A Phase Ib/II Study of BYL719 and Cetuximab in Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma

NCT ID: NCT01602315

Last Updated: 2020-12-29

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

TERMINATED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

179 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-11-12

Study Completion Date

2016-09-16

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

This was a multi-center, open-label, Phase Ib dose escalation /Phase II study in recurrent or metastatic head and neck squamous cell carcinoma (RM HNSCC) patients considered to be resistant, ineligible or intolerant to platinum-based chemotherapy. The Phase Ib included three arms. Three different methods of administration and two different BYL719 formulations were studied to determine the MTD and/or RP2D of BYL719 in combination with cetuximab:

Arm A - film-coated whole tablets were orally administered to patients who were able to swallow the tablets; Arm B - a drinkable suspension prepared from crushed film-coated tablets was administered orally to patients with swallowing dysfunction Arm C - a suspension from a dispersible tablet administered via G-tube, in patients with swallowing dysfunction. Arm C was used to investigate the pharmacokinetics (PK), compared to Arm A (film coated tablet), and safety of the dispersible tablet of the dispersible tablet formulation of BYL719.

The Phase II investigated the clinical efficacy of BYL719 and consisted of an open label, randomized Phase II part investigating BYL719 in combination with cetuximab compared to cetuximab alone in patients resistant or intolerant to platinum and naïve to cetuximab (Scheme 1: Arm 1 and Arm 2), and a non-randomized Phase II part Scheme 2: Arm 3. In addition, patients who experienced disease progression in Arm 2 (cetuximab) were allowed to switch to the combination regimen (cross-over, Arm 2B). The safety of the BYL719 in combination with cetuximab was also further characterized in Arms 1, 2B and 3.

Patients were treated until progression of disease), unacceptable toxicity, or withdrawal of informed consent, whichever occurred first (except for phase II Arm 2 had the opportunity to crossover to the combination treatment (Arm 2B). In the follow-up period all patients had to complete the safety follow-up assessments within 30 days after the last dose of the study treatment. Patients who did not have disease progression at the time of discontinuation of study treatment were radiologically followed for disease status until disease progression, initiation of subsequent anticancer therapies, or death, whichever occurred first. In addition, all patients enrolled in Phase II were followed for survival.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Recurrent Head and Neck Squamous Cell Carcinoma Metastatic Head and Neck Squamous Cell Carcinoma

Keywords

Explore important study keywords that can help with search, categorization, and topic discovery.

BYL719 PI3K inhibitor PIK3CA cetuximab EGFR HNSCC RM HNSCC platinum-based chemotherapy (RM HNSCC) patients resistant or ineligible/intolerant to platinum-based chemotherapy swallowing dysfunction G-tube alpelisib

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Phase Ib: A-BYL719 FC whole tab+cetux

Oral film-coated tablets without swallowing dysfunction.

Group Type EXPERIMENTAL

BYL719 as film-coated (FC) whole tablets

Intervention Type DRUG

Oral alpha-specific PI3K inhibitor

cetuximab

Intervention Type BIOLOGICAL

Recombinant chimeric monoclonal antibody driven against EGFR

Phase II: 2-Cetuximab

Cetuximab in patients naive to cetuximab (phase ll)

Group Type EXPERIMENTAL

cetuximab

Intervention Type BIOLOGICAL

Recombinant chimeric monoclonal antibody driven against EGFR

Phase Ib: B-BYL719 FC drink sus+cetux

Crushed film-coated (FC) tablets as an oral suspension with swallowing dysfunction.

