Solid Tumors Harboring Somatic HER2 or EGFR Exon 18 MutationsPhase 2SUMMITNCT01953926

Basket Study of Neratinib in Participants With Solid Tumors Harboring Somatic HER2 or EGFR Exon 18 Mutations

A Phase 2 study testing Neratinib, Fulvestrant and Trastuzumab in people with Solid Tumors Harboring Somatic HER2 or EGFR Exon 18 Mutations, run by Puma Biotechnology, Inc. at 60 sites in 13 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

Search all trials

Trial summary

Who is it for?: Ages 18 years and older
Conditions the study is for: Solid Tumors Harboring Somatic HER2 or EGFR Exon 18 Mutations
What is being tested?: Neratinib, Fulvestrant and Trastuzumab
Type of treatment: Pill or oral medicine, Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 582 participants
Where: 60 sitesin 13 countries
Study dates: 30 Sep 2013to 2 Jan 2023
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Provide written informed consent
Histologically confirmed cancers for which no curative therapy exists
Documented HER2 or EGFR exon 18 mutation
Participants must agree and commit to use appropriate methods of contraception as outlined in the protocol
At least one measurable lesion, defined by RECIST v1.1

Who cannot join

Participants harboring ineligible somatic HER2 mutations
Prior treatment with any HER2-directed tyrosine kinase inhibitor (e.g., lapatinib, afatinib, dacomitinib, neratinib) is excluded with the following exception: patients with EGFR exon 18 mutated NSCLC who may have received afatinib, osimertinib, or other pan HER or EGFR TKIs remain eligible
Participants who are receiving any other anticancer agents
Symptomatic or unstable brain metastases
Women who are pregnant or breast-feeding
There are additional inclusion and exclusion criteria. The study center will determine if criteria for participation are met.

Trial locations and contacts

60 sites in 13 countries.

Country

City, postcode or hospital

60 sites in 13 countries

United States30 sites

University of Michigan
Ann Arbor, Michigan, 48109
Winship Cancer Institute, Emory University
Atlanta, Georgia, 30322
University of Alabama at Birmingham
Birmingham, Alabama, 35249
Dana Farber Cancer Institute
Boston, Massachusetts, 02215
Roswell Park Comprehensive Cancer Center
Buffalo, New York, 14263
Northwestern University
Chicago, Illinois, 60611
UT Southwestern Medical Center
Dallas, Texas, 75390
City of Hope
Duarte, California, 91010
The West Clinic
Germantown, Tennessee, 38138
Saint Francis Cancer Center-Bon Secours
Greenville, South Carolina, 29607
MD Anderson Cancer Center
Houston, Texas, 77030
Mayo Clinic Florida
Jacksonville, Florida, 32224
Gundersen Center for Cancer and Blood Disorders
La Crosse, Wisconsin, 54601
University of California, San Diego
La Jolla, California, 92093
University of Southern California
Los Angeles, California, 90089
University of California, Los Angeles
Los Angeles, California, 90095
University of Wisconsin Carbone Cancer Center
Madison, Wisconsin, 53792
University of Miami
Miami, Florida, 33136
Vanderbilt-Ingram Cancer Center
Nashville, Tennessee, 37232
Ochsner Clinic Foundation
New Orleans, Louisiana, 70121
Memorial Sloan-Kettering Cancer Center
New York, New York, 10065
Stanford Cancer Center
Palo Alto, California, 94304
Mayo Clinic Arizona
Phoenix, Arizona, 85054
UPMC Magee-Woman's Hospital, Women's Cancer Center
Pittsburgh, Pennsylvania, 15213
Mayo Clinic
Rochester, Minnesota, 55905
Metro Minnesota Community Oncology Research Consortium
Saint Louis Park, Minnesota, 55416
UCSF Helen Diller Family Comprehensive Cancer Center
San Francisco, California, 94115
Seattle Cancer Care Alliance
Seattle, Washington, 98109
Washington University
St Louis, Missouri, 63110
Kaiser Permanente NoCal (STRATA)
Vallejo, California, 94589

Other trials in United States

Spain9 sites

Hospital Universitario Quiron Dexeus
Barcelona, 08028
Hospital Universitario Vall d'Hebron
Barcelona, 08035
Hospital Universitari Clinic Barcelona
Barcelona, 08036
Hospital Clinico Universitario San Carlos
Madrid, 28040
Hospital Universitario Fundacion Jimenez Diaz
Madrid, 28040
Hospital Universitario Madrid Sanchinarro (START Madrid)
Madrid, 28050
Hospital Universitario Quiron Madrid
Madrid, 28223
Instituto Valenciano de Oncologia
Valencia, 46009
Hospital Clinico Universitario de Valencia
Valencia, 46010

Other trials in Spain

Israel5 sites

Hadassah Medical Center
Jerusalem, 9112001
Davidoff Cancer Center, Rabin Medical Center
Petah Tikva, 4941494
Sheba Medical Center
Ramat Gan, 5262000
Kaplan Medical Center
Rehovot, 7661041
Sourasky Medical Center
Tel Aviv, 6423906

Other trials in Israel

France4 sites

Institut Bergonié
Bordeaux, 33076
Centre Leon Berard
Lyon, 69393
Institut Curie - Hopital Rene Huguenin
Saint-Cloud, Île-de-France Region, 92210
Institut Gustave Roussy
Villejuif, Paris, 94800

Other trials in France

Italy4 sites

Azienda Socio Sanitaria Territoriale di Cremona
Cremona, 26100
Istituto Europeo di Oncologia (IEO) I.R.C.C.S.
Milan, 20141
Fondazione Policlinico Universitario Gemelli I.R.C.C.S.
Roma, 00168
AOU Citta della Salute e della Scienza di Torino
Torino, 10126

