Metastatic Appendiceal AdenocarcinomaPhase 1/2NCT06207305
A Phase I/II Study of Intraperitoneal Paclitaxel in Patients With Metastatic Appendiceal Adenocarcinoma
A Phase 1/2 study testing Dexamethasone, Diphenhydramine and Famotidine in people with Metastatic Appendiceal Adenocarcinoma and Intraperitoneal Paclitaxel, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
To find the recommended dose of the drug paclitaxel that can be given intraperitoneally (given directly into the abdominal cavity) to participants with metastatic appendiceal adenocarcinoma. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Metastatic Appendiceal Adenocarcinoma, Intraperitoneal Paclitaxel
- What is being tested?
- Dexamethasone, Diphenhydramine and Famotidine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 39 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Jan 2024to 2 Jan 2028
- Phase
- Phase 1/2
- 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
- Age 18 years and above. There will be no upper age restriction
- ECOG performance status ≤ 2
- Participants must have histologically confirmed diagnosis of unresectable locally metastatic appendiceal adenocarcinoma
- Metastatic disease in the peritoneal cavity and not a candidate for cytoreductive surgery
- Participants must have adequate organ and marrow function as defined below:
- leukocytes ≥3000/mcL absolute neutrophil count ≥1,500/mcL platelets ≥75,000/mcL total bilirubin ≤ institutional upper limit of normal (ULN) creatinine ≤ 1.5X institutional ULN
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- 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 metastases outside the peritoneal cavity are not eligible for enrollment
- Participants with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial
- The effects of PTX on the developing human fetus are unknown. For this reason, and because Taxane agents as well as other therapeutic agents used in this trial are known to be teratogenic, 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. (Refer to Pregnancy Assessment Policy MD Anderson Institutional Policy # CLN1114). This includes all female patients, between the onset of menses (as early as 8 years of age) and 55 years unless the patient presents with an applicable exclusionary factor which may be one of the following:
- Postmenopausal (no menses in greater than or equal to 12 consecutive months)
- History of hysterectomy or bilateral salpingo-oophorectomy
- Ovarian failure (Follicle Stimulating Hormone and Estradiol in menopausal range, who have received Whole Pelvic Radiation Therapy)
- History of bilateral tubal ligation or another surgical sterilization procedure Approved methods of birth control are as follows: Hormonal contraception (i.e., birth control pills, injection, implant, transdermal patch, vaginal ring), Intrauterine device (IUD), Tubal Ligation or hysterectomy, Subject/Partner post vasectomy, Implantable or injectable contraceptives, and condoms plus spermicide. Not engaging in sexual activity for the total duration of the trial and the drug washout period is an acceptable practice; however periodic abstinence, the rhythm method, and the withdrawal method are not acceptable methods of birth control. 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 paclitaxel administration.
- Ability to understand and the willingness to sign a written informed consent document
- 14) English and non-English-speaking participants
Who cannot join
- Active infection such as pneumonia or wound infections that would preclude protocol therapy
- Participants with unstable angina or New York Heart Association (NYHA) Grade II or greater congestive heart failure
- Participants deemed unable to comply with study and/or follow-up procedures (i.e., cognitive impairment)
- Participants with a known hypersensitivity to protocol systemic chemotherapy that was life-threatening, required hospitalization or prolongation of existing hospitalization, or resulted in persistent or significant disability or incapacity
- Previous surgery that would preclude safe diagnostic laparoscopy with port placement
- Participants who have not recovered from adverse events (AE) due to prior anti-cancer therapy (i.e., have residual toxicities > Grade 1) with the exception of alopecia
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- Participants who are receiving any other investigational agents
- Participants with metastases outside the peritoneal cavity
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to PTX or other agents used in study
- Participants with psychiatric illness/social situations that would limit compliance with study requirements Participants who are pregnant
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
MD Anderson Cancer Center
Houston, Texas, 77303
Beth Helmink, MD, 832-696-5784, bhelmink@mdanderson.org
Beth Helmink, MD
About this study
To find the recommended dose of the drug paclitaxel that can be given intraperitoneally (given directly into the abdominal cavity) to participants with metastatic appendiceal adenocarcinoma.
