CompletedNCT02369458Updated Nov 18, 2025Results posted

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 interventional study of Mitomycin-C and Pegfilgrastim in Squamous Cell Carcinoma of the Head and Neck and Squamous Cell Carcinoma, Head and Neck, sponsored by Washington University School of Medicine. Completed at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-11-18.

Sponsored by Washington University School of Medicine · Phase 2, Interventional, and Treatment

Phase: Phase 2
Study type: Interventional
Enrollment: 48
Allocation: Randomized
Ages: 18 Years and older
Sex: All

Study 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. Based on the preclinical and clinical data presented, the investigators propose that mitomycin C will have anti-tumor activity in these patients.

Conditions studied

Squamous Cell Carcinoma of the Head and Neck
Squamous Cell Carcinoma, Head and Neck

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Squamous Cell Carcinoma of Head and Neck 1,680

In context

Squamous Cell Carcinoma of Head and Neck

1,680 studies on the registry are indexed under Squamous Cell Carcinoma of Head and Neck; 539 are open to participants now.

This study's enrollment of 48 is close to the median of 49 across 1,432 interventional studies indexed under Squamous Cell Carcinoma of Head and Neck.

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Lead sponsor

Washington University School of Medicine is the lead sponsor of 1,765 studies on the registry; 271 are open to participants now.

Of its 324 completed or terminated interventional studies of FDA-regulated products, 212 (65%) have results posted.

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

Who can participate

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

Inclusion criteria

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
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).

Exclusion criteria

Exclusion Criteria:

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.

Study design

Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel assignment
Masking: None (open label)
Enrollment: 48 participants (actual)

Study arms

Experimental
Cohort A: p16+ OPSCC
* 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)
Drug: Mitomycin-C · Drug: Pegfilgrastim
Experimental
Cohort 2: p16- HNSCC
* 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)
Drug: Mitomycin-C · Drug: Pegfilgrastim

Interventions

DrugMitomycin-C
Also known as: Mitosol, Mitomycin
DrugPegfilgrastim
Also known as: •Neulasta®, GCSF

What researchers measure

Primary outcomes

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.
Time frame: Approximately 6 months (median 5.6 months with full range of 0.1-33.7 months)
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.
Time frame: Approximately 6 months (median 4.0 months with full range of 0.5-12.0 months)

Secondary outcomes

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.
Time frame: Approximately 6 months (median 5.6 months with full range of 0.1-33.7 months)
Number of Participants With Grade 3/4/5 Adverse Events
-Using CTCAE Version 3.0
Time frame: 28 days after completion of treatment (median length of follow-up was 96 days, full range of 3-463 days)
Overall Survival (OS)
-Defined as the date of first treatment to the date of death, last date alive, or date of patient withdrawal.
Time frame: 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)

Other outcomes

Quality of Life as Measured by the EORTC QLQ-C30
-EORTC QLQ-C30: this has a total score, one general QOL, and one "within the last week" subscale, as well as a general health item and a single overall QOL item. This study does not use current empirical guidelines for the EORTC-QLQ-30 global score with the understanding that both the magnitude and variance of scores vary considerably from patient to patient, from one time point to another and by such factors as disease condition, age, and comorbidity. The participants can choose from 1-4 with 1 being Not At All and 4 being Very Much.
Time frame: Baseline, every 5 weeks, and end of treatment (estimated at 6 months)
Quality of Life as Measured by the Cognitive Failures Questions (CFQ)
-Cognitive Failures Questions (CFQ) - has 3 subscales describing perception, memory, and motor function. A change in 1 standard deviation will be considered a perceptible difference. The participants can choose a scale from 0-4 with 0 being Never and 4 being Very Often.
Time frame: Baseline, every 5 weeks, and end of treatment (estimated at 6 months)

Results

Posted May 18, 2023

Participant flow

Participant flow — Overall Study
Milestone	Cohort A: p16+ OPSCC	Cohort B: p16- HNSCC
Started	35	13
Completed	34	13
Not completed	1	0
Withdrew: Determined to be not eligible	1	0

Outcome measures

PrimaryTumor 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.

Time frame:: Approximately 6 months (median 5.6 months with full range of 0.1-33.7 months)
Reported as:: Count of participants · Participants

Tumor Response Rate (TRR)
Participants	Cohort A: p16+ OPSCC	Cohort B: p16- HNSCC
Tumor Response Rate (TRR)	3	0

PrimaryTumor 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.

Time frame:: Approximately 6 months (median 4.0 months with full range of 0.5-12.0 months)
Reported as:: Count of participants · Participants

Tumor Response Rate (TRR) for Participants Enrolled Post October 2020
Participants	Cohort A: p16+ OPSCC	Cohort B: p16- HNSCC
Tumor Response Rate (TRR) for Participants Enrolled Post October 2020	0	—

SecondaryProgression-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.

