What side effects are associated with this type of intervention?

Common treatments for Squamous Cell Carcinoma (SCC) include platinum-based chemotherapies (e.g., cisplatin), 5-fluorouracil (5-FU), and taxanes. These treatments are associated with a range of side effects. Cisplatin can cause nephrotoxicity, ototoxicity, and severe nausea and vomiting. 5-FU is known for causing mucositis, diarrhea, and myelosuppression. Taxanes, such as paclitaxel, often lead to peripheral neuropathy, myelosuppression, and hypersensitivity reactions. Mitomycin C, a DNA crosslinking agent, shares some similar side effects, including myelosuppression and potential nephrotoxicity.

What prior FDA approvals exist?

For the treatment of Squamous Cell Carcinoma (SCC), several FDA-approved drugs have been utilized. Cisplatin, often used in combination with other agents like 5-FU, is a cornerstone in the treatment of various SCCs due to its DNA crosslinking properties. Additionally, cetuximab, an EGFR inhibitor, has been approved for use in combination with platinum-based chemotherapy for recurrent or metastatic head and neck SCC. Pembrolizumab, an immune checkpoint inhibitor targeting PD-1, has also been approved for recurrent or metastatic SCC, particularly in cases resistant to platinum and cetuximab. These treatments share a common goal of targeting the cancer cells' ability to proliferate and survive, similar to the mechanism of action of Mitomycin C.

What other types of research exist?

Promising novel alternatives to Mitomycin C for Squamous Cell Carcinoma patients include: 1) Vismodegib, a hedgehog pathway inhibitor, particularly for locally advanced basal cell carcinoma. 2) Checkpoint inhibitors, which are emerging as a new treatment approach for PD-L1-positive recurrent/metastatic squamous cell carcinoma of the head and neck (R/M SCCHN). 3) Valproic Acid (VPA) in combination with Cisplatin and Cetuximab, which has shown potential in preclinical studies and is currently being evaluated in clinical trials.

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Alkylating agents

Mitomycin C for Head and Neck Cancer

Phase 2

Waitlist Available

Led By Peter Oppelt, M.D.

Research Sponsored by Washington University School of Medicine

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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.

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).

Must not have

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.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up approximately 6 months (median 5.6 months with full range of 0.1-33.7 months)

Awards & highlights

No Placebo-Only Group

Summary

This study is evaluating whether a drug which has been used for decades to treat cancer may have the potential to help patients with incurable head and neck cancer.

Who is the study for?

This trial is for adults with incurable head and neck squamous cell carcinoma (HNSCC) that has worsened despite previous treatments. Participants must have proper liver, kidney, and blood function, agree to use contraception, and not be pregnant or breastfeeding. Those with controlled brain metastases may qualify.

What is being tested?

The study tests Mitomycin-C's effectiveness in patients whose HNSCC hasn't responded to standard therapies like platin, 5-FU, cetuximab, and taxane. Pegfilgrastim is also used to support white blood cell counts during treatment.

What are the potential side effects?

Mitomycin-C can cause side effects such as fatigue, nausea, low blood counts leading to increased infection risk or bleeding problems. Pegfilgrastim might cause bone pain or pain at the injection site.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My cancer can be measured by scans or physical exam.

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My cancer is in the head or neck area and cannot be cured with surgery or has spread.

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My cancer progressed after treatment with platinum and immunotherapy.

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My kidney function is normal or only slightly above normal.

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I can care for myself but may not be able to do any physical work.

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I have tissue available for p16 testing.

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I am 18 years old or older.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I do not have any serious illnesses that could interfere with the study.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ approximately 6 months (median 5.6 months with full range of 0.1-33.7 months)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~approximately 6 months (median 5.6 months with full range of 0.1-33.7 months)

This trial's timeline: 3 weeks for screening, Varies for treatment, and approximately 6 months (median 5.6 months with full range of 0.1-33.7 months) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Tumor Response Rate (TRR)

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

Secondary study objectives

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

Progression-free Survival (PFS)

Other study objectives

Quality of Life as Measured by the Cognitive Failures Questions (CFQ)

Quality of Life as Measured by the EORTC QLQ-C30

Side effects data

From 2017 Phase 2 trial • 100 Patients • NCT02016898

31%

Wound leak

31%

Hypotony

11%

Cystic blebs

Choroidal effusion

Shallowing of anterior chamber

Hyphema

Suprachoroidal hemorrhage

100%

80%

60%

40%

20%

Study treatment Arm

Irrigation Placement of Mitomycin-C

Sponge Placement of Mitomycin-C

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Cohort A: p16+ OPSCCExperimental Treatment2 Interventions

* 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)

Group II: Cohort 2: p16- HNSCCExperimental Treatment2 Interventions

* 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)

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Mitomycin-C

2011

Completed Phase 2

~240

Pegfilgrastim

2013

Completed Phase 3

~4440

0 / 8Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Squamous Cell Carcinoma (SCC) include DNA crosslinking agents like Mitomycin C, which work by forming covalent bonds between DNA strands, thereby inhibiting DNA replication and transcription, leading to cell death. Other treatments include platinum-based chemotherapies (e.g., cisplatin), which cause DNA damage and apoptosis, and taxanes (e.g., docetaxel), which disrupt microtubule function, preventing cell division. These mechanisms are crucial for SCC patients as they target rapidly dividing cancer cells, aiming to reduce tumor size and prevent metastasis, ultimately improving survival outcomes.

Histopathological findings in oesophageal carcinoma with and without preoperative chemotherapy.[Metastatic nasopharynx cancer at diagnosis: clinical and prognostic (study of 51 cases)].Occult breast cancer presenting as metastatic adenocarcinoma of unknown primary: clinical presentation, immunohistochemistry, and molecular analysis.