Other trials in Italy

Australia1 site

Peter MacCallum Cancer Centre
East Melbourne, Victoria, 8006

Other trials in Australia

Belgium1 site

UZ Leuven
Leuven, 3000

Other trials in Belgium

Canada1 site

British Columbia Cancer Center
Vancouver, British Columbia, V5Z 4E6

Other trials in Canada

Denmark1 site

University Hospital, Rigshospitalet
Copenhagen, DK-2100

Other trials in Denmark

Ireland1 site

St. Vincent's University Hospital
Dublin, Leinster, D04 T6F4

Other trials in Ireland

Serbia1 site

Institute for Oncology and Radiology of Serbia
Belgrade, 11000

Other trials in Serbia

South Korea1 site

Yonsei University Health System, Serverance Hospital
Seodaemun-Gu, Seoul, 120-752

Other trials in South Korea

United Kingdom1 site

Royal Free Hospital
London, NW3 2QG

Other trials in United Kingdom

About this study

Official title: An Open-Label, Phase 2 Basket Study of Neratinib in Patients With Solid Tumors With Somatic Activating HER Mutations

Read the detailed description

This is an open-label, multicenter, multinational, Phase 2 basket study exploring the efficacy and safety of neratinib as monotherapy or in combination with other therapies in participants with HER (EGFR, HER2) mutation-positive solid tumors. The study has a basket design and includes several cohorts, either defined by an actionable somatic mutation or by actionable mutation and tumor histology, including HER2 mutant breast, HER2 mutant cervical, HER2 mutant salivary gland, and EGFR Exon 18 mutant Non-small cell lung cancers.

The trial will consist of a screening period, a treatment period, and an end of treatment visit occurring when neratinib is discontinued for any reason, a safety follow-up visit occurring 28 days after the last dose of neratinib and a survival follow-up period.

Conditions: Solid Tumors Harboring Somatic HER2 or EGFR Exon 18 Mutations
Being tested: Neratinib, Fulvestrant, Trastuzumab, Paclitaxel
Keywords: Neratinib, Nerlynx, Breast, Solid Tumors, Cancer, HER2 mutations, EGFR mutations, Fulvestrant, Trastuzumab, Cervical, Salivary, ERBB2, Exon 18, Metastatic, HR Positive, Lung, Non-Small Cell Lung Cancer (NSCLC)
Medical subject headings: Neoplasms, Neoplasm Metastasis, Carcinoma, Non-Small-Cell Lung

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

NeratinibDrug
Also called Nerlynx
240 mg administered orally, once daily with food, continuously in 28 day cycles
FulvestrantDrug
Also called Faslodex
500 mg administered as two 5 mL injections on Days 1, 15, and 29; then once every 4 weeks thereafter month, then Day 1 of every 4 week cycle
TrastuzumabDrug
Also called Herceptin
Initial dose of 8 mg/kg of trastuzumab administered IV on Day 1, followed by 6 mg/kg IV once every 3 weeks thereafter
PaclitaxelDrug
Also called Taxol
80mg/m^2 administered IV on Days 1, 8, and 15 of every 4 week cycle

Study groups in NCT01953926
Study group	Role	What they receive
Neratinib monotherapy	Experimental	Neratinib monotherapy in HER2 mutated cancers including cervical, salivary gland, and lung cancers containing EGFR exon 18 mutations. Cohorts closed to enrollment in prior amendments: HER2 mutant cancers including bladder/urinary, colorectal, endometrial, breast HR-positive, TNBC HR-negative, lung, gastroesophageal, biliary, and ovarian; HER3 mutant solid tumor NOS; HER4 mutant solid tumor NOS; fibrolamellar carcinoma and EGFR brain. Receives: Drug: Neratinib
Neratinib and Trastuzumab	Experimental	Neratinib and Trastuzumab in HER2 mutated (TNBC, HR-negative) breast cancers. Cohorts closed to enrollment in in prior amendments: colorectal, lung cancer HER2 mutant. Receives: Drug: Neratinib; Drug: Trastuzumab
Neratinib, Fulvestrant and Trastuzumab (Randomized)	Experimental	Neratinib, Fulvestrant and Trastuzumab or Fulvestrant and Trastuzumab or Fulvestrant alone in HER2 mutated (HR-positive with prior CDK4/6i) breast cancers. Receives: Drug: Neratinib; Drug: Fulvestrant; Drug: Trastuzumab
Neratinib, Fulvestrant and Trastuzumab (Non-Randomized)	Experimental	Neratinib, Fulvestrant and Trastuzumab in HER2 mutated (HR-positive with or without CDK4/6i) breast cancers. Receives: Drug: Neratinib; Drug: Fulvestrant; Drug: Trastuzumab
Neratinib and Paclitaxel	Experimental	Neratinib and Paclitaxel in HER2 mutated bladder/urinary tract cancers. Receives: Drug: Neratinib; Drug: Paclitaxel
Neratinib and Fulvestrant	Experimental	Neratinib and Fulvestrant in HER2 mutated (HR-positive) breast cancers. Receives: Drug: Neratinib; Drug: Fulvestrant

What is being measured

The primary outcome is the main question the study is designed to answer.

