Clinical Trials

An up-to-date list of clinical trials currently enrolling patients with appendix cancer and/or pseudomyxoma peritonei from appendiceal origin is provided below.

The ACPMP Research Foundation attempts to keep this list as updated and complete as possible.  Please note, however, that the primary public sources from which this list is compiled do not always reflect the most recent changes to enrollment status, and there may be changes to the enrollment status or new trials added that are not yet reflected in the periodic updates provided.  Nevertheless, the below list of clinical trials, and the contact information provided for each, is intended to provide sufficient baseline information for use by any individual interested in further exploring potential eligibility and participation.

For more information on clinical trials for appendix cancer and/or pseudomyxoma peritonei or to update any of the information below, please contact Deborah Shelton.


Clinical studies (often also commonly referred to as clinical trials or clinical research) can have many different objectives, depending on the specific study.  Such objectives may include, for example, developing new treatments, identifying causes of a disease or condition, studying trends, and evaluating ways in which genetics may be related to a disease or condition.

Typically, a new therapy or procedure is first tested in the laboratory and in animal studies, and, if the results merit further investigation, it is then moved into a clinical trial (ie., tested in humans).  The clinical trial testing is conducted in phases (generally Phase 1, 2, and 3, each of which is briefly explained below).  Depending on the particular phase, clinical trials are designed to obtain more detailed information about the investigational therapy, including its safety profile, risks, and effectiveness.

Sometimes a patient decides to participate in a clinical trial because none of the treatments otherwise available have worked, or they find them to have side effects that are not tolerable.  In this way, clinical trials can sometimes provide another option when standard therapies have failed.  Other patients participate in a clinical trial because they want to contribute to the advancement of medical knowledge.  Often, an individual that decides to participate in a clinical trial does so with both goals in mind.

The study of an investigational drug in humans proceeds in phases, with the information learned from each phase used to build upon the next: generally Phase 1, 2, and 3.

A phase 1 study is early testing in humans.  Its purpose is to determine dosing, safety and tolerance of an investigational drug and to gather information about its biological activity within the body.

Once an acceptable margin of safety is established for the investigational drug, Phase 2 begins.  A Phase 2 study is generally the first study in which primary measurements will be made to determine efficacy of the investigational drug.  In terms of the a study’s objectives, though, the line between a Phase 1 and Phase 2 study can sometimes be somewhat blurred, particularly in the case of oncology trials where there is a small study population and interest in expediting development process while appropriately safeguarding patient safety.

A Phase 3 study is a large, pivotal safety and effectiveness trial.  Information gathered during this stage includes additional evidence of safety and efficacy, long-term tolerance, drug interactions, etc.

As noted above, clinical trials – particularly for new cancer therapies – are sometimes collapsed.  In other words, some researchers design these trials to combine to phases into a single study protocol (e.g., Phase 1/2, or Phase 2/3).  The reason for this type of design is to help facilitate a more seamless transition between phases, potentially allowing research questions to be answered more quickly or with fewer patients.

The National Cancer Institute (NCI) provides the following examples of benefits and risks of participating in a clinical trial that should be considered in deciding whether a clinical trial may be right for you:

Potential Benefits: may include having access to a new therapy that is not otherwise available; close monitoring by the clinical research team; being among the first to benefit from a therapy that is determined to be more effective than the standard treatment; and helping researchers learn more about your type of cancer; and benefitting the larger patient population in the future.

Potential Risks: may include the investigational treatment may be inferior to the standard treatment, or may not work for the individual patient at all; there may be unanticipated side effects or worse side effects than the standard treatment; increased doctors visits and associated expenses, including travel and logistical support; extra tests may be required that may be both time-consuming and uncomfortable; and a patient’s health insurance may not cover all patient costs in a clinical trial.

For additional information, see NIH's Clinical Trials Information for Patients and Caregivers.


