related nonhematologic grade 3 or any grade 4 AE according to the Common Terminology Criteria for Adverse Events version 3.0 except: Grade 3 nausea, vomiting, diarrhea, or rash unless subject was receiving appropriate medical therapy.

Pharmacokinetics; safety and tolerability
Pharmacokinetics; safety and tolerability; influence of food on the same.
Secondary Endpoints
Intervention for Symptomatic Metastatic Central Nervous System Disease
From randomization date to first intervention for symptomatic metastatic CNS disease, assessed up to 59 months.The result is based on primary analysis data cut.
Objective Response Rate (ORR) - Central Assessment (ITT Population With Measurable Disease at Screening)
From randomization date to first confirmed Complete or Partial Response, whichever came earlier, up to 42 months.The result is based on primary analysis data cut.
Clinical Benefit Rate (CBR) - Central Assessment (ITT Population With Measurable Disease at Screening)
From randomization date to either first confirmed CR or PR or Stable Disease, whichever came earlier, up to 42 months.The result is based on primary analysis data cut.
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
neratinib plus capecitabineEXPERIMENTALneratinib 240 mg orally, once daily with food, continuously in 21 day cycles, and capecitabine 1500 mg/m\^2 daily in 2 evenly divided doses, orally with water within 30 minutes after a meal, taken on days 1 to 14 of each 21 day cycle.
lapatinib plus capecitabineACTIVE_COMPARATORlapatinib 1250 mg orally, once daily, continuously in 21 day cycles, and capecitabine 2000 mg/m\^2 daily in 2 evenly divided doses, orally with water within 30 minutes after a meal, taken on days 1 to 14 of each 21 day cycle.
NeratinibEXPERIMENTAL240 mg orally daily for one year
PlaceboPLACEBO_COMPARATORorally daily for one year
Adjuvant Neratinib, Crofelemer, LoperamideEXPERIMENTALParticipants will receive 240mg neratinib to be taken continuously in 21-day cycles once a day for up to 55 weeks on study with no rest between cycles unless related to toxicity. Participants will receive Neratinib and may also be prescribed standard of care maintenance adjuvant trastuzumab (duration of maintenance trastuzumab is at the discretion of the treating physician), for up to 55 weeks. If applicable, after the completion of trastuzumab maintenance therapy (determined by treating physician), neratinib may continue as monotherapy to complete a maximum of 55 weeks. Participants will also receive 125mg of prophylactic Crofelemer and 4 mg of prophylactic loperamide as needed in the first 2 cycles.
Adjuvant Neratinib, LoperamideEXPERIMENTALParticipants will receive 120 mg of neratinib on days 1-7 with subsequent increase in dose by 40 mg every 7 days until the full dose of 240 mg a day is reached within the first cycle (up to 240mg) to be taken continuously in 21-day cycles once a day for up to 55 weeks on study with no rest between cycles unless related to toxicity. Participants will receive Neratinib and may also be prescribed standard of care maintenance adjuvant trastuzumab (duration of maintenance trastuzumab is at the discretion of the treating physician), for up to 55 weeks. If applicable, after the completion of trastuzumab maintenance therapy (determined by treating physician), neratinib may continue as monotherapy to complete a maximum of 55 weeks. Participants will also receive 4 mg of prophylactic loperamide to be taken as needed.
Part I: Neratinib OnlyEXPERIMENTAL-Patients will receive neratinib PO daily on Days 1-28. Each cycle is 4 weeks. Treatment continues in the absence of disease progression or unacceptable toxicity.
Part II: Neratinib Only (ER-)EXPERIMENTAL-Patients will receive neratinib PO daily on Days 1-28. Each cycle is 4 weeks. Treatment continues in the absence of disease progression or unacceptable toxicity.
Part II: Neratinib + Fulvestrant (ER+, fulvestrant-naive)EXPERIMENTAL-Patients will receive neratinib PO daily on Days 1-28 and fulvestrant on Day 1 of each cycle (and C1D15). Each cycle is 4 weeks. Treatment continues in the absence of disease progression or unacceptable toxicity.
Part II: Neratinib + Fulvestrant (ER+. prior fulvestrant-tx)EXPERIMENTAL-Patients will receive neratinib PO daily on Days 1-28 and fulvestrant on Day 1 of each cycle (and C1D15). Each cycle is 4 weeks. Treatment continues in the absence of disease progression or unacceptable toxicity.