Confirmed Objective Response Rate (ORR) by Independent Central Review (Breast Cancer With Prior CDK46i Cohort)
Percentage of participants who are confirmed by independent central review to have achieved complete response (CR) or partial response (PR) according to Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 (HR+, HER2 negative metastatic breast cancer cohorts).
Per Response Evaluation Criteria in Sold Tumors Criteria (RECISTv1.1) for target lesions and assessed by MRI or CT: Complete response(CR),Disappearance of all target lesions; Partial response(PR),>=30% decrease in sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR
Measured: From enrollment date to first confirmed Complete or Partial Response, whichever came earlier, assessed up to 58 months
Confirmed Objective Response Rate (ORR) by Investigator Review (Cervical Cancer Cohort)
Percentage of participants who are confirmed by investigator review to have achieved complete response (CR) or partial response (PR) according to Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 (cervical cancer cohort).
Per Response Evaluation Criteria in Sold Tumors Criteria (RECISTv1.1) for target lesions and assessed by MRI or CT: Complete response(CR),Disappearance of all target lesions; Partial response(PR),>=30% decrease in sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR
Measured: From enrollment date to first confirmed Complete or Partial Response, whichever came earlier, assessed up to 58 months
Objective Response Rate (ORR) at First Assessment by Investigator Review (All Other Cohorts)
Percentage of participants who achieve CR or PR per Response Evaluation Criteria in Sold Tumors Criteria (RECIST) v1.1, or other defined response criteria, at the first scheduled tumor assessment (all other cohorts), per RECIST (if assessed) or PERCIST.
RECISTv1.1 for target lesions and assessed by MRI or CT: Complete response(CR),Disappearance of all target lesions; Partial response(PR),>=30% decrease in sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR
PERCISTv1.0: Complete Metabolic Response - Complete resolution of 18F-FDG uptake within measurable target lesion so that it is less than mean liver activity and indistinguishable from surrounding background blood-pool levels
Partial Metabolic Response - Reduction of minimum of 30% in target measurable tumour 18F-FDG SULpeak. Absolute drop in SUL must be at least 0.8 SUL units, as well. No new lesions.
Positive Metabolic Response - Participants having either "Complete Metabolic Response" or "Part
Measured: From first treatment date to first Complete or Partial Response, whichever came earlier, assessed up to 8 or 9 weeks

Secondary outcomes (9)

Confirmed Objective Response Rate (ORR) by Investigator Review (Breast Cancer With Prior CDK46i Cohort)
Percentage of participants who are confirmed by investigator review to have achieved complete response (CR) or partial response (PR) according to Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 (HR+, HER2 negative metastatic breast cancer cohorts).
Per Response Evaluation Criteria in Sold Tumors Criteria (RECISTv1.1) for target lesions and assessed by MRI or CT: Complete response(CR),Disappearance of all target lesions; Partial response(PR),>=30% decrease in sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR
Measured: From enrollment date to first confirmed Complete or Partial Response, whichever came earlier, assessed up to 58 months
Confirmed Objective Response Rate (ORR) by Investigator Review (All Other Cohorts)
Percentage of participants who achieve CR or PR per RECIST v1.1, or metabolic complete response via PERCIST v1.0.
For RECIST, A complete or partial response that is confirmed no less than 4-weeks after the criteria for response are initially met. PERCIST criteria were used for patients without RECIST assessments.
Measured: From first treatment date to confirmed Complete or Partial Response, assessed up to 58 months.
Duration of Response (DOR) by Independent Central Review (Breast Cancer With Prior CDK46i Cohort)
Time from which measurement criteria are met for response (whichever status is recorded first) until the first date of documented disease progression or death. Disease progression assessed by RECIST criteria, or for PERCIST for those participants who did not have RECIST performed.
Measured: From first response to first disease progression or death, assessed up to 58 months
Duration of Response (DOR) by Investigator Review (All Cohorts)
Time from which measurement criteria are met for response (whichever status is recorded first) until the first date of documented disease progression or death. Disease progression assessed by RECIST criteria, or for PERCIST for those participants who did not have RECIST performed.
Measured: From first response to first disease progression or death, assessed up to 58 months
Clinical Benefit Rate (CBR) by Independent Central Review (Breast Cancer With Prior CDK46i Cohort)
Percentage of participants with CR + PR + stable disease ≥16, or ≥24 weeks for breast cancer, from the date of enrollment.
Measured: From enrollment date to first documented response or stable disease ≥16, or ≥24 weeks for breast cancer, assessed up to 58 months
Clinical Benefit Rate (CBR) by Investigator Review (All Cohorts)
Percentage of participants with CR + PR + stable disease ≥16, or ≥24 weeks for breast cancer, from the date of enrollment.
Measured: From enrollment date to first documented response or stable disease ≥16, or ≥24 weeks for breast cancer, assessed up to 58 months
Progression-Free Survival (PFS) by Independent Central Review (Breast Cancer With Prior CDK46i Cohort)
Number of months between first dose date and the first date on which recurrence, progression, or death due to any cause, is documented, censored at the last tumor assessment or at the initiation of new anticancer therapy. Progression was defined by RECIST criteria for those participants with RECIST assessments; and PERCIST criteria for other participants.
Measured: From enrollment date until the date of first documented progression, or date of death from any cause, whichever came first, assessed up to 58 months
Progression-Free Survival (PFS) by Investigator Review (All Cohorts)
Number of months between first dose date and the first date on which recurrence, progression, or death due to any cause, is documented, censored at the last tumor assessment or at the initiation of new anticancer therapy. Progression was defined by RECIST criteria for those participants with RECIST assessments; and PERCIST criteria for other participants.
Measured: From enrollment date until the date of first documented progression, or date of death from any cause, whichever came first, assessed up to 58 months
Number of Participants With Treatment-Emergent Adverse Events
The safety of neratinib in patients as measured by the incidence of treatment-emergent adverse events (TEAE), including serious adverse events (SAEs), in study participants. TEAEs are any adverse event that occurred on or after first dose of investigational product and up to 28 days after the last dose
Measured: From first dose through 28 days after the last dose, assessed up to 75 months.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

30 Sep 20132 Jan 2023

First posted1 Oct 2013 (estimated)
Study start30 Sep 2013
Primary completion2 Jan 2023
Study completion2 Jan 2023

Why it stopped: The study was terminated to align with the sponsor's current development plans for neratinib. The decision was not based on any new efficacy or safety data for neratinib.

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (10)Papers the sponsor has linked to this study.
Other trials for Solid Tumors Harboring Somatic HER2 or EGFR Exon 18 MutationsStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 582 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01953926
Sponsor's protocol ID: PUMA-NER-5201
Other IDs: 2013-002872-42
Acronym: SUMMIT
Status last verified: Feb 2024
First submitted: 26 Sep 2013
Last updated: 12 Mar 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: 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.

