NCT01946854

Brief Summary

Appendiceal cancer is a rare disease that does not cause many symptoms. As such, doctors are not sure if chemotherapy actually has an effect on the disease. The goal of this clinical research study is to learn more about the effects that chemotherapy may have on appendiceal cancer.

Trial Health

55
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
30

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Jul 2013

Longer than P75 for not_applicable

Geographic Reach
1 country

1 active site

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 16, 2013

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

September 12, 2013

Completed
8 days until next milestone

First Posted

Study publicly available on registry

September 20, 2013

Completed
12.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 31, 2026

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 31, 2026

Completed
Last Updated

November 18, 2025

Status Verified

November 1, 2025

Enrollment Period

12.6 years

First QC Date

September 12, 2013

Last Update Submit

November 17, 2025

Conditions

Gastrointestinal Neoplasms

Keywords

GastrointestinalMetastatic Well-differentiated Mucinous Appendiceal Adenocarcinomas with Pseudomyxoma PeritoneaAppendiceal cancerSurgically unresectableChemotherapyObservationQuestionnaires

Outcome Measures

Primary Outcomes (1)

  • Tumor Growth Rate

    Primary objective is to determine if difference in tumor growth rate for each patient, as measured by mpRECIST, between observation and systemic chemotherapy periods is ≥5%. Growth rate determined as the percent reduction from the start of the period to the 6 month measurement in that period.

    6 months

Study Arms (2)

Observation Arm

OTHER

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

Behavioral: Questionnaire

Chemotherapy Group

ACTIVE COMPARATOR

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.

Behavioral: Questionnaire

Interventions

QuestionnaireBEHAVIORAL

Completion of 3 questionnaires at baseline and at final visit about quality of life, and 1 questionnaire about symptoms, diagnosis, treatment, prognosis, and patient's relationship with caregivers. These will take about 35-50 minutes to complete. Completion of 3 quality of life questionnaires every 3 months for 1 year.

Chemotherapy GroupObservation Arm

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Patients must have histological evidence of a metastatic well differentiated or moderately differentiated mucinous appendiceal epithelial neoplasm (AEN).
  • Radiographic images demonstrating the presence of mucinous peritoneal carcinomatosis (PMP).
  • Patients must not be considered a candidate for a complete surgical cytoreductive surgery. This determination will be made through either discussion at MD Anderson peritoneal surface malignancy multidisciplinary review or consultation with MD Anderson peritoneal surgeon.
  • Eastern Cooperative Oncology Group (ECOG) Performance Status 0-2.
  • Age \>/= 18 years old.
  • 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.
  • Adequate bone marrow function as evidenced by: Hemoglobin \>/= 9.0 g/dl; Platelet \>/= 75,000 cells/mm\^3; Absolute neutrophil count \>/= 1000/mm\^3.
  • Women must not be pregnant or lactating. Women of childbearing potential must have a negative Beta-HCG serum pregnancy test and agree to refrain from breast-feeding, as specified in the informed consent given the unknown risk of teratogenicity of agents in the study. Patients of childbearing potential agree to use an effective form of contraception during chemotherapy and for 90 days following the last chemotherapy treatment.
  • Patients must agree to participate and be able to understand and provide informed consent to participate in the trial.

You may not qualify if:

  • Concurrent uncontrolled medical illness that is deemed by the investigator to have potential to interfere with the delivery of chemotherapy for a six month time period.
  • Patients who are receiving concurrent investigational therapy or who have received investigational therapy within 30 days of the first scheduled day of protocol treatment.
  • The presence of complete or partial bowel obstruction based upon clinical assessment.
  • Ongoing use of total parental nutrition.
  • The presence of a concurrent non-appendiceal metastatic cancer.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Publications (1)

  • Shen JP, Yousef AM, Zeineddine FA, Zeineddine MA, Tidwell RS, Beaty KA, Scofield LC, Rafeeq S, Hornstein N, Lano E, Eng C, Matamoros A, Foo WC, Uppal A, Scally C, Mansfield P, Taggart M, Raghav KP, Overman MJ, Fournier K. Efficacy of Systemic Chemotherapy in Patients With Low-grade Mucinous Appendiceal Adenocarcinoma: A Randomized Crossover Trial. JAMA Netw Open. 2023 Jun 1;6(6):e2316161. doi: 10.1001/jamanetworkopen.2023.16161.

    PMID: 37261831DERIVED

Related Links

MeSH Terms

Conditions

Gastrointestinal NeoplasmsAppendiceal Neoplasms

Interventions

Surveys and Questionnaires

Condition Hierarchy (Ancestors)

Digestive System NeoplasmsNeoplasms by SiteNeoplasmsDigestive System DiseasesGastrointestinal DiseasesCecal NeoplasmsIntestinal NeoplasmsCecal DiseasesIntestinal Diseases

Intervention Hierarchy (Ancestors)

Data CollectionEpidemiologic MethodsInvestigative TechniquesHealth Care Evaluation MechanismsQuality of Health CareHealth Care Quality, Access, and EvaluationPublic HealthEnvironment and Public Health

Study Officials

  • Keith F. Fournier, MD

    M.D. Anderson Cancer Center

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
not applicable
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
OTHER
Intervention Model
CROSSOVER
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 12, 2013

First Posted

September 20, 2013

Study Start

July 16, 2013

Primary Completion

January 31, 2026

Study Completion

January 31, 2026

Last Updated

November 18, 2025

Record last verified: 2025-11

Locations

US(1)