Crossover: Neratinib + TrastuzumabEXPERIMENTAL-If a participant experiences disease progression during Part I or Part II following neratinib, trastuzumab (or FDA approved biosimilar) may be added to the treatment regimen. Trastuzumab may be administered intravenously with a loading dose of 6 mg/kg then 4 mg/kg every 2 weeks. A cycle will be defined as 28 days. Patients will continue to receive neratinib PO daily on Days 1-28. Each cycle is 4 weeks.
Crossover: Neratinib + Fulvestrant + TrastuzumabEXPERIMENTAL-If a participant experiences disease progression during Part I or Part II following neratinib, trastuzumab (or FDA approved biosimilar) may be added to the treatment regimen. Trastuzumab may be administered intravenously with a loading dose of 6 mg/kg then 4 mg/kg every 2 weeks. A cycle will be defined as 28 days. Patients will receive neratinib PO daily on Days 1-28 and fulvestrant on Day 1 of each cycle (and C1D15). Each cycle is 4 weeks.
Arm AEXPERIMENTAL1. Neratinib 240 mg (six 40mg tablets) orally once daily for 13 cycles (C) (1 C = 28 days), unless patient discontinues earlier. 2. Mandatory loperamide 4 mg (2 tablets/capsules) orally, 3 times a day (total 12 mg a day) starting Day (D) 1 of neratinib and for the first 14 days. Then, 4 mg (2 tablets/capsules) orally, 2 times a day (total 8 mg a day) until the end of C2 (D56); thereafter, loperamide will be administered PRN (without exceeding 16 mg per day).
Arm BEXPERIMENTAL1. Neratinib 120 mg for Week 1 (C1D1 - C1D7), followed by 160 mg neratinib for Week 2 (C1D8 - C1D14), followed by 240 mg neratinib for Week 3 and thereafter for 13 cycles inclusive, until cycle 13 day 28 (unless patient discontinues earlier). 2. Loperamide to be administered PRN only (without exceeding 16 mg per day).
Arm CEXPERIMENTAL1. Neratinib 240 mg (six 40-mg tablets) orally once daily for 13 C, unless patient discontinues earlier. 2. Mandatory loperamide 4 mg (2 tablets/capsules) orally, 3 times a day (total 12 mg a day) for the first 14 days. After the first 14 days, 4 mg (2 tablets/capsules) orally, 2 times a day (total 8 mg a day) to complete a total of 28 days. 3. Mandatory colesevelam 1,875 mg (three 625-mg capsules orally), 2 times a day for the first month (28 days). After day 28, any prophylaxis or treatment for diarrhoea could be administered PRN, if loperamide not to exceed 16 mg per day.
AEXPERIMENTALWeeks 1-3\* patients receive either (a) Neratinib, (b) Letrozole or Anastrozole or (c) Neratinib + Letrozole or Anastrozole Weeks 4-24 patients receive Neratinib + Letrozole or Anastrozole and Trastuzumab \*Starting drug intervention varies for the first 3 weeks depending on arms: a, b, and c by randomization.
TemozolomideACTIVE_COMPARATOR* Daily Radiation for a maximum of 49 days. * Temozolomide will be administered orally on a daily dosing schedule * Temozolomide will be administered approximately 2-3 hours before each session of radiotherapy * Temozolomide will also be administered post radiation for up to 6 cycles (5 days/cycle)
Neratinib with TemozolomideEXPERIMENTAL* Daily Radiation for a maximum of 49 days. * Temozolomide will be administered orally on a daily dosing schedule * Temozolomide will be administered approximately 2-3 hours before each session of radiotherapy * Neratinib will be taken post radiation at a daily oral pre-determine dose
QBS10072SEXPERIMENTALDaily Radiation for a maximum of 49 days. QBS10072S will be administered on Day 1 of Radiation Treatment QBS10072S will be administered post- radiation for up to 6 cycles
Abemaciclib with TemozolomideEXPERIMENTAL* Daily Radiation for a maximum of 49 days. * Temozolomide will be administered orally on a daily dosing schedule during radiation * Temozolomide will be administered approximately 2-3 hours before each session of radiotherapy * Abemaciclib will be taken post radiation at a twice daily oral pre-determined dose
CC-115EXPERIMENTAL* Twice daily oral dosing of CC-115 * Daily Radiation for a maximum of 49 days * CC115 will also be taken twice daily post radiation
LoperamideEXPERIMENTAL240 mg Neratinib orally once daily with food for thirteen 28-day cycles. Loperamide daily for two 28-day cycles and then as needed.
