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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine if systemic chemotherapy can effectively treat appendiceal cancer, a rare cancer with few symptoms. Participants will be divided into two groups: one will receive chemotherapy for six months followed by six months of observation, while the other will be observed first and then receive chemotherapy. The study seeks individuals diagnosed with a specific type of appendiceal cancer that has spread to the abdominal lining and cannot be surgically removed. As an unphased trial, it offers patients the chance to contribute to significant research that could lead to new treatment options for appendiceal cancer.

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.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that chemotherapy, which affects the whole body, is usually well-tolerated by patients. One study found that 50% of patients experienced stable disease after 12 months of treatment, indicating the treatment can work without worsening the condition. However, specific safety information about using this type of chemotherapy for appendiceal cancer in humans is limited. Chemotherapy is a common treatment for many cancers, and its safety is well-studied. Participants should consult the medical team running the trial with any questions or concerns. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about this chemotherapy approach for appendiceal cancer because it offers flexibility and personalization in treatment. Unlike standard options that often follow a rigid protocol, this method allows the medical oncologist to tailor the type of fluoropyrimidine-based chemotherapy to each patient, potentially improving outcomes. Additionally, the trial's structure—either starting with observation or immediate treatment—provides valuable insights into the timing and effectiveness of chemotherapy, which could lead to more refined strategies for managing this rare cancer.

What evidence suggests that chemotherapy might be an effective treatment for appendiceal cancer?

Research has shown that the benefits of chemotherapy for appendiceal cancer remain unclear. Some studies found that patients with low-grade mucinous appendiceal adenocarcinoma did not benefit significantly from chemotherapy. In certain cases, tumors grew slightly during treatment, though this change was not substantial. The outlook for patients with appendiceal cancer varies greatly, with survival rates differing widely. In this trial, one group of participants will receive chemotherapy for six months, while another group will be observed for six months before starting chemotherapy. Overall, current evidence does not strongly support chemotherapy as an effective treatment for this type of cancer. ^{1 6 7 8 9}

Who Is on the Research Team?

Keith F. Fournier, MD

Principal Investigator

M.D. Anderson Cancer Center

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10236240/

Efficacy of Systemic Chemotherapy in Patients With Low- ... These findings suggest that patients with low-grade mucinous appendiceal adenocarcinoma did not derive clinically meaningful benefit from ...

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/37261831/

Efficacy of Systemic Chemotherapy in Patients With ... - PubMed Tumor growth while receiving chemotherapy increased 8.4% (95% CI, 1.5% to 15.3%) from baseline but was not significantly different than tumor ...

3. esmoopen.com esmoopen.com/article/S2059-7029(23)00854-2/fulltext

Defining a role for systemic chemotherapy in local and ... Prognosis from small retrospective and heterogeneous cohorts is poor, with a 5-year survival ranging from 14% to 59%. Due to changing ...

4. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0022480422007193

Association of Systemic Chemotherapy Approaches With ... While there is limited evidence regarding the efficacy of systemic chemotherapy in appendiceal cancer specifically, several studies have demonstrated improved ...

5. gioncologynow.com gioncologynow.com/post/investigating-systemic-chemotherapy-in-appendiceal-adenocarcinomas

Investigating Systemic Chemotherapy in Appendiceal ... Systemic chemotherapy did not show any benefit in patients with inoperable low-grade mucinous appendiceal adenocarcinoma.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9310237/

The role of chemotherapy in the treatment of advanced ... This review aims to evaluate the literature for patients who received intraperitoneal and systemic chemotherapy for appendiceal cancers.

7. mdpi.com mdpi.com/2072-6694/17/20/3297

Survival Outcomes and Prognostic Factors in Metastatic ... This study evaluated real-world outcomes in patients with advanced appendiceal adenocarcinoma who received palliative chemotherapy. The results support ...

8. dailynews.ascopubs.org dailynews.ascopubs.org/do/appendix-cancer-not-same-colorectal-cancer-implications-systemic-chemotherapy

Appendix Cancer Is Not the Same as Colorectal Cancer Median survival is not yet reached after 17.6 months of follow-up, and 50% of evaluable patients had stable disease after 12 months on treatment ...

9. withpower.com withpower.com/trial/phase-gastrointestinal-neoplasms-6-2013-9237f

Chemotherapy for Appendiceal Cancer · Info for Participants Is systemic chemotherapy safe for humans? The research articles do not provide specific safety data for systemic chemotherapy in humans, but they discuss its ...