ClinicalTrials.gg
CompletedNCT04366713Updated Feb 10, 2023Results posted

A Study to Characterize Colon Pathology in Patients With HER2 Amplified Breast Cancer Treated With Neratinib

A Phase 2 interventional study of Neratinib and Capecitabine in HER2 Amplified Breast Cancer, sponsored by Puma Biotechnology, Inc.. Completed at 1 site in Portugal. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2023-02-10.

Sponsored by Puma Biotechnology, Inc. · Phase 2, Interventional, and Other

Phase
Phase 2
Study type
Interventional
Enrollment
6
Allocation
Not applicable
Ages
18 Years and older
Sex
All
01

Study summary

This study will investigate colon pathology in patients with HER2-positive breast cancer treated with neratinib. Colonoscopy will be performed after eligibility has been confirmed, prior to administration of the first dose of neratinib, and after 28 days of neratinib treatment.

Read the detailed description

This is an open-label, phase 2 study that will investigate colon pathology in patients with HER2-positive breast cancer treated with neratinib as monotherapy.

All patients will receive neratinib for the first 28 days as a single daily dose of 240 mg.

Colonoscopy will be performed after eligibility has been confirmed, but prior to administration of the first dose of neratinib and at Day 30 (± 3 days) the conclusion of Cycle 1 (28 days).

Following the second study colonoscopy procedure:

  • For patients being treated for stage 1 to 3c breast cancer in the extended adjuvant setting, neratinib will continue to be administered at a single daily dose of 240 mg until completion of one year of therapy from start of treatment, or until disease recurrence (as determined by the Investigator), death, unacceptable toxicity, or other specified withdrawal criterion.
  • For patients being treated for metastatic breast cancer (mBC), capecitabine will be introduced after the second study colonoscopy procedure at a dose of 750mg/m2 twice daily for 14 days of each 21 day treatment cycle, with neratinib administered continuously throughout at 240 mg daily, until disease progression, death, unacceptable toxicity, or other specified withdrawal criterion.

All patients will receive loperamide diarrhea prophylaxis daily for one 28-day cycle and then as needed.

02

Conditions studied

  • HER2 Amplified Breast Cancer

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Keywords

  • HER2-Positive
  • Breast Cancer
  • Colon Pathology
  • Colonoscopy
03

In context

Breast Neoplasms

14,825 studies on the registry are indexed under Breast Neoplasms; 3,319 are open to participants now.

This study's enrollment of 6 is below the median of 72 across 10,896 interventional studies indexed under Breast Neoplasms.

Browse Breast Neoplasms studies →

Lead sponsor

Puma Biotechnology, Inc. is the lead sponsor of 38 studies on the registry; 3 are open to participants now.

Of its 17 completed or terminated interventional studies of FDA-regulated products, 17 (100%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  1. Aged ≥18 years.
  2. confirmed stage 1 through stage 4 primary adenocarcinoma of the breast.
  3. Documented overexpression or gene-amplified tumor by a validated approved method.
  4. Participants with confirmed stage 1 to stage 3c breast cancer receiving extended treatment with neratinib monotherapy must have completed a course of prior adjuvant trastuzumab or experienced side effects that resulted in early discontinuation of trastuzumab that have since resolved.
  5. Participants with mBC must have had at least 2 prior HER2-directed regimens.
  6. (LVEF) ≥50% measured by multiple-gated acquisition scan (MUGA) or echocardiogram (ECHO).
  7. (ECOG) status of 0 to 1.
  8. Negative β-human chorionic gonadotropin (hCG) pregnancy test for premenopausal women of reproductive capacity (those who are biologically capable of having children) and for women less than 12 months after menopause. [Women are considered if they are ≥12 months without menses, in the absence of endocrine or anti-endocrine therapies.]
  9. Women of must agree and commit to the use of a highly effective non-hormonal method of , i.e., intrauterine device, bilateral tubal ligation, vasectomized partner, or abstinence (only when it is the preferred lifestyle of the participant), from the time of until 28 days after the last dose of the products. Men (male participant) with a female partner of childbearing potential must agree and commit to use condom and the female partner must agree and commit to use a highly effective method of contraception (i.e., any of the above methods, or for females, hormonal contraception associated with inhibition of ovulation) while on treatment and for 3 months after last dose of investigational products.
  10. Recovery (i.e., to Grade 1 or baseline) from all adverse events related to prior therapies (excluding alopecia, neuropathy, and nail changes).
  11. No major bleeding diathesis or use of anticoagulants that would pose a high risk for endoscopic procedure.
  12. Provide written, informed consent to participate in the study and follow the study procedures.