Budesonide and LoperamideEXPERIMENTAL240 mg Neratinib orally once daily with food for thirteen 28-day cycles. Anti-inflammatory treatment for 1 cycle and Loperamide to be administered daily for two 28-day cycles and then as needed, thereafter.
Colestipol and LoperamideEXPERIMENTAL240 mg Neratinib orally once daily with food for thirteen 28-day cycles. Colestipol for 1 cycle and loperamide to be administered 1 cycle and then as needed, thereafter.
Colestipol with Loperamide as neededEXPERIMENTAL240 mg Neratinib orally once daily with food for thirteen 28-day cycles. Colestipol for 1 cycle and loperamide to be administered as needed.
Neratinib Dose Escalation 1EXPERIMENTAL120 mg Neratinib for Week 1, followed by 160 mg Neratinib starting for Week 2, followed by 240 mg Neratinib starting at Week 3 and thereafter (C1D15 to End of Treatment). Loperamide administered as needed.
Neratinib Dose Escalation 2EXPERIMENTAL160 mg neratinib for the first 2 weeks, followed by 200 mg neratinib for the next 2 weeks, followed by 240 mg neratinib thereafter (C2D1 to End of treatment. Loperamide will be administered on an as-needed basis only.
neratinib monotherapyEXPERIMENTAL240 mg once daily with food, continuously in 21 day cycles
neratinib plus temsirolimusEXPERIMENTAL240 mg neratinib plus 8 mg temsirolimus IV with optional dose escalation to 15 mg temsirolimus
neratinib plus paclitaxelEXPERIMENTAL -
trastuzumab plus paclitaxelACTIVE_COMPARATOR -
Neratinib 240 mg, with prior trastuzumabEXPERIMENTALNeratinib administered with 80 mg capsules and 40 mg coated tablets taken orally in prescribed dose of 240 mg daily, as long as tolerated and disease does not worsen.
Neratinib 240 mg, no prior trastuzumabEXPERIMENTALNeratinib administered with 80 mg capsules and 40 mg coated tablets taken orally in prescribed dose of 240 mg daily, as long as tolerated and disease does not worsen.
Dose Level -1EXPERIMENTALNeratinib 160 mg and Niraparib 100 mg by mouth once daily for 28 day cycles.
Dose Level 1EXPERIMENTALNeratinib 160 mg and Niraparib 200 mg by mouth once daily for 28 day cycles.
Dose Level 2EXPERIMENTALNeratinib 200 mg and Niraparib 200 mg by mouth once daily for 28 day cycles.
Dose Level 3EXPERIMENTALNeratinib 240 mg and Niraparib 200 mg by mouth once daily for 28 day cycles.
Dose Level 4EXPERIMENTALNeratinib 240 mg and Niraparib 300 mg by mouth once daily for 28 day cycles.
Phase 1b: Platinum Resistant Expansion CohortEXPERIMENTALThis portion of the study provides for cohort expansion to observe for 4 month or greater progression-free survival in patients with platinum resistant ovarian cancer treated at the recommended phase 2 dose (RP2D) determined in Phase I.
Neratinib + Divalproex Sodium - Dose Escalation CohortEXPERIMENTALNeratinib by mouth (PO) once daily + Divalproex Sodium (Valproate) by mouth (PO) twice daily on days 1-28 of each course.
ColonEXPERIMENTALColon Cancer (RAS-mutated) - Phase II dose expansion at recommended phase II dose (RP2D)
Glioblastoma (GBM)EXPERIMENTALGlioblastoma with a RAS-mutation or EGFR alteration at RP2D
Ocular Melanoma (OM)EXPERIMENTALPhase II dose expansion at RP2D
Other CancerEXPERIMENTAL"Other Cancer" (RAS-mutated) at RP2D
Pancreatic CancerEXPERIMENTALRAS-mutated pancreatic cancer at RP2D
Treatment A (Period 1) and Treatment B (Period 2)EXPERIMENTALTreatment A (240mg Neratinib x1) Treatment B (30mg Lansoprazole X 7 days + 240mg Neratinib x1)
Neratinib + CapecitabineEXPERIMENTALNeratinib + Capecitabine
Temsirolimus plus NeratinibEXPERIMENTALThis is an open-label, single arm, dose-escalation phase I-II study to determine the maximum tolerated dose (MTD) of temsirolimus with daily neratinib, and to determine the safety and efficacy of this combination when given to patients with advanced breast carcinoma. Patients with trastuzumab-refractory HER2-amplified disease or triple negative disease will be enrolled in both phases of this clinical trial.