Read the detailed description
Primary (Phase I):
- To assess the maximum tolerated dose (MTD) of paclitaxel via IP route given every 14 days in subjects with metastatic appendiceal adenocarcinoma Primary (Phase II):
- To assess the pathologic and radiographic objective response rate of paclitaxel via IP route in participants with metastatic appendiceal adenocarcinoma
Secondary Objectives
- To assess the progression-free and overall survival of metastatic appendiceal adenocarcinoma treated with IP paclitaxel. Although the clinical benefit of this drug has not yet been established, the intent of offering this treatment is to provide a possible therapeutic benefit, and thus the participants will be carefully monitored for tumor response and symptom relief in addition to safety and tolerability
- To assess the pharmacokinetics of IP PTX
- To assess the change in PCI following IP PTX in patients with metastatic appendiceal adenocarcinoma
- To assess rate of initially unresectable participants with metastatic appendiceal adenocarcinoma able to undergo CRS / HIPEC after IP PTX
- To assess the rate of conversion from positive to negative cytology in peritoneal fluid following IP PTX in participants with metastatic appendiceal adenocarcinoma
- To assess the prognostic value of circulating tumor DNA (ctDNA) in participants with metastatic appendiceal adenocarcinoma and the correlation of quantitative ctDNA measurement with radiographic and pathologic response
- To generate PDX and PDO models of appendiceal adenocarcinoma and evaluate their ability to predict response of human tumors
- To evaluate the effect of IP PTX on the transcriptomic state of appendiceal adenocarcinoma and the tumor microenvironment (TME) through comparison of pre- and post-treatment specimens
- To assess the impact of GNAS, KRAS, TP53, and APC mutation on response to IP PTX therapy
- To assess the impact of mucinous, signet ring cell, and goblet cell histology on response to IP PTX therapy
- Being tested
- Dexamethasone, Diphenhydramine, Famotidine, Paclitaxel
- Medical subject headings
- Adenocarcinoma, Mucinous
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DexamethasoneDrug
Also called Decadron
Given by PO
DiphenhydramineDrug
Also called Benadryl®
Given by PO
FamotidineDrug
Also called Pepcid®
Given by PO
PaclitaxelDrug
Also called Taxol
Given by PO
| Study group | Role | What they receive |
|---|---|---|
| Phase I/Phase II | Experimental | Participants will be assigned to a study phase (Phase I or Phase II) based on when participants join this study. Up to 4 groups of at least 3 participants will be enrolled in Phase I of the study, and up to 15 participants will be enrolled in Phase II.
If you are enrolled in Phase I, the dose of paclitaxel you receive will depend on when the participants join this study. The first group of participants will receive the lowest dose level of paclitaxel. Each new group will receive a higher dose of paclitaxel than the group before it, if no intolerable side effects were seen. This will continue until the highest tolerable dose of paclitaxel given intraperitoneally is found.
Participants who are enrolled in Phase II, participants will receive paclitaxel at the recommended dose that was found in Phase I. Receives: Drug: Dexamethasone; Drug: Diphenhydramine; Drug: Famotidine; Drug: Paclitaxel |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and adverse events (AEs)
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
Measured: Through study completion; an average of 1 year.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jan 2024
- Study start30 Jan 2024
- Primary completion2 Jan 2028 (estimated)
- Study completion2 Jan 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Metastatic Appendiceal AdenocarcinomaStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 39 participants (estimated)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Beth Helmink, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06207305
- Sponsor's protocol ID
- 2023-0860
- Other IDs
- NCI-2024-00080
- Status last verified
- Jul 2026
- First submitted
- 5 Jan 2024
- Last updated
- 16 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Study of Intraperitoneal Paclitaxel in Patients With Metastatic Appendiceal Adenocarcinoma
- ClinicalTrials.gov ID
- NCT06207305
- Acronym / sponsor ID
- 2023-0860
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 39 participants (estimated)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Study start
- 30 Jan 2024 (actual)
- Primary completion
- 2 Jan 2028 (estimated)
- Study completion
- 2 Jan 2028 (estimated)
- First posted
- 16 Jan 2024 (actual)
- 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.
To find the recommended dose of the drug paclitaxel that can be given intraperitoneally (given directly into the abdominal cavity) to participants with metastatic appendiceal adenocarcinoma.