The compilation of clinical-trial information provided is organized as follows: (1) the name of the clinical trial, and the study identifier and link to the registry providing more detailed information; (2) a brief summary of the protocol/objectives of the trial, (3) key baseline eligibility criteria, and (4) study contact information.  Please note that the eligibility criteria for any given clinical trial are typically extensive, and thus not detailed in the list of clinical trials provided here. All of the inclusion and exclusion criteria can be accessed, however, through the link provided for that particular trial and should be discussed further with your physician and/or the contact person listed for that clinical trial.  Examples of common eligibility for a clinical study of a new investigational cancer drug include (a) having a specific type or stage of cancer; (b) having received or not received a certain kind of prior therapy; (c) having specific genetic changes in your tumor; (d) medical history; and (e) current health status.

Each clinical trial is assigned a specific identifier; the identifier is referred to formally as the NCT number in the U.S. and is the registry number as provided in the listing for that trial at  The NCT number and the corresponding link to the full listing on is provided for each clinical trial listed here where applicable.

Nivolumab and Ipilimumab in Mucinous Colorectal and Appendiceal Tumors (NCT03693846)

Location: University of Pennsylvania, Abramson Cancer Center, Philadelphia, Pennsylvania.

Brief description: This is a Phase 2 clinical trial of patients with mucinous adenocarcinoma of the appendix (or colon, rectum).  A total of 21 patients will be enrolled.  Treatment will consist of nivolumab  (brand name OPDIVO) every 4 weeks and ipilimumab (brand name YERVOY) every 8 weeks until disease progression, unacceptable toxicity, or 2 years of therapy.

Baseline Eligibility Criteria, include, but are not limited to:

  • metastatic disease confined to the peritoneum only;
  • prior systemic chemotherapy with FOLFOX and FOLFIRI unless contraindicated or refused;
  • immunohistochemistry testing demonstrates microsatellite stable and/or mismatch repair proficient;
  • no bowel obstruction within the past 60 days.

Contact Person(s): Nurse Navigator Trish Gambino; Study Nurse Lisa DiCicco.

Note: Dr. Thomas Karasic, one of the investigators for this trial, spoke about the trial at the Drexel ACPMP Patient-Practitioner Symposium: “Innovations in the Multidisciplinary Management of PMP/Appendiceal Cancer,” Saturday April 6, 2019, 7:30 am to 12:30 pm, Philadelphia, Pennsylvania.  The ACPMP Research Foundation was the co-host and sponsor of this educational event.

Phase 1/2 Study to Investigate the Safety, Biologic, and Antitumor Activity of ONCOS-102 in combination with Durvalumab (brand name IMFINZI) in Patients with Peritoneal Malignancies (NCT02963831)

Location(s): Ludwig Cancer Institute - Research Facilities in Miami, Florida; Buffalo, New York; Toledo, Ohio, and Charlottesville, Virginia.

Brief description: This is a Phase 1/2 study in patients with peritoneal disease, including from appendiceal carcinoma.  A total of approximately 78 participants will be enrolled.  Treatment will consist of ONCOS-102 (an investigational drug) administered intraperitoneally once a week for a total of 6 weeks, and durvalumb (brand name IMFINZI) administered by IV infusion once every four weeks for a total of 12 four-week cycles.

Baseline Eligibility Criteria, include, but are not limited to:

  • peritoneal malignancy, including cancer originating from the appendix;
  • failed on prior standard chemotherapy with no additional therapy options available known to prolong survival;
  • willing to undergo a core needle biopsy during screening and during Study Week 5;
  • no ongoing bowel perforation or presence of bowel fistula or abscess, or history of small or large bowel obstruction within 3 months of registering for the clinical trial, including persons with palliative gastric drainage catheters;  NOTE: palliative diverting ileostomy or colostomy ARE ALLOWED if patient has been symptom-free for more than 3 months.

Contact Person(s)Lisa Shohara and/or Danielle McCabe, Tel. 212-450-1515

The MATCH Screening Trial: Targeted Therapy Directed by Genetic Testing in Treating Patients with Advanced Refractory Solid Tumors, Lymphomas, or Multiple Myeloma (NCT02465060)

Location(s):  National Cancer Institute, Bethesda, Maryland, and over a thousand other participating institutions throughout the U.S.