Neratinib and VinorelbineEXPERIMENTALNeratinib: 240 mg administered daily by mouth continuously, Vinorelbine: 25 mg/m\^2 administered IV on Day 1 and 8 of 21 day cycle
1EXPERIMENTALNeratinib alone
2EXPERIMENTALNeratinib plus rifampin
Neratinib and Temsirolimus (Dose level 1)EXPERIMENTALNeratinib 120 mg and Temsirolimus 15 mg: Neratinib 40 mg tablets administered orally daily for as long as tolerated and the disease under study does not worsen. Temsirolimus administered intravenously once weekly for as long as tolerated and the disease under study does not worsen
Neratinib and Temsirolimus (Dose level 2)EXPERIMENTALNeratinib 120 mg and Temsirolimus 25 mg: Neratinib 40 mg tablets administered orally daily for as long as tolerated and the disease under study does not worsen. Temsirolimus administered intravenously once weekly for as long as tolerated and the disease under study does not worsen
Neratinib and Temsirolimus (Dose level 3)EXPERIMENTALNeratinib 120 mg and Temsirolimus 50 mg: Neratinib 40 mg tablets administered orally daily for as long as tolerated and the disease under study does not worsen. Temsirolimus administered intravenously once weekly for as long as tolerated and the disease under study does not worsen
Neratinib and Temsirolimus (Dose level 4)EXPERIMENTALNeratinib 120 mg and Temsirolimus 75 mg: Neratinib 40 mg tablets administered orally daily for as long as tolerated and the disease under study does not worsen. Temsirolimus administered intravenously once weekly for as long as tolerated and the disease under study does not worsen
Neratinib and Temsirolimus (Dose level 5)EXPERIMENTALNeratinib 160 mg and Temsirolimus 15 mg: Neratinib 40 mg tablets administered orally daily for as long as tolerated and the disease under study does not worsen. Temsirolimus administered intravenously once weekly for as long as tolerated and the disease under study does not worsen
Neratinib and Temsirolimus (Dose level 6)EXPERIMENTALNeratinib 160 mg and Temsirolimus 25 mg: Neratinib 40 mg tablets administered orally daily for as long as tolerated and the disease under study does not worsen. Temsirolimus administered intravenously once weekly for as long as tolerated and the disease under study does not worsen
Neratinib and Temsirolimus (Dose level 7)EXPERIMENTALNeratinib 160 mg and Temsirolimus 50 mg: Neratinib 40 mg tablets administered orally daily for as long as tolerated and the disease under study does not worsen. Temsirolimus administered intravenously once weekly for as long as tolerated and the disease under study does not worsen
Neratinib and Temsirolimus (Dose level 8)EXPERIMENTALNeratinib 160 mg and Temsirolimus 75 mg: Neratinib 40 mg tablets administered orally daily for as long as tolerated and the disease under study does not worsen. Temsirolimus administered intravenously once weekly for as long as tolerated and the disease under study does not worsen
Neratinib and Temsirolimus (Dose level 9)EXPERIMENTALNeratinib 200 mg and Temsirolimus 15 mg: Neratinib 40 mg tablets administered orally daily for as long as tolerated and the disease under study does not worsen. Temsirolimus administered intravenously once weekly for as long as tolerated and the disease under study does not worsen
Neratinib and Temsirolimus (Dose level 10)EXPERIMENTALNeratinib 200 mg and Temsirolimus 25 mg: Neratinib 40 mg tablets administered orally daily for as long as tolerated and the disease under study does not worsen. Temsirolimus administered intravenously once weekly for as long as tolerated and the disease under study does not worsen