Time frame:: Approximately 6 months (median 5.6 months with full range of 0.1-33.7 months)
Reported as:: Median · months

Progression-free Survival (PFS)
months	Cohort A: p16+ OPSCC	Cohort B: p16- HNSCC
Progression-free Survival (PFS)	2.2 (1.6 to 3.4)	2.1 (1.0 to 3.0)

SecondaryNumber of Participants With Grade 3/4/5 Adverse Events

-Using CTCAE Version 3.0

Time frame:: 28 days after completion of treatment (median length of follow-up was 96 days, full range of 3-463 days)
Reported as:: Count of participants · Participants

Number of Participants With Grade 3/4/5 Adverse Events
Participants	Cohort A: p16+ OPSCC and Cohort B: p16- OPSCC
Number of Participants With Grade 3/4/5 Adverse Events	26

SecondaryOverall Survival (OS)

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

Time frame:: 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)
Reported as:: Median · months

Overall Survival (OS)
months	Cohort A: p16+ OPSCC	Cohort B: p16- HNSCC
Overall Survival (OS)	6.6 (3.5 to 9.9)	3.2 (1.5 to 9.4)

Other pre-specifiedQuality of Life as Measured by the EORTC QLQ-C30

-EORTC QLQ-C30: this has a total score, one general QOL, and one "within the last week" subscale, as well as a general health item and a single overall QOL item. This study does not use current empirical guidelines for the EORTC-QLQ-30 global score with the understanding that both the magnitude and variance of scores vary considerably from patient to patient, from one time point to another and by such factors as disease condition, age, and comorbidity. The participants can choose from 1-4 with 1 being Not At All and 4 being Very Much.

Time frame:: Baseline, every 5 weeks, and end of treatment (estimated at 6 months)

Results for this outcome have not been posted.

Other pre-specifiedQuality of Life as Measured by the Cognitive Failures Questions (CFQ)

-Cognitive Failures Questions (CFQ) - has 3 subscales describing perception, memory, and motor function. A change in 1 standard deviation will be considered a perceptible difference. The participants can choose a scale from 0-4 with 0 being Never and 4 being Very Often.

Time frame:: Baseline, every 5 weeks, and end of treatment (estimated at 6 months)

Results for this outcome have not been posted.

Adverse events

Collected over -Adverse events were collected from start of treatment through 28 days following day of last treatment (median length of follow-up was 96 days, full range of 3-463 days). -Adverse events and all cause mortality were not collected separately by cohort.. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
Group	Deaths	Serious	Other
Cohort A: p16+ OPSCC and Cohort B: p16- HNSCC	47/47 (100%)	21/47 (44.7%)	47/47 (100%)

Most frequent serious events

Showing 10 of 24

Most frequent serious events
Event	Cohort A: p16+ OPSCC and Cohort B: p16- HNSCC
DyspneaRespiratory, thoracic and mediastinal disorders	4/47
DysphagiaGastrointestinal disorders	2/47
SepsisInfections and infestations	2/47
DehydrationMetabolism and nutrition disorders	2/47
Respiratory failureRespiratory, thoracic and mediastinal disorders	2/47
Injury to carotid arteryCardiac disorders	1/47
Pericardial effusionCardiac disorders	1/47
Restrictive cardiomyopathyCardiac disorders	1/47
Sinus tachycardiaCardiac disorders	1/47
Double visionEye disorders	1/47

Most frequent other events

Showing 10 of 105

Most frequent other events
Event	Cohort A: p16+ OPSCC and Cohort B: p16- HNSCC
AnemiaBlood and lymphatic system disorders	45/47
Lymphocyte count decreasedInvestigations	42/47
FatigueGeneral disorders	29/47
HypoalbuminemiaMetabolism and nutrition disorders	27/47
HyponatremiaMetabolism and nutrition disorders	21/47
Platelet count decreasedInvestigations	19/47
Alkaline phosphatase increasedInvestigations	17/47
DysphagiaGastrointestinal disorders	16/47
DyspneaRespiratory, thoracic and mediastinal disorders	16/47
HypocalcemiaMetabolism and nutrition disorders	13/47

Baseline characteristics

Age, Continuous
Age, Continuous(years)	Cohort A: p16+ OPSCC	Cohort B: p16- HNSCC	Total
Median	61 (45 to 81)	61 (54 to 68)	61 (45 to 81)

Sex: Female, Male
Sex: Female, Male(Participants)	Cohort A: p16+ OPSCC	Cohort B: p16- HNSCC	Total
Female	1	5	6
Male	34	8	42

Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)	Cohort A: p16+ OPSCC	Cohort B: p16- HNSCC	Total
Hispanic or Latino	0	0	0
Not Hispanic or Latino	35	13	48
Unknown or Not Reported	0	0	0

Race (NIH/OMB)
Race (NIH/OMB)(Participants)	Cohort A: p16+ OPSCC	Cohort B: p16- HNSCC	Total
American Indian or Alaska Native	0	0	0
Asian	0	0	0
Native Hawaiian or Other Pacific Islander	0	0	0
Black or African American	0	5	5
White	35	8	43
More than one race	0	0	0
Unknown or Not Reported	0	0	0

Region of Enrollment
Region of Enrollment(participants)	Cohort A: p16+ OPSCC	Cohort B: p16- HNSCC	Total
United States	35	13	48

Study locations

1 site

Washington University School of Medicine
St Louis, Missouri 63110, United States

References and documents

Study documents

Protocol and statistical analysis plan · Mar 9, 2023

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Nov 18, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT02369458
Lead sponsor: Washington University School of Medicine
Responsible party: Sponsor
First posted: Feb 24, 2015
Start date: Apr 14, 2015
Primary completion: Jun 1, 2022
Completion: Nov 1, 2024
Results posted: May 18, 2023
Last update: Nov 18, 2025

Study contacts

Peter Oppelt, M.D.

principal investigator · Washington University School of Medicine

Oversight

Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

View the source record on ClinicalTrials.gov ↗

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