Publications

Hyman DM, Piha-Paul SA, Won H, Rodon J, Saura C, Shapiro GI, Juric D, Quinn DI, Moreno V, Doger B, Mayer IA, Boni V, Calvo E, Loi S, Lockhart AC, Erinjeri JP, Scaltriti M, Ulaner GA, Patel J, Tang J, Beer H, Selcuklu SD, Hanrahan AJ, Bouvier N, Melcer M, Murali R, Schram AM, Smyth LM, Jhaveri K, Li BT, Drilon A, Harding JJ, Iyer G, Taylor BS, Berger MF, Cutler RE Jr, Xu F, Butturini A, Eli LD, Mann G, Farrell C, Lalani AS, Bryce RP, Arteaga CL, Meric-Bernstam F, Baselga J, Solit DB. HER kinase inhibition in patients with HER2- and HER3-mutant cancers. Nature. 2018 Feb 8;554(7691):189-194. doi: 10.1038/nature25475. Epub 2018 Jan 31.
PubMed 29420467
Smyth LM, Piha-Paul SA, Won HH, Schram AM, Saura C, Loi S, Lu J, Shapiro GI, Juric D, Mayer IA, Arteaga CL, de la Fuente MI, Brufksy AM, Spanggaard I, Mau-Sorensen M, Arnedos M, Moreno V, Boni V, Sohn J, Schwartzberg LS, Gonzalez-Farre X, Cervantes A, Bidard FC, Gorelick AN, Lanman RB, Nagy RJ, Ulaner GA, Chandarlapaty S, Jhaveri K, Gavrila EI, Zimel C, Selcuklu SD, Melcer M, Samoila A, Cai Y, Scaltriti M, Mann G, Xu F, Eli LD, Dujka M, Lalani AS, Bryce R, Baselga J, Taylor BS, Solit DB, Meric-Bernstam F, Hyman DM. Efficacy and Determinants of Response to HER Kinase Inhibition in HER2-Mutant Metastatic Breast Cancer. Cancer Discov. 2020 Feb;10(2):198-213. doi: 10.1158/2159-8290.CD-19-0966. Epub 2019 Dec 5.
PubMed 31806627
Li BT, Gandhi L, Ravichandran V, Besse B, Mazieres J, Shapiro GI, Boni V, Waqar SN, Park H, Quinn DI, Martinez A, Stemmer SM, Cortot AB, Barlesi F, Quoix E, Burkard ME, Selcuklu SD, Hunt C, Donoghue MTA, Kris MG, Bebchuk J, Eli LD, McCulloch L, Solit DB, Janne PA. Efficacy of Neratinib-Based Therapy in ERBB2-Mutant Lung Adenocarcinomas: Findings From 2 International Phase 2 Studies. Clin Lung Cancer. 2026 Mar;27(2):214-226.e5. doi: 10.1016/j.cllc.2025.09.009. Epub 2025 Oct 1.
PubMed 41193346
Friedman CF, D'Souza A, Bello Roufai D, Tinker AV, de Miguel M, Gambardella V, Goldman J, Loi S, Melisko ME, Oaknin A, Spanggaard I, Shapiro GI, ElNaggar AC, Panni S, Ravichandran V, Frazier AL, DiPrimeo D, Eli LD, Solit DB. Targeting HER2-mutant metastatic cervical cancer with neratinib: Final results from the phase 2 SUMMIT basket trial. Gynecol Oncol. 2024 Feb;181:162-169. doi: 10.1016/j.ygyno.2023.12.004. Epub 2024 Jan 11.
PubMed 38211393
Jhaveri K, Eli LD, Wildiers H, Hurvitz SA, Guerrero-Zotano A, Unni N, Brufsky A, Park H, Waisman J, Yang ES, Spanggaard I, Reid S, Burkard ME, Vinayak S, Prat A, Arnedos M, Bidard FC, Loi S, Crown J, Bhave M, Piha-Paul SA, Suga JM, Chia S, Saura C, Garcia-Saenz JA, Gambardella V, de Miguel MJ, Gal-Yam EN, Rapael A, Stemmer SM, Ma C, Hanker AB, Ye D, Goldman JW, Bose R, Peterson L, Bell JSK, Frazier A, DiPrimeo D, Wong A, Arteaga CL, Solit DB. Neratinib + fulvestrant + trastuzumab for HR-positive, HER2-negative, HER2-mutant metastatic breast cancer: outcomes and biomarker analysis from the SUMMIT trial. Ann Oncol. 2023 Oct;34(10):885-898. doi: 10.1016/j.annonc.2023.08.003. Epub 2023 Aug 18.
PubMed 37597578
Harding JJ, Piha-Paul SA, Shah RH, Murphy JJ, Cleary JM, Shapiro GI, Quinn DI, Brana I, Moreno V, Borad M, Loi S, Spanggaard I, Park H, Ford JM, Arnedos M, Stemmer SM, de la Fouchardiere C, Fountzilas C, Zhang J, DiPrimeo D, Savin C, Duygu Selcuklu S, Berger MF, Eli LD, Meric-Bernstam F, Jhaveri K, Solit DB, Abou-Alfa GK. Antitumour activity of neratinib in patients with HER2-mutant advanced biliary tract cancers. Nat Commun. 2023 Feb 6;14(1):630. doi: 10.1038/s41467-023-36399-y.
PubMed 36746967
Shishido SN, Masson R, Xu L, Welter L, Prabakar RK, D' Souza A, Spicer D, Kang I, Jayachandran P, Hicks J, Lu J, Kuhn P. Disease characterization in liquid biopsy from HER2-mutated, non-amplified metastatic breast cancer patients treated with neratinib. NPJ Breast Cancer. 2022 Feb 18;8(1):22. doi: 10.1038/s41523-022-00390-5.
PubMed 35181666
Oaknin A, Friedman CF, Roman LD, D'Souza A, Brana I, Bidard FC, Goldman J, Alvarez EA, Boni V, ElNaggar AC, Passalacqua R, Do KTM, Santin AD, Keyvanjah K, Xu F, Eli LD, Lalani AS, Bryce RP, Hyman DM, Meric-Bernstam F, Solit DB, Monk BJ. Neratinib in patients with HER2-mutant, metastatic cervical cancer: Findings from the phase 2 SUMMIT basket trial. Gynecol Oncol. 2020 Oct;159(1):150-156. doi: 10.1016/j.ygyno.2020.07.025. Epub 2020 Jul 25.
PubMed 32723675
Ulaner GA, Saura C, Piha-Paul SA, Mayer I, Quinn D, Jhaveri K, Stone B, Shahin S, Mann G, Dujka M, Bryce R, Meric-Bernstam F, Solit DB, Hyman DM. Impact of FDG PET Imaging for Expanding Patient Eligibility and Measuring Treatment Response in a Genome-Driven Basket Trial of the Pan-HER Kinase Inhibitor, Neratinib. Clin Cancer Res. 2019 Dec 15;25(24):7381-7387. doi: 10.1158/1078-0432.CCR-19-1658. Epub 2019 Sep 23.
PubMed 31548342
Hanker AB, Brewer MR, Sheehan JH, Koch JP, Sliwoski GR, Nagy R, Lanman R, Berger MF, Hyman DM, Solit DB, He J, Miller V, Cutler RE Jr, Lalani AS, Cross D, Lovly CM, Meiler J, Arteaga CL. An Acquired HER2T798I Gatekeeper Mutation Induces Resistance to Neratinib in a Patient with HER2 Mutant-Driven Breast Cancer. Cancer Discov. 2017 Jun;7(6):575-585. doi: 10.1158/2159-8290.CD-16-1431. Epub 2017 Mar 8.
PubMed 28274957