Group Type EXPERIMENTAL

cetuximab

Intervention Type BIOLOGICAL

Recombinant chimeric monoclonal antibody driven against EGFR

BYL719 drink suspension

Intervention Type DRUG

Oral alpha-specific PI3K inhibitor

Phase II: 3-BYL719 + Cetuximab

BYL719 + cetuximab in patients resistant to cetuximab. BYL719 can be administered as FC whole/crushed only or DT via G-tube in addition, depending on the Phase Ib results

Group Type EXPERIMENTAL

BYL719 as film-coated (FC) whole tablets

Intervention Type DRUG

Oral alpha-specific PI3K inhibitor

cetuximab

Intervention Type BIOLOGICAL

Recombinant chimeric monoclonal antibody driven against EGFR

BYL719 drink suspension

Intervention Type DRUG

Oral alpha-specific PI3K inhibitor

Phase II: 1-BYL719 + Cetuximab

BYL719 + Cetuximab in Patients naive to cetuximab. BYL719 can be administered as FC whole/crushed only or DT via G-tube in addition, depending on the Phase Ib results

Group Type EXPERIMENTAL

BYL719 as film-coated (FC) whole tablets

Intervention Type DRUG

Oral alpha-specific PI3K inhibitor

cetuximab

Intervention Type BIOLOGICAL

Recombinant chimeric monoclonal antibody driven against EGFR

BYL719 drink suspension

Intervention Type DRUG

Oral alpha-specific PI3K inhibitor

Phase Ib: C-BYL719 DT+cetux

Dispersible tablet with swallowing dysfunction administered via a gastrostomy tube (G-tube)

Group Type EXPERIMENTAL

BYL719 as dispersible tablets (DT)

Intervention Type DRUG

New formulation of the oral alpha-specific PI3K inhibitor

cetuximab

Intervention Type BIOLOGICAL

Recombinant chimeric monoclonal antibody driven against EGFR

Phase II: Cross over

patients received BYL719 at RP2D in combination with cetuximab.

Group Type EXPERIMENTAL

BYL719 as film-coated (FC) whole tablets

Intervention Type DRUG

Oral alpha-specific PI3K inhibitor

cetuximab

Intervention Type BIOLOGICAL

Recombinant chimeric monoclonal antibody driven against EGFR

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

BYL719 as film-coated (FC) whole tablets

Oral alpha-specific PI3K inhibitor

Intervention Type DRUG

BYL719 as dispersible tablets (DT)

New formulation of the oral alpha-specific PI3K inhibitor

Intervention Type DRUG

cetuximab

Recombinant chimeric monoclonal antibody driven against EGFR

Intervention Type BIOLOGICAL

BYL719 drink suspension

Oral alpha-specific PI3K inhibitor

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

NVP-BYL719 alpelisib NVP-BYL719 erbitux NVP-BYL719

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Age ≥ 18 years
* Patients with histologically/cytologically-confirmed HNSCC
* Patients must be resistant to platinum-based chemotherapy, or be ineligible (due to medical comorbidities) or intolerant to platinum-based therapy per medical history
* For Phase Ib, there is no restriction on the number of prior therapies for recurrent or metastatic disease
* For Phase II, patients may have received a maximum of 1 prior line of therapy for recurrent or metastatic disease
* For Phase Ib, prior cetuximab or other EGFR-targeted antibody therapy is allowed regardless of the prior treatment settings.
* For Phase II, Arms 1 and 2, prior cetuximab or other EGFR-targeted antibody therapy is allowed only if administered in the induction setting, or concurrently with radiation in the curative setting, with the last dose of cetuximab administered at least 12 months prior to starting the study treatment. For Arm 3, prior cetuximab must have been administered in the curative, recurrent or metastatic disease setting and disease progression documented within 9 months of the last dose of cetuximab administered in that setting. This regimen (including both platinum and cetuximab) must be the most recent anti-neoplastic treatment regimen administered.
* Patients with swallowing dysfunction who are unable to swallow BYL719 whole tablets and are not using feeding tubes for study drug administration can participate in the Phase Ib Arm B. For the Phase II, these patients with swallowing dysfunction may participate if able to drink the suspension and results of Arm B confirm the use of this method. Patients with swallowing dysfunction requiring G tube (G/PEG tube) for study drug administration may participate in Phase II if Arm C confirms dispersible tablet via G tube administration is permitted if the administration of drinkable suspension of BYL719 is allowed to be used in Phase II.
* Availability of a representative tumor specimen. Patients enrolled in Arm 3 of Phase II must have disease sites amenable to biopsy unless prior agreement between Novartis and the Investigator.
* At least one measurable or non-measurable lesion as per RECIST 1.1 criteria for patients in Phase Ib; Measurable disease as determined by RECIST v1.1 for Phase II patients
* World Health Organization (WHO) Performance Status (PS) ≤ 2
* Adequate organ function
* Negative serum pregnancy test.

