NCT03803774

Brief Summary

This phase I trial studies the side effects and best dose of birinapant when given together with intensity modulated re-irradiation therapy (IMRRT) in treating patients with head and neck squamous cell carcinoma that has come back at or near the same place as the original (primary) tumor (locally recurrent). Birinapant may stop the growth of tumor cells by blocking inhibitor of apoptosis (IAP), a protein needed for tumor cell survival. IMRRT uses thin beams of radiation of different intensities that are aimed at the tumor from many angles. This type of re-irradiation therapy reduces the damage to healthy tissue near the tumor. Giving birinapant with IMRRT may lower the chance of head and neck squamous cell carcinoma growing or spreading.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
13

participants targeted

Target at below P25 for phase_1

Timeline
2mo left

Started Sep 2019

Longer than P75 for phase_1

Geographic Reach
1 country

33 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress97%
Sep 2019Jul 2026

First Submitted

Initial submission to the registry

January 14, 2019

Completed
1 day until next milestone

First Posted

Study publicly available on registry

January 15, 2019

Completed
8 months until next milestone

Study Start

First participant enrolled

September 25, 2019

Completed
4.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 15, 2023

Completed
8 months until next milestone

Results Posted

Study results publicly available

July 23, 2024

Completed
2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

July 9, 2026

Expected
Last Updated

April 29, 2026

Status Verified

January 1, 2026

Enrollment Period

4.1 years

First QC Date

January 14, 2019

Results QC Date

July 1, 2024

Last Update Submit

April 9, 2026

Conditions

Outcome Measures

Primary Outcomes (2)

  • Incidence of Dose-limiting Toxicities (DLTs) and Grades 1-5 Serious and/or Non-serious Toxicities Related (Except for Unrelated and Unlikely) to Intervention

    Adverse events will be graded using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. A DLT is defined as any of the following adverse events possibly attributed to the combination of birinapant and radiotherapy that occur within 42 days after treatment. Any grade 5 toxicities. Any grade ≥ 4 hematologic toxicity, except lymphopenia. Any grade ≥ 3 non-hematologic toxicity except for nausea or vomiting managed with supportive care over 2 weeks. ≥ grade 3 prolonged (\> 7 days) serum amylase or lipase elevation, aspartate aminotransferase elevation, and/or alanine aminotransferase elevation. Any grade toxicity that mandates discontinuation of birinapant treatment for more than 2 weeks. Grade 1 is mild. Grade 2 is moderate. Grade 3 is severe. Grade 4 is life-threatening. Grade 5 is death related to adverse event.

    Up to 42 days post-treatment

  • Maximum Tolerated Dose (MTD) of Birinapant

    MTD is defined as the dose level at which no more than 1 of up to 6 participants experience dose limiting- toxicity (DLT) during 42 days after the start of therapy, and the dose below that at which at least 2 (of =\< 6) participants have DLT as a result of the drug. A DLT is defined as any of the following adverse events possibly attributed to the combination of birinapant and radiotherapy that occur within 42 days after treatment. Any grade 5 toxicities. Any grade ≥ 4 hematologic toxicity except lymphopenia. Any grade ≥ 3 non-hematologic toxicity except for nausea or vomiting managed with supportive care over 2 weeks. ≥ grade 3 prolonged (\> 7 days) serum amylase or lipase elevation, aspartate aminotransferase elevation, and/or alanine aminotransferase elevation. Any grade toxicity that mandates discontinuation of birinapant treatment for more than 2 weeks. Adverse events were graded using the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.

    Up to 42 days

Secondary Outcomes (11)

  • Response Rate

    From the start of the treatment until response assessment by positron emission tomography (PET)-computed tomography (CT), assessed at 3 months post-treatment

  • Local-regional Control

    Up to 24 months post-treatment

  • Progression-free Survival (PFS)

    From start of treatment to time of progression or death, whichever occurs first, assessed up to 24 months post-treatment

  • Overall Survival (OS)

    Up to 24 months post-treatment

  • Fas-associated Protein With Death Domain (FADD) Copy Gain in Tumor Tissue and/or in Blood Associated With Response

    At baseline

  • +6 more secondary outcomes

Other Outcomes (1)

  • Number of Participants With Serious and/or Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0)

    An average of 611 days.

Study Arms (1)

Treatment (IMRRT, birinapant)

EXPERIMENTAL

Beginning on day 1, patients undergo IMRRT 5 days a week (Monday-Friday). Patients also receive birinapant IV over 30 minutes on days 2 and 9 of each cycle. Treatment repeats every 3 weeks for up to 2 cycles in the absence of disease progression or unacceptable toxicity.

