AzTrials
AzTrials

Squamous Cell Carcinoma of the Head and NeckPhase 2NCT02369458

Mitomycin C in Patients With Incurable p16 Positive Oropharyngeal and p16 Negative Head and Neck Squamous Cell Carcinoma (HNSCC) Resistant to Standard Therapies

A Phase 2 study testing Mitomycin-C and Pegfilgrastim in people with Squamous Cell Carcinoma of the Head and Neck and Squamous Cell Carcinoma, Head and Neck, run by Washington University School of Medicine at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

No agent is known to have efficacy in patients with incurable HNSCC that progressed with prior platin, 5-FU, cetuximab and taxane. Herein lies the unmet need to be addressed by this trial. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Mitomycin-C and Pegfilgrastim
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
48 participants
Where
1 siteUnited States
Study dates
14 Apr 2015to 1 Nov 2024
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

  • Histologically or cytologically confirmed incurable HNSCC of the oral cavity, oropharynx, larynx, hypopharynx, and/or Level 1-3 neck node with non-cutaneous SCC and unknown primary. "Incurable" is defined as metastatic disease or a local or regional recurrence in a previously irradiated site that is unresectable (or patient declines resection).
  • Progression following platin and immunotherapy given for incurable disease.
  • Measurable disease defined as lesions that can be accurately measured in at least one dimension (longest diameter to be recorded) as ≥ 10 mm with CT scan, as ≥ 20 mm by chest x-ray, or ≥ 10 mm with calipers by clinical exam per RECIST 1.1.
  • Tissue available (either initial diagnostic or recurrent tissue specimen) for p16 testing.
  • At least 18 years of age.
  • ECOG performance status ≤ 3
Show all 14Show fewer
  • Adequate hematologic, renal, and hepatic function as defined below:
  • Absolute neutrophil count ≥ 1,000/mcl
  • Platelets ≥ 75,000/mcl
  • Total bilirubin ≤ 1.5 mg/dL
  • AST(SGOT)/ALT(SGPT) ≤ 2.5 x ULN, alkaline phosphatase ≤ 2.5 x ULN, unless bone metastasis is present in the absence of liver metastasis
  • Creatinine below ULN (males 0.7-1.30 mg/dl; females 0.6-1.10 mg/dl) OR Creatinine clearance ≥ 60 mL/min/1.73 m2 for patients with creatinine levels above institutional normal
  • Women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control, abstinence) prior to study entry, for the duration of study participation, and for 1 month after completing treatment. Should a woman become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately.
  • Ability to understand and willingness to sign an IRB approved written informed consent document (or that of legally authorized representative, if applicable).

Who cannot join

  • Other active malignancy with the exception of basal cell or squamous cell carcinoma of the skin which were treated with local resection only, carcinoma in situ of the cervix, or synchronous H\&N primaries.
  • Currently receiving any other investigational agents.
  • 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 and/or breastfeeding. Patient must have a negative pregnancy test within 7 days of start of study treatment.
  • Known active central nervous system (CNS) metastases. Subjects with previously treated brain metastases may participate provided they are stable (without evidence of progression by imaging for at least four weeks prior to the first dose of trial treatment and any neurologic symptoms have returned to baseline), have no evidence of new or enlarging brain metastases, and are not using steroids for at least 28 days prior to treatment.
  • A history of allergic reactions attributed to compounds of similar chemical or biologic composition to mitomycin C or other agents used in the study.
  • Known HIV-positivity on combination antiretroviral therapy because of the potential for pharmacokinetic interactions with the study drugs. In addition, these patients are at increased risk of lethal infections when treated with marrow-suppressive therapy. Appropriate studies will be undertaken in patients receiving combination antiretroviral therapy when indicated.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Washington University School of Medicine

    St Louis, Missouri, 63110

Other trials in United States

About this study

Official title: Phase II Trial of Mitomycin C in Patients With Incurable p16 Positive Oropharyngeal and p16 Negative Head and Neck Squamous Cell Carcinoma (HNSCC) Resistant to Standard Therapies

No agent is known to have efficacy in patients with incurable HNSCC that progressed with prior platin, 5-FU, cetuximab and taxane. Herein lies the unmet need to be addressed by this trial. Based on the preclinical and clinical data presented, the investigators propose that mitomycin C will have anti-tumor activity in these patients.