Exclusion criteria

EXCLUSION CRITERIA:

  1. Participants with confirmed stage 1 to stage 3c currently receiving chemotherapy, radiation therapy, immunotherapy, or biotherapy for breast cancer.
  2. Participants with mBC who have received prior capecitabine or directed tyrosine kinase inhibitor (TKI) therapy.
  3. Currently using drugs that have been implicated as causing microscopic colitis/watery diarrhea, such as acarbose, aspirin, proton pump inhibitors, nonsteroidal anti-inflammatory drugs (NSAIDs), histamine H2 receptor antagonists, selective serotonin reuptake inhibitors, and ticlopidine (Pardi, 2017).
  4. Major surgery within \<28 days of starting treatment or received chemotherapy, agents, or other cancer therapy, except hormonal therapy (e.g., tamoxifen, aromatase inhibitors), \<14 days prior to the initiation of investigational products.
  5. Active uncontrolled cardiac disease, including cardiomyopathy, congestive heart failure ( functional classification of ≥2; including individuals who currently use digitalis, beta-blockers, or calcium channel blockers specifically for congestive heart failure), unstable angina, myocardial infarction within 12 months of enrollment, or ventricular arrhythmia.
  6. Interval (QTc) interval >0.450 seconds (males) or >0.470 (females), or known history of QTc prolongation or Torsade de Pointes (TdP).
  7. Diagnosis of inflammatory bowel disease
  8. Screening laboratory assessments outside the following limits:

    Laboratory Parameters Required Limit for Exclusion (ANC) \<1,000/µl (\<1.0 x 109/L) count \<50,000/µl (\<100 x 109/L) \<8 g/dL (transfusions allowed) Transfusions must be at least 14 days prior to initiation of treatment Total >1.5 x institutional (ULN) (in case of known Gilbert's syndrome, \<2 x ULN is allowed) (AST) and/or alanine aminotransferase (ALT) >2.5 x institutional ULN (>5 x ULN if liver are present) Creatinine \<30 mL/min (as calculated by Cockcroft-Gault formula A or Modification of Diet in Renal Disease formula B) (INR) >1.5 a Cockcroft and Gault, 1976 b Levey et al, 1999

  9. Active, unresolved infections.
  10. Participants with a second , other than adequately treated non-melanoma skin cancers, in situ melanoma or in situ cervical cancer. Participants with other non-mammary malignancies must have been disease free for at least 5 years.
  11. Currently pregnant or breast-feeding.
  12. Significant chronic gastrointestinal disorder with diarrhea as a major symptom (eg, Crohn's disease, malabsorption, or Grade ≥2 National Cancer Institute [NCI] Version 4.0 [CTCAE v.4.0] diarrhea of any etiology at baseline).
  13. Clinically active infection with or virus.
  14. Evidence of significant medical illness, abnormal laboratory finding, or psychiatric illness/social situations that could, in the Investigator's judgment, make the person inappropriate for this study.
  15. Known to any component of the investigational products; known allergies to any of the medications or components of medications used in the trial.
  16. Unable or unwilling to swallow tablets
Terms in these criteria, in plain language(27)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Histologically or cytologically confirmed

The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).

Your pathology report is the document that shows this.

HER2

HER2 is a protein, made from the ERBB2 gene, that drives the growth of some cancers. A study may look for a level of the protein, extra copies of the gene, or a particular change in it.

In breast cancer, protein tests are read as HER2-positive, HER2-low or HER2-negative; other cancers use their own rules. Check which test result the study requires.

Adjuvant and neoadjuvant

Neoadjuvant treatment is given before the main treatment (usually surgery), often to shrink a tumour. Adjuvant treatment is given after it, to lower the chance of the cancer coming back.

LVEF (left ventricular ejection fraction)

The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.

ECOG performance status

A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.

0
Fully active, no restrictions.
1
Limited in strenuous activity, but walking and able to do light work, such as housework or office work.
2
Walking and able to look after yourself, but unable to work; up and about more than half of waking hours.
3
Able to look after yourself only in part; in bed or a chair more than half of waking hours.
4
Unable to look after yourself at all; in bed or a chair all day.

Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.

Postmenopausal

Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

Clinically significant

Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.

NYHA class (heart failure)

The New York Heart Association’s four classes of how much heart failure limits physical activity.

I
No limits: ordinary activity causes no undue tiredness or breathlessness.
II
Slight limits: comfortable at rest, ordinary activity causes symptoms.
III
Marked limits: comfortable at rest, less than ordinary activity causes symptoms.
IV
Symptoms even at rest; any activity makes them worse.

Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.

QT and QTc

On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.

The study may name a correction formula and a limit in milliseconds, often around 450–480.

Absolute neutrophil count (ANC)

The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.

It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.

Platelets

Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.

Hemoglobin (Hb)

The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).

Bilirubin

A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.

Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.

ULN (upper limit of normal)

The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.

“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.

ALT and AST (liver enzymes)

Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.

Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.

Metastases

Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.

Creatinine clearance (CrCl)

How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.

INR, PT and aPTT (clotting tests)

Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.

Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.

Cancer (malignancy)

Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.

Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.

CTCAE grade

The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.

1
Mild.
2
Moderate.
3
Severe.
4
Life-threatening.
5
Death due to the event.

“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.

Hepatitis B tests

Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.

Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.

Hepatitis C tests

Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.

After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.

Hypersensitivity

An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.

05

Study design

Phase
Phase 2
Primary purpose
Other
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
6 participants (actual)

Study arms

  • Experimental
    Neratinib

    Neratinib with loperamide prophylaxis, and capecitabine for participants treated for metastatic breast cancer

    Drug: Neratinib · Drug: Capecitabine · Drug: Loperamide

Interventions

  • Administered orally once daily as a single daily dose of 240 mg

    Also known as: Nerlynx

  • Administered orally twice daily at 750 mg/m\^2 for 14 days of each 21 day treatment cycle

  • Administered orally for prophylaxis for 28 days and then as needed

06

What researchers measure

Primary outcomes

  1. Changes in Colon Pathology

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

    Time frame: From baseline to second colonoscopy, which is 88 days after start of neratinib treatment.

Secondary outcomes

  1. Incidence and Severity of Diarrhea

    Incidence and severity of treatment emergent (TEAE) diarrhea will be summarized according to the NCI-CTCAE version 4.0 in the first cycle of neratinib treatment, which is from the time of the first colonoscopy to the second colonoscopy, or 28 days for subjects with only one colonoscopy. Incidence is defined as the number of patients experiencing diarrhea divided by the number of patients at risk.

    Time frame: From baseline to second colonoscopy, which is 88 days after start of neratinib treatment.

07

Results

Posted Feb 10, 2023

Participant flow

Patients with stage 1 to 3c disease receiving extended adjuvant therapy are anticipated to participate in the study for approximately 1 year: 1 month for screening and 12 months of treatment, and 1 month of safety follow up. Patients with mBC are anticipated to participate in the study for an average of 12 month: 1 month of screening, 9.5 months for treatment, and 1 month of safety follow up.

Participant flow — Overall Study
MilestoneBreast Cancer Participants Treated With Neratinib
Started6
Treated5
Completed4
Not completed2

Outcome measures

PrimaryChanges in Colon Pathology

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

Time frame:
From baseline to second colonoscopy, which is 88 days after start of neratinib treatment.
Reported as:
Number · participants
Changes in Colon Pathology
participantsNeratinib Patients With 2 Colonoscopies
No Significant Findings2
Mild Changes2
SecondaryIncidence and Severity of Diarrhea

Incidence and severity of treatment emergent (TEAE) diarrhea will be summarized according to the NCI-CTCAE version 4.0 in the first cycle of neratinib treatment, which is from the time of the first colonoscopy to the second colonoscopy, or 28 days for subjects with only one colonoscopy. Incidence is defined as the number of patients experiencing diarrhea divided by the number of patients at risk.