Procedure: Biopsy ProcedureDrug: BirinapantProcedure: Computed TomographyRadiation: Intensity-Modulated Radiation TherapyProcedure: Magnetic Resonance ImagingProcedure: Positron Emission Tomography

Interventions

Undergo biopsy

Also known as: Biopsy, BIOPSY_TYPE, Bx
Treatment (IMRRT, birinapant)

Given IV

Also known as: TL32711
Treatment (IMRRT, birinapant)

Undergo CT scan

Also known as: CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography, Computerized axial tomography (procedure), Computerized Tomography, Computerized Tomography (CT) scan, CT, CT Scan, Diagnostic CAT Scan, Diagnostic CAT Scan Service Type, tomography
Treatment (IMRRT, birinapant)

Undergo IMRRT

Also known as: IMRT, Intensity modulated radiation therapy (procedure), Intensity Modulated RT, Intensity-Modulated Radiotherapy, Radiation, Intensity-Modulated Radiotherapy
Treatment (IMRRT, birinapant)

Undergo MRI

Also known as: Magnetic Resonance, Magnetic Resonance Imaging (MRI), Magnetic resonance imaging (procedure), Magnetic Resonance Imaging Scan, Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance, MR, MR Imaging, MRI, MRI Scan, MRIs, NMR Imaging, NMRI, Nuclear Magnetic Resonance Imaging, sMRI, Structural MRI
Treatment (IMRRT, birinapant)

Undergo PET scan

Also known as: Medical Imaging, Positron Emission Tomography, PET, PET Scan, Positron emission tomography (procedure), Positron Emission Tomography Scan, Positron-Emission Tomography, PT
Treatment (IMRRT, birinapant)

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Patients must have histologically or cytologically confirmed locally recurrent HNSCC, including nasopharyngeal or sinonasal cancer for whom re-irradiation for local control is considered standard of care
  • Patients with human papillomavirus (HPV)-negative or HPV-positive head and neck cancer are eligible
  • Patients who have had prior treatment with immune therapies are eligible
  • Patients must have received curative-intent platinum- and/or cetuximab-based chemoradiotherapy or radiotherapy alone
  • Patients must have completed their last treatment dose with chemotherapy or immunotherapy at least 4 weeks (6 weeks for nitrosoureas or mitomycin C) before enrolling on study
  • Patients must have completed their last treatment dose with radiotherapy at least 6 months before enrolling on study
  • Patients who have had major surgery must be fully recovered and require a recovery period of at least 4 weeks prior to enrolling on study
  • Age \>= 18 years
  • Eastern Cooperative Oncology Group (ECOG) performance status =\< 2
  • Hemoglobin \>= 9 g/dL (transfusion permitted)
  • Absolute neutrophil count \>= 1,500/mcL
  • Platelets \>= 100,000/mcL
  • Total bilirubin within 1.5 x the upper limit of normal (ULN) institutional limits
  • Aspartate aminotransferase (AST) (serum glutamic-oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase \[SGPT\]) =\< 2.5 x institutional upper limit of normal
  • Serum creatinine =\< 1.5 x upper limit of normal (ULN), OR: Creatinine clearance \>= 50 mL/min according to Cockcroft Gault formula or other institutional methods
  • +5 more criteria

You may not qualify if:

  • Eligibility for curative-intent surgery, unless the patient is considered a poor surgical candidate related to resectability, functional outcome, or prefers non-surgical therapy
  • More than 2 lines of palliative systemic therapy (platinum-, taxane- or cetuximab-based chemotherapy or immunotherapy)
  • Patients who are receiving any other investigational agents
  • Patients with known brain metastases should be excluded from this clinical trial because of their poor prognosis and because they often develop progressive neurologic dysfunction that would confound the evaluation of neurologic and other adverse events
  • History of allergic reactions attributed to compounds of similar chemical or biologic composition to birinapant
  • Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
  • Pregnant women are excluded from this study because birinapant may have potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with birinapant, breastfeeding should be discontinued prior to enrollment. A negative pregnancy test is required for women of childbearing potential. Women who are postmenopausal (age-related amenorrhea \>= 12 consecutive months, or who had undergone hysterectomy or bilateral oophorectomy are exempt from pregnancy testing. If necessary, to confirm postmenopausal status, a follicle stimulating hormone (FSH) level may be included at screening
  • Human immunodeficiency virus (HIV) positive patients on combination antiretroviral therapy are ineligible because of the potential for pharmacokinetic interactions with birinapant
  • Patients requiring the use of anti-tumor necrosis factor (anti TNF) therapies, such as infliximab, or patients who have received treatment with anti-TNF therapies within 5 half-lives of the drug (48 days for infliximab, 55 days for golimumab, 70 days for certolizumab and adalimumab, and 16 days for etanercept)
  • Patients with previous exposure to birinapant