Study at a glance

From the registry record, last updated by the sponsor on 12 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open-Label, Phase 2 Basket Study of Neratinib in Patients With Solid Tumors With Somatic Activating HER Mutations
ClinicalTrials.gov ID: NCT01953926
Acronym / sponsor ID: SUMMIT / PUMA-NER-5201
Conditions: Solid Tumors Harboring Somatic HER2 or EGFR Exon 18 Mutations
Keywords: Neratinib, Nerlynx, Breast, Solid Tumors, Cancer, HER2 mutations, EGFR mutations, Fulvestrant
Phase: Phase 2
Study type: Interventional
Recruitment status: TerminatedReason stopped: The study was terminated to align with the sponsor's current development plans for neratinib. The decision was not based on any new efficacy or safety data for neratinib.
Enrolment: 582 participants (actual)
Lead sponsor: Puma Biotechnology, Inc.Industry (pharma, biotech, devices)
Study start: 30 Sep 2013 (actual)
Primary completion: 2 Jan 2023 (actual)
Study completion: 2 Jan 2023 (actual)
First posted: 1 Oct 2013 (estimated)
Results: Posted 12 Mar 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 6

Arm	Type	Receives
Neratinib monotherapy	Experimental	Drug: NeratinibNeratinib monotherapy in HER2 mutated cancers including cervical, salivary gland, and lung cancers containing EGFR exon 18 mutations. Cohorts closed to enrollment in prior amendments: HER2 mutant cancers including bladder/urinary, colorectal, endometrial, breast HR-positive, TNBC HR-negative, lung, gastroesophageal, biliary, and ovarian; HER3 mutant solid tumor NOS; HER4 mutant solid tumor NOS; fibrolamellar carcinoma and EGFR brain.
Neratinib and Trastuzumab	Experimental	Drug: Neratinib, Drug: TrastuzumabNeratinib and Trastuzumab in HER2 mutated (TNBC, HR-negative) breast cancers. Cohorts closed to enrollment in in prior amendments: colorectal, lung cancer HER2 mutant.
Neratinib, Fulvestrant and Trastuzumab (Randomized)	Experimental	Drug: Neratinib, Drug: Fulvestrant, Drug: TrastuzumabNeratinib, Fulvestrant and Trastuzumab or Fulvestrant and Trastuzumab or Fulvestrant alone in HER2 mutated (HR-positive with prior CDK4/6i) breast cancers.
Neratinib, Fulvestrant and Trastuzumab (Non-Randomized)	Experimental	Drug: Neratinib, Drug: Fulvestrant, Drug: TrastuzumabNeratinib, Fulvestrant and Trastuzumab in HER2 mutated (HR-positive with or without CDK4/6i) breast cancers.
Neratinib and Paclitaxel	Experimental	Drug: Neratinib, Drug: PaclitaxelNeratinib and Paclitaxel in HER2 mutated bladder/urinary tract cancers.
Neratinib and Fulvestrant	Experimental	Drug: Neratinib, Drug: FulvestrantNeratinib and Fulvestrant in HER2 mutated (HR-positive) breast cancers.

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (5 listed)

Provide written informed consent
Histologically confirmed cancers for which no curative therapy exists
Documented HER2 or EGFR exon 18 mutation
Participants must agree and commit to use appropriate methods of contraception as outlined in the protocol
At least one measurable lesion, defined by RECIST v1.1

Key exclusion criteria (6 listed)

Participants harboring ineligible somatic HER2 mutations
Prior treatment with any HER2-directed tyrosine kinase inhibitor (e.g., lapatinib, afatinib, dacomitinib, neratinib) is excluded with the following exception: patients with EGFR exon 18 mutated NSCLC who may have received afatinib, osimertinib, or other pan HER or EGFR TKIs remain eligible
Participants who are receiving any other anticancer agents
Symptomatic or unstable brain metastases
Women who are pregnant or breast-feeding