Brief description:  This is a large Phase 2 precision medicine trial to study the treatment directed by genetic testing in patients with solid tumors, including appendix, that have progressed following at least one line of standard treatment or for which there is no agreed upon treatment approach.  Approximately, a total of 6,452 participants will be enrolled across the numerous arms.  The thinking underlying this study is that patients with certain tumor mutations and other generic abnormalities may benefit more from treatment that targets that tumor’s particular generic abnormality.  Identifying those genetic abnormalities may help doctors plan better subsequent treatment for those patients.

In terms of steps to enroll, here is how it works.  First, the physician at one of the participating clinical trial sites orders genomic sequencing from one of the designated commercial labs.  Second, the lab tests the tumor sample for gene abnormalities for one or more being studied as part of the trial.  Third, if the patient does have an abnormality that matches one of the numerous ones being studied, the lab contacts the ordering physician and provides him or her with a written referral to the trial.  Fourth, if the patient and his or her physician decide to pursue the MATCH trial as a treatment option, the physician uses that written referral from the lab to register the patient for a screening assessment.  Fifth, the study personnel review the referral and formally assign the patient to the treatment arm for that generic abnormality and notify the patient.  And Sixth, once the patient is enrolled in the treatment arm, the patient is treated as long as the tumor is shrinking or remains stable.

Baseline Eligibility Criteria, include, but are not limited to:

  • have medically (histologically) documented solid tumor (disease agnostic; this study is directed at certain generic mutations) requiring therapy and meet either 1. or 2. below:
    • 1.  have progressed following at least one line of standard systemic therapy and there is no other approval/standard therapy available that has been shown to prolong overall survival.  Patients who cannot receive other standard therapy due to medical issues are eligible so long as the other eligibility criteria are met.  Note:  If the patient is currently receiving therapy, regardless of whether it is considered standard, the clinician must have assessed that the current therapy is no longer benefitting the patient.; or
    • 2.  have disease for which no standard treatment exists that has been shown to prolong survival; and
  • have measurable disease;
  • have received results from one of the designated laboratories indicating a “rare variant” of tumor that has been identified by participating commercial lab mutation of interest for this trial;
  • be able to swallow tablets or capsules; a patient with any GI disease that would impair ability to swallow, retain, or absorb drug is not eligible.

Contact Person(s):  Contact information is site-specific; review the site locations list to identify listed contact for the site(s) of interest to you.  If interested in participating at the NCI site, Bethesda, Maryland, please contact Deborah Shelton for specific contact information.

Dose Escalation and Expansion Study of GSK3359609 in Subjects with Selected Advanced Solid Tumors (INDUCE-1) (NCT02723955)

Locations:  Multiple throughout U.S. and outside of U.S.  U.S. sites in CA, FLA, NY, OK, PA, TN.  Participating sites outside of U.S. in Australia; Canada; France; Netherlands; and Spain.  For specific participating sites, contact Deborah Shelton.

Brief description:  This is a Phase 1 clinical trial of patients with advanced or recurrent solid tumors, including appendiceal.  A total of approximately 500 patients will be enrolled.  Treatment will consist of an investigational drug administered as a stand-alone therapy and in combination with pembrolizumab (brand name KEYTRUDA)  or specified chemotherapy regimens.

Baseline Eligibility Criteria, include, but are not limited to:

  • invasive appendiceal malignancy (or others as specified) that has been  diagnosed as locally advanced/metastatic or relapsed/refractory;
  • disease has progressed after standard therapy for the specific tumor type, or for which standard therapy has proven to be ineffective, intolerable, or is considered inappropriate, or no further standard therapy exists;
  • recent history (within 6 months) of acute diverticulitis, inflammatory bowel disease, intra-abdominal abscess, or gastrointestinal obstruction
  • recent history (within 6 months) of uncontrolled symptomatic ascites or pleural effusions.