Neratinib and Temsirolimus (Dose level 11)EXPERIMENTALNeratinib 200 mg and Temsirolimus 50 mg: Neratinib 40 mg tablets administered orally daily for as long as tolerated and the disease under study does not worsen. Temsirolimus administered intravenously once weekly for as long as tolerated and the disease under study does not worsen
Neratinib and Temsirolimus (Dose level 12)EXPERIMENTALNeratinib 240 mg and Temsirolimus 15 mg: Neratinib 40 mg tablets administered orally daily for as long as tolerated and the disease under study does not worsen. Temsirolimus administered intravenously once weekly for as long as tolerated and the disease under study does not worsen
3EXPERIMENTAL80-mg capsule
Neratinib and Capecitabine (Dose Level 1)EXPERIMENTALNeratinib 160 mg and Capecitabine 1500 mg/m\^2
Neratinib and Capecitabine (Dose Group 2)EXPERIMENTALNeratinib 240 mg and Capecitabine 1500 mg/m\^2
Neratinib and Capecitabine (Dose Group 3)EXPERIMENTALNeratinib 240 mg and Capecitabine 2000 mg/m\^2
Neratinib and Capecitabine (Dose Group 4)EXPERIMENTALNeratinib 200 mg and Capecitabine 2000 mg/m\^2
Neratinib and Capecitabine (Dose Group 5)EXPERIMENTALNeratinib 160 mg and Capecitabine 2000 mg/m\^2
Neratinib and Capecitabine MTD (Dose Group 6)EXPERIMENTALNeratinib and Capecitabine Maximum Tolerated Dose without prior lapatinib
Neratinib and Capecitabine MTD (Dose Group 7)EXPERIMENTALNeratinib and Capecitabine Maximum Tolerated Dose with prior lapatinib
Nera 160 + PacEXPERIMENTALNeratinib 160 mg + Paclitaxel 80 mg/m\^2
Nera 240 + PacEXPERIMENTALNeratinib 240 mg + Paclitaxel 80 mg/m\^2
QDEXPERIMENTALOnce daily
BIDEXPERIMENTALTwice daily
neratinib 160 mg + vinorelbineEXPERIMENTALneratinib 160 mg tablets administered daily by mouth, vinorelbine 25 mg/m\^2 administered IV on day 1 and day 8 of 21 day cycle
neratinib 240 mg + vinorelbineEXPERIMENTALneratinib 240 mg tablets administered daily by mouth, vinorelbine 25 mg/m\^2 administered IV on day 1 and day 8 of 21 day cycle
neratinib 240 mg + vinorelbine, No Prior LapatinibEXPERIMENTALneratinib 240 mg tablets administered daily by mouth, vinorelbine 25 mg/m\^2 administered IV on day 1 and day 8 of 21 day cycle
neratinib 240 mg + vinorelbine, Prior LapatinibEXPERIMENTALneratinib 240 mg tablets administered daily by mouth, vinorelbine 25 mg/m\^2 administered IV on day 1 and day 8 of 21 day cycle
Neratinib 80 mgEXPERIMENTAL -
Neratinib 160 mgEXPERIMENTAL -
Neratinib 240 mgEXPERIMENTAL -
Neratinib 320 mgEXPERIMENTAL -
Neratinib 40 mgEXPERIMENTAL -
Neratinib 120 mgEXPERIMENTAL -
Neratinib 180 mgEXPERIMENTAL -
Neratinib 400 mgEXPERIMENTAL -
Neratinib 320 mg MTDEXPERIMENTAL -
Interventions
NameTypeDescription
neratinibDRUG -
capecitabineDRUG -
lapatinibDRUG -
placeboOTHER -
TrastuzumabBIOLOGICALGiven Intravenously (IV)
LoperamideDRUGGiven PO
CrofelemerDRUGGiven PO
Diphenoxylate/AtropineDRUGAllowed for participants experiencing refractory diarrhea
FulvestrantDRUG -
Tumor biopsyPROCEDURE-Optional at baseline and disease progression
Research blood samplePROCEDURE-Baseline, cycle 1 day 15, cycle 2 day 1, cycle 3 day 1, day 1 of each odd cycle, end of treatment (progression)
ColesevelamDRUGColesevelam capsules orally, 2 times a day for the first month (28 days).
Letrozole (L) or Anastrozole (A)DRUGL: (2.5 mg) OR A: (1 mg) orally daily (up to a maximum of 24 weeks)\*
TemozolomideDRUGTemzolomide capsules
QBS10072SDRUGQBS10072S administered intravenously
AbemaciclibDRUGAbemaciclib tablets
CC-115DRUGCC-115 tablets
ColestipolDRUG2 g twice daily with or without food for one 28 day cycle
BudesonideDRUG9 mg extended release tablets once daily with or without food for 28 days
temsirolimusDRUG -
PaclitaxelDRUGPaclitaxel - 80 mg/m² IV administered on days 1, 8, and 15 of a 28-day cycle. Treatment will be administered until documented disease progression, symptomatic deterioration, unacceptable toxicity, death or withdrawal of consent.
