Safety and Efficacy of PIPAC Using Single Agent Mitomycin in Solid Tumors

NCT07487168 | Not Yet Recruiting Phase 1 DMC FDA Drug FDA Device

• 1 other identifier: MCC-23064
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

This single-center, Phase 1 dose-escalation study will evaluate the safety, tolerability, and recommended Phase 2 dose (RP2D) of pressurized intraperitoneal aerosol chemotherapy with mitomycin C (PIPAC-MMC) for patients with unresectable peritoneal carcinomatosis from gastrointestinal primaries (colorectal, high-grade appendiceal, or small bowel). Up to three PIPAC procedures are planned at 8-week intervals while patients continue 5-fluorouracil/leucovorin (5-FU/LV) between procedures. The trial uses a Bayesian optimal interval (BOIN) design to determine dose-limiting toxicities (DLTs) and maximum tolerated dose (MTD). Pharmacokinetics (PK), pharmacodynamics (PD), and quality of life (QoL) will be assessed.

Conditions

Peritoneal Carcinomatosis

Keywords

Appendiceal cancer; Peritoneal disease; Colorectal cancer; Pressurized Intraperitoneal Aerosolized Chemotherapy

Outcome Measures

Primary Outcomes (1)

• Maximum Tolerated Dose (MTD)

  The MTD of PIPAC-MMC will be determined to establish the recommended phase 2 dose (RP2D).

  Up to 12 Months

Secondary Outcomes (2)

• Event-Free Survival (EFS)

  Up to 24 Months

• Quality of Life

  Up to 12 Months

Study Arms (1)

Mitomycin C Dose Escalation

EXPERIMENTAL

All participants are assigned to receive PIPAC-MMC. Dose escalation occurs via sequential cohorts using the BOIN model. No participant changes dose once assigned; each receives up to three PIPAC procedures at their assigned dose level.

Drug: Mitomycin C (MMC); Device: Pressurized Intraperitoneal Aerosolized Chemotherapy (PIPAC)

Interventions

Mitomycin C (MMC) DRUG

Delivered intraperitoneally as aerosol under laparoscopy.

Mitomycin C Dose Escalation

Pressurized Intraperitoneal Aerosolized Chemotherapy (PIPAC) DEVICE

A drug delivery approach in which the antineoplastic agent is delivered directly into the peritoneal cavity solution under laparoscopic pressures (12 mm HG).

Also known as: CapnoPen®

Mitomycin C Dose Escalation

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants must have histologically confirmed peritoneal disease from colorectal, small bowel, or high grade appendiceal adenocarcinoma. High grade appendiceal cancers include moderate or poorly differentiated mucinous or non-mucinous adenocarcinoma, signet ring cell adenocarcinoma, or goblet cell adenocarcinoma. This can be established by image guided biopsy, diagnostic laparoscopy, or previous surgery.
• Patients must be ineligible for CRS/HIPEC through one of the following criteria: a) PCI score ≥16. b) Inability to achieve complete cytoreduction due to extent of disease. c) Significant small bowel involvement precluding a complete CRS. d) Unresectable disease in porta hepatis, pelvic side wall or other critical structure. e) Patients who decline invasive cytoreduction.
• Participants must be 18 years of age or older.
• Participants must have completed at least 4 months of first-line systemic therapy (5-FU based approach with or without biologic therapy).
• Participants must have Eastern Cooperative Oncology Group (ECOG) Performance status of 0 or 1.
• Participants must have adequate organ and marrow function as defined below: a) absolute neutrophil count ≥1500/mcL. b) platelets ≥100,000/mcL. c) total bilirubin ≤ institutional upper limit of normal (ULN). d) AST(SGOT)/ALT(SGPT) ≤3 × institutional ULN. e) creatinine ≤ 1.5 institutional ULN. or f) glomerular filtration rate (GFR) ≥60 mL/min/1.73 m2.
• Human immunodeficiency virus (HIV)-infected participants on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial.
• For participants with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated.
• Participants with a history of hepatitis C virus (HCV) infection must have been treated and cured. For participants with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load.
• Participants with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should undergo a clinical risk assessment of cardiac function using the New York Heart Association Functional Classification (see Appendix C).
• To be eligible for this trial, participants should be class 2B or better.
• MMC is a known teratogen, for this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of study drug administration. For women of child-bearing potential a negative urine pregnancy test is required on the morning of surgery.
• Ability to understand and the willingness to sign a written informed consent document.

You may not qualify if:

• Participants who have received targeted therapy, immunotherapy, or radiotherapy within 4 weeks (6 weeks for VEGF inhibitors, nitrosoureas or mitomycin C) prior to entering the study.
• Participants who have not recovered from adverse events (AEs) due to prior anti-cancer therapy (i.e., have residual toxicities \> Grade 1), except for alopecia and chemotherapy induced peripheral neuropathy \< 2.
• Patients with extensive metastatic liver disease (\>50% liver volume) are excluded from the trial as are patients with brain metastases. Patients with non-peritoneal metastatic disease are otherwise eligible provided they meet the survival expectations of \>6 months.
• Patients with brain metastases are excluded
• Patients with bowel obstruction or need for nutritional support (i.e., TPN or tube feeds).
• Participants who are receiving any other investigational agents or enrolled on other research protocols that may interfere with compliance with requirements of the study.
• History of allergic reactions or poor tolerance attributed to compounds of similar chemical or biologic composition to MMC, fluoropyrimidines, or anesthesia medications.
• Participants with uncontrolled intercurrent illnesses.
• Participants with psychiatric illness/social situations that would limit compliance with study requirements.
• Pregnant women are excluded from this study because MMC is an antibiotic alkylating antineoplastic agent with the 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 MMC, breastfeeding should be discontinued if the mother is treated with MMC.
• Inability to safely perform laparoscopy due to previously noted adhesions or extensive prior surgery which the treating surgeon feels would exclude safe abdominal access.
• Life expectancy less than 6 months.
• Patients with history of thromboembolic complications that cannot discontinue for the perioperative duration.

Sponsors & Collaborators

• H. Lee Moffitt Cancer Center and Research Institute: lead

Study Sites (1)

Moffitt Cancer Center

Tampa, Florida, 33612, United States

Location

MeSH Terms

Conditions

Peritoneal Neoplasms; Appendiceal Neoplasms; Peritoneal Diseases; Colorectal Neoplasms

Interventions

Mitomycin

Condition Hierarchy (Ancestors)

Abdominal Neoplasms; Neoplasms by Site; Neoplasms; Digestive System Neoplasms; Digestive System Diseases; Cecal Neoplasms; Intestinal Neoplasms; Gastrointestinal Neoplasms; Gastrointestinal Diseases; Cecal Diseases; Intestinal Diseases; Colonic Diseases; Rectal Diseases

Intervention Hierarchy (Ancestors)

Mitomycins; Indolequinones; Quinones; Organic Chemicals; Azirines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Indoles; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Sean Dineen, MD

  Moffitt Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Valentina Diaz Aranzazu

CONTACT

813-745-8536; valentina.diazaranzazu@moffitt.org

Study Design

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

Study Record Dates

• First Submitted: March 17, 2026
• First Posted: March 23, 2026
• Study Start (Estimated): August 1, 2026
• Primary Completion (Estimated): March 1, 2028
• Study Completion (Estimated): March 1, 2028
• Last Updated: May 15, 2026

Record last verified: 2026-05

Locations

US (1)