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
NeratinibAlso: Nerlynx240 mg administered orally, once daily with food, continuously in 28 day cycles	Drug	Neratinib and Fulvestrant, Neratinib and Paclitaxel, Neratinib and Trastuzumab, Neratinib monotherapy, Neratinib, Fulvestrant and Trastuzumab (Non-Randomized), Neratinib, Fulvestrant and Trastuzumab (Randomized)
FulvestrantAlso: Faslodex500 mg administered as two 5 mL injections on Days 1, 15, and 29; then once every 4 weeks thereafter month, then Day 1 of every 4 week cycle	Drug	Neratinib and Fulvestrant, Neratinib, Fulvestrant and Trastuzumab (Non-Randomized), Neratinib, Fulvestrant and Trastuzumab (Randomized)
TrastuzumabAlso: HerceptinInitial dose of 8 mg/kg of trastuzumab administered IV on Day 1, followed by 6 mg/kg IV once every 3 weeks thereafter	Drug	Neratinib and Trastuzumab, Neratinib, Fulvestrant and Trastuzumab (Non-Randomized), Neratinib, Fulvestrant and Trastuzumab (Randomized)
PaclitaxelAlso: Taxol80mg/m^2 administered IV on Days 1, 8, and 15 of every 4 week cycle	Drug	Neratinib and Paclitaxel

Primary outcome

Confirmed Objective Response Rate (ORR) by Independent Central Review (Breast Cancer With Prior CDK46i Cohort)
Time frame: From enrollment date to first confirmed Complete or Partial Response, whichever came earlier, assessed up to 58 months
Percentage of participants who are confirmed by independent central review to have achieved complete response (CR) or partial response (PR) according to Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 (HR+, HER2 negative metastatic breast cancer cohorts). Per Response Evaluation Criteria in Sold Tumors Criteria (RECISTv1.1) for target lesions and assessed by MRI or CT: Complete response(CR),Disappearance of all target lesions; Partial response(PR),>=30% decrease in sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR
Confirmed Objective Response Rate (ORR) by Investigator Review (Cervical Cancer Cohort)
Time frame: From enrollment date to first confirmed Complete or Partial Response, whichever came earlier, assessed up to 58 months
Percentage of participants who are confirmed by investigator review to have achieved complete response (CR) or partial response (PR) according to Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 (cervical cancer cohort). Per Response Evaluation Criteria in Sold Tumors Criteria (RECISTv1.1) for target lesions and assessed by MRI or CT: Complete response(CR),Disappearance of all target lesions; Partial response(PR),>=30% decrease in sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR
Objective Response Rate (ORR) at First Assessment by Investigator Review (All Other Cohorts)
Time frame: From first treatment date to first Complete or Partial Response, whichever came earlier, assessed up to 8 or 9 weeks
Percentage of participants who achieve CR or PR per Response Evaluation Criteria in Sold Tumors Criteria (RECIST) v1.1, or other defined response criteria, at the first scheduled tumor assessment (all other cohorts), per RECIST (if assessed) or PERCIST. RECISTv1.1 for target lesions and assessed by MRI or CT: Complete response(CR),Disappearance of all target lesions; Partial response(PR),>=30% decrease in sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR PERCISTv1.0: Complete Metabolic Response - Complete resolution of 18F-FDG uptake within measurable target lesion so that it is less than mean liver activity and indistinguishable from surrounding background blood-pool levels Partial Metabolic Response - Reduction of minimum of 30% in target measurable tumour 18F-FDG SULpeak. Absolute drop in SUL must be at least 0.8 SUL units, as well. No new lesions. Positive Metabolic Response - Participants having either "Complete Metabolic Response" or "Part

9 secondary outcomes

Confirmed Objective Response Rate (ORR) by Investigator Review (Breast Cancer With Prior CDK46i Cohort)
Time frame: From enrollment date to first confirmed Complete or Partial Response, whichever came earlier, assessed up to 58 months
Confirmed Objective Response Rate (ORR) by Investigator Review (All Other Cohorts)
Time frame: From first treatment date to confirmed Complete or Partial Response, assessed up to 58 months.
Duration of Response (DOR) by Independent Central Review (Breast Cancer With Prior CDK46i Cohort)
Time frame: From first response to first disease progression or death, assessed up to 58 months
Duration of Response (DOR) by Investigator Review (All Cohorts)
Time frame: From first response to first disease progression or death, assessed up to 58 months
Clinical Benefit Rate (CBR) by Independent Central Review (Breast Cancer With Prior CDK46i Cohort)
Time frame: From enrollment date to first documented response or stable disease ≥16, or ≥24 weeks for breast cancer, assessed up to 58 months
Clinical Benefit Rate (CBR) by Investigator Review (All Cohorts)
Time frame: From enrollment date to first documented response or stable disease ≥16, or ≥24 weeks for breast cancer, assessed up to 58 months
Progression-Free Survival (PFS) by Independent Central Review (Breast Cancer With Prior CDK46i Cohort)
Time frame: From enrollment date until the date of first documented progression, or date of death from any cause, whichever came first, assessed up to 58 months
Progression-Free Survival (PFS) by Investigator Review (All Cohorts)
Time frame: From enrollment date until the date of first documented progression, or date of death from any cause, whichever came first, assessed up to 58 months
Number of Participants With Treatment-Emergent Adverse Events
Time frame: From first dose through 28 days after the last dose, assessed up to 75 months.

Results and safety

Results posted to the registry on 12 Mar 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Neratinib	318	231
Neratinib + Fulvestrant	45	31
Neratinib + Paclitaxel	23	14
Neratinib + Trastuzumab	92	70
Neratinib + Fulvestrant + Trastuzumab	90	32
Fulvestrant	7	2
Fulvestrant + Trastuzumab	7	0

Primary outcome: Confirmed Objective Response Rate (ORR) by Independent Central Review (Breast Cancer With Prior CDK46i Cohort)

Time frame: From enrollment date to first confirmed Complete or Partial Response, whichever came earlier, assessed up to 58 months. Number. Unit: percentage of participants

Measure	Breast HR+ w Prior CDK4/6i (Neratinib + Fulvestrant + Trastuzumab) n = 59	Breast HR+ w. Prior CDK4/6i (Fulvestrant) n = 7	Breast HR+ With Prior CDK4/6i (Fulvestrant + Trastuzumab) n = 7
Value	40.7	0	14.3