Contact Person(s):  US GSK Clinical Trials Call Center at 877-379-3718, or for specific site locations/contact information anywhere within or outside of U.S., please contact Deborah Shelton.

My Pathway:  A Study Evaluating Herceptin/Perjeta, Tarceva, Zelboraf/Cotellic, Erivedge, Alecensa, and Tecentriq Treatment Targeted Against Certain Molecular Alterations in Participants with Advanced Solid Tumors  (NCT02091141)

Location(s):  51 locations throughout the U.S.  For specific participating sites, contact Deborah Shelton.

Brief description:  This is a Phase 2 study in patients with metastatic cancer, including of appendiceal origin.  A total of approximately 765 participants will be enrolled.  Treatment will depend on specific treatment arm, which, in turn depends on that participant’s specific tumor mutation or gene expression abnormality as demonstrated by the molecular-testing results from a CLIA-certified laboratory (using tissue and/or blood, depending on treatment arm).  Specific mutations being tested specified below under “Baseline Eligibility Criteria.”

Baseline Eligibility Criteria, include, but are not limited to:

  • metastatic cancer (including of appendiceal origin);
  • Have received standard first-line therapy for metastatic cancer (except for a tumor type for which no first-line therapy exists) and for whom a trial of targeted therapy is considered the best available treatment option.  (NOTE:  To be eligible, should not have any available therapies that will convey clinical benefit and/or are not suitable options per the treating physician’s judgment);
  • no prior treatment with the specific assigned study drug or any other drug sharing that same molecular target;
  • Mutations being studied as part of trial:  EGFR-activating; BRAF V600; hedgehog pathway relevant mutation; ALK; and/or elevated tissue tumor mutational burden.  (Note:  HER2 is being studied only in biliary cancer, salivary cancer, and bladder cancer.)
  • for all treatment arms (except the one for elevated tissue tumor mutational burden, must have the ability to swallow pills (i.e., drug is atezolizumab (brand name TECENTRIQ)) and not have refractory nausea and vomiting, malabsorption, external biliary shunt, or significant bowel resection that would preclude absorption of the oral investigational drug.

Contact Person(s):  Pam Mangat

Mesothlin-Targeted Immunotoxin LMB-100 in Combination with Tofacitinib in Persons with Previously Treated Pancreatic Adenocarcinoma, Cholangiocarcinoma and Other Mesothelin Expressing Solid Tumors (NCT04034238)

Location:  National Cancer Institute (Bethesda, Maryland)

Brief description:  This is a Phase 1 study of patients with solid tumors that express the protein mesothelin.  The primary objective of the study is to determine a tolerable and safe dose of the investigational drug, LMB-100, in combination with an FDA-approved drug (approved for other uses such as arthritis and ulcerative colitis) called tofacitinib (brand name Xeljanz).  This study will enroll approximately 45 participants.

Baseline Eligibility Criteria, include, but are not limited to:

  • Any solid tumor for which at least 20% of tumor cells express mesothelin. [Note:  With patient consent, NCI can screen any appendiceal patients to determine whether their tumor is mesothelin-positive and, thus, eligible to enroll in this clinical trial.];
  • No curative therapy exists for the patient;
  • Must have received at least one prior standard chemotherapy regimen for advanced disease OR be ineligible to receive available standard therapy, or have received all available standard therapies available for prior treatment of early stage disease;
  • If disease is deficient MisMatch Repair (dMMR) or Micro-Satellite Instability High (MSI-H), must have received at least one prior anti-PD1 therapy, OR be ineligible due to other medical conditions, or have refused anti-PD1 therapy.

Contact Person(s)Maureen E. Edgerly, R.N. - Tel. 240-760-6013

Note: NCI reached out to ACPMP for assistance and to build awareness for this trial. Click here for blog post. 

Feasibility of the LUM Imaging System for Detection of Peritoneal Surface Malignancies (NCT03834272)

Location: Massachusetts General Hospital Cancer Center, Boston, Massachusetts.