Neratinib 160 mgDRUGEscalating doses to determine recommended phase 2 dose (RP2D)
Neratinib 200 mgDRUGDetermined RP2D dose
Neratinib 240 mgDRUGEscalating doses to determine recommended phase 2 dose (RP2D)
Niraparib 100 mgDRUGEscalating doses to determine recommended phase 2 dose (RP2D)
Niraparib 200 mgDRUGEscalating doses to determine recommended phase 2 dose (RP2D)
Niraparib 300 mgDRUGPhase 1: Escalating doses to determine recommended phase 2 dose (RP2D)
Niraparib at RP2DDRUGPhase 1b: Determined dose
Neratinib at RP2DDRUGPhase 1b: Determined dose
Divalproex SodiumDRUGCombination of Neratinib and Divalproex Sodium (Valproate) will be given to patients with advanced solid tumors (dose escalation) and Ras-mutated cancers (dose expansion).
LansoprazoleDRUG -
VinorelbineDRUG -
DigoxinDRUG -
Neratinib (HKI-272)DRUGNeratinib 40-mg oral tablets. SINGLE DOSE of 120-mg (3 x 40-mg tablets)
MoxifloxacinDRUG -
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Eligibility Criteria
Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites252

Inclusion Criteria: * Aged ≥18 years at signing of informed consent. * Histologically confirmed MBC, current stage IV. * Documented HER2 overexpression or gene-amplified tumor immunohistochemistry 3+ or 2+, with confirmatory fluorescence in situ hybridization (FISH) +. * Prior treatment with at lea...

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Competitive Landscape -Breast Cancer 404 trials

Top 20 of 97 competitors

CompanyTickerTrialsLead PhaseDrugs
AstraZeneca PLCAZN47PHASE3Fulvestrant, Capivasertib
Merck & Co., Inc.MRK12PHASE3Pembrolizumab, Paclitaxel, Doxorubicin, Epirubicin, Cyclophosphamide
Eli Lilly and CompanyLLY27PHASE3Abemaciclib, Standard Adjuvant Endocrine Therapy
BioNTech SE Sponsored ADRBNTX7PHASE3DB-1303/BNT323, T-DM1
Gilead Sciences, Inc.GILD13PHASE3Sacituzumab Govitecan-hziy, Eribulin, Capecitabine Product, Gemcitabine, Vinorelbine
Novartis AG Sponsored ADRNVS30PHASE3Ribociclib
Olema Pharmaceuticals, Inc.OLMA5PHASE3Palazestrant, Fulvestrant, Anastrozole, Letrozole, Exemestane
Pfizer Inc.PFE34PHASE3ARV-471, Fulvestrant
BeOne Medicines Ltd. Sponsored ADRONC5PHASE3BGB-43395, Letrozole, Abemaciclib, Palbociclib, Ribociclib
Jazz Pharmaceuticals Public Limited CompanyJAZZ3PHASE3Zanidatamab, Trastuzumab, Eribulin, Vinorelbine, Gemcitabine
Celcuity Inc.CELC3PHASE3Gedatolisib, Palbociclib, Fulvestrant, Alpelisib
Relay Therapeutics, Inc.RLAY2PHASE3RLY-2608, Capivasertib, Fulvestrant
GSK plc Sponsored ADRGSK2PHASE3Niraparib
Greenwich LifeSciences, Inc.GLSI1PHASE3GLSI-100
Bristol-Myers Squibb CompanyBMY5PHASE2Iza-bren, Nab-paclitaxel, Paclitaxel, Capecitabine, Carboplatin
BriaCell Therapeutics CorpBCTX2PHASE3SV-BR-1-GM, Cyclophosphamide, Interferon infiltration of the inoculation site, Retifanlimab, Treatment of Physician's Choice
Incyte CorporationINCY4PHASE2Ruxolitinib, Capecitabine, Regorafenib
Natera, Inc.NTRA3PHASE2Discontinuation of the anti-HER2 maintenance therapy
Puma Biotechnology, Inc.PBYI3PHASE2Neratinib, Loperamide, Colesevelam
Immutep Ltd Sponsored ADRIMMP1PHASE2eftilagimod alpha, Paclitaxel
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT02977780primaryCompletionDate: changed
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