Time frame:
From baseline to second colonoscopy, which is 88 days after start of neratinib treatment.
Reported as:
Number · percentage of participants
Incidence and Severity of Diarrhea
percentage of participantsSafety
TEAE Diarrhea80
Serious Diarrhea0

Adverse events

Collected over From time of first dose, through 28 days after last dose, assessed up to 16 months.. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Safety0/5 (0%)2/5 (40%)5/5 (100%)
Most frequent serious events
Most frequent serious events
EventSafety
Pericardial effusionCardiac disorders1/5
PneumoniaInfections and infestations1/5
Spinal fractureInjury, poisoning and procedural complications1/5
Pleural effusionRespiratory, thoracic and mediastinal disorders1/5
Most frequent other events
Showing 10 of 16
Most frequent other events
EventSafety
DiarrhoeaGastrointestinal disorders5/5
VomitingGastrointestinal disorders3/5
DizzinessNervous system disorders2/5
ThrombocytopeniaBlood and lymphatic system disorders1/5
Abdominal distensionGastrointestinal disorders1/5
Abdominal painGastrointestinal disorders1/5
ConstipationGastrointestinal disorders1/5
Faeces hardGastrointestinal disorders1/5
Faeces softGastrointestinal disorders1/5
FlatulenceGastrointestinal disorders1/5

Baseline characteristics

Safety population (treated patients)

Age, Continuous
Age, Continuous(years)Neratinib
Mean45.6 ± 14.8
Sex: Female, Male
Sex: Female, Male(Participants)Neratinib
Female5
Male0
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Neratinib
American Indian or Alaska Native0
Asian0
Native Hawaiian or Other Pacific Islander0
Black or African American0
White5
More than one race0
Unknown or Not Reported0
Region of Enrollment
Region of Enrollment(Participants)Neratinib
Portugal5
08

Study locations

1 site
  • Hospital CUF Descobertas
    Lisboa, 1998-018, Portugal
09

References and documents

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

Individual participant data

Plan to share: Yes — Puma Biotechnology is committed to sharing clinical trial data and information to help physicians and patients make informed treatment decisions, and to help qualified researchers advance scientific knowledge. In accordance with legal and regulatory requirements, Puma publishes study protocol information and clinical study results on clinical trial registries, including ClinicalTrials.gov and EU Clinical Trials Register. Puma also publishes information about clinical studies in peer-reviewed scientific journals and shares data in scientific meetings. Puma commits to safeguarding confidentiality and patient privacy throughout the clinical trial data and information sharing process. Any patient-level data will be anonymized to protect personally identifiable information. Qualified researchers and study participants may submit requests for other study documentation and clinical trial data to clinicaltrials@pumabiotechnology.com for consideration.

Supporting information: Study protocol, Statistical analysis plan, Informed consent form, Clinical study report

Time frame: Clinical study documents and clinical trial data may be requested by qualified researchers and study participants for studies that have been completed for at least 18 months, and for which the indication of the drug has been approved in the US and/or EU, as applicable. Requests will be accepted for up to 24 months after the criteria described in this section are met.

Access criteria: Requestors must provide organizational contact information; a detailed research plan, including outcomes; timeline for completion of the research; qualifications of the research team; funding source; and potential conflicts of interest. Puma will not provide access to patient-level data if there is a reasonable likelihood that individual patients could be identified, or in cases where confidentiality or consent provisions prohibit transfer of data or information to third parties. Additionally, Puma will not disclose information that jeopardizes intellectual property rights or divulges confidential commercial information.

Data sharing details ↗ · pumabiotechnology.com

10

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Ran from Jun 2020 to primary completion in Dec 2021, 17 months.

Results were first posted in Feb 2023, 1 year 1 month after primary completion.

Of 232 industry-sponsored phase 2 interventional studies under Breast Neoplasms, half reached primary completion within 34 months of starting (the median), counting those still running; a quarter within 22 and three quarters within 53. This study's 17 months is below that.

Counting every sponsor, half of 1,302 studies reached it within 51 months; a quarter within 30 and three quarters within 82.

How these figures are worked out, and which studies are left out of them.

Registered
Mar 23, 2020
First posted
Apr 29, 2020
StartActual
Jun 30, 2020
Primary completionActual
Dec 28, 2021
CompletionActual
Dec 28, 2021
Results first posted
Feb 10, 2023
Last update
Feb 10, 2023
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Feb 10, 2023, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT04366713
Responsible party
Sponsor
First posted
Apr 29, 2020
Start date
Jun 30, 2020
Primary completion
Dec 28, 2021
Completion
Dec 28, 2021
Results posted
Feb 10, 2023
Last update
Feb 10, 2023

Study contacts

Chief Scientific Officer
study director · Puma Biotechnology, Inc.

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Jan 2023. You cannot join it, but the record below documents what was studied.

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Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

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Studies of the drug tested here: Neratinib

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