Detailed description
Primary (Phase I):
- To assess the maximum tolerated dose (MTD) of paclitaxel via IP route given every 14 days in subjects with metastatic appendiceal adenocarcinoma Primary (Phase II):
- To assess the pathologic and radiographic objective response rate of paclitaxel via IP route in participants with metastatic appendiceal adenocarcinoma
Secondary Objectives
- To assess the progression-free and overall survival of metastatic appendiceal adenocarcinoma treated with IP paclitaxel. Although the clinical benefit of this drug has not yet been established, the intent of offering this treatment is to provide a possible therapeutic benefit, and thus the participants will be carefully monitored for tumor response and symptom relief in addition to safety and tolerability
- To assess the pharmacokinetics of IP PTX
- To assess the change in PCI following IP PTX in patients with metastatic appendiceal adenocarcinoma
- To assess rate of initially unresectable participants with metastatic appendiceal adenocarcinoma able to undergo CRS / HIPEC after IP PTX
- To assess the rate of conversion from positive to negative cytology in peritoneal fluid following IP PTX in participants with metastatic appendiceal adenocarcinoma
- To assess the prognostic value of circulating tumor DNA (ctDNA) in participants with metastatic appendiceal adenocarcinoma and the correlation of quantitative ctDNA measurement with radiographic and pathologic response
- To generate PDX and PDO models of appendiceal adenocarcinoma and evaluate their ability to predict response of human tumors
- To evaluate the effect of IP PTX on the transcriptomic state of appendiceal adenocarcinoma and the tumor microenvironment (TME) through comparison of pre- and post-treatment specimens
- To assess the impact of GNAS, KRAS, TP53, and APC mutation on response to IP PTX therapy
- To assess the impact of mucinous, signet ring cell, and goblet cell histology on response to IP PTX therapy
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Phase I/Phase II | Experimental | Drug: Dexamethasone, Drug: Diphenhydramine, Drug: Famotidine, Drug: PaclitaxelParticipants will be assigned to a study phase (Phase I or Phase II) based on when participants join this study. Up to 4 groups of at least 3 participants will be enrolled in Phase I of the study, and up to 15 participants will be enrolled in Phase II. If you are enrolled in Phase I, the dose of paclitaxel you receive will depend on when the participants join this study. The first group of participants will receive the lowest dose level of paclitaxel. Each new group will receive a higher dose of paclitaxel than the group before it, if no intolerable side effects were seen. This will continue until the highest tolerable dose of paclitaxel given intraperitoneally is found. Participants who are enrolled in Phase II, participants will receive paclitaxel at the recommended dose that was found in Phase I. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- Age 18 years and above. There will be no upper age restriction
- ECOG performance status ≤ 2
- Participants must have histologically confirmed diagnosis of unresectable locally metastatic appendiceal adenocarcinoma
- Metastatic disease in the peritoneal cavity and not a candidate for cytoreductive surgery
- Participants must have adequate organ and marrow function as defined below:
Key exclusion criteria (10 listed)
- Active infection such as pneumonia or wound infections that would preclude protocol therapy
- Participants with unstable angina or New York Heart Association (NYHA) Grade II or greater congestive heart failure
- Participants deemed unable to comply with study and/or follow-up procedures (i.e., cognitive impairment)
- Participants with a known hypersensitivity to protocol systemic chemotherapy that was life-threatening, required hospitalization or prolongation of existing hospitalization, or resulted in persistent or significant disability or incapacity
- Previous surgery that would preclude safe diagnostic laparoscopy with port placement
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 |
|---|---|---|
| DexamethasoneAlso: DecadronGiven by PO | Drug | Phase I/Phase II |
| DiphenhydramineAlso: Benadryl®Given by PO | Drug | Phase I/Phase II |
| FamotidineAlso: Pepcid®Given by PO | Drug | Phase I/Phase II |
| PaclitaxelAlso: TaxolGiven by PO | Drug | Phase I/Phase II |
Primary outcome
Safety and adverse events (AEs)
Time frame: Through study completion; an average of 1 year.
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
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 16 Jul 2026.
- MD Anderson Cancer CenterLink 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.
Beth Helmink, 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 Phase 1/2 study testing Dexamethasone, Diphenhydramine and Famotidine in people with Metastatic Appendiceal Adenocarcinoma and Intraperitoneal Paclitaxel, 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 18 inclusion and 10 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: looking for participants now, as of the sponsor's last update on 16 Jul 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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Contact Beth Helmink, MD ((832) 696-5784, bhelmink@mdanderson.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and adverse events (AEs) (time frame: Through study completion; an average of 1 year.). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 16 Jul 2026.