Population analysed: Cohort of cancer type and treatment

Primary outcome: Confirmed Objective Response Rate (ORR) by Investigator Review (Cervical Cancer Cohort)

Time frame: From enrollment date to first confirmed Complete or Partial Response, whichever came earlier, assessed up to 58 months. Number. Unit: percentage of participants

Measure	Cervical (Neratinib) n = 22
Value	18.2

Population analysed: Cohort of cancer type and treatment

Primary outcome: Objective Response Rate (ORR) at First Assessment by Investigator Review (All Other Cohorts)

Time frame: From first treatment date to first Complete or Partial Response, whichever came earlier, assessed up to 8 or 9 weeks. Number. Unit: percentage of participant

Measure	Breast Cancer (Neratinib) n = 36	Breast Cancer HR+ (Neratinib + Fulvestrant) n = 45	Breast Cancer HR+ (Neratinib + Fulvestrant + Trastuzumab) n = 31	Breast Cancer HR- (Neratinib + Trastuzumab) n = 21	Lung Cancer HER2 Mutant (Neratinib) n = 26	Lung Cancer HER2 Mutant (Neratinib + Trastuzumab) n = 52	Lung Cancer EGFR Mutant Exon 18 (Neratinib) n = 31	Biliary Tract Cancer (Neratinib) n = 25	Bladder/Urinary Tract Cancer (Neratinib) n = 16	Bladder/Urinary Tract Cancer (Neratinib + Paclitaxel) n = 22	Brain Cancer (Neratinib) n = 38	Colorectal Cancer (Neratinib) n = 12	Colorectal Cancer (Neratinib + Trastuzumab) n = 19	Endometrial Cancer (Neratinib) n = 7	Ovarian Cancer (Neratinib) n = 10	Salivary Gland Cancer (Neratinib) n = 11	Gastroesophageal Cancer (Neratinib) n = 7	Fibrolamellar Carcinoma (FLC) (Neratinib) n = 15	HER2 NOS Cancer (Neratinib) n = 42	HER3 NOS Cancer (Neratinib) n = 16	HER4 NOS Cancer (Neratinib) n = 3
Value	36.1	42.2	48.4	33.3	3.8	15.4	19.4	12.0	0	13.6	0	0	5.3	0	0	36.4

9 secondary outcomes are reported in the full results.

Adverse events

Time frame: All events from day 1 of study drug through 28 days after last dose are included, up to 75 months.. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Neratinib	144 of 317 (45.4%)	304 of 317 (95.9%)	231 of 317
Neratinib + Fulvestrant	12 of 45 (26.7%)	45 of 45 (100%)	31 of 45
Neratinib + Paclitaxel	13 of 22 (59.1%)	20 of 22 (90.9%)	14 of 22
Neratinib + Trastuzumab	45 of 92 (48.9%)	92 of 92 (100%)	71 of 92
Neratinib + Fulvestrant + Trastuzumab	28 of 90 (31.1%)	89 of 90 (98.9%)	32 of 90
Fulvestrant	5 of 7 (71.4%)	7 of 7 (100%)	2 of 7
Fulvestrant + Trastuzumab	0 of 7 (0%)	7 of 7 (100%)	0 of 7