Being tested
Mitomycin-C, Pegfilgrastim
Medical subject headings
Squamous Cell Carcinoma of Head and Neck

What participants receive

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

  • Mitomycin-CDrug

    Also called Mitosol, Mitomycin

  • PegfilgrastimDrug

    Also called •Neulasta®, GCSF

Study groups in NCT02369458
Study groupRoleWhat they receive
Cohort A: p16+ OPSCCExperimental* Mitomycin C given on Day 1 every 5 weeks (each cycle is 5 weeks). * Pegfilgrastim will be given on Day 2 of each cycle (subcutaneous injection)
Receives: Drug: Mitomycin-C; Drug: Pegfilgrastim
Cohort 2: p16- HNSCCExperimental* Mitomycin C given on Day 1 every 5 weeks (each cycle is 5 weeks). * Pegfilgrastim will be given on Day 2 of each cycle (subcutaneous injection)
Receives: Drug: Mitomycin-C; Drug: Pegfilgrastim

What is being measured

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

  1. Tumor Response Rate (TRR)

    • TRR will be evaluated separately in p16- (HPV-unrelated) HNSCC patients and in p16+ (HPV positive) OPSCC patients using two optimal two-stage Simon designs. In both cases, the expected TRR is 10%. A TRR of 30% is considered a clinically significant increase.
    • RECIST 1.1 will be used for this outcome.

    Measured: Approximately 6 months (median 5.6 months with full range of 0.1-33.7 months)

  2. Tumor Response Rate (TRR) for Participants Enrolled Post October 2020

    • TRR will be evaluated in p16+ (HPV positive) OPSCC HNSCC patients
    • RECIST 1.1 will be used for this outcome.

    Measured: Approximately 6 months (median 4.0 months with full range of 0.5-12.0 months)

Secondary outcomes (3)

  1. Progression-free Survival (PFS)

    • PFS is defined as the duration of time from start of treatment to time of first radiologic confirmation of progression or death, whichever occurs first.
    • Progressive disease per RECIST 1.1
    • Target lesions - At least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if that is the smallest on study). In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm.
    • Non-target lesions - Appearance of one or more new lesions and/or unequivocal progression of existing non-target lesions. Unequivocal progression should not normally trump target lesion status. It must be representative of overall disease status change, not a single lesion increase.

    Measured: Approximately 6 months (median 5.6 months with full range of 0.1-33.7 months)

  2. Number of Participants With Grade 3/4/5 Adverse Events

    -Using CTCAE Version 3.0

    Measured: 28 days after completion of treatment (median length of follow-up was 96 days, full range of 3-463 days)

  3. Overall Survival (OS)

    -Defined as the date of first treatment to the date of death, last date alive, or date of patient withdrawal.

    Measured: Through completion of follow-up (median length of follow-up Cohort A= 6.6 months IQR 2.7-12.0 months, median length of follow-up Cohort B=3.2 months IQR 1.5-9.4 months)

Timeline

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

  1. First posted24 Feb 2015 (estimated)
  2. Study start14 Apr 2015
  3. Primary completion1 Jun 2022
  4. Study completion1 Nov 2024

Resources

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
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
48 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02369458
Sponsor's protocol ID
201503060
Status last verified
Nov 2025
First submitted
16 Feb 2015
Last updated
18 Nov 2025
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Mitomycin-C and Pegfilgrastim in people with Squamous Cell Carcinoma of the Head and Neck and Squamous Cell Carcinoma, Head and Neck, run by Washington University School of Medicine at 1 site in United States.

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 14 inclusion and 7 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: finished, as of the sponsor's last update on 18 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. 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 18 May 2023). 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?

Tumor Response Rate (TRR) (time frame: Approximately 6 months (median 5.6 months with full range of 0.1-33.7 months)). The study lists 2 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 NCT02369458.

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 18 Nov 2025.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in