Exclusion Criteria

* Prior treatment with PI3K-inhibitors
* Patients with a prior serious infusion reaction to cetuximab
* Patients with uncontrolled CNS tumor metastatic involvement
* Clinically significant cardiac disease or impaired cardiac function
* Patients with diabetes mellitus
* Impaired GI function or GI disease
* History of another malignancy within 2 years prior to starting study treatment
* Pregnant or nursing (lactating) women
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Novartis Investigative Site

Sacramento, California, United States

Site Status

Novartis Investigative Site

San Francisco, California, United States

Site Status

Novartis Investigative Site

Aurora, Colorado, United States

Site Status

Novartis Investigative Site

Jacksonville, Florida, United States

Site Status

Novartis Investigative Site

Orlando, Florida, United States

Site Status

Novartis Investigative Site

Atlanta, Georgia, United States

Site Status

Novartis Investigative Site

Boston, Massachusetts, United States

Site Status

Novartis Investigative Site

St Louis, Missouri, United States

Site Status

Novartis Investigative Site

New York, New York, United States

Site Status

Novartis Investigative Site

New York, New York, United States

Site Status

Novartis Investigative Site

Philadelphia, Pennsylvania, United States

Site Status

Novartis Investigative Site

Charleston, South Carolina, United States

Site Status

Novartis Investigative Site

Nashville, Tennessee, United States

Site Status

Novartis Investigative Site

Houston, Texas, United States

Site Status

Novartis Investigative Site

Melbourne, Victoria, Australia

Site Status

Novartis Investigative Site

Murdoch, Western Australia, Australia

Site Status

Novartis Investigative Site

Toronto, Ontario, Canada

Site Status

Novartis Investigative Site

Lyon, , France

Site Status

Novartis Investigative Site

Toulouse, , France

Site Status

Novartis Investigative Site

Shatin, New Territories, Hong Kong, Hong Kong

Site Status

Novartis Investigative Site

Maastricht, , Netherlands

Site Status

Novartis Investigative Site

Nijmegen, , Netherlands

Site Status

Novartis Investigative Site

Singapore, , Singapore

Site Status

Novartis Investigative Site

Seoul, Korea, South Korea

Site Status

Novartis Investigative Site

Seoul, Korea, South Korea

Site Status

Novartis Investigative Site

Seoul, , South Korea

Site Status

Novartis Investigative Site

Tainan City, Taiwan ROC, Taiwan

Site Status

Novartis Investigative Site

Kuei-Shan Chiang, Taoyuan/ Taiwan ROC, Taiwan

Site Status

Novartis Investigative Site

Taipei, , Taiwan

Site Status

Countries

Review the countries where the study has at least one active or historical site.

Argentina Brazil United States Australia Canada France Hong Kong Netherlands Singapore South Korea Taiwan

References

Explore related publications, articles, or registry entries linked to this study.

Razak ARA, Wang HM, Chang JY, Ahn MJ, Munster P, Blumenschein G Jr, Solomon B, Lim DW, Hong RL, Pfister D, Saba NF, Lee SH, van Herpen C, Quadt C, Bootle D, Blumenstein L, Demanse D, Delord JP. A Phase 1b/2 Study of Alpelisib in Combination with Cetuximab in Patients with Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma. Target Oncol. 2023 Nov;18(6):853-868. doi: 10.1007/s11523-023-00997-z. Epub 2023 Oct 25.

Reference Type DERIVED
PMID: 37875771 (View on PubMed)

Related Links

Access external resources that provide additional context or updates about the study.

https://www.novctrd.com/ctrdweb/trialresult/trialresults/pdf?trialResultId=16267

Results for CBYL719X2104 can be found on the Novartis Clinical Trial Results Website

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

2011-006017-34

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CBYL719X2104

Identifier Type: -

Identifier Source: org_study_id