Chemotherapy for Appendiceal Cancer

Overseen By Keith F. Fournier, MD

Age: 18+

Sex: Any

Trial Phase: Academic

Sponsor: M.D. Anderson Cancer Center

Disqualifiers: Uncontrolled illness, Bowel obstruction, Others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 6

30 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What data supports the effectiveness of the treatment Systemic Chemotherapy for Appendiceal Cancer?

Research shows that systemic chemotherapy, often used in combination with other treatments, can lead to a response in some patients with advanced appendiceal cancer, with a response rate of 39% and stable disease in 36% of cases. Additionally, the survival outcomes for patients receiving systemic chemotherapy are similar to those seen in treatments for metastatic colorectal cancer. ^{1 2 3 4 5}

Is systemic chemotherapy safe for humans?

The research articles do not provide specific safety data for systemic chemotherapy in humans, but they discuss its use in treating appendiceal cancer, often in combination with other treatments like surgery and heated chemotherapy. This suggests it is generally considered safe enough to be used in clinical settings, though specific safety outcomes are not detailed. ^{1 2 4 6 7}

How does systemic chemotherapy differ from other treatments for appendiceal cancer?

Systemic chemotherapy is unique for appendiceal cancer because there are no standard treatments specifically for this condition, making it a novel approach. It involves using drugs that travel through the bloodstream to reach and treat cancer cells throughout the body, which can be different from localized treatments like surgery or radiation. ^{8 9 10 11 12}

What is the purpose of this trial?

This trial is investigating the effects of chemotherapy on patients with appendiceal cancer, a rare disease with few symptoms. Because appendiceal cancer is rare and varies greatly, there are no established guidelines for using chemotherapy. Chemotherapy uses drugs to kill cancer cells by targeting their rapid growth. The study will compare two approaches: starting with chemotherapy or waiting before starting treatment.

Research Team

Keith F. Fournier, MD

Principal Investigator

M.D. Anderson Cancer Center

Eligibility Criteria

This trial is for adults with a rare type of appendiceal cancer that has spread and isn't suitable for complete surgical removal. Participants must have specific blood cell counts, not be pregnant or breastfeeding, able to answer quality-of-life questionnaires, and agree to use effective contraception.

Inclusion Criteria

Patients must be able to understand and provide answers to the European Organisation for Research and Treatment of Cancer (EORTC) QLQ-C30/OV-28 QOL questionnaires in order to participate in the trial.

My scans show I have mucinous peritoneal carcinomatosis.

I am not pregnant or breastfeeding and agree to use birth control during and after treatment.

Exclusion Criteria

I have a blockage in my intestines.

I am currently on IV nutrition.

I don't have any health issues that could interfere with 6 months of chemotherapy.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Observation

Participants are observed for 6 months to monitor tumor growth rate

6 months

Every 3 months (+/- 2 weeks)

Chemotherapy

Participants receive chemotherapy for 6 months, type determined by standard of care

6 months

Every 3 months (+/- 2 weeks)

End-of-Treatment

End-of-treatment visit with physical exam, performance status, blood tests, and imaging

1 visit

Long-Term Follow-Up

Participants are followed annually to check medical records and conduct a brief call

Annually

Treatment Details

Interventions

• Systemic Chemotherapy

Trial Overview The study aims to understand the effects of systemic chemotherapy on metastatic well-differentiated mucinous appendiceal adenocarcinomas with pseudomyxoma peritonei. It involves patients completing questionnaires to assess their quality of life during treatment.

Participant Groups

Group I: Observation Arm Experimental Treatment 1 Intervention

Patients observed for 6 months, then will receive chemotherapy for 6 months.

Group II: Chemotherapy Group Active Control 1 Intervention

Patients receive chemotherapy for 6 months, then observed for 6 months. The exact type of fluoropyrimidine-based chemotherapy is not mandated and final treatment decisions will be left to the medical oncologist who is administering the chemotherapy. All chemotherapy adjustments will be done by the treating medical oncologist according to standard of care.

Who Is Running the Clinical Trial?

M.D. Anderson Cancer Center

Lead Sponsor

Trials

3,107

Recruited

1,813,000+

References

1. Cyprus pubmed.ncbi.nlm.nih.gov

Perioperative systemic chemotherapy for peritoneal mucinous appendiceal carcinomas treated with cytoreductive surgery & HIPEC. [2019]

2. United States pubmed.ncbi.nlm.nih.gov

Systemic chemotherapy and survival in patients with metastatic low-grade appendiceal mucinous adenocarcinoma. [2020]

3. United States pubmed.ncbi.nlm.nih.gov

Systemic therapy for advanced appendiceal adenocarcinoma: an analysis from the NCCN Oncology Outcomes Database for colorectal cancer. [2019]

4. United States pubmed.ncbi.nlm.nih.gov

Improved Survival with Anti-VEGF Therapy in the Treatment of Unresectable Appendiceal Epithelial Neoplasms. [2018]

5. England pubmed.ncbi.nlm.nih.gov

Synchronous extraperitoneal and intraperitoneal dissemination of appendix cancer. [2007]

6. United States pubmed.ncbi.nlm.nih.gov

Adverse Events Postoperatively Had No Impact on Long-Term Survival of Patients Treated with Cytoreductive Surgery with Heated Intraperitoneal Chemotherapy for Appendiceal Cancer with Peritoneal Metastases. [2018]

7. United States pubmed.ncbi.nlm.nih.gov

Impact of adjuvant chemotherapy on outcomes in appendiceal cancer. [2021]

8. United States pubmed.ncbi.nlm.nih.gov

The use of paclitaxel and cisplatin in a patient with epithelial ovarian cancer and human immunodeficiency virus. [2015]

9. Greece pubmed.ncbi.nlm.nih.gov

Predictive factors for tumor response to chemotherapy in patients with pancreatic cancer. [2015]

10. Switzerland pubmed.ncbi.nlm.nih.gov

Chemotherapy for inoperable advanced or metastatic cholangiocarcinoma: retrospective analysis of 78 cases in a single center over four years. [2022]

11. Germany pubmed.ncbi.nlm.nih.gov

[Anthracyclines in therapy of ovarian carcinoma: a systematic review of primary and 2nd-line therapy after platinum]. [2018]

12. England pubmed.ncbi.nlm.nih.gov

A systematic overview of chemotherapy effects in ovarian cancer. [2019]