Brief description: This is a Phase 1/2 study of patients with metastases of the peritoneum from primary gastrointestinal cancer or appendiceal cancer, ovarian cancer, or mesothelioma. A total of 18 patients will be enrolled. This study is being conducted to test if the investigational drug (LUM015) can be given to patients at a dose that allows tumor tissue to be identified by the LUM Imaging System during surgery. Treatment will include intraoperative injection of an investigational drug (LUM015) and in vivo imaging with a handheld optical head.

Baseline Eligibility Criteria, include, but are not limited to:

  • metastases to the peritoneum from appendiceal cancer, gastrointestinal cancer, ovarian cancer, or mesothelioma;
  • must be scheduled for surgical resection (cytoreduction);
  • no investigational drugs within 30 days prior to surgery;
  • no history of allergic reaction to any oral or intravenous contrast agents.

Contact Person(s): Principal Investigator James Cusack, MD or Study Coordinator Bridget Kelly  – Tel.617-724-4093

Injection of Bromelain and Acetylcysteine in combination into recurrent mucinous tumor or pseudomyxoma peritonei: a phase 1/2 study (Australian New Zealand Clinical Trials Registry (ANZCTR) Universal Trial Number (UTN) U1111-1203-1657)

Location(s): St. George Hospital – Kogarah, Australia

Brief description: This is a Phase 1/2 study in patients with mucinous peritoneal tumor, including PMP that are not suitable for cytoreductive surgery and hyperthermic intraperitoneal chemotherapy (CRS/HIPEC) or other potentially beneficial surgery.   A total of approximately 60 participants will be enrolled.  The combination of Bromelain and Acetylcysteine will be injected directly into the tumor or peritoneal cavity via a drain and allowed to dwell for 24 hours with the expectation that the tumor will be dissolved by the drug combination administered.  The tumor will then be drained and a repeat treatment will be considered.  The dose of the drug is dependent on the calculated tumor dimensions and volume.

Baseline Eligibility Criteria, include, but are not limited to:

  • patients with psuedomyxoma Peritonei (PMP or mucinous soft to intermediate grade tumor who are not candidates for CRS/HIPEC or other surgery;
    • Note:  Having hard tumor in one region does not exclude treatment in another area provided the appearance is mucinous.
  • in the case of a target lesion (versus free intraperitoneal), the tumor must be safely accessible percutaneously;
  • no suspected fistula of the tumor into the GI tract, invading or abutting major vessel or other area of concern;
  • no infected tumor (pus on aspiration or indicated on blood test).

Contact Person(s)Sarah Valle, Tel. +61291132070

Note: Currently enrolling in Australia only; U.S. and European are forthcoming.

Intraperitoneal Oxaliplatin in Combo with IV mFOLFRI for Peritoneal Carcinomatosis from Colorectal & Appendiceal Cancer (IPOX-FOLFRI) (NCT04158349)

Location:  Huntsman Cancer Institute, Salt Lake City, Utah.

Brief description: This is a Phase 1 study to determine the maximum tolerated dose (MTD) of intraperitoneal oxaliplatin when given in combination with mFOLFRI, a standardized chemotherapy regimen utilizing 5-fluorouracil, leucovorin, and irinotecan.  This study will be halted once the maximum tolerated dose has been established.  During therapy, patients will be monitored for dose-limiting toxicities and adverse events.  Upon completion or cessation of participation, patients will be followed until resolution of any ongoing study treatment-related adverse events, initiation of alternative treatment, and survival.  Approximately 18 participants will be enrolled.

Baseline Eligibility Criteria include, but are not limited to:

  • Confirmed appendiceal adenocarcinoma (or colon, rectal) with peritoneal dissemination of disease within 6 months or more after initial diagnosis.
  • Have Stage IV peritoneal-based disease only; must not have any metastases outside the peritoneal cavity.
  • Willing and able to to undergo placement of an intraperitoneal catheter (if do not already have one).
  • Willing to return for follow-up.

Contact:  Susan Sharry at 801-585-3453 (phone) or (e-mail)

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