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 12 Mar 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 29420467Hyman DM, Piha-Paul SA, Won H, Rodon J, Saura C, Shapiro GI, Juric D, Quinn DI, Moreno V, Doger B, Mayer IA, Boni V, Calvo E, Loi S, Lockhart AC, Erinjeri JP, Scaltriti M, Ulaner GA, Patel J, Tang J, Beer H, Selcuklu SD, Hanrahan AJ, Bouvier N, Melcer M, Murali R, Schram AM, Smyth LM, Jhaveri K, Li BT, Drilon A, Harding JJ, Iyer G, Taylor BS, Berger MF, Cutler RE Jr, Xu F, Butturini A, Eli LD, Mann G, Farrell C, Lalani AS, Bryce RP, Arteaga CL, Meric-Bernstam F, Baselga J, Solit DB. HER kinase inhibition in patients with HER2- and HER3-mutant cancers. Nature. 2018 Feb 8;554(7691):189-194. doi: 10.1038/nature25475. Epub 2018 Jan 31.
Background: PubMed 31806627Smyth LM, Piha-Paul SA, Won HH, Schram AM, Saura C, Loi S, Lu J, Shapiro GI, Juric D, Mayer IA, Arteaga CL, de la Fuente MI, Brufksy AM, Spanggaard I, Mau-Sorensen M, Arnedos M, Moreno V, Boni V, Sohn J, Schwartzberg LS, Gonzalez-Farre X, Cervantes A, Bidard FC, Gorelick AN, Lanman RB, Nagy RJ, Ulaner GA, Chandarlapaty S, Jhaveri K, Gavrila EI, Zimel C, Selcuklu SD, Melcer M, Samoila A, Cai Y, Scaltriti M, Mann G, Xu F, Eli LD, Dujka M, Lalani AS, Bryce R, Baselga J, Taylor BS, Solit DB, Meric-Bernstam F, Hyman DM. Efficacy and Determinants of Response to HER Kinase Inhibition in HER2-Mutant Metastatic Breast Cancer. Cancer Discov. 2020 Feb;10(2):198-213. doi: 10.1158/2159-8290.CD-19-0966. Epub 2019 Dec 5.
Derived from this study: PubMed 41193346Li BT, Gandhi L, Ravichandran V, Besse B, Mazieres J, Shapiro GI, Boni V, Waqar SN, Park H, Quinn DI, Martinez A, Stemmer SM, Cortot AB, Barlesi F, Quoix E, Burkard ME, Selcuklu SD, Hunt C, Donoghue MTA, Kris MG, Bebchuk J, Eli LD, McCulloch L, Solit DB, Janne PA. Efficacy of Neratinib-Based Therapy in ERBB2-Mutant Lung Adenocarcinomas: Findings From 2 International Phase 2 Studies. Clin Lung Cancer. 2026 Mar;27(2):214-226.e5. doi: 10.1016/j.cllc.2025.09.009. Epub 2025 Oct 1.
Derived from this study: PubMed 38211393Friedman CF, D'Souza A, Bello Roufai D, Tinker AV, de Miguel M, Gambardella V, Goldman J, Loi S, Melisko ME, Oaknin A, Spanggaard I, Shapiro GI, ElNaggar AC, Panni S, Ravichandran V, Frazier AL, DiPrimeo D, Eli LD, Solit DB. Targeting HER2-mutant metastatic cervical cancer with neratinib: Final results from the phase 2 SUMMIT basket trial. Gynecol Oncol. 2024 Feb;181:162-169. doi: 10.1016/j.ygyno.2023.12.004. Epub 2024 Jan 11.
Derived from this study: PubMed 37597578Jhaveri K, Eli LD, Wildiers H, Hurvitz SA, Guerrero-Zotano A, Unni N, Brufsky A, Park H, Waisman J, Yang ES, Spanggaard I, Reid S, Burkard ME, Vinayak S, Prat A, Arnedos M, Bidard FC, Loi S, Crown J, Bhave M, Piha-Paul SA, Suga JM, Chia S, Saura C, Garcia-Saenz JA, Gambardella V, de Miguel MJ, Gal-Yam EN, Rapael A, Stemmer SM, Ma C, Hanker AB, Ye D, Goldman JW, Bose R, Peterson L, Bell JSK, Frazier A, DiPrimeo D, Wong A, Arteaga CL, Solit DB. Neratinib + fulvestrant + trastuzumab for HR-positive, HER2-negative, HER2-mutant metastatic breast cancer: outcomes and biomarker analysis from the SUMMIT trial. Ann Oncol. 2023 Oct;34(10):885-898. doi: 10.1016/j.annonc.2023.08.003. Epub 2023 Aug 18.
Derived from this study: PubMed 36746967Harding JJ, Piha-Paul SA, Shah RH, Murphy JJ, Cleary JM, Shapiro GI, Quinn DI, Brana I, Moreno V, Borad M, Loi S, Spanggaard I, Park H, Ford JM, Arnedos M, Stemmer SM, de la Fouchardiere C, Fountzilas C, Zhang J, DiPrimeo D, Savin C, Duygu Selcuklu S, Berger MF, Eli LD, Meric-Bernstam F, Jhaveri K, Solit DB, Abou-Alfa GK. Antitumour activity of neratinib in patients with HER2-mutant advanced biliary tract cancers. Nat Commun. 2023 Feb 6;14(1):630. doi: 10.1038/s41467-023-36399-y.
Derived from this study: PubMed 35181666Shishido SN, Masson R, Xu L, Welter L, Prabakar RK, D' Souza A, Spicer D, Kang I, Jayachandran P, Hicks J, Lu J, Kuhn P. Disease characterization in liquid biopsy from HER2-mutated, non-amplified metastatic breast cancer patients treated with neratinib. NPJ Breast Cancer. 2022 Feb 18;8(1):22. doi: 10.1038/s41523-022-00390-5.
Derived from this study: PubMed 32723675Oaknin A, Friedman CF, Roman LD, D'Souza A, Brana I, Bidard FC, Goldman J, Alvarez EA, Boni V, ElNaggar AC, Passalacqua R, Do KTM, Santin AD, Keyvanjah K, Xu F, Eli LD, Lalani AS, Bryce RP, Hyman DM, Meric-Bernstam F, Solit DB, Monk BJ. Neratinib in patients with HER2-mutant, metastatic cervical cancer: Findings from the phase 2 SUMMIT basket trial. Gynecol Oncol. 2020 Oct;159(1):150-156. doi: 10.1016/j.ygyno.2020.07.025. Epub 2020 Jul 25.
Derived from this study: PubMed 31548342Ulaner GA, Saura C, Piha-Paul SA, Mayer I, Quinn D, Jhaveri K, Stone B, Shahin S, Mann G, Dujka M, Bryce R, Meric-Bernstam F, Solit DB, Hyman DM. Impact of FDG PET Imaging for Expanding Patient Eligibility and Measuring Treatment Response in a Genome-Driven Basket Trial of the Pan-HER Kinase Inhibitor, Neratinib. Clin Cancer Res. 2019 Dec 15;25(24):7381-7387. doi: 10.1158/1078-0432.CCR-19-1658. Epub 2019 Sep 23.
Derived from this study: PubMed 28274957Hanker AB, Brewer MR, Sheehan JH, Koch JP, Sliwoski GR, Nagy R, Lanman R, Berger MF, Hyman DM, Solit DB, He J, Miller V, Cutler RE Jr, Lalani AS, Cross D, Lovly CM, Meiler J, Arteaga CL. An Acquired HER2T798I Gatekeeper Mutation Induces Resistance to Neratinib in a Patient with HER2 Mutant-Driven Breast Cancer. Cancer Discov. 2017 Jun;7(6):575-585. doi: 10.1158/2159-8290.CD-16-1431. Epub 2017 Mar 8.
HER Kinase Inhibition in Patients With HER2- And HER3-mutant CancersLink provided by the sponsor.
Efficacy and Determinants of Response to HER Kinase Inhibition in HER2 -Mutant Metastatic Breast CancerLink provided by the sponsor.
Individual participant dataSharing planned: 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.

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Chief Scientific Officer study director

Puma Biotechnology, Inc.

60 sites in 13 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 12 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 60 sites in United States, Spain, Israel, France, Italy and 8 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 12 Mar 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Confirmed Objective Response Rate (ORR) by Independent Central Review (Breast Cancer With Prior CDK46i Cohort) (time frame: From enrollment date to first confirmed Complete or Partial Response, whichever came earlier, assessed up to 58 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01953926, which also lists 10 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Mar 2024; AzTrials last refreshed its copy on 7 Nov 2026.