Gastrointestinal NeoplasmsNCT01946854
Crossover Trial of Systemic Chemotherapy in Patients With Metastatic Well-Differentiated Mucinous Appendiceal Adenocarcinomas With Pseudomyxoma Peritonei
A study testing Questionnaire in people with Gastrointestinal Neoplasms, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Gastrointestinal Neoplasms
- What is being tested?
- Questionnaire
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- 16 Jul 2013to 31 Jan 2028
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Texas MD Anderson Cancer Center
Houston, Texas, 77030
About this study
Official title: A Randomized Crossover Trial of Systemic Chemotherapy in Patients With Metastatic Well-Differentiated Mucinous Appendiceal Adenocarcinomas With Pseudomyxoma Peritonei
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.
Read the detailed description
Study Groups:
If you are found to be eligible to take part in this study, you will be randomly assigned (as in the flip of a coin) to 1 of 2 study groups (cohorts):
- If you are in Cohort A, you will first be observed for 6 months, then you will receive chemotherapy for 6 months.
- If you are in Cohort B, you will first receive chemotherapy for 6 months, then you will be observed for 6 months.
You will have an equal chance of being assigned to either cohort. No matter which cohort you are in, the type of chemotherapy you receive will depend on the standard of care for the disease and what your doctor thinks is in your best interest. You will sign a separate consent form for chemotherapy that explains how the drug is given, as well as its risks and benefits.
Study Visits:
Every 3 months (+/- 2 weeks) for 12 months:
- You will have a physical exam, including measurement of your weight and blood pressure.
- Your performance status will be recorded.
- Blood (about 2 tablespoons) will be drawn for routine tests.
- You will complete the 3 questionnaires about your quality of life.
- You will have an MRI or a CT scan to check the status of the disease.
Length of Stud:
You will be on study for up to 12 months. You will no longer be able to take part in this study if the disease gets worse, if intolerable side effects occur, or if you are unable to follow study directions.
Your active participation on the study will be over after the end-of-treatment visit.
End-of-Treatment Visit:
The end-of-treatment visit will be the same as your 4th study visit. Twelve (12) months (+/- 4 weeks) after you began the study, the following tests and procedures will be performed:
- You will have a physical exam, including measurement of your weight and blood pressure.
- Your performance status will be recorded.
- Blood (about 2 tablespoons) will be drawn for routine tests.
- You will complete the questionnaires you completed at screening.
- If one was not performed within 4 weeks before this visit, you will have an MRI or a CT scan to check the status of the disease.
Long-Term Follow-Up:
After the end-of-treatment visit, you will be followed 1 time each year by checking your medical record and seeing how you are doing. If nothing is in your medical record, you will be called and asked about how you are doing. This call should last about 5-10 minutes.
This is an investigational study.
Up to 30 patients take part in this study. All will be enrolled at MD Anderson.
- Conditions
- Gastrointestinal Neoplasms
- Being tested
- Questionnaire
- Keywords
- Gastrointestinal, Metastatic Well-differentiated Mucinous Appendiceal Adenocarcinomas with Pseudomyxoma Peritonea, Appendiceal cancer, Surgically unresectable, Chemotherapy, Observation, Questionnaires
- Medical subject headings
- Gastrointestinal Neoplasms, Appendiceal Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
QuestionnaireBehavioural
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.
| Study group | Role | What they receive |
|---|---|---|
| Observation Arm | Other | Patients observed for 6 months, then will receive chemotherapy for 6 months. Receives: 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. Receives: Behavioral: Questionnaire |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Sep 2013 (estimated)
- Study start16 Jul 2013
- Primary completion31 Jan 2028 (estimated)
- Study completion31 Jan 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Gastrointestinal NeoplasmsStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Keith F. Fournier, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01946854
- Sponsor's protocol ID
- 2012-1050
- Other IDs
- NCI-2015-00850
- Status last verified
- Jun 2026
- First submitted
- 12 Sep 2013
- Last updated
- 15 Jun 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
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.
PubMed 37261831
Study at a glance
From the registry record, last updated by the sponsor on 15 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Crossover Trial of Systemic Chemotherapy in Patients With Metastatic Well-Differentiated Mucinous Appendiceal Adenocarcinomas With Pseudomyxoma Peritonei
- ClinicalTrials.gov ID
- NCT01946854
- Acronym / sponsor ID
- 2012-1050
- Conditions
- Gastrointestinal Neoplasms
- Keywords
- Gastrointestinal, Metastatic Well-differentiated Mucinous Appendiceal Adenocarcinomas with Pseudomyxoma Peritonea, Appendiceal cancer, Surgically unresectable, Chemotherapy, Observation, Questionnaires
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Study start
- 16 Jul 2013 (actual)
- Primary completion
- 31 Jan 2028 (estimated)
- Study completion
- 31 Jan 2028 (estimated)
- First posted
- 20 Sep 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Study Groups:
If you are found to be eligible to take part in this study, you will be randomly assigned (as in the flip of a coin) to 1 of 2 study groups (cohorts):
- If you are in Cohort A, you will first be observed for 6 months, then you will receive chemotherapy for 6 months.
- If you are in Cohort B, you will first receive chemotherapy for 6 months, then you will be observed for 6 months.
You will have an equal chance of being assigned to either cohort. No matter which cohort you are in, the type of chemotherapy you receive will depend on the standard of care for the disease and what your doctor thinks is in your best interest. You will sign a separate consent form for chemotherapy that explains how the drug is given, as well as its risks and benefits.
Study Visits:
Every 3 months (+/- 2 weeks) for 12 months:
- You will have a physical exam, including measurement of your weight and blood pressure.
- Your performance status will be recorded.
- Blood (about 2 tablespoons) will be drawn for routine tests.
- You will complete the 3 questionnaires about your quality of life.
- You will have an MRI or a CT scan to check the status of the disease.
Length of Stud:
You will be on study for up to 12 months. You will no longer be able to take part in this study if the disease gets worse, if intolerable side effects occur, or if you are unable to follow study directions.
Your active participation on the study will be over after the end-of-treatment visit.
End-of-Treatment Visit:
The end-of-treatment visit will be the same as your 4th study visit. Twelve (12) months (+/- 4 weeks) after you began the study, the following tests and procedures will be performed:
- You will have a physical exam, including measurement of your weight and blood pressure.
- Your performance status will be recorded.
- Blood (about 2 tablespoons) will be drawn for routine tests.
- You will complete the questionnaires you completed at screening.
- If one was not performed within 4 weeks before this visit, you will have an MRI or a CT scan to check the status of the disease.
Long-Term Follow-Up:
After the end-of-treatment visit, you will be followed 1 time each year by checking your medical record and seeing how you are doing. If nothing is in your medical record, you will be called and asked about how you are doing. This call should last about 5-10 minutes.
This is an investigational study.
Up to 30 patients take part in this study. All will be enrolled at MD Anderson.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Observation Arm | Other | Behavioral: QuestionnairePatients observed for 6 months, then will receive chemotherapy for 6 months. |
| Chemotherapy Group | Active comparator | Behavioral: QuestionnairePatients 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 may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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.
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| QuestionnaireCompletion 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. | Behavioural | Chemotherapy Group, Observation Arm |
Primary outcome
Tumor Growth Rate
Time frame: 6 months
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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jun 2026.
- Derived from this study: PubMed 37261831Shen 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.
- University of Texas MD Anderson Cancer Center WebsiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Keith F. Fournier, MD principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Questionnaire in people with Gastrointestinal Neoplasms, run by M.D. Anderson Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 15 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Tumor Growth Rate (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01946854, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Jun 2026.