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (33)

University of Alabama at Birmingham Cancer Center

Birmingham, Alabama, 35233, United States

Location

Los Angeles General Medical Center

Los Angeles, California, 90033, United States

Location

USC / Norris Comprehensive Cancer Center

Los Angeles, California, 90033, United States

Location

UC Irvine Health/Chao Family Comprehensive Cancer Center

Orange, California, 92868, United States

Location

UM Sylvester Comprehensive Cancer Center at Coral Gables

Coral Gables, Florida, 33146, United States

Location

UM Sylvester Comprehensive Cancer Center at Deerfield Beach

Deerfield Beach, Florida, 33442, United States

Location

University of Miami Miller School of Medicine-Sylvester Cancer Center

Miami, Florida, 33136, United States

Location

UM Sylvester Comprehensive Cancer Center at Plantation

Plantation, Florida, 33324, United States

Location

Moffitt Cancer Center-International Plaza

Tampa, Florida, 33607, United States

Location

Moffitt Cancer Center - McKinley Campus

Tampa, Florida, 33612, United States

Location

Moffitt Cancer Center

Tampa, Florida, 33612, United States

Location

University of Kansas Clinical Research Center

Fairway, Kansas, 66205, United States

Location

HaysMed

Hays, Kansas, 67601, United States

Location

University of Kansas Cancer Center

Kansas City, Kansas, 66160, United States

Location

Lawrence Memorial Hospital

Lawrence, Kansas, 66044, United States

Location

The University of Kansas Cancer Center - Olathe

Olathe, Kansas, 66061, United States

Location

University of Kansas Cancer Center-Overland Park

Overland Park, Kansas, 66210, United States

Location

Mercy Hospital Pittsburg

Pittsburg, Kansas, 66762, United States

Location

Salina Regional Health Center

Salina, Kansas, 67401, United States

Location

University of Kansas Health System Saint Francis Campus

Topeka, Kansas, 66606, United States

Location

University of Kansas Hospital-Westwood Cancer Center

Westwood, Kansas, 66205, United States

Location

University of Kentucky/Markey Cancer Center

Lexington, Kentucky, 40536, United States

Location

NCI - Center for Cancer Research

Bethesda, Maryland, 20892, United States

Location

University Health Truman Medical Center

Kansas City, Missouri, 64108, United States

Location

University of Kansas Cancer Center - North

Kansas City, Missouri, 64154, United States

Location

University of Kansas Cancer Center - Lee's Summit

Lee's Summit, Missouri, 64064, United States

Location

University of Kansas Cancer Center at North Kansas City Hospital

North Kansas City, Missouri, 64116, United States

Location

NYU Langone Hospital - Long Island

Mineola, New York, 11501, United States

Location

Laura and Isaac Perlmutter Cancer Center at NYU Langone

New York, New York, 10016, United States

Location

Wake Forest Baptist Health - Wilkes Medical Center

Wilkesboro, North Carolina, 28659, United States

Location

Wake Forest University Health Sciences

Winston-Salem, North Carolina, 27157, United States

Location

Ohio State University Comprehensive Cancer Center

Columbus, Ohio, 43210, United States

Location

Huntsman Cancer Institute/University of Utah

Salt Lake City, Utah, 84112, United States

Location

MeSH Terms

Interventions

BiopsybirinapantRadiotherapy, Intensity-ModulatedMagnetic Resonance Spectroscopy

Intervention Hierarchy (Ancestors)

CytodiagnosisCytological TechniquesClinical Laboratory TechniquesDiagnostic Techniques and ProceduresDiagnosisSpecimen HandlingDiagnostic Techniques, SurgicalSurgical Procedures, OperativeInvestigative TechniquesRadiotherapy, ConformalRadiotherapy, Computer-AssistedRadiotherapyTherapeuticsSpectrum AnalysisChemistry Techniques, Analytical

Results Point of Contact

Title
Dr. Vassiliki Saloura
Organization
National Cancer Institute

Study Officials

  • Vassiliki Saloura

    National Cancer Institute LAO

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
LTE60
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
NIH
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 14, 2019

First Posted

January 15, 2019

Study Start

September 25, 2019

Primary Completion

November 15, 2023

Study Completion (Estimated)

July 9, 2026

Last Updated

April 29, 2026

Results First Posted

July 23, 2024

Record last verified: 2026-01

Data Sharing

IPD Sharing
Will share

NCI is committed to sharing data in accordance with NIH policy. For more details on how clinical trial data is shared, access the link to the NIH data sharing